[1]
Kunz, W.; Keller, H.; Muckter, H. N-phthalyl-glutamic acid imide; experimental studies on a new synthetic product with sedative properties. Arzneimittelforschung, 1956, 6, 426-430.
[2]
McBride, W.G. Thalidomide and congenital abnormalities. Lancet, 1961, 2, 1358.
[3]
Miller, M.T.; Stromland, K. Teratogen update: Thalidomide: A review with a focus on ocular findings and new potential uses. Teratology, 1999, 60, 306-321.
[4]
Knobloch, J.; Ruther, U. Shedding light on an old mystery: Thalidomide suppresses survival pathways to induce limb defects. Cell Cycle, 2008, 7, 1121-1127.
[5]
Kim, J.H.; Scialli, A.R. Thalidomide: The tragedy of birth defects and the effective treatment of disease. Toxicol. Sci., 2011, 122, 1-6.
[6]
Ito, T.; Handa, H. Deciphering the mystery of thalidomide teratogenicity. Congenit. Anom. (Kyoto), 2012, 52, 1-7. [Kyoto].
[7]
Singhal, S.; Mehta, J.; Desikan, R.; Ayers, D.; Roberson, P.; Eddlemon, P.; Munshi, N.; Anaissie, E.; Wilson, C.; Dhodapkar, M.; Zeddis, J.; Barlogie, B. Antitumor activity of thalidomide in refractory multiple myeloma. N. Engl. J. Med., 1999, 341, 1565-1571.
[8]
Richardson, P.; Anderson, K. Thalidomide and dexamethasone: a new standard of care for initial therapy in multiple myeloma. J. Clin. Oncol., 2006, 24, 334-336.
[9]
Xu, M.; Hou, Y.; Sheng, L.; Peng, J. Therapeutic effects of thalidomide in haematological disorders: A review. Front. Med., 2013, 7, 290-300.
[10]
Mark, T.M.; Bowman, I.A.; Rossi, A.C.; Shah, M.; Rodriguez, M.; Quinn, R.; Pearse, R.N.; Zafar, F.; Pekle, K.; Jayabalan, D.; Ely, S.; Coleman, M.; Chen-Kiang, S.; Niesvizky, R. Thalidomide, clarithromycin, lenalidomide and dexamethasone therapy in newly diagnosed, symptomatic multiple myeloma. Leuk. Lymphoma, 2014, 55, 2842-2849.
[11]
Schey, S.; Brown, S.R.; Tillotson, A.L.; Yong, K.; Williams, C.; Davies, F.; Morgan, G.; Cavenagh, J.; Cook, G.; Cook, M.; Orti, G.; Morris, C.; Sherratt, D.; Flanagan, L.; Gregory, W.; Cavet, J. Myeloma UK Early Phase Clinical Trial Network. Bendamustine, thalidomide and dexamethasone combination therapy for relapsed/refractory myeloma patients: results of the MUKone randomized dose selection trial. Br. J. Haematol., 2015, 170, 336-348.
[12]
Sonneveld, P.; Asselbergs, E.; Zweegman, S.; van der Holt, B.; Kersten, M.J.; Vellenga, E.; van Marwijk-Kooy, M.; Broyl, A.; de Weerdt, O.; Lonergan, S.; Palumbo, A.; Lokhorst, H. Phase 2 study of carfilzomib, thalidomide, and dexamethasone as induction/consolidation therapy for newly diagnosed multiple myeloma. Blood, 2015, 125, 449-456.
[13]
Smith, S.M.; Grinblatt, D.; Johnson, J.L.; Niedzwiecki, D.; Rizzieri, D.; Bartlett, N.L.; Cheson, B.D. Cancer and Leukemia Group B. Thalidomide has limited single-agent activity in relapsed or refractory indolent non-Hodgkin lymphomas: a phase II trial of the Cancer and Leukemia Group B. Br. J. Haematol., 2008, 140, 313-319.
[14]
Wu, H.; Zhao, C.; Gu, K.; Jiao, Y.; Hao, J.; Sun, G. Thalidomide plus chemotherapy exhibit enhanced efficacy in the clinical treatment of T-cell non-Hodgkin’s lymphoma: A prospective study of 46 cases. Mol. Clin. Oncol., 2014, 2, 695-700.
[15]
Damaj, G.; Lefrère, F.; Delarue, R.; Varet, B.; Furman, R.; Hermine, O. Thalidomide therapy induces response in relapsed mantle cell lymphoma. Leukemia, 2003, 17, 1914-1915.
[16]
Richardson, S.J.; Eve, H.E.; Copplestone, J.A.; Dyer, M.J.; Rule, S.A. Activity of thalidomide and lenalidomide in mantle cell lymphoma. Acta Haematol., 2010, 123, 21-29.
[17]
Awan, F.T.; Johnson, A.J.; Lapalombella, R.; Hu, W.; Lucas, M.; Fischer, B.; Byrd, J.C. Thalidomide and lenalidomide as new therapeutics for the treatment of chronic lymphocytic leukemia. Leuk. Lymphoma, 2010, 51, 27-38.
[18]
Pointon, J.C.; Eagle, G.; Bailey, J.; Evans, P.; Allsup, D.; Greenman, J. Thalidomide enhances cyclophosphamide and dexamethasone-mediated cytotoxicity towards cultured chronic lymphocytic leukaemia cells. Oncol. Rep., 2010, 24, 1315-1321.
[19]
Giannopoulos, K.; Mertens, D.; Stilgenbauer, S. Treating chronic lymphocytic leukemia with thalidomide and lenalidomide. Expert Opin. Pharmacother., 2011, 12, 2857-2864.
[20]
Luo, X.; Xu, Y.; Li, B.; Qin, T.; Zhang, P.; Zhang, H.; Fang, L.; Pan, L.; Hu, N.; Qu, S.; Zhang, Y.; Huang, G.; Gale, R.P.; Xiao, Z. Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response. Blood Cancer J., 2018, 8, 9.
[21]
Millrine, D.; Kishimoto, T. A brighter side to lenalidomide: its potential use in immunological disorders. Trends Mol. Med., 2017, 23, 348-361.
[22]
Raza, A.; Meyer, P.; Dutt, D.; Zorat, F.; Lisak, L.; Nascimben, F.; du Randt, M.; Kaspar, C.; Goldberg, C.; Loew, J.; Dar, S.; Gezer, S.; Venugopal, P.; Zeldis, J. Thalidomide produces transfusion independence in long standing refractory anemias of patients with myelodysplastic syndromes. Blood, 2001, 98, 958-965.
[23]
Strupp, C.; Germing, U.; Aivado, M.; Misgeld, E.; Haas, R.; Gattermann, N. Thalidomide for the treatment of patients with myelodysplastic syndromes. Leukemia, 2002, 16, 1-6.
[24]
Musto, P. Thalidomide therapy for myelodysplastic syndromes: Current status and future perspectives. Leuk. Res., 2004, 28, 325-332.
[25]
Invernizzi, R.; Travaglino, E.; De Amici, M.; Brugnatelli, S.; Ramajoli, I.; Rovati, B.; Benatti, C.; Ascari, E. Thalidomide treatment reduces apoptosis levels in bone marrow cells from patients with myelodysplastic syndromes. Leuk. Res., 2005, 29, 641-647.
[26]
Musto, P. Thalidomide therapy in adult patients with myelodysplastic syndrome: A north central cancer treatment group phase II trial. Cancer, 2007, 109, 1211-1212.
[27]
Hayashi, K.; Hattori, K.; Toi, F. Thalidomide is a highly effective treatment of MDS: A single-hospital experience in Japan. Int. J. Hematol., 2010, 91, 725-727.
[28]
Chung, S.Y.; Lin, S.F.; Chen, P.M.; Chang, M.C.; Kao, W.Y.; Chao, T.Y.; Hsiao, L.T.; Yen, C.C.; Yang, M.H.; Hwang, W.S.; Lin, T.L.; Chiou, T.J.; Chang, C.S. Thalidomide for the treatment of myelodysplastic syndrome in Taiwan: results of a phase II trial. Anticancer Res., 2012, 32, 3415-3419.
[29]
Zhao, W.H.; Zeng, Q.C.; Huang, B.T.; Li, B.S.; Chen, R.L. Decitabine plus thalidomide yields more sustained survival rates than decitabine monotherapy for risk-tailored elderly patients with myelodysplastic syndrome. Leuk. Res., 2015, 39, 424-428.
[30]
Corral, L.G.; Haslett, P.A.; Muller, G.V.; Chen, R.; Wong, L.M.; Ocampo, C.J.; Patterson, R.T.; Stirling, D.I.; Kaplan, G. Differential cytokine modulation and T cell activation by two distinct classes of thalidomide analogues that are potent inhibitors of TNF-alpha. J. Immunol., 1999, 163, 380-386.
[31]
Vallet, S.; Palumbo, A.; Raje, N.; Boccadoro, M.; Anderson, K.C. Thalidomide and lenalidomide: mechanism-based potential drug combinations. Leuk. Lymphoma, 2008, 49, 1238-1245.
[32]
Kotla, V.; Goel, S.; Nischal, S.; Heuck, C.; Vivek, K.; Das, B.; Verma, A. Mechanism of action of lenalidomide in hematological malignancies. J. Hematol. Oncol., 2009, 2, 36.
[33]
Sedlarikova, L.; Kubiczkova, L.; Sevcikova, S.; Hajek, R. Mechanism of immunomodulatory drugs in multiple myeloma. Leuk. Res., 2012, 36, 1218-1224.
[34]
Vo, M.C.; Anh-NguyenThi, T.; Lee, J.H.; Nguyen-Pham, T.N.; Lakshmi, T.J.; Jung, S.H.; Kim, J.H.; Lee, J.J. Lenalidomide enhances the function of dendritic cells generated from patients with multiple myeloma. Exp. Hematol., 2017, 46, 48-55.
[35]
Galustian, C.; Meyer, B.; Labarte, M.C.; Dredge, K.; Klaschka, D.; Henry, J.; Todryk, S.; Chen, R.; Muller, G.; Stirling, D.; Schafer, P.; Bartlett, J.B.; Dalgleish, A.G. The anti-cancer agents lenalidomide and pomalidomide inhibit the proliferation and function of T regulatory cells. Cancer Immunol. Immunother., 2009, 58, 1033-1045.
[36]
Davies, F.; Baz, R. Lenalidomide mode of action: linking bench and clinical findings. Blood Rev., 2010, 24(Suppl. 1), S13-S19.
[37]
Dredge, K.; Marriott, J.B.; Macdonald, C.D.; Man, H.W.; Chen, R.; Muller, G.W.; Stirling, D.; Dalgleish, A.G. Novel thalidomide analogues display anti-angiogenic activity independently of immunomodulatory effects. Br. J. Cancer, 2002, 87, 1166-1172.
[38]
Dredge, K.; Horsfall, R.; Robinson, S.P.; Zhang, L.H.; Lu, L.; Tang, Y.; Shirley, M.A.; Muller, G.; Schafer, P.; Stirling, D.; Dalgleish, A.G.; Bartlett, J.B. Orally administrated lenalidomide (CC-5013) is anti-angiogenic in vivo and inhibits endothelial cell migration and Akt phosphorylation in vitro. Microvasc. Res., 2005, 69, 56-63.
[39]
Dankbar, B.; Padró, T.; Leo, R.; Feldmann, B.; Kropff, M.; Mesters, R.M.; Serve, H.; Berdel, W.E.; Kienast, J. Vascular endothelial growth factor and interleukin-6 in paracrine tumor-stromal cell interactions in multiple myeloma. Blood, 2000, 95, 2630-2636.
[40]
Escoubet-Lozach, L.; Lin, I.L.; Jensen-Pergakes, K.; Brady, H.A.; Gandhi, A.K.; Schafer, P.H.; Muller, G.W.; Worland, P.J.; Chan, K.W.; Verhelle, D. Pomalidomide and lenalidomide induce p21 WAF-1 expression in both lymphoma and multiple myeloma through a LSD1-mediated epigenetic mechanism. Cancer Res., 2009, 69, 7347-7356.
[41]
Mitsiades, N.; Mitsiades, C.S.; Poulaki, V.; Chauhan, D.; Richardson, P.G.; Hideshima, T.; Munshi, N.C.; Treon, S.P.; Anderson, K.C. Apoptotic signaling induced by immunomodulatory thalidomide analogs in human multiple myeloma cells: Therapeutic implications. Blood, 2002, 99, 4525-4530.
[42]
Chang, X.; Zhu, Y.; Shi, C.; Stewart, A.K. Mechanism of immunomodulatory drugs´ action in the treatment of multiple myeloma. Acta Biochim. Biophys. Sin. (Shanghai), 2014, 46, 240-253.
[43]
Wiernik, P.H.; Lossos, I.S.; Tuscano, J.M.; Justice, G.; Vose, J.M.; Cole, C.E.; Lam, W.; McBride, K.; Wride, K.; Pietronigro, D.; Takeshita, K.; Ervin-Haynes, A.; Zeldis, J.B.; Habermann, T.M. Lenalidomide monotherapy in relapsed or refractory aggressive non-Hodgkin’s lymphoma. J. Clin. Oncol., 2008, 26, 4952-4957.
[44]
Habermann, T.M.; Lossos, I.S.; Justice, G.; Vose, J.M.; Wiernik, P.H.; McBride, K.; Wride, K.; Ervin-Haynes, A.; Takeshita, K.; Pietronigro, D.; Zeldis, J.B.; Tuscano, J.M. Lenalidomide oral monotherapy produces a high response rate patients with relapsed or refractory mantle cell lymphoma. Br. J. Haematol., 2009, 145, 344-349.
[45]
Witzig, T.E.; Vose, J.M.; Zinzani, P.L.; Reeder, C.B.; Buckstein, R.; Polikoff, J.A.; Bouabdallah, R.; Haioun, C.; Tilly, H.; Guo, P.; Pietronigro, D.; Ervin-Haynes, A.L.; Czuczman, M.S. An international phase II trial of single agent lenalidomide for relapsed or refractory aggressive B-cell non-Hodgkin’s lymphoma. Ann. Oncol., 2011, 22, 1622-1627.
[46]
Eve, H.E.; Carey, S.; Richardson, S.J.; Heise, C.C.; Mamidipudi, V.; Shi, T.; Radford, J.A.; Auer, R.L.; Bullard, S.H.; Rule, S.A. Single-agent lenalidomide in relapsed / refractory mantle cell lymphoma: Results from a UK phase II study suggest activity and possible gender differences. Br. J. Haematol., 2012, 159, 154-163.
[47]
Pan, B.; Lentzsch, S. The application and biology of immunomodulatory drugs (IMiDs) in cancer. Pharmacol. Ther., 2012, 136, 56-68.
[48]
Dawar, R.; Hernandez-Ilizalilutti, F. The emerging role of lenalidomide in the management of mantle cell lymphoma (MCL). Best Pract. Res. Clin. Haematol., 2012, 25, 185-190.
[49]
Goy, A.; Sinha, R.; Williams, M.E.; Kalayoglu Besisik, S.; Drach, J.; Ramchandren, R.; Zhang, L.; Cicero, S.; Fu, T.; Witzig, T.E. Single-agent lenalidomide in patients with mantle-cell lymphoma who relapsed or progressed after or were refractory to bortezomib: Phase II MCL-001 (EMERGE) study. J. Clin. Oncol., 2013, 31, 3688-3695.
[50]
Zinzani, P.L.; Vose, J.M.; Czuczman, M.S.; Reeder, C.B.; Haioun, C.; Polikoff, J.; Tilly, H.; Zhang, L.; Prandi, K.; Li, J.; Witzig, T.E. Long-term follow-up of lenalidomide in relapsed/refractory mantle-cell lymphoma: subset analysis of the NHL-003 study. Ann. Oncol., 2013, 24, 2892-2897.
[51]
Zhang, L.H.; Kosek, J.; Wang, M.; Heise, C.; Schafer, P.H.; Chopra, R. Lenalidomide efficacy in activated B-cell-like subtype diffuse large B-cell lymphoma is dependent upon IRF4 and cereblon expression. Br. J. Haematol., 2013, 160, 487-502.
[52]
Kritharis, A.; Coyle, M.; Sharma, J.; Evens, A.M. Lenalidomide in non-Hodgkin lymphoma: Biological perspectives and therapeutic opportunities. Blood, 2015, 125, 2471-2476.
[53]
Chen, C.I. Lenalidomide alone and in combination for chronic lymphocytic leukemia. Curr. Hematol. Malig. Rep., 2013, 8, 7-13.
[54]
Kater, A.P.; Tonino, S.H.; Egle, A.; Ramsay, A.G. How does lenalidomide target the chronic lymphocytic leukemia microenvironment? Blood, 2014, 124, 2184-2189.
[55]
Liberati, A.M.; Vitolo, U.; Palumbo, A.; Cortelezzi, A. Lenalidomide in the treatment of lymphoproliferative disorders and multiple myeloma. Adv. Hematol., 2013, 2013, 812605.
[56]
Yang, B.; Yu, R.L.; Chi, X.H.; Lu, X.C. Lenalidomide treatment for multiple myeloma: Systematic review and meta analysis of randomized controlled trials. PLoS One, 2013, 8, e64354.
[57]
Dimopoulos, M.A.; Terpos, E.; Niesvizky, R. How lenalidomide is changing the treatment of patients with multiple myeloma. Crit. Rev. Oncol. Hematol., 2013, 88(Suppl. 1), S23-S35.
[58]
Cives, M.; Simone, V.; Brunetti, O.; Longo, V.; Silvestris, F. Novel lenalidomide-based combinations for treatment of multiple myeloma. Crit. Rev. Oncol. Hematol., 2013, 85, 9-20.
[59]
Zago, M.; Oehrlein, K.; Rendl, C.; Hahn-Ast, C.; Kanz, L.; Weisel, K. Lenalidomide in relapsed and refractory multiple myeloma disease: Feasibility and benefits of long-term treatment. Ann. Hematol., 2014, 93, 1993-1999.
[60]
Popat, R.; Khan, I.; Dickson, J.; Cheesman, S.; Smith, D.; D’Sa, S.; Rabin, N.; Yong, K. An alternative dosing strategy of lenalidomide for patients with relapsed multiple myeloma. Br. J. Haematol., 2015, 168, 148-151.
[61]
Moreau, P.; Attal, M.; Facon, T. Frontline therapy of multiple myeloma. Blood, 2015, 125, 3076-3084.
[62]
Zagouri, F.; Terpos, E.; Kastritis, E.; Dimopoulos, M.A. An update on the use of lenalidomide for the treatment of multiple myeloma. Expert Opin. Pharmacother., 2015, 16, 1865-1877.
[63]
Kim, Y.; Schmidt-Wolf, I.G. Lenalidomide in multiple myeloma. Expert Rev. Anticancer Ther., 2015, 15, 491-497.
[64]
Kuroda, J.; Kobayashi, T.; Taniwaki, M. Prognostic indicators of lenalidomide for multiple myeloma: consensus and controversy. Expert Rev. Anticancer Ther., 2015, 15, 787-804.
[65]
Fink, E.C.; Ebert, B.L. The novel mechanism of lenalidomide activity. Blood, 2015, 126, 2366-2369.
[66]
Lacy, M.Q.; Hayman, S.R.; Gertz, M.A.; Dispenzieri, A.; Buadi, F.; Kumar, S.; Greipp, P.R.; Lust, J.A.; Russell, S.J.; Dingli, D.; Kyle, R.A.; Fonseca, R.; Bergsagel, P.L.; Roy, V.; Mikhael, J.R.; Stewart, A.K.; Laumann, K.; Allred, J.B.; Mandrekar, S.J.; Rajkumar, S.V. Pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma. J. Clin. Oncol., 2009, 27, 5008-5014.
[67]
Lacy, M.Q.; Hayman, S.R.; Gertz, M.A.; Short, K.D.; Dispenzieri, A.; Kumar, S.; Greipp, P.R.; Lust, J.A.; Russell, S.J.; Dingli, D.; Zeldenrust, S.; Fonseca, R.; Bergsagel, P.L.; Roy, V.; Mikhael, J.R.; Stewart, A.K.; Laumann, K.; Allred, J.B.; Mandrekar, S.J.; Rajkumar, S.V.; Buadi, F. Pomalidomide (CC4047) plus low-dose dexamethasone (Pom/dex) is active and well tolerated in lenalidomide refractory multiple myeloma (MM). Leukemia, 2010, 24, 1934-1939.
[68]
Schey, S.; Ramasamy, K. Pomalidomide therapy for myeloma. Expert Opin. Investig. Drugs, 2011, 20, 691-700.
[69]
Richardson, P.G.; Siegel, D.; Baz, R.; Kelley, S.L.; Munshi, N.C.; Laubach, J.; Sullivan, D.; Alsina, M.; Schlossman, R.; Ghobrial, I.M.; Doss, D.; Loughney, N.; McBride, L.; Bilotti, E.; Anand, P.; Nardelli, L.; Wear, S.; Larkins, G.; Chen, M.; Zaki, M.H.; Jacques, C.; Anderson, K.C. Phase 1 study of pomalidomide MTD, safety, and efficacy in patients with refractory multiple myeloma who have received lenalidomide and bortezomib. Blood, 2013, 121, 1961-1967.
[70]
Leleu, X.; Attal, M.; Arnulf, B.; Moreau, P.; Traulle, C.; Marit, G.; Mathiot, C.; Petillon, M.O.; Macro, M.; Roussel, M.; Pegourie, B.; Kolb, B.; Stoppa, A.M.; Hennache, B.; Bréchignac, S.; Meuleman, N.; Thielemans, B.; Garderet, L.; Royer, B.; Hulin, C.; Benboubker, L.; Decaux, O.; Escoffre-Barbe, M.; Michallet, M.; Caillot, D.; Fermand, J.P.; Avet-Loiseau, H.; Facon, T. Intergroupe Francophone du Myélome. Pomalidomide plus low-dose dexamethasone is active and well tolerated in bortezomib and lenalidomide-refractory multiple myeloma: Intergroupe Francophone du Myelome 2009-02. Blood, 2013, 121, 1968-1975.
[71]
Miguel, J.S.; Weisel, K.; Moreau, P.; Lacy, M.; Song, K.; Delforge, M.; Karlin, L.; Goldschmidt, H.; Banos, A.; Oriol, A.; Alegre, A.; Chen, C.; Cavo, M.; Garderet, L.; Ivanova, V.; Martinez-Lopez, J.; Belch, A.; Palumbo, A.; Schey, S.; Sonneveld, P.; Yu, X.; Sternas, L.; Jacques, C.; Zaki, M.; Dimopoulos, M. Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003): a randomised open-label phase 3 trial. Lancet Oncol., 2013, 14, 1055-1066.
[72]
Richardson, P.G.; Siegel, D.; Vij, R.; Hofmeister, C.C.; Baz, R.; Jagannath, S.; Chen, C.; Lonial, S.; Jakubowiak, A.; Bahlis, N.; Song, K.; Belch, A.; Raje, N.; Shustik, C.; Lentzsch, S.; Lacy, M.; Mikhael, J.; Matous, J.; Vesole, D.; Chen, M.; Zaki, M.H.; Jacques, C.; Yu, Z.; Anderson, K.C. Pomalidomide alone or in combination with low-dose dexamethasone in relapsed and refractory multiple myeloma: a randomized phase 2 study. Blood, 2014, 123, 1826-1832.
[73]
Clark, S.M.; Steinbach, A.; Clemmons, A.B. Pomalidomide for the treatment of multiple myeloma. J. Adv. Pract. Oncol., 2014, 5, 51-56.
[74]
Dimopoulos, M.A.; Leleu, X.; Palumbo, A.; Moreau, P.; Delforge, M.; Cavo, M.; Ludwig, H.; Morgan, G.J.; Davies, F.E.; Sonneveld, P.; Schey, S.A.; Zweegman, S.; Hansson, M.; Weisel, K.; Mateos, M.V.; Facon, T.; Miguel, J.F. Expert panel consensus statement on the optimal use of pomalidomide in relapsed and refractory multiple myeloma. Leukemia, 2014, 28, 1573-1585.
[75]
Mark, T.M.; Coleman, M.; Niesvizky, R. Preclinical and clinical results with pomalidomide in the treatment of relapsed / refractory multiple myeloma. Leuk. Res., 2014, 38, 517-524.
[76]
Leleu, X.; Karlin, L.; Macro, M.; Hulin, C.; Garderet, L.; Roussel, M.; Arnulf, B.; Pegourie, B.; Kolb, B.; Stoppa, A.M.; Brechiniac, S.; Marit, G.; Thielemans, B.; Onraed, B.; Mathiot, C.; Banos, A.; Lacotte, L.; Tiab, M.; Dib, M.; Fuzibet, J.G.; Petillon, M.O.; Rodon, P.; Wetterwald, M.; Royer, B.; Legros, L.; Benboubker, L.; Decaux, O.; Escoffre-Barbe, M.; Caillot, D.; Fermand, J.P.; Moreau, P.; Attal, M.; Avet-Loiseau, H.; Facon, T. Intergroupe Francophone du Myélome (IFM). Pomalidomide plus low-dose dexamethasone in multiple myeloma with deletion 17p and/or translocation (4;14): IFM 2010-02 trial results. Blood, 2015, 125, 1411-1417.
[77]
San Miguel, J.F.; Weisel, K.C.; Song, K.W.; Delforge, M.; Karlin, L.; Goldschmidt, H.; Moreau, P.; Banos, A.; Oriol, A.; Garderet, L.; Cavo, M.; Ivanova, V.; Alegre, A.; Martinez-Lopez, J.; Chen, C.; Renner, C.; Bahlis, N.J.; Yu, X.; Teasdale, T.; Sternas, L.; Jacques, C.; Zaki, M.H.; Dimopoulos, M.A. Impact of prior treatment and depth of response on survival in MM-003, a randomized phase 3 study comparing pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone in relapsed/refractory multiple myeloma. Haematologica, 2015, 100, 1334-1339.
[78]
Krämer, I.; Engelhardt, M.; Fichtner, S.; Neuber, B.; Medenhoff, S.; Bertsch, U.; Hillengass, J.; Raab, M.S.; Hose, D.; Ho, A.D.; Goldschmidt, H.; Hundemer, M. Lenalidomide enhances myeloma-specific T-cell responses in vivo and in vitro. OncoImmunology, 2016, 5, e1139662.
[79]
Neuber, B.; Dai, J.; Waraich, W.A.; Neuber, B.; Dai, J.; Waraich, W.A. Lenalidomide overcomes the immunosuppression of regulátory CD8+CD28- T-cells. Oncotarget, 2017, 8, 98200-98214.
[80]
Giuliani, M.; Janji, B.; Berchem, G. Activation of NK cells and disruption of PD-L1/PD-1 axis: two different ways for lenalidomide to block myeloma progression. Oncotarget, 2017, 8, 24031-24044.
[81]
Ito, T.; Ando, H.; Suzuki, T.; Ogura, T.; Hotta, K.; Imamura, Y.; Yamaguchi, Y.; Handa, H. Identification of a primary target of thalidomide teratogenicity. Science, 2010, 327, 1345-1350.
[82]
Lopez-Girona, A.; Mendy, D.; Ito, T.; Miller, K.; Gandhi, A.K.; Kang, J.; Karasawa, S.; Carmel, G.; Jackson, P.; Abbasian, M.; Mahmoudi, A.; Cathers, B.; Rychak, E.; Gaidarova, S.; Chen, R.; Schafer, P.H.; Handa, H.; Daniel, T.O.; Evans, J.F.; Chopra, R. Cereblon is a direct protein target for immunomodulatory and antiproliferative activities of lenalidomide and pomalidomide. Leukemia, 2012, 26, 2326-2335.
[83]
Chang, X.B.; Stewart, A.K. What is the functional role of the thalidomide binding protein cereblon? Int. J. Biochem. Mol. Biol., 2011, 2, 287-294.
[84]
Fischer, E.S.; Böhm, K.; Lydeard, J.R.; Yang, H.; Stadler, M.B.; Cavadini, S.; Nagel, J.; Serluca, F.; Acker, V.; Lingaraju, G.M.; Tichkule, R.B.; Schebesta, M.; Forrester, W.C.; Schirle, M.; Hassiepen, U.; Ottl, J.; Hild, M.; Beckwith, R.E.; Harper, J.W.; Jenkins, J.L.; Thomä, N.H. Structure of the DDB1-CRBN E3 ubiquitin ligase in complex with thalidomide. Nature, 2014, 512, 49-53.
[85]
Chamberlain, P.P.; Lopez-Girona, A.; Miller, K.; Carmel, G.; Pagarigan, B.; Chie-Leon, B.; Rychak, E.; Corral, L.G.; Ren, Y.J.; Wang, M.; Riley, M.; Delker, S.L.; Ito, T.; Ando, H.; Mori, T.; Hirano, Y.; Handa, H.; Hakoshima, T.; Daniel, T.O.; Cathers, B.E. Stucture of the human cereblon-DDB1-lenalidomide complex reveals basis for responsiveness to thalidomide analogs. Nat. Struct. Mol. Biol., 2014, 21, 803-809.
[86]
Lupas, A.N.; Zhu, H.; Korycinski, M. The thalidomide-binding domain of cereblon defines the CULT domain family and is a new member of the β-tent fold. PLOS Comput. Biol., 2015, 11, e1004023.
[87]
Hartmann, M.D.; Boichenko, I.; Coles, M.; Lupas, A.N.; Hernandez Alvarez, B. Structural dynamics of cereblon ligand binding domain.PLOS ONE, 2015, 10, e 0128342,
[88]
Hershko, A.; Ciechanover, A. The ubiquitin system. Annu. Rev. Biochem., 1998, 67, 425-479.
[89]
Pickart, C.M. Mechanisms underlying ubiquitination. Annu. Rev. Biochem., 2001, 70, 503-533.
[90]
Glickman, M.H.; Ciechanover, A. The ubiquitin-proteasome proteolytic pathway: destruction for the sake of construction. Physiol. Rev., 2002, 82, 373-428.
[91]
Pickart, C.M.; Cohen, R.E. Proteasomes and their kin: proteases in the machine age. Nat. Rev. Mol. Cell Biol., 2004, 5, 177-187.
[92]
Golab, J.; Bauer, T.M.; Daniel, V.; Naujokat, C. Role of the ubiquitin-proteasome pathway in the diagnosis of human diseases. Clin. Chim. Acta, 2004, 340, 27-40.
[93]
Hershko, A. Review: Nobel Lecture. The ubiquitin system for protein degradation and some of its roles in the control of the cell division cycle. Cell Death Differ., 2005, 12, 1191-1197.
[94]
Rose, I. Review: Nobel Lecture. Ubiquitin at Fox Chase. Cell Death Differ., 2005, 12, 1162-1166.
[95]
Ciechanover, A. Intracellular protein degradation from a vague idea through the lysosome and the ubiquitin-proteasome system and on to human diseases and drug targeting: Nobel Lecture, December 8, 2004. Ann. N. Y. Acad. Sci., 2007, 1116, 1-28.
[96]
Ciechanover, A. Tracing the history of the ubiquitin proteolytic system: The pioneering article. Biochem. Biophys. Res. Commun., 2009, 387, 1-10.
[97]
Orlowski, R.Z. The role of the ubiquitin-proteasome pathway in apoptosis. Cell Death Differ., 1999, 6, 303-313.
[98]
Wójcik, C. Regulation of apoptosis by the ubiquitin and proteasome pathway. J. Cell. Mol. Med., 2002, 6, 25-48.
[99]
Kinyamu, H.K.; Chen, J.; Archer, T.K. Linking the ubiquitin-proteasome pathway to chromatin remodeling/modification by nuclear receptors. J. Mol. Endocrinol., 2005, 34, 281-297.
[100]
Sun, Y. E3 ubiquitin ligases as cancer targets and biomarkers. Neoplasia, 2006, 8, 645-654.
[101]
Nalepa, G.; Rolfe, M.; Wade Harper, J. Drug discovery in the ubiquitin-proteasome system. Nat. Rev. Drug Discov., 2006, 5, 596-613.
[102]
Kitagawa, K.; Kotake, Y.; Kitagawa, M. Ubiquitin-mediated control of oncogene and tumor suppressor gene products. Cancer Sci., 2009, 100, 1374-1381.
[103]
Bassermann, T.; Eichner, R.; Pagano, M. The ubiquitin proteasome system-Implications for cell cycle control and the targeted treatment of cancer. Biochim. Biophys. Acta, 2014, 1843, 150-162.
[104]
Metzger, M.B.; Pruneda, J.N.; Klevit, R.E.; Weissman, A.M. RING-type E3 ligases: Master manipulators of E2 ubiquitin-conjugating enzymes and ubiquitination. Biochim. Biophys. Acta, 2014, 1843, 47-60.
[105]
Ciechanover, A.; Stanhill, A. The complexity of recognition of ubiquitinated substrates by 26 S proteasome. Biochim. Biophys. Acta, 2014, 1843, 86-96.
[106]
Krӧnke, J.; Udeshi, N.D.; Narla, A.; Grauman, P.; Hurst, S.N.; McConkey, M.; Svinkina, T.; Heckl, D.; Comer, E.; Li, X.; Ciarlo, C.; Hartman, E.; Munshi, N.; Schenone, M.; Schreiber, S.L.; Carr, S.A.; Ebert, B.L. Lenalidomide causes selective degradation of IKZF1 and IKZF3 in multiple myeloma cells. Science, 2014, 343, 301-305.
[107]
Lu, G.; Middleton, R.E.; Sun, H.; Naniong, M.; Ott, C.J.; Mitsiades, C.S.; Wong, K.K.; Bradner, J.E.; Kaelin, W.G., Jr The myeloma drug lenalidomide promotes the cereblon-dependent destruction of Ikaros proteins. Science, 2014, 343, 305-309.
[108]
Gandhi, A.K.; Kang, J.; Havens, C.G.; Conklin, T.; Ning, Y.; Wu, L.; Ito, T.; Ando, H.; Waldman, M.F.; Thakurta, A.; Klippel, A.; Handa, H.; Daniel, T.O.; Schafer, P.H.; Chopra, R. Immunomodulatory agents lenalidomide and pomalidomide co-stimulate T cells by inducing degradation of T cell repressors Ikaros and Aiolos via modulation of the E3 ubiquitin ligase complex CRL4CRBN. Br. J. Haematol., 2014, 164, 811-821.
[109]
Stewart, A.K. How thalidomide works against cancer. Science, 2014, 343, 256-257.
[110]
Zhu, Y.X.; Braggio, E.; Shi, C.X.; Kortuem, K.M.; Bruins, L.A.; Schmidt, J.E.; Chang, X.B.; Langlais, P.; Luo, M.; Jedlowski, P.; LaPlant, B.; Laumann, K.; Fonseca, R.; Bergsagel, P.L.; Mikhael, J.; Lacy, M.; Champion, M.D.; Stewart, A.K. Identification of cereblon-binding proteins and relationship with response and survival after IMiDs in multiple myeloma. Blood, 2014, 124, 536-545.
[111]
Semeraro, M.; Galluzzi, L. Novel insights into the mechanism of action of lenalidomide.Oncoimmunology, 2014, 3, e 28386,
[112]
Keevan, J.; Figg, W.D. New mechanism of lenalidomide activity. Cancer Biol. Ther., 2014, 15, 968-969.
[113]
Shaffer, A.L.; Tolga Emre, N.C.; Lamy, L.; Ngo, V.N.; Wright, G.; Xiao, W.; Powell, J.; Dave, S.; Yu, X.; Zhao, H.; Zeng, Y.; Chen, B.; Epstein, J.; Staudt, L.M. IRF4 addiction in multiple myeloma. Nature, 2008, 454, 226-231.
[114]
Shaffer, A.L.; Tolga Emre, N.C.; Romesser, P.B.; Staudt, L.M. IRF4: Immunity. Malignancy! Therapy? Clin. Cancer Res., 2009, 15, 2954-2961.
[115]
Lopez-Girona, A.; Heintel, D.; Zhang, L.H.; Mendy, D.; Gaidarova, S.; Brady, H.; Bartlett, J.B.; Schafer, P.H.; Schreder, M.; Bolomsky, A.; Hilgarth, B.; Zojer, N.; Gisslinger, H.; Ludwig, H.; Daniel, T.; Jäger, U.; Chopra, R. Lenalidomide downregulates the cell survival factor, interferon regulatory factor-4, providing a potential mechanistic link for predicting response. Br. J. Haematol., 2011, 154, 325-336.
[116]
Krönke, J.; Fink, E.C.; Hollenbach, P.W.; MacBeth, K.J.; Hurst, S.N.; Udeshi, N.D.; Chamberlain, P.P.; Mani, D.R.; Man, H.W.; Gandhi, A.K.; Svinkina, T.; Schneider, R.K.; McConkey, M.; Järås, M.; Griffiths, E.; Wetzler, M.; Bullinger, L.; Cathers, B.E.; Carr, S.A.; Chopra, R.; Ebert, B.L. Lenalidomide induces ubiquitination and degradation of CK1α in del(5q) MDS. Nature, 2015, 523, 183-188.
[117]
Bjorklund, C.C.; Lu, L.; Kang, J.; Hagner, P.R.; Havens, C.G.; Amatangelo, M.; Wang, M.; Ren, Y.; Couto, S.; Breider, M.; Ning, Y.; Gandhi, A.K.; Daniel, T.O.; Chopra, R.; Klippel, A.; Thakurta, A.G. Rate of CRL4 (CRBN) substrate Ikaros and Aiolos degradation underlies differential activity of lenalidomide and pomalidomide in MM cells by regulation of c-Myc and IRF4. Blood Cancer J., 2015, 5, e 354.
[118]
Petroski, M.D.; Deshales, R.J. Function and regulation of cullin-RING ubiquitin ligases. Nat. Rev. Mol. Cell Biol., 2005, 6, 9-20.
[119]
Jin, J.; Arias, E.E.; Chen, J.; Wade Harper, J.; Walter, J.C. A family of diverse Cul4-Ddb1-interacting proteins includes Cdt2, which is required for S phase destruction of the replication factor Cdt1. Mol. Cell, 2006, 23, 709-721.
[120]
Li, T.; Chen, X.; Garbutt, K.C.; Zhou, P.; Zheng, N. Structure of DDB1 in complex with a ParamyxovirusV protein: viral hijack of a propeller cluster in ubiquitin ligase. Cell, 2006, 124, 105-117.
[121]
Angers, S.; Li, T.; Yi, X.; MacCoss, M.J.; Moon, R.T.; Zheng, N. Molecular architecture and assembly of the DDB1-CUL4A ubiquitin ligase machinery. Nature, 2006, 443, 590-593.
[122]
Lee, J.; Zhou, P. DCAFs, the missing link of the CUL4-DDB1 ubiquitin ligase. Mol. Cell, 2007, 26, 775-780.
[123]
Catic, A. Culling for survival. Blood, 2008, 112, 211-212.
[124]
Waning, D.L.; Li, B.; Jia, N.; Naaldijk, Y.; Goebel, W.S. HogenEsch, H.; Chun, K.T. Cul 4A is required for hematopoietic cell viability and its deficiency leads to apoptosis. Blood, 2008, 112, 320-329.
[125]
Jackson, S.; Xiong, Y. CRL4s: The CUL4-RING E3 ubiquitin ligases. Trends Biochem. Sci., 2009, 34, 562-570.
[126]
Sugasawa, K. The CUL4 enigma: Culling DNA repair factors. Mol. Cell, 2009, 34, 403-404.
[127]
Lee, J.; Zhou, P. Pathogenic role of the CRL4 ubiquitin ligase in human disease. Front. Oncol., 2012, 2, 1-7.
[128]
Zhao, Y.; Sun, Y. Cullin-RING ligases as attractive anti-cancer targets. Curr. Pharm. Des., 2013, 19, 3215-3225.
[129]
Pan, Z.Q.; Kentsis, A.; Dias, D.C.; Yamoah, K.; Wu, K. Nedd8 on cullin: building an express way to protein destruction. Oncogene, 2004, 23, 1985-1997.
[130]
Merlet, J.; Burger, J.; Gomes, J.E.; Pintard, L. Regulation of cullin-RING E3 ubiquitin-ligases by neddylation and dimerization. Cell. Mol. Life Sci., 2009, 66, 1924-1938.
[131]
Lydeard, J.R.; Schulman, B.A.; Harper, J.W. Building and remodelling cullin-ring E3 ubiquitin ligases. EMBO Rep., 2013, 14, 1050-1061.
[132]
Kortűm, K.M.; Zhu, Y.X.; Shi, C.X.; Jedlowski, P.; Stewart, A.K. Cereblon binding molecules in multiple myeloma. Blood Rev., 2015, 29, 329-334.
[133]
Xu, Q.; Hou, Y.X.; Langlais, P.; Erickson, P.; Zhu, J.; Shi, C.X.; Luo, M.; Zhu, Y.; Xu, Y.; Mandarino, L.J.; Stewart, K.; Chang, X.B. Expression of the cereblon binding protein argonaute 2 plays an important role for multiple myeloma cell growth and survival. BMC Cancer, 2016, 16, 297.
[134]
He, Y.J.; McCall, C.M.; Hu, J.; Zeng, Y.; Xiong, Y. DDB1 functions as a linker to recruit receptor WD40 proteins to CUL4-ROC1 ubiquitin ligases. Genes Dev., 2006, 20, 2949-2954.
[135]
Iovine, B.; Iannella, M.L.; Bevilacqua, M.A. Damage-specific DNA binding protein 1 (DDB1): a protein with a wide range of functions. Int. J. Biochem. Cell Biol., 2011, 43, 1664-1667.
[136]
Komatsu, M.; Kageyama, S.; Ichimura, Y. p62/SQSTM1/A170: physiology and pathology. Pharmacol. Res., 2012, 66, 457-462.
[137]
Fuchs, O.; Bokorova, R.; Vostry, M.; Kostečka, A.; Polak, J. Cereblon and its role in the treatment of multiple myeloma by lenalidomide and pomalidomide. Int. J. Hematol. Dis, 2014, 1, 13-20.
[138]
Zhu, Y.X.; Braggio, E.; Shi, C.X.; Bruins, L.A.; Schmidt, J.E.; Van Wier, S.; Chang, X.B.; Bjorklund, C.C.; Fonseca, R.; Bergsagel, P.L.; Orlowski, R.Z.; Stewart, A.K. Cereblon expression is required for the antimyeloma activity of lenalidomide and pomalidomide. Blood, 2011, 118, 4771-4779.
[139]
Broyl, A.; Kuiper, R.; van Duin, M.; van der Holt, B.; el Jarari, L.; Bertsch, U.; Zweegman, S.; Buijs, A.; Hose, D.; Lokhorst, H.M.; Goldschmidt, H.; Sonneveld, P. Dutch-Belgian HOVON group;German GMMG Group. High cereblon expression is associated with better survival in patients with newly diagnosed multiple myeloma treated with thalidomide maintenance. Blood, 2013, 121, 624-627.
[140]
Heintel, D.; Rocci, A.; Ludwig, H.; Bolomsky, A.; Caltagirone, S.; Schreder, M.; Pfeifer, S.; Gisslinger, H.; Zojer, N.; Jäger, U.; Palumbo, A. High expression of cereblon (CRBN) is associated with improved clinical response in patients with multiple myeloma treated with lenalidomide and dexamethasone. Br. J. Haematol., 2013, 161, 695-700.
[141]
Lodé, L.; Amiot, M.; Maiga, S.; Touzeau, C.; Menard, A.; Magrangeas, F.; Minvielle, S.; Pellat-Deceunynck, C.; Bene, M.C.; Moreau, P. Cereblon expression in multiple myeloma: not ready for prime time. Br. J. Haematol., 2013, 163, 282-284.
[142]
Gandhi, A.K.; Mendy, D.; Waldman, M.; Chen, G.; Rychak, E.; Miller, K.; Gaidarova, S.; Ren, Y.; Wang, M.; Breider, M.; Carmel, G.; Mahmoudi, A.; Jackson, P.; Abbasian, M.; Cathers, B.E.; Schafer, P.H.; Daniel, T.O.; Lopez-Girona, A.; Thakurta, A.; Chopra, R. Measuring cereblon as a biomarker of response or resistance to lenalidomide and pomalidomide requires use of standardized reagents and understanding of gene complexity. Br. J. Haematol., 2014, 164, 233-244.
[143]
Pearse, R.N. IMiDs: Not for the CRBN weak. Leuk. Res., 2014, 38, 21-22.
[144]
Schuster, S.R.; Kortuem, K.M.; Zhu, Y.X.; Braggio, E.; Shi, C.X.; Bruins, L.A.; Schmidt, J.E.; Ahmann, G.; Kumar, S.; Rajkumar, S.V.; Mikhael, J.; Laplant, B.; Champion, M.D.; Laumann, K.; Barlogie, B.; Fonseca, R.; Bergsagel, P.L.; Lacy, M.; Stewart, A.K. The clinical significance of cereblon expression in multiple myeloma. Leuk. Res., 2014, 38, 23-28.
[145]
Zhu, Y.X.; Kortuem, K.M.; Stewart, A.K. Molecular mechanism of action of immune-modulatory drugs thalidomide, lenalidomide and pomalidomide in multiple myeloma. Leuk. Lymphoma, 2013, 54, 683-687.
[146]
Jonasova, A.; Bokorova, R.; Polak, J.; Vostry, M.; Kostecka, A.; Hajkova, H.; Neuwirtova, R.; Siskova, M.; Sponerova, D.; Cermak, J.; Mikulenkova, D.; Cervinek, L.; Brezinova, J.; Michalova, K.; Fuchs, O. High level of full-length cereblon mRNA in lower risk myelodysplastic syndrome with isolated 5q deletion is implicated in the efficacy of lenalidomide. Eur. J. Haematol., 2015, 95, 27-34.
[147]
Fuchs, O.; Polak, J.; Bokorova, R.; Kostecka, A.; Vostry, M.; Neuwirtova, R.; Siskova, M.; Stopka, T.; Lauermannova, M.; Soukupova-Maaloufova, J.; Salek, C.; Mikulenkova, D.; Cermak, J.; Brezinova, J.; Zemanova, Z.; Michalova, K.; Jonasova, A. High level of full-length cereblon messenger RNA and protein is important for lenalidomide efficacy in lower risk MDS patients.Abstract
227 from the 14th International Symposium on Myelodysplastic
Syndromes. Leuk. Res., 2017, 55(Suppl 1), S 132.
[148]
Lee, K.J.; Lee, K.M.; Jo, S.; Kang, K.W.; Park, C.S. Induction of cereblon by NF-E2-related factor 2 in neuroblastoma cells exposed to hypoxia-reoxygenation. Biochem. Biophys. Res. Commun., 2010, 399, 711-715.
[149]
Lionetti, M.; Agnelli, L.; Lombardi, L.; Tassone, P.; Neri, A. MicroRNAs in the pathobiology of multiple myeloma. Curr. Cancer Drug Targets, 2012, 12, 823-837.
[150]
Wu, P.; Agnelli, L.; Walker, B.A.; Todoerti, K.; Lionetti, M.; Johnson, D.C.; Kaiser, M.; Mirabella, F.; Wardell, C.; Gregory, W.M.; Davies, F.E.; Brewer, D.; Neri, A.; Morgan, G.J. Improved risk stratification in myeloma using a microRNA-based classifier. Br. J. Haematol., 2013, 162, 348-359.
[151]
Bi, C.; Chng, W.J. MicroRNA: important player in the pathobiology of multiple myeloma. Biomed. Res. Inst, 2014, 2014, 521586.
[152]
Greenberg, A.J.; Walters, D.K.; Kumar, S.K.; Rajkumar, S.V.; Jelinek, D.F. Responsiveness of cytogenetically discrete human myeloma cell lines to lenalidomide: lack of correlation with cereblon and interferon regulatory factor 4 expression levels. Eur. J. Haematol., 2013, 91, 504-513.
[153]
Huang, S.Y.; Lin, C.W.; Lin, H.H.; Yao, M.; Tang, J.L.; Wu, S.J.; Chen, Y.C.; Lu, H.Y.; Hou, H.A.; Chen, C.Y.; Chou, W.C.; Tsay, W.; Chou, S.J.; Tien, H.F. Expression of cereblon protein assessed by immunohistochemical staining in myeloma cells is associated with superior response of thalidomide- and lenalidomide-based treatment, but not bortezomib-based treatment, in patients with multiple myeloma. Ann. Hematol., 2014, 93, 1371-1380.
[154]
Chang, X.; Xu, Q.; Hou, Y.; Li, C.; Xu, Y.; Stewart, A.K. Mouse monoclonal antibodies generated from full length human cereblon: Detection of cereblon protein in patients with multiple myeloma. Int. J. Mol. Sci., 2017, 18, E1999.
[155]
Eichner, R.; Heider, M.; Fernández-Sáiz, V.; van Bebber, F.; Garz, A.K.; Lemeer, S.; Rudelius, M.; Targosz, B.S.; Jacobs, L.; Knorn, A.M.; Slawska, J.; Platzbecker, U.; Germing, U.; Langer, C.; Knop, S.; Einsele, H.; Peschel, C.; Haass, C.; Keller, U.; Schmid, B.; Götze, K.S.; Kuster, B.; Bassermann, F. Immunomodulatory drugs disrupt the cereblon-CD147-MCT1 axis to exert antitumor activity and teratogenicity. Nat. Med., 2016, 22, 735-743.
[156]
Krönke, J.; Kuchenbauer, F.; Kull, M.; Teleanu, V.; Bullinger, L.; Bunjes, D.; Greiner, A.; Kolmus, S.; Köpff, S.; Schreder, M.; Mügge, L.O.; Straka, C.; Engelhardt, M.; Döhner, H.; Einsele, H.; Bassermann, F.; Bargou, R.; Knop, S.; Langer, C. IKZF1 expression is a prognostic marker in newly diagnosed standard-risk multiple myeloma treated with lenalidomide and intensive chemotherapy: a study of the German Myeloma Study Group (DSMM). Leukemia, 2017, 31, 1363-1367.
[157]
Krönke, J.; Knop, S.; Langer, C. Prognostic impact of Ikaros expression in lenalidomide-treated multiple myeloma. Oncotarget, 2017, 8, 106163-106164.
[158]
Catley, L.; Weisberg, E.; Tai, Y.T.; Atadja, P.; Remiszewski, S.; Hideshima, T.; Mitsiades, N.; Shringarpure, R.; LeBlanc, R.; Chauhan, D.; Munshi, N.C.; Schlossman, R.; Richardson, P.; Griffin, J.; Anderson, K.C. NVP-LAQ824 is a potent novel histone deacetylase inhibitor with significant activity against multiple myeloma. Blood, 2003, 102, 2615-2622.
[159]
Mitsiades, C.S.; Mitsiades, N.S.; McMullan, C.J.; Poulaki, V.; Shringarpure, R.; Hideshima, T.; Akiyama, M.; Chauhan, D.; Munshi, N.; Gu, X.; Bailey, C.; Joseph, M.; Libermann, T.A.; Richon, V.M.; Marks, P.A.; Anderson, K.C. Transcriptional signature of histone deacetylase inhibition in multiple myeloma: biological and clinical implications. Proc. Natl. Acad. Sci. USA, 2004, 101, 540-545.
[160]
Catley, L.; Weisberg, M.E.; Kiziltepe, T.; Tai, Y.T.; Hideshima, T.; Neri, P.; Tassone, P.; Atadja, P.; Chauhan, D.; Munshi, N.C.; Anderson, K.C. Aggresome induction by proteasome inhibitor bortezomib and alpha-tubulin hyperacetylation by tubulin deacetylase (TDAC) inhibitor LBH589 are synergistic in myeloma cells. Blood, 2006, 108, 3441-3449.
[161]
Richardson, P.G.; Harvey, R.D.; Laubach, J.P.; Moreau, P.; Lonial, S.; San-Miguel, J.F. Panobinostat for the treatment of relapsed or relapsed/refractory multiple myeloma: Pharmacology and clinical outcomes. Expert Rev. Clin. Pharmacol., 2016, 91, 35-48.
[162]
Sivaraj, D.; Green, M.M.; Gasparetto, C. Panobinostat for the management of multiple myeloma. Future Oncol., 2017, 13, 477-488.
[163]
Laubach, J.P.; San-Miguel, J.F.; Hungria, V.; Hou, J.; Moreau, P.; Lonial, S.; Lee, J.H.; Einsele, H.; Alsina, M.; Richardson, P.G. Deacetylase inhibitors: an advance in myeloma therapy? Expert Rev. Hematol., 2017, 10, 229-237.
[164]
Hideshima, T.; Cottini, F.; Ohguchi, H.; Jakubikova, J.; Gorgun, G.; Mimura, N.; Tai, Y.T.; Munshi, N.C.; Richardson, P.G.; Anderson, C.K. Rational combination treatment with histone deacetylase inhibitors and immunomodulatory drugs in multiple myeloma. Blood Cancer J., 2015, 5, e312.
[165]
Li, S.; Pal, R.; Monaghan, S.A.; Schafer, P.; Ouyang, H.; Mapara, M.; Galson, D.L.; Lentzsch, S. IMiD immunomodulatory compounds block C/EBPβ translation through eIF4E down-regulation resulting in inhibition of MM. Blood, 2011, 117, 5157-5165.
[166]
Chesi, M.; Robbiani, D.F.; Sebag, M.; Chng, W.J.; Affer, M.; Tiedemann, R.; Valdez, R.; Palmer, S.E.; Haas, S.S.; Stewart, A.K.; Fonseca, R.; Kremer, R.; Cattoretti, G.; Bergsagel, P.L. AID-dependent activation of a MYC transgene induces multiple myeloma in a conditional mouse model of post-germinal center malignancies. Cancer Cell, 2008, 13, 167-180.
[167]
Chesi, M.; Matthews, G.M.; Garbitt, V.M.; Palmer, S.E.; Shortt, J.; Lefebure, M.; Stewart, A.K.; Johnstone, R.W.; Bergsagel, P.L. Drug response in a genetically engineered mouse model of multiple myeloma is predictive of clinical efficacy. Blood, 2012, 120, 376-385.
[168]
Affer, M.; Chesi, M.; Chen, W.D.; Keats, J.J.; Demchenko, Y.N.; Roschke, A.V.; Van Wier, S.; Fonseca, R.; Bergsagel, P.L.; Kuehl, W.M. Promiscuous MYC locus rearrangements hijack enhancers but mostly super-enhancers to dysregulate MYC expression in multiple myeloma. Leukemia, 2014, 28, 1725-1735.
[169]
Walker, B.A.; Wardell, C.P.; Brioli, A.; Boyle, E.; Kaiser, M.F.; Begum, D.B.; Dahir, N.B.; Johnson, D.C.; Ross, F.M.; Davies, F.E.; Morgan, G.J. Translocations at 8q24 juxtapose MYC with genes that harbor superenhancers resulting in overexpression and poor prognosis in myeloma patients. Blood Cancer J., 2014, 4, e191.
[170]
Gopalakrishnan, R.; Matta, H.; Tolani, B.; Triche, T., Jr; Chaudhary, P.M. Immunomodulatory drugs target IKZF1-IRF4-MYC axis in primary effusion lymphoma in a cereblon-dependent manner and display synergistic cytotoxicity with BRD4 inhibitors. Oncogene, 2016, 35, 1797-1810.
[171]
Ramsay, A.G.; Johnson, A.J.; Lee, A.M.; Gorgün, G.; Le Dieu, R.; Blum, W.; Byrd, J.C.; Gribben, J.G. Chronic lymphocytic leukemia T cells show impaired immunological synapse formation that can be reversed with an immunomodulating drug. J. Clin. Invest., 2008, 118, 2427-2437.
[172]
Idler, I.; Giannopoulos, K.; Zenz, T.; Bhattacharya, N.; Nothing, M.; Döhner, H.; Stilgenbauer, S.; Mertens, D. Lenalidomide treatment of chronic lymphocytic leukaemia patients reduces regulatory T cells and induces Th17 T helper cells. Br. J. Haematol., 2010, 148, 940-950.
[173]
Skórka, K.; Bhattacharya, N.; Wlasiuk, P.; Kowal, M.; Mertens, D.; Dmoszyńska, A.; Giannopoulos, K. Thalidomide regulation of NF-κB proteins limits Tregs activity in chronic lymphocytic leukemia. Adv. Clin. Exp. Med., 2014, 23, 25-32.
[174]
Lee, B.N.; Gao, H.; Cohen, E.N.; Badoux, X.; Wierda, W.G.; Estrov, Z.; Faderl, S.H.; Keating, M.J.; Ferrajoli, A.; Reuben, J.M. Treatment with lenalidomide modulates T-cell immunophenotype and cytokine poduction in patients with chronic lymphocytic leukemia. Cancer, 2011, 117, 3999-4008.
[175]
Kasyapa, C.S.; Sher, T.; Chanan-Khan, A.A. Multiple myeloma and immunomodulation: Regulating the regulatory cells. Leuk. Lymphoma, 2012, 53, 1253-1254.
[176]
Chanan-Khan, A.; Miller, K.C.; Musial, L.; Lawrence, D.; Padmanabhan, S.; Takeshita, K.; Porter, C.W.; Goodrich, D.W.; Bernstein, Z.P.; Wallace, P.; Spaner, D.; Mohr, A.; Byrne, C.; Hernandez-Ilizaliturri, F.; Chrystal, C.; Starostik, P.; Czuczman, M.S. Clinical efficiacy of lenalidomide in patients with relapsed or refractory chronic lymphocytic leukemia: Results of a phase II study. J. Clin. Oncol., 2006, 24, 5343-5349.
[177]
Ferrajoli, A.; Lee, B.N.; Schlette, E.J.; O’Brien, S.M.; Gao, H.; Wen, S.; Wierda, W.G.; Estrov, Z.; Faderl, S.; Cohen, E.N.; Li, C.; Reuben, J.M.; Keating, M.J. Lenalidomide induces complete and partial remissions in patients with relapsed and refractory chronic lymphocytic leukemia. Blood, 2008, 111, 5291-5297.
[178]
Wendtner, C.M.; Hillmen, P.; Mahadevan, D.; Bühler, A.; Uharek, L.; Coutré, S.; Frankfurt, O.; Bloor, A.; Bosch, F.; Furman, R.R.; Kimby, E.; Gribben, J.G.; Gobbi, M.; Dreisbach, L.; Hurd, D.D.; Sekeres, M.A.; Ferrajoli, A.; Shah, S.; Zhang, J.; Moutouh-de Parseval, L.; Hallek, M.; Heerema, N.A.; Stilgenbauer, S.; Chanan-Khan, A.A. Final results of a multicenter phase I study of lenalidomide in patients with relapsed or refractory chronic lymhocytic leukemia. Leuk. Lymphoma, 2012, 53, 417-423.
[179]
Wendtner, C.; Hallek, M.; Fraser, G.; Michallet, A.S.; Hillmen, P.; Dürig, J.; Kalaycio, M.; Gribben, J.G.; Stilgenbauer, S.; Buhler, A.; Kipps, T.J.; Purse, B.; Zhang, J.; De Bedout, S.; Mei, J.; Chanan-Khan, A. Safety and efficacy of different lenalidomide starting doses in patients with relapsed or refractory chronic lymphocytic leukemia: results of an international multicenter double-blinded randomized phase II trial. Leuk. Lymphoma, 2016, 57, 1291-1299.
[180]
Batoo, S.A.; Hernandez-Ilizaliturri, F. The emerging role of lenalidomide in the management of lymphoid malignancies. Ther. Adv. Hematol., 2011, 2, 45-53.
[181]
Blumel, S.; Broadway-Duren, J. Approaches to managing safety with lenalidomide in hematologic malignancies. Adv. Practitioner, 2014, 5, 269-279.
[182]
Cortelezzi, A.; Sciumé, M.; Reda, G. Lenalidomide in the treatment of chronic lymphocytic leukemia. Adv. Hematol., 2012, Article ID 393864.
[183]
James, D.F.; Werner, L.; Brown, J.R.; Wierda, W.G.; Barrientos, J.C.; Castro, J.E.; Greaves, A.; Johnson, A.J.; Rassenti, L.Z.; Rai, K.R.; Neuberg, D.; Kipps, T.J. Lenalidomide and rituximab for the initial treatment of patients with chronic lymphocytic leukemia: a multicentre clinical-translational study from the chronic lymphocytic leukemia research consortium. J. Clin. Oncol., 2014, 32, 2067-2073.
[184]
Mato, A.R.; Foon, K.A.; Feldman, T.; Schuster, S.J.; Svoboda, J.; Chow, K.F.; Valentinetti, M.; Mrkulic, M.; Azzollini, K.; Gadaleta, G.; Bhattacharyya, P.K.; Zenreich, J.; Pascual, L.N.; Yannotti, K.; Kdiry, S.; Howlett, C.; Strelec, L.; Porter, D.; Bejot, C.; Goy, A. Reduced-dose fludarabine, cyclophosphamide, and rituximab (FCR-Lite) plus lenalidomide, followed by lenalidomide consolidation/maintenance, in previously untreated chronic lymphocytic leukemia. Am. J. Hematol., 2015, 90, 487-492.
[185]
Fecteau, J.F.; Corral, L.G.; Ghia, E.M.; Gaidarova, S.; Futalan, D.; Bharati, I.S.; Cathers, B.; Schwaederlé, M.; Cui, B.; Lopez-Girona, A.; Messmer, D.; Kipps, T.J. Lenalidomide inhibits the proliferation of CLL cells via a cereblon/p21(WAF1, CIP1)-dependent mechanism independent of functional p53. Blood, 2014, 124, 1637-1644.
[186]
Jamroziak, K.; Szemraj, J.; Robak, T.; Tukiendorf, A.; Giannopoulos, K. Cereblon expression predicts clinical response in chronic lymphocytic leukemia treated with a thalidomide/ fludarabine regimen. Leuk. Lymphoma, 2015, 56, 808-810.
[187]
Schulz, A.; Durr, C.; Zenz, T.; Döhner, H.; Stilgenbauer, S.; Lichter, P.; Seiffert, M. Lenalidomide reduces survival of chronic lymphocytic leukemia cells in primary cocultures by altering the myeloid microenvironment. Blood, 2013, 121, 2503-2511.
[188]
Fiorcari, S.; Martinelli, S.; Bulgarelli, J.; Audrito, V.; Zucchini, P.; Colaci, E.; Potenza, L.; Narni, F.; Luppi, M.; Deaglio, S.; Marasca, R.; Maffei, R. Lenalidomide interferes with tumor-promoting properties of nurse-like cells in chronic lymphocytic leukemia. Haematologica, 2015, 100, 253-262.
[189]
Shaim, H.; Estrov, Z.; Harris, F.; Hernandez Sanabria, M.; Liu, Z.; Ruvolo, P.; Thompson, P.A.; Ferrajoli, A.; Daher, M.; Burger, J.; Muftuoglu, M.; Imahashi, N.; Li, L.; Liu, E.; Alsuliman, A.S.; Basar, R.; Nassif Kerbauy, L.; Sobieski, C.; Gokdemir, E.; Kondo, K.; Wierda, W.; Keating, M.; Shpall, E.J.; Rezvani, K. The CXCR4-STAT3-IL-10 pathway controls the immunoregulatory function of chronic lymphocytic leukemia and is modulated by lenalidomide. Front. Immunol., 2018, 8, 1773.
[190]
Maffei, R.; Fiorcari, S.; Martinelli, S.; Benatti, S.; Bulgarelli, J.; Rizzotto, L.; Debbia, G.; Santachiara, R.; Rigolin, G.M.; Forconi, F.; Rossi, D.; Laurenti, L.; Palumbo, G.A.; Vallisa, D.; Cuneo, A.; Gaidano, G.; Luppi, M.; Marasca, R. Increased SHISA3 expression characterizes chronic lymphocytic leukemia patients sensitive to lenalidomide. Leuk. Lymphoma, 2018, 59, 423-433.
[191]
Takahashi, K.; Hu, B.; Wang, F.; Yan, Y.; Kim, E.; Vitale, C.; Patel, K.P.; Strati, P.; Gumbs, C.; Little, L.; Tippen, S.; Song, X.; Zhang, J.; Jain, N.; Thompson, P.; Garcia-Manero, G.; Kantarjian, H.; Estrov, Z.; Do, K.A.; Keating, M.; Burger, J.A.; Ferrajoli, A.; Futreal, P.A.; Wierda, W.G. Clinical implications of cancer gene mutations in patients with chronic lymphocytic leukemia treated with lenalidomide. Blood, 2018, 131, 1820-1832.
[192]
González-Rodríguez, A.P.; Payer, A.R.; Acebes-Huerta, A.; Huergo-Zapico, L.; Villa-Alvarez, M.; Gonzalez-García, E.; Gonzalez, S. Lenalidomide and chronic lymphocytic leukemia. BioMed Res. Int., 2013, 2013, 932010.
[193]
Maffei, R.; Colaci, E.; Fiorcari, S.; Martinelli, S.; Potenza, L.; Luppi, M.; Marasca, R. Lenalidomide in chronic lymphocytic leukemia: the present and future in the era of tyrosine kinase inhibitors. Crit. Rev. Oncol. Hematol., 2016, 97, 291-302.
[194]
Itchaki, G.; Brown, J.R. Lenalidomide in the treatment of chronic lymphocytic leukemia. Expert Opin. Investig. Drugs, 2017, 26, 633-650.
[195]
Siegel, R.; Ma, J.; Zou, Z.; Jemal, A. Cancer statistics, 2014. CA Cancer J. Clin., 2014, 64, 9-29.
[196]
Verhelle, D.; Corral, L.G.; Wong, K.; Mueller, J.H.; Moutouh-de Parseval, L.; Jensen-Pergakes, K.; Schafer, P.H.; Chen, R.; Glezer, E.; Ferguson, G.D.; Lopez-Girona, A.; Muller, G.W.; Brady, H.A.; Chan, K.W. Lenalidomide and CC-4047 inhibit the proliferation of malignant B cells while expanding normal CD34+ progenitor cells. Cancer Res., 2007, 67, 746-755.
[197]
Witzig, T.E.; Wiernik, P.H.; Moore, T.; Reeder, C.; Cole, C.; Justice, G.; Kaplan, H.; Voralia, M.; Pietronigro, D.; Takeshita, K.; Ervin-Haynes, A.; Zeldis, J.B.; Vose, J.M. Lenalidomide oral monotherapy produces durable responses in relapsed or refractory indolent non-Hodgkin’s lymphoma. J. Clin. Oncol., 2009, 27, 5404-5409.
[198]
Wiernik, P.H.; Lossos, I.S.; Tuscano, J.M.; Justice, G.; Vose, J.M.; Cole, C.E.; Lam, W.; McBride, K.; Wride, K.; Pietronigro, D.; Takeshita, K.; Ervin-Haynes, A.; Zeldis, J.B.; Habermann, T.M. Lenalidomide monotherapy in relapsed or refractory aggressive non-Hodgkin’s lymphoma. J. Clin. Oncol., 2008, 26, 4952-4957.
[199]
Witzig, T.E.; Nowakowski, G.S.; Habermann, T.M.; Goy, A.; Hernandez-Ilizaliturri, F.J.; Chiappella, A.; Vitolo, U.; Fowler, N.; Czuczman, M.S. A comprehensive review of lenalidomide therapy for B-cell non-Hodgkin lymphoma. Ann. Oncol., 2015, 26, 1667-1677.
[200]
Wang, M.; Fayad, L.; Wagner-Bartak, N.; Zhang, L.; Hagemeister, F.; Neelapu, S.S.; Samaniego, F.; McLaughlin, P.; Fanale, M.; Younes, A.; Cabanillas, F.; Fowler, N.; Newberry, K.J.; Sun, L.; Young, K.H.; Champlin, R.; Kwak, L.; Feng, L.; Badillo, M.; Bejarano, M.; Hartig, K.; Chen, W.; Chen, Y.; Byrne, C.; Bell, N.; Zeldis, J.; Romaguera, J. Lenalidomide in combination with rituximab for patients with relapsed or refractory mantle-cell lymphoma: a phase I/II clinical trial. Lancet Oncol., 2012, 13, 716-723.
[201]
Ahmadi, T.; Chong, E.A.; Gordon, A.; Aqui, N.A.; Nasta, S.D.; Svoboda, J.; Mato, A.R.; Schuster, S.J. Combined lenalidomide, low-dose dexamethasone, and rituximab achieves durable responses in rituximab-resistant indolent and mantle cell lymphomas. Cancer, 2014, 120, 222-228.
[202]
Chong, E.A.; Ahmadi, T.; Aqui, N.A.; Svoboda, J.; Nasta, S.D.; Mato, A.R.; Walsh, K.M.; Schuster, S.J. Combination of lenalidomide and rituximab overcomes rituximab resistance in patients with indolent B-cell and mantle cell lymphomas. Clin. Cancer Res., 2015, 21, 1835-1842.
[203]
Yang, Y.; Shaffer, A.L., III; Emre, N.C.; Ceribelli, M.; Zhang, M.; Wright, G.; Xiao, W.; Powell, J.; Platig, J.; Kohlhammer, H.; Young, R.M.; Zhao, H.; Yang, Y.; Xu, W.; Buggy, J.J.; Balasubramanian, S.; Mathews, L.A.; Shinn, P.; Guha, R.; Ferrer, M.; Thomas, C.; Waldmann, T.A.; Staudt, L.M. Exploiting synthetic lethality for the therapy of ABC diffuse large B cell lymphoma. Cancer Cell, 2012, 21, 723-737.
[204]
Avivi, I.; Goy, A. Refining the mantle cell lymphoma paradigm: Impact of novel therapies on current practice. Clin. Cancer Res., 2015, 21, 3853-3861.
[205]
Gribben, J.G.; Fowler, N.; Morschhauser, F. Mechanisms of action of lenalidomide in B-cell non-Hodgkin lymphoma. J. Clin. Oncol., 2015, 33, 2803-2811.
[206]
Otáhal, P.; Průková, D.; Král, V.; Fabry, M.; Vočková, P.; Latečková, L.; Trněný, M.; Klener, P. Lenalidomide enhances antitumor functions of chimeric antigen receptor modified T cells. OncoImmunology, 2015, 5, e1115940.
[207]
Arora, M.; Gowda, S.; Tuscano, J. A comprehensive review of lenalidomide in B-cell non-Hodgkin lymphoma. Ther. Adv. Hematol., 2016, 7, 209-221.
[208]
Garciaz, S.; Coso, D.; Schiano de Colella, J.M.; Bouabdallah, R. Lenalidomide for the treatment of B-cell lymphoma. Expert Opin. Investig. Drugs, 2016, 25, 1103-1116.
[209]
Martin, P.; Jung, S.H.; Pitcher, B.; Bartlett, N.L.; Blum, K.A.; Shea, T.; Hsi, E.D.; Ruan, J.; Smith, S.E.; Leonard, J.P.; Cheson, B.D. A phase II trial of lenalidomide plus rituximab in previously untreated follicular non-Hodgkin’s lymphoma (NHL): CALGB 50803 (Aliance). Ann. Oncol., 2017, 28, 2806-2812.
[210]
Witzig, T.E.; Luigi Zinzani, P.; Habermann, T.M.; Tuscano, J.M.; Drach, J.; Ramchandren, R.; Kalayoglu Besisik, S.; Takeshita, K.; Casadebaig Bravo, M.L.; Zhang, L.; Fu, T.; Goy, A. Long-term analysis of phase II studies of single-agent lenalidomide in relapsed/refractory mantle cell lymphoma. Am. J. Hematol., 2017, 92, E575-E583.
[211]
Henry, J.Y.; Labarthe, M.C.; Meyer, B.; Dasgupta, P.; Dalgleish, A.G.; Galustian, C. Enhanced cross-priming of naive CD8+ T cells by dendritic cells treated by the IMiDs® immunomodulatory compounds lenalidomide and pomalidomide. Immunology, 2013, 139, 377-385.
[212]
Schafer, P.H.; Gandhi, A.K.; Loveland, M.A.; Chen, R.S.; Man, H.W.; Schnetkamp, P.P.; Wolbring, G.; Govinda, S.; Corral, L.G.; Payvandi, F.; Muller, G.W.; Stirling, D.I. Enhancement of cytokine production and AP-1 transcriptional activity in T cells by thalidomide-related immunomodulatory drugs. J. Pharmacol. Exp. Ther., 2003, 305, 1222-1232.
[213]
Martiniani, R.; Di Loreto, V.; Di Sano, C.; Lombardo, A.; Liberati, A.M. Biological activity of lenalidomide and its underlying therapeutic effects in multiple myeloma. Adv. Hematol., 2012, 2012, 842945.
[214]
Mitsiades, C.S.; Mitsiades, N.S.; Richardson, P.G.; Munshi, N.C.; Anderson, K.C. Multiple myeloma: a prototypic disease model for the characterization and therapeutic targeting of interactions between tumor cells and their local microenvironment. J. Cell. Biochem., 2007, 101, 950-968.
[215]
Ghosh, N.; Grunwald, M.R.; Fasan, O.; Bhutani, M. Expanding role of lenalidomide in hematologic malignancies. Cancer Manag. Res., 2015, 7, 105-119.
[216]
Bianchi, G.; Richardson, P.G.; Anderson, K.C. Promising therapies in multiple myeloma. Blood, 2015, 126, 300-310.
[217]
Naymagon, L.; Abdul-Hay, M. Novel agents in the treatment of multiple myeloma: a review about the future. J. Hematol. Oncol., 2016, 9, 52.
[218]
Rajan, A.M.; Kumar, S. New investigational drugs with single-agent activity in multiple myeloma. Blood Cancer J., 2016, 6, e451.
[219]
Oscio, E.M.; Mitsiades, C.S.; Orlowski, R.Z.; Anderson, K.C. Future agents and treatment directions in multiple myeloma. Expert Rev. Hematol., 2014, 7, 127-141.
[220]
El-Amm, J.; Tabbara, I.A. Emerging therapies in multiple myeloma. Am. J. Clin. Oncol., 2015, 38, 315-321.
[221]
Broijl, A.; Sonneveld, P. An update in the treatment options for multiple myeloma in nontransplant eligible patients. Expert Opin. Pharmacother., 2015, 16, 1945-1957.
[222]
Pratt, G. An oral proteasome inhibitor for multiple myeloma. Lancet Oncol., 2014, 15, 1417-1418.
[223]
Kumar, S.K.; Berdeja, J.G.; Niesvizky, R.; Lonial, S.; Laubach, J.P.; Hamadani, M.; Stewart, A.K.; Hari, P.; Roy, V.; Vescio, R.; Kaufman, J.L.; Berg, D.; Liao, E.; Di Bacco, A.; Estevam, J.; Gupta, N.; Hui, A.M.; Rajkumar, V.; Richardson, P.G. Safety and tolerability of ixazomib, an oral proteasome inhibitor, in combination with lenalidomide and dexamethasone in patients with previously untreated multiple myeloma: an open-label phase 1/2 study. Lancet Oncol., 2014, 15, 1503-1512.
[224]
Richardson, P.G.; Baz, R.; Wang, M.; Jakubowiak, A.J.; Laubach, J.P.; Harvey, R.D.; Talpaz, M.; Berg, D.; Liu, G.; Yu, J.; Gupta, N.; Di Bacco, A.; Hui, A.M.; Lonial, S. Phase 1 study of twice-weekly ixazomib, an oral proteasome inhibitor, in relapsed/refractory multiple myeloma patients. Blood, 2014, 124, 1038-1046.
[225]
Kumar, S.K.; Bensinger, W.I.; Zimmerman, T.M.; Reeder, C.B.; Berenson, J.R.; Berg, D.; Hui, A.M.; Gupta, N.; Di Bacco, A.; Yu, J.; Shou, Y.; Niesvizky, R. Phase 1 study of weekly dosing with the investigational oral proteasome inhibitor ixazomib in relapsed/refractory multiple myeloma. Blood, 2014, 124, 1047-1055.
[226]
Richardson, P.G.; Moreau, P.; Laubach, J.P.; Gupta, N.; Hui, A.M.; Anderson, K.C.; San Miguel, J.F.; Kumar, S. The investigational proteasome inhibitor ixazomib for the treatment of multiple myeloma. Future Oncol., 2015, 11, 1153-1168.
[227]
Gentile, M.; Offidani, M.; Vigna, E.; Corvatta, L.; Recchia, A.G.; Morabito, L.; Morabito, F.; Gentili, S. Ixazomib for the treatment of multiple myeloma. Expert Opin. Investig. Drugs, 2015, 24, 1287-1298.
[228]
Stewart, A.K.; Rajkumar, S.V.; Dimopoulos, M.A.; Masszi, T.; Špička, I.; Oriol, A.; Hájek, R.; Rosiñol, L.; Siegel, D.S.; Mihaylov, G.G.; Goranova-Marinova, V.; Rajnics, P.; Suvorov, A.; Niesvizky, R.; Jakubowiak, A.J.; San-Miguel, J.F.; Ludwig, H.; Wang, M.; Maisnar, V.; Minarik, J.; Bensinger, W.I.; Mateos, M.V.; Ben-Yehuda, D.; Kukreti, V.; Zojwalla, N.; Tonda, M.E.; Yang, X.; Xing, B.; Moreau, P.; Palumbo, A. ASPIRE Investigators. Carfilzomib, lenalidomide, and dexamethasone for relapsed multiple myeloma. N. Engl. J. Med., 2014, 372, 142-152.
[229]
Sugumar, D.; Keller, J.; Vij, R. Targeted treatments for multiple myeloma: specific role of carfilzomib. Pharmacogenomics Personal Med, 2015, 8, 23-33.
[230]
Muchtar, E.; Gertz, M.A.; Magen, H. A practical review on carfilzomib in multiple myeloma. Eur. J. Haematol., 2016, 96, 564-577.
[231]
Shi, C-X.; Kortűm, K.M.; Zhu, Y.X.; Jedlowski, P.; Bruins, L.; Braggio, E.; Stewart, A.K. Proteasome inhibitors block Ikaros degradation by lenalidomide in multiple myeloma. Haematologica, 2015, 100, e315-e317.
[232]
Atanackovic, D.; Luetkens, T.; Kröger, N. Coinhibitory molecule PD-1 as a potential target for the immunotherapy of multiple myeloma. Leukemia, 2014, 28, 993-1000.
[233]
Pardoll, D.M. The blockade of immune checkpoints in cancer immunotherapy. Nat. Rev. Cancer, 2012, 12, 252-264.
[234]
Dolan, D.E.; Gupta, S. PD-1 pathway inhibitors: Changing the landscape of cancer immunotherapy. Cancer Contr., 2014, 21, 231-237.
[235]
Philips, G.K.; Atkins, M. Therapeutic uses of anti-PD-1 and anti-PD-L1 antibodies. Int. Immunol., 2014, 27, 39-46.
[236]
List, A.; Kurtin, S.; Roe, D.J.; Buresh, A.; Mahadevan, D.; Fuchs, D.; Rimsza, L.; Heaton, R.; Knight, R.; Zeldis, J.B. Efficacy of lenalidomide in myelodysplastic syndromes. N. Engl. J. Med., 2005, 352, 549-557.
[237]
List, A.; Dewald, G.; Bennett, J.; Giagounidis, A.; Raza, A.; Feldman, E.; Powell, B.; Greenberg, P.; Thomas, D.; Stone, R.; Reeder, C.; Wride, K.; Patin, J.; Schmidt, M.; Zeldis, J.; Knight, R. Myelodysplastic Syndrome-003 Study Investigators. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N. Engl. J. Med., 2006, 355, 1456-1465.
[238]
Kuendgen, A.; Lauseker, M.; List, A.F.; Fenaux, P.; Giagounidis, A.A.; Brandenburg, N.A.; Backstrom, J.; Glasmacher, A.; Hasford, J.; Germing, U. International Working Group on MDS with del. (5q). Lenalidomide does not increase AML progression risk in RBC transfusion-dependent patients with low- or intermediate-1-risk MDS with del(5q): A comparative analysis. Leukemia, 2013, 27, 1072-1079.
[239]
List, A.F.; Wride, K.; Dewald, G.; Bennett, J.M.; Giagounidis, A.; Kurtin, S.; Knight, R.D. Cytogenetic response to lenalidomide is
associated with improved survival in patients with chromosome 5q
deletion. Leuk. Res., 2007, 31 (Suppl. 1), S38, Abstract C028
[240]
Ades, L.; Le Bras, F.; Sebert, M.; Kelaidi, C.; Lamy, T.; Dreyfus, F.; Eclache, V.; Delaunay, J.; Bouscary, D.; Visanica, S.; Turlure, P.; Bresler, A.G.; Cabrol, M.P.; Banos, A.; Blanc, M.; Vey, N.; Delmer, A.; Wattel, E.; Chevret, S.; Fenaux, P. Treatment with lenalidomide does not appear to increase the risk of progression in lower risk myelodysplastic syndromes with 5q deletion. A comparative analysis by the Groupe Francophone des Myelodysplasies. Haematologica, 2012, 97, 213-218.
[241]
List, A.F.; Bennett, J.M.; Sekeres, M.A.; Skikne, B.; Fu, T.; Shammo, J.M.; Nimer, S.D.; Knight, R.D.; Giagounidis, A. MDS- 003 Study Investigators. Extended survival and reduced risk of AML progression in erythroid-responsive lenalidomide-treated patients with lower-risk del(5q) MDS. Leukemia, 2014, 28, 1033-1040.
[242]
Komrokji, R.S.; List, A.F. Short and long-term benefits of lenalidomide treatment in patients with lower-risk del(5q) myelodysplastic syndromes. Ann. Oncol., 2016, 27, 62-68.
[243]
Giagounidis, A.; Fenaux, P.; Mufti, G.J.; Muus, P.; Platzbecker, U.; Sanz, G.; Cripe, L.; Von Lilienfeld-Toal, M.; Wells, R.A. Practical recommendations on the use of lenalidomide in the management of myelodysplastic syndromes. Ann. Hematol., 2008, 87, 345-352.
[244]
Giagounidis, A.A.; Kulasekararaj, A.; Germing, U.; Radkowski, R.; Haase, S.; Petersen, P.; Göhring, G.; Büsche, G.; Aul, C.; Mufti, G.J.; Platzbecker, U. Long-term transfusion independence in del(5q) MDS patients who discontinue lenalidomide. Leukemia, 2012, 26, 855-858.
[245]
Göhring, G.; Lange, K.; Hofmann, W.; Nielsen, K.V.; Hellström-Lindberg, E.; Roy, L.; Morgan, M.; Kreipe, H.; Büsche, G.; Giagounidis, A.; Schlegelberger, B. Telomere shortening, clonal evolution and disease progression in myelodysplastic syndrome patients with 5q deletion treated with lenalidomide. Leukemia, 2012, 26, 356-358.
[246]
Jädersten, M.; Saft, L.; Pellagatti, A.; Göhring, G.; Wainscoat, J.S.; Boultwood, J.; Porwit, A.; Schlegelberger, B.; Hellström-Lindberg, E. Clonal heterogeneity in the 5q- syndrome: p53 expressing progenitors prevail during lenalidomide treatment and expand at disease progression. Haematologica, 2009, 94, 1762-1766.
[247]
Jädersten, M.; Saft, L.; Smith, A.; Kulasekararaj, A.; Pomplun, S.; Göhring, G.; Hedlund, A.; Hast, R.; Schlegelberger, B.; Porwit, A.; Hellström-Lindberg, E.; Mufti, G.J. TP53 mutations in low-risk myelodysplastic syndromes with del(5q) predict disease progression. J. Clin. Oncol., 2011, 29, 1971-1979.
[248]
Kulasekararaj, A.G.; Smith, A.E.; Mian, S.A.; Mohamedali, A.M.; Krishnamurthy, P.; Lea, N.C.; Gäken, J.; Pennaneach, C.; Ireland, R.; Czepulkowski, B.; Pomplun, S.; Marsh, J.C.; Mufti, G.J. TP53 mutations in myelodysplastic syndrome are strongly correlated with aberrations of chromosome 5, and correlate with adverse prognosis. Br. J. Haematol., 2013, 160, 660-672.
[249]
Gamez, S.; Ali, A.; Raza, A. Implications of TP53 gene mutations
in myelodysplastic syndromes: a review. J. Blood Disorders, 2015,
2, id. 1028.
[250]
Belickova, M.; Vesela, J.; Jonasova, A.; Pejsova, B.; Votavova, H.; Merkerova, M.D.; Zemanova, Z.; Brezinova, J.; Mikulenkova, D.; Lauermannova, M.; Valka, J.; Michalova, K.; Neuwirtova, R.; Cermak, J. TP53 mutation variant allele frequency is a potential predictor for clinical outcome of patients with lower-risk myelodysplastic syndromes. Oncotarget, 2016, 7, 36266-36279.
[251]
Lodé, L.; Ménard, A.; Flet, L.; Richebourg, S.; Loirat, M.; Eveillard, M.; Le Bris, Y.; Godon, C.; Theisen, O.; Gagez, A.L.; Cartron, G.; Commes-Maerten, T.; Villemagne, B.; Luycx, O.; Godmer, P.; Pellat-Deceunynck, C.; Soussi, T.; Béné, M.C.; Delaunay, J.; Peterlin, P. Emergence and evolution of TP53 mutations are a key feature of disease progression in myelodysplastic patients with lower-risk del(5q) treated with lenalidomide. Haematologica, 2018, 103, e143-e146.
[252]
Tehranchi, R.; Woll, P.S.; Anderson, K.; Buza-Vidas, N.; Mizukami, T.; Mead, A.J.; Astrand-Grundström, I.; Strömbeck, B.; Horvat, A.; Ferry, H.; Dhanda, R.S.; Hast, R.; Rydén, T.; Vyas, P.; Göhring, G.; Schlegelberger, B.; Johansson, B.; Hellström-Lindberg, E.; List, A.; Nilsson, L.; Jacobsen, S.E. Persistent malignant stem cells in del(5q) myelodysplasia in remission. N. Engl. J. Med., 2010, 363, 1025-1037.
[253]
Melchert, M.; Kale, V.; List, A. The role of lenalidomide in the treatment of patients with chromosome 5q deletion and other myelodysplastic syndromes. Curr. Opin. Hematol., 2007, 14, 123-129.
[254]
List, A.F. Lenalidomide – The Phoenix Rises. N. Engl. J. Med., 2007, 357, 2183-2186.
[255]
Kurtin, S.E.; List, A.F. Durable long-term responses in patients with myelodysplastic syndromes treated with lenalidomide. Clin. Lymphoma Myeloma, 2009, 9, E10-E13.
[256]
Komrokji, R.S.; List, A.F. Lenalidomide for teatment of myelodysplastic syndromes: current status and future directions. Hematol. Oncol. Clin. North Am., 2010, 24, 377-388.
[257]
Raza, A.; Reeves, J.A.; Feldman, E.J.; Dewald, G.W.; Bennett, J.M.; Deeg, H.J.; Dreisbach, L.; Schiffer, C.A.; Stone, R.M.; Greenberg, P.L.; Curtin, P.T.; Klimek, V.M.; Shammo, J.M.; Thomas, D.; Knight, R.D.; Schmidt, M.; Wride, K.; Zeldis, J.B.; List, A.F. Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1 risk myelodysplastic syndromes with karyotypes other than deletion 5q. Blood, 2008, 111, 86-93.
[258]
Hoefsloot, L.H.; van Amelsvoort, M.P.; Broeders, L.C.; van der Plas, D.C.; van Lom, K.; Hoogerbrugge, H.; Touw, I.P.; Löwenberg, B. Erythropoietin-induced activation of STAT5 is impaired in the myelodysplastic syndrome. Blood, 1997, 89, 1690-1700.
[259]
Santini, V. Anemia as the main manifestation of MDS. Semin. Hematol., 2015, 52, 348-356.
[260]
Sibon, D.; Cannas, G.; Baracco, F.; Prebet, T.; Vey, N.; Banos, A.; Besson, C.; Corm, S.; Blanc, M.; Slama, B.; Perrier, H.; Fenaux, P.; Wattel, E. Groupe Francophone des Myélodysplasies. Lenalidomide in lower-risk myelodysplastic syndromes with karyotypes other than deletion 5q and refractory to erythropoiesis-stimulating agents. Br. J. Haematol., 2012, 156, 619-625.
[261]
Toma, A.; Kosmider, O.; Chevret, S.; Delaunay, J.; Stamatoullas, A.; Rose, C.; Beyne-Rauzy, O.; Banos, A.; Guerci-Bresler, A.; Wickenhauser, S.; Caillot, D.; Laribi, K.; De Renzis, B.; Bordessoule, D.; Gardin, C.; Slama, B.; Sanhes, L.; Gruson, B.; Cony-Makhoul, P.; Chouffi, B.; Salanoubat, C.; Benramdane, R.; Legros, L.; Wattel, E.; Tertian, G.; Bouabdallah, K.; Guilhot, F.; Taksin, A.L.; Cheze, S.; Maloum, K.; Nimuboma, S.; Soussain, C.; Isnard, F.; Gyan, E.; Petit, R.; Lejeune, J.; Sardnal, V.; Renneville, A.; Preudhomme, C.; Fontenay, M.; Fenaux, P.; Dreyfus, F. Lenalidomide with or without erythropoietin in transfusion dependent erythropoiesis-stimulating agent-refractory lower risk MDS without 5q deletion. Leukemia, 2016, 30, 897-905.
[262]
Basiorka, A.A.; Mc Graw, K.L.; De Ceunick, L.; Griner, L.N.; Zhang, L.; Clark, J.A.; Caceres, G.; Sokol, L.; Komrokji, R.S.; Reuther, G.W.; Wei, S.; Tavernier, J.; List, A.F. Lenalidomide stabilizes the erythropoietin receptor by inhibiting the E3 ubiquitin ligase RNF41. Cancer Res., 2016, 76, 3531-3540.
[263]
Jing, X.; Infante, J.; Nachtman, R.G.; Jurecic, R. E3 ligase FLRF (Rnf41) regulates differentiation of hematopoietic progenitors by governing steady – state levels of cytokine and retinoic acid receptors. Exp. Hematol., 2008, 36, 1110-1120.
[264]
Wauman, J.; De Ceunick, L.; Vanderroost, N.; Lievens, S.; Tavernier, J. RNF41 (Nrdp1) controls type 1 cytokine receptor degradation and ectodomain shedding. J. Cell Sci., 2011, 124, 921-932.
[265]
McGraw, K.L.; Fuhler, G.M.; Johnson, J.O.; Clark, J.A.; Caceres, G.C.; Sokol, L.; List, A.F. Erythropoietin receptor signaling is membrane dependent. PLoS One, 2012, 7, e34477.
[266]
McGraw, K.L.; Basiorka, A.A.; Johnson, J.O.; Clark, J.; Caceres, G.; Padron, E.; Heaton, R.; Ozawa, Y.; Wei, S.; Sokol, L.; List, A.F. Lenalidomide induces lipid raft assembly to enhance erythropoietin receptor signaling in myelodysplastic syndrome progenitors. PLoS One, 2014, 9, e114249.
[267]
Santini, V.; Almeida, A.; Giagounidis, A.; Gröpper, S.; Jonasova, A.; Vey, N.; Mufti, G.J.; Buckstein, R.; Mittelman, M.; Platzbecker, U.; Shpilberg, O.; Ram, R.; Del Cañizo, C.; Gattermann, N.; Ozawa, K.; Risueño, A.; MacBeth, K.J.; Zhong, J.; Séguy, F.; Hoenekopp, A.; Beach, C.L.; Fenaux, P. Randomized phase III study of lenalidomide versus placebo in RBC transfusion-dependent patients with lower risk non-del(5q) myelodysplastic syndromes and ineligible for or refractory to erythropoiesis-stimulating agents. J. Clin. Oncol., 2016, 34, 2988-2996.
[268]
Vlachos, A.; Farrar, J.E.; Atsidaftos, E.; Muir, E.; Narla, A.; Markello, T.C.; Singh, S.A.; Landowski, M.; Gazda, H.T.; Blanc, L.; Liu, J.M.; Ellis, S.R.; Arceci, R.J.; Ebert, B.L.; Bodine, D.M.; Lipton, J.M. Diminutive somatic deletions in the 5q region led to a phenotype atypical of clasical 5q- syndrome. Blood, 2013, 122, 2487-2490.
[269]
Ebert, B.L.; Pretz, J.; Bosco, J.; Chang, C.Y.; Tamayo, P.; Galili, N.; Raza, A.; Root, D.E.; Attar, E.; Ellis, S.R.; Golub, T.R. Identification of RPS14 as a 5q- syndrome gene by RNA interference screen. Nature, 2008, 451, 252-253.
[270]
Pellagatti, A.; Hellström-Lindberg, E.; Giagounidis, A.; Perry, J.; Malcovati, L.; Della Porta, M.G.; Jädersten, M.; Killick, S.; Fidler, C.; Cazzola, M.; Wainscoat, J.S.; Boultwood, J. Haploinsufficiency of RPS14 in 5q- syndrome is associated with deregulation of ribosomal- and translation-related genes. Br. J. Haematol., 2008, 142, 57-64.
[271]
Sohal, D.; Pellagatti, A.; Zhou, L.; Mo, Y.; Opalinska, J.B.; Alencar, C.; Heuck, C.; Wickrema, A.; Friedman, E.; Greally, J.; Ebert, B.L.; Warner, J.; Boultwood, J.; Verma, A. Downregulation of ribosomal
proteins is seen in non 5q- MDS. Blood, 2008, 112, ASH
Meeting Abstract 854.
[272]
Wu, L.; Li, X.; Xu, F.; Zhang, Z.; Chang, C.; He, Q. Low RPS14 expression in MDS without 5q- aberration confers higher apoptosis rate of nucleated erythrocytes and predicts prolonged survival and possible response to lenalidomide in lower risk non-5q- patients. Eur. J. Haematol., 2013, 90, 486-493.
[273]
Czibere, A.G.; Bruns, I.; Junge, B.; Singh, R.; Kobbe, G.; Haas, R.; Germing, U. Low RPS14 expression is common in myelodysplastic syndromes without 5q- aberration and defines a subgroup of patients with prolonged survival. Haematologica, 2009, 94, 1453-1455.
[274]
Dutt, S.; Narla, A.; Lin, K.; Mullally, A.; Abayasekara, N.; Megerdichian, C.; Wilson, F.H.; Currie, T.; Khanna-Gupta, A.; Berliner, N.; Kutok, J.L.; Ebert, B.L. Haploinsufficiency for ribosomal protein genes causes selective activation of p53 in human erythroid progenitor cells. Blood, 2011, 117, 2567-2576.
[275]
Zhang, Y.; Lu, H. Signaling to p53: ribosomal proteins find their way. Cancer Cell, 2009, 16, 369-377.
[276]
Fumagalli, S.; Di Cara, A.; Neb-Gulati, A.; Natt, F.; Schwemberger, S.; Hall, J.; Babcock, G.F.; Bernardi, R.; Pandolfi, P.P.; Thomas, G. Absence of nucleolar disruption after impairment of 40S ribosome biogenesis reveals an rpL11-translation-dependent mechanism of p53 induction. Nat. Cell Biol., 2009, 11, 501-508.
[277]
Pellagatti, A.; Marafioti, T.; Paterson, J.C.; Barlow, J.L.; Drynan, L.F.; Giagounidis, A.; Pileri, S.A.; Cazzola, M.; McKenzie, A.N.; Wainscoat, J.S.; Boultwood, J. Induction of p53 and up-regulation of the p53 pathway in the human 5q- syndrome. Blood, 2010, 115, 2721-2723.
[278]
Danilova, N.; Kumagai, A.; Lin, J. p53 upregulation is a frequent response to deficiency of cell-essential genes. PLoS One, 2010, 5, e15938.
[279]
Fumagalli, S.; Thomas, G. The role of p53 in ribosomopathies. Semin. Hematol., 2011, 48, 97-105.
[280]
Cazzola, M. Myelodysplastic syndrome with isolated 5q deletion (5q- syndrome). A clonal stem cell disorder characterized by defective ribosome biogenesis. Haematologica, 2008, 93, 967-972.
[281]
Barlow, J.L.; Drynan, L.F.; Trim, N.L.; Erber, W.N.; Warren, A.J.; McKenzie, A.N. New insights into 5q- syndrome as a ribosomopathy. Cell Cycle, 2010, 9, 4286-4293.
[282]
Ebert, B.L. Deletion 5q in myelodysplastic syndrome: a paradigm for the study of hemizygous deletions in cancer. Leukemia, 2009, 23, 1252-1256.
[283]
Narla, A.; Ebert, B.L. Ribosomopathies: human disorders of ribosome dysfunction. Blood, 2010, 115, 3196-3205.
[284]
Ebert, B.L.; Galili, N.; Tamayo, P.; Bosco, J.; Mak, R.; Pretz, J.; Tanguturi, S.; Ladd-Acosta, C.; Stone, R.; Golub, T.R.; Raza, A. An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome. PLoS Med., 2008, 5, e35.
[285]
Kerdivel, G.; Chesnais, V.; Becht, E.; Toma, A.; Cagnard, N.; Dumont, F.; Rousseau, A.; Fenaux, P.; Chevret, S.; Chapuis, N.; Boeva, V.; Fridman, W.H.; Fontenay, M.; Kosmider, O. Lenalidomide-mediated erythroid improvement in non-del(5q) Myelodysplastic syndromes is associated with bone marrow immuno-remodeling. Leukemia, 2018, 32, 558-562.
[286]
Adès, L.; Boehrer, S.; Prebet, T.; Beyne-Rauzy, O.; Legros, L.; Ravoet, C.; Dreyfus, F.; Stamatoullas, A.; Chaury, M.P.; Delaunay, J.; Laurent, G.; Vey, N.; Burcheri, S.; Mbida, R.M.; Hoarau, N.; Gardin, C.; Fenaux, P. Efficacy and safety of lenalidomide in intermediate-2 or high-risk myelodysplastic syndromes with 5q deletion: results of a phase 2 study. Blood, 2009, 113, 3947-3952.
[287]
Cheson, B.D.; Greenberg, P.L.; Bennett, J.M.; Lowenberg, B.; Wijermans, P.W.; Nimer, S.D.; Pinto, A.; Beran, M.; de Witte, T.M.; Stone, R.M.; Mittelman, M.; Sanz, G.F.; Gore, S.D.; Schiffer, C.A.; Kantarjian, H. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood, 2006, 108, 419-425.
[288]
Möllgård, L.; Saft, L.; Treppendahl, M.B.; Dybedal, I.; Nørgaard, J.M.; Astermark, J.; Ejerblad, E.; Garelius, H.; Dufva, I.H.; Jansson, M.; Jädersten, M.; Kjeldsen, L.; Linder, O.; Nilsson, L.; Vestergaard, H.; Porwit, A.; Grønbæk, K.; Hellström-Lindberg, E. Clinical effect of increasing doses of lenalidomide in high-risk myelodysplastic syndrome and acute myeloid leukemia with chromosome 5 abnormalities. Haematologica, 2011, 96, 963-971.
[289]
Fenaux, P.; Giagounidis, A.; Selleslag, D.; Beyne-Rauzy, O.; Mufti, G.J.; Mittelman, M.; Muus, P.; te Boekhorst, P.; Sanz, G.; del Canizo, C.; Guerci-Bresler, A.; Schlegelberger, B.; Aul, C.; Kreipe, H.; Goehring, G.; Knight, R.; Francis, J.; Fu, T.; Hellström-Lindberg, E. RBC transfusion independence and safety profile of
lenalidomide 5 or 10 mg in pts with low- or int-1-risk MDS with
del5q: results from a randomized phase III trial (MDS-004). Blood,
2009, 114, ASH Abstract 944.
[290]
Fenaux, P.; Giagounidis, A.; Selleslag, D.; Beyne-Rauzy, O.; Mittelman, M.; Muus, P.; Knight, R.D.; Fu, T.; Hellstrom-Lindberg, E. Safety of lenalidomide (LEN) from a randomized phase III trial (MDS-004) in low-/int-1-risk myelodysplastic syndromes (MDS) with a del(5q) abnormality. J. Clin. Oncol., 2010, 28(Suppl.), 6598.
[291]
Fenaux, P.; Giagounidis, A.; Selleslag, D.; Beyne-Rauzy, O.; Mufti, G.; Mittelman, M.; Muus, P.; Te Boekhorst, P.; Sanz, G.; Del Cañizo, C.; Guerci-Bresler, A.; Nilsson, L.; Platzbecker, U.; Lübbert, M.; Quesnel, B.; Cazzola, M.; Ganser, A.; Bowen, D.; Schlegelberger, B.; Aul, C.; Knight, R.; Francis, J.; Fu, T.; Hellström-Lindberg, E. MDS-004 Lenalidomide del5q Study Group. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with low-/intermediate-1-risk myelodysplastic syndromes with del5q. Blood, 2011, 118, 3765-3776.
[292]
Sekeres, M.A. Lenalidomide in MDS: 4th time’s a charm. Blood, 2011, 118, 3757-3758.
[293]
Revicki, D.A.; Brandenburg, N.A.; Muus, P.; Yu, R.; Knight, R.; Fenaux, P. Health-related quality of life outcomes of lenalidomide in transfusion-dependent patients with low- or intermediate-1-risk myelodysplastic syndromes with a chromosome 5q deletion: results from a randomized clinical trial. Leuk. Res., 2013, 37, 259-265.
[294]
Le Bras, F.; Sebert, M.; Kelaidi, C.; Lamy, T.; Dreyfus, F.; Delaunay, J.; Banos, A.; Blanc, M.; Vey, N.; Schmidt, A.; Visanica, S.; Eclache, V.; Turlure, P.; Beyne-Rauzy, O.; Guerci, A.; Delmer, A.; de Botton, S.; Rea, D.; Fenaux, P.; Adès, L. Treatment by lenalidomide in lower risk myelodysplastic syndrome with 5q deletion-the GFM experience. Leuk. Res., 2011, 35, 1444-1448.
[295]
Cheson, B.D.; Bennett, J.M.; Kantarjian, H.; Pinto, A.; Schiffer, C.A.; Nimer, S.D.; Löwenberg, B.; Beran, M.; de Witte, T.M.; Stone, R.M.; Mittelman, M.; Sanz, G.F.; Wijermans, P.W.; Gore, S.; Greenberg, P.L. World Health Organization(WHO) international working group. Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood, 2000, 96, 3671-3674.
[296]
Tiu, R.V.; Sekeres, M.A. Lenalidomide in del 5q MDS: Responses and side effects revisited. Leuk. Res., 2011, 35, 1440-1441.
[297]
Harada, H.; Watanabe, M.; Suzuki, K.; Yanagita, S.; Suzuki, T.; Yoshida, Y.; Kimura, A.; Tsudo, M.; Matsuda, A.; Tohyama, K.; Taniwaki, M.; Takeshita, K.; Takatoku, M.; Ozawa, K. Lenalidomide is active in Japanese patients with symptomatic anemia in low- or intermediate-1 risk myelodysplastic syndromes with a deletion 5q abnormality. Int. J. Hematol., 2009, 90, 353-360.
[298]
Matsuda, A.; Taniwaki, M.; Jinnai, I.; Harada, H.; Watanabe, M.; Suzuki, K.; Yanagita, S.; Suzuki, T.; Yoshida, Y.; Kimura, A.; Tsudo, M.; Tohyama, K.; Takatoku, M.; Ozawa, K. Morphologic analysis in myelodysplastic syndromes with del(5q) treated with lenalidomide. A Japanese multiinstitutional study. Leuk. Res., 2012, 36, 575-580.
[299]
Sánchez-Garcia, J.; del Canizo, C. Lorenzo, I.; Nomdedeu, B.; Luño, E.; de Paz, R.; Xicoy, B.; Valcárcel, D.; Brunet, S.; Marco-Betes, V.; García-Pintos, M.; Osorio, S.; Tormo, M.; Bailén, A.; Cerveró, C.; Ramos, F.; Diez-Campelo, M.; Such, E.; Arrizabalaga, B.; Azaceta, G.; Bargay, J.; Arilla, M.J.; Falantes, J.; Serrano-López, J.; Sanz, G.F.; Spanish Group on Myelodysplastic Syndromes (GES MD). Multivariate time-dependent comparison of the impact of lenalidomide in lower-risk myelodysplastic syndromes with chromosome 5q deletion. Br. J. Haematol., 2014, 166, 189-201.
[300]
Cerqui, E.; Pelizzari, A.; Schieppati, F.; Borlenghi, E.; Pagani, C.; Bellotti, D.; Lamorgese, C.; Boiocchi, L.; Sottini, A.; Imberti, L.; Rossi, G. Lenalidomide in patients with red blood cell transfusion-dependent myelodysplastic syndrome and del(5q): a single-centre “real-world” experience. Leuk. Lymphoma, 2015, 56, 3129-3134.
[301]
Sharma, S.V.; Lee, D.Y.; Li, B.; Quinlan, M.P.; Takahashi, F.; Maheswaran, S.; McDermott, U.; Azizian, N.; Zou, L.; Fischbach, M.A.; Wong, K.K.; Brandstetter, K.; Wittner, B.; Ramaswamy, S.; Classon, M.; Settleman, J. A chromatin-mediated reversible drug-tolerant state in cancer cell subpopulations. Cell, 2010, 141, 69-80.
[302]
Giagounidis, A.A.N. Lenalidomide for del(5q) and non-del(5q) myelodysplastic syndromes. Semin. Hematol., 2012, 49, 312-322.
[303]
List, A.F.; Lancet, J.E.; Melchert, M.; Lush, R.; Yu, J.; Chen, N.; Schmidt, M.; Knight, R. Two-stage pharmacokinetic efficacy
study of lenalidomide alone or combined with recombinant
erythropoietin (EPO) in lower risk MDS EPO-failures [PK-002].
Blood, 2007, 110, ASH Meeting Abstract 4626.
[304]
Park, S.; Vassilieff, D.; Bardet, V.; Viguié, F.; Dreyfus, F. Efficacy of the association of lenalidomide to erythropoiesis-stimulating agents in del (5q) MDS patients refractory to single-agent lenalidomide. Leukemia, 2010, 24, 1960-1977.
[305]
Emanuel, P.D.; Wang, Z.; Cai, D. TLK199 (TelintraTM), a novel
glutathione analog inhibitor of GST P1-1, causes proliferation and
maturation of bone marrow precursor cells and correlates with
clinical improvement in myelodysplastic syndrome (MDS) patients
in a phase 2a study. Blood, 2004, 104, ASH Meeting Abstract
2372.
[306]
Galili, N.; Tamayo, P.; Botvinnik, O.B.; Mesirov, J.P.; Brooks, M.R.; Brown, G.; Raza, A. Prediction of response to therapy with ezatiostat in lower risk myelodysplastic syndrome. J. Hematol. Oncol., 2012, 5, 20.
[307]
Raza, A.; Galili, N.; Smith, S.; Godwin, J.; Lancet, J.; Melchert, M.; Jones, M.; Keck, J.G.; Meng, L.; Brown, G.L.; List, A. Phase 1 multicenter dose-escalation study of ezatiostat hydrochloride (TLK199 tablets), a novel glutathione analog prodrug, in patients with myelodysplastic syndrome. Blood, 2009, 113, 6533-6540.
[308]
Raza, A.; Galili, N.; Callander, N.; Ochoa, L.; Piro, L.; Emanuel, P.; Williams, S.; Burris, H., III; Faderl, S.; Estrov, Z.; Curtin, P.; Larson, R.A.; Keck, J.G.; Jones, M.; Meng, L.; Brown, G.L. Phase 1-2a multicenter dose-escalastion study of ezatiostat hydrochloride liposomes for injection (Telintra, TLK199), a novel glutathione analog prodrug in patients with myelodysplastic syndrome. J. Hematol. Oncol., 2009, 2, 20.
[309]
Quddus, F.; Clima, J.; Seedham, H.; Sajjad, G.; Galili, N.; Raza, A. Oral ezatiostat HCl (TLK199) and myelodysplastic syndrome: a case report of sustained hematologic response following an abbreviated exposure. J. Hematol. Oncol., 2010, 3, 16.
[310]
Raza, A.; Galili, N.; Mulford, D.; Smith, S.E.; Brown, G.L.; Steensma, D.P.; Lyons, R.M.; Boccia, R.; Sekeres, M.A.; Garcia-Manero, G.; Mesa, R.A. Phase 1 dose-ranging study of oral ezatiostat hydrochloride (Telintra, TLK 199) in combination with lenalidomide (Revlimid) in patients with non-deletion(5q) low to intermediate-1 risk myelodysplastic syndrome (MDS). J. Hematol. Oncol., 2012, 5, 18.
[311]
Lyons, R.M.; Wilks, S.T.; Young, S.; Brown, G.L. Oral ezatiostat HCL (Telintra, TLK 199) and idiopathic chronic neutropenia (ICN): a case report of complete response of a patient with G-CSF resistant ICN following treatment with ezatiostat, a glutathione S-transferase P1-1 (GSTP1-1) inhibitor. J. Hematol. Oncol., 2011, 4, 43.
[312]
Raza, A.; Galili, N.; Smith, S.E.; Godwin, J.; Boccia, R.V.; Myint, H.; Mahadevan, D.; Mulford, D.; Rarick, M.; Brown, G.L.; Schaar, D.; Faderl, S.; Komrokji, R.S.; List, A.F.; Sekeres, M. A phase 2 randomized multicenter study of 2 extended dosing schedules of oral ezatiostat in low to intermediate-1 risk myelodysplastic syndrome. Cancer, 2012, 118, 2138-2147.
[313]
Sekeres, M.A.; List, A.F.; Cuthbertson, D.; Paquette, R.; Ganetzky, R.; Latham, D.; Paulic, K.; Afable, M.; Saba, H.I.; Loughran, T.P. Jr.; Maciejewski, J.P. Phase I combination trial of lenalidomide and azacitidine in patients with higher-risk myelodysplastic syndromes. J. Clin. Oncol., 2010, 28, 2253-2258.
[314]
Sekeres, M.A.; O’Keefe, C.; List, A.F.; Paulic, K.; Afable, M., II; Englehaupt, R.; Maciejewski, J.P. Demonstration of additional benefit in adding lenalidomide to azacitidine in patients with higher-risk myelodysplastic syndromes. Am. J. Hematol., 2011, 86, 102-103.
[315]
Garcia-Manero, G.; Daver, N.G.; Borthakur, G.; Konopleva, M.; Ravandi, F.; Wierda, W.G.; Estrov, Z.; Faderl, S.; Kadia, T.; Rey, K.; Cheung, C.; Kantarjian, H.M. Phase I study of the combination
of 5-azacitidine sequentially with high-dose lenalidomide in
higher-risk myelodysplastic syndrome (MDS) and acute myelogenous
leukemia (AML). Blood, 2011, 118, ASH Meeting Abstract
2613.
[316]
Bally, C.; Itzykson, R.; Gruson, B.; Dreyfus, F.; Siguret, V.; Taksin, A.L.; de Renzis, B.; Legros, L.; Thomas, X.; Bouabdallah, K.; Chaibi, P.; Kelaidi, C.; Fenaux, P.; Ades, L. Azacitidine (AZA) after
failure of lenalidomide (LEN) in low/int-1 risk MDS with del
5q. Blood, 2011, 118, ASH Meeting Abstract 2786
[317]
Platzbecker, U.; Ganster, C.; Neesen, J.; Kuendgen, A.; Götze, K.; Bug, G.; Shirneshan, K.; Braulke, F.; Röllig, C.; Naumann, R.; Bűssemaker, E.; Giagounidis, A.; Hofmann, W.K.; Germing, U.; vHaase, D.; Ehninger, G. Safety and efficacy of a combination of
5-azacitidine followed by lenalidomide in high-risk MDS or AML
patients with del(5q) cytogenetic abnormalities-results of the
“AZALE” trial. Blood, 2011, 118, ASH Meeting Abstract 3799
[318]
Wei, A.H.; Tan, P.T.; Walker, P.A.; Avery, S.; Patil, S.S.; Schwarer, A.P.; Janusewicz, E.; Harrison, S.J.; Ho, W.K.; Tam, C.S.; Spencer, A. A phase Ib dose escalation safety analysis of lenalidomide and azacitidine maintenance therapy for poor risk AML.Blood, 2011, 118, ASH Meeting Abstract 3625
[319]
Sekeres, M.A.; Tiu, R.V.; Komrokji, R.; Lancet, J.; Advani, A.S.; Afable, M.; Englehaupt, R.; Juersivich, J.; Cuthbertson, D.; Paleveda, J.; Tabarroki, A.; Visconte, V.; Makishima, H.; Jerez, A.; Paquette, R.; List, A.F.; Maciejewski, J.P. Phase 2 study of the lenalidomide and azacitidine combination in patients with higher-risk myelodysplastic syndromes. Blood, 2012, 120, 4945-4951.
[320]
Pollyea, D.A.; Kohrt, H.E.; Gallegos, L.; Figueroa, M.E.; Abdel-Wahab, O.; Zhang, B.; Bhattacharya, S.; Zehnder, J.; Liedtke, M.; Gotlib, J.R.; Coutre, S.; Berube, C.; Melnick, A.; Levine, R.; Mitchell, B.S.; Medeiros, B.C. Safety, efficacy and biological predictors of response to sequential azacitidine and lenalidomide for elderly patients with acute myeloid leukemia. Leukemia, 2012, 26, 893-901.
[321]
Scherman, E.; Malak, S.; Perot, C.; Gorin, N.C.; Rubio, M.T.; Isnard, F. Interest of the association azacitidine-lenalidomide as frontline therapy in high-risk myelodysplasia or acute myeloid leukemia with complex karyotype. Leukemia, 2012, 26, 822-824.
[322]
Pollyea, D.A.; Zehnder, J.; Coutre, S.; Pollyea, D.A.; Zehnder, J.; Coutre, S. Sequential azacitidine plus lenalidomide combination for elderly patients with untreated acute myeloid leukemia. Haematologica, 2013, 98, 591-596.
[323]
Platzbecker, U.; Germing, U. Combination of azacitidine and lenalidomide in myelodysplastic syndromes or acute myeloid leukemia-a wise liason? Leukemia, 2013, 27, 1813-1819.
[324]
Zeidan, A.M.; Gore, S.D.; Komrokji, R.S. Higher-risk myelodysplastic syndromes with del(5q): is sequential azacitidine-lenalidomide combination the way to go? Expert Rev. Hematol., 2013, 6, 251-254.
[325]
Ramsingh, G.; Westervelt, P.; Cashen, A.F.; Uy, G.L.; Stockerl-Goldstein, K.; Abboud, C.N.; Bernabe, N.; Monahan, R.; DiPersio, J.F.; Vij, R. A phse 1study of concomitant high-dose lenalidomide and 5- azacitidine induction in the treatment of AML. Leukemia, 2013, 27, 725-728.
[326]
Ornstein, M.C.; Mukherjee, S.; Sekeres, M.A. More is better: Combination therapies for myelodysplastic syndromes. Best Pract. Res. Clin. Haematol., 2015, 28, 22-31.
[327]
Wei, A.; Tan, P.; Perruzza, S.; Govindaraj, C.; Fleming, S.; McManus, J.; Avery, S.; Patil, S.; Stevenson, W.; Plebanski, M.; Spencer, A. Maintenance lenalidomide in combination with 5-azacitidine as post-remision therapy for acute myeloid leukemia. Br. J. Haematol., 2015, 169, 199-210.
[328]
Ganster, C.; Shirneshan, K.; Salinas-Riester, G.; Braulke, F.; Schanz, J.; Platzbecker, U.; Haase, D. Influence of total genomic alteration and chromosomal fragmentation on response to a combination of azacitidine and lenalidomide in a cohort of patients with very high risk MDS. Leuk. Res., 2015, 39, 1079-1087.
[329]
Todaro, J.; Weinschenker-Bollmann, P.; Rother, E.T.; del Giglio, A. Azacitidine and lenalidomide as an alternative treatment for refractory acute myeloid leukemia: a case report. Sao Paulo Med. J., 2015, 133, 271-274.
[330]
Narayan, R.; Garcia, J.S.; Percival, M.E.M.; Berube, C.; Coutre, S.; Gotlib, J.; Greenberg, P.; Liedtke, M.; Hewitt, R.; Regan, K.; Williamson, C.; Doykan, C.; Cardone, M.H.; McMillan, A.; Medeiros, B.C. Sequential azacitidine plus lenalidomide in previously treated elderly patients with acute myeloid leukemia and higher risk myelodysplastic syndrome. Leuk. Lymphoma, 2016, 57, 609-615.
[331]
Finelli, C.; Clissa, C.; Follo, M.; Stanzani, M.; Parisi, S.; Mongiorgi, S.; Barraco, M.; Avanziani, P.; Bosi, C.; Castagnazi, B.; Candoni, A.; Crugnola, M.; Giannini, M.B.; Gobbi, M.; Leonardi, G.; Rigolin, G.M.; Russo, D.; Tosi, P.; Visani, G.; Cocco, L.; Cavo, M. Azacitidine and lenalidomide (combined vs sequential
treatment in higher-risk myelodysplastic syndromes. Long-term results
of a randomized phase II multicenter study. Blood, 2016, 128,
ASH Meeting Abstract 3169
[332]
Broudy, V.C.; Lin, N.L. AMG 531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine, 2004, 25, 52-60.
[333]
Wang, B.; Nichol, J.L.; Sullivan, J.T. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin. Pharmacol. Ther., 2004, 76, 628-638.
[334]
Frampton, J.E.; Lyseng-Williamson, K.A. Romiplostim. Drugs, 2009, 69, 307-317.
[335]
Keating, G.M. Romiplostim: a review of its use in immune thrombocytopenia. Drugs, 2012, 72, 415-435.
[336]
Kantarjian, H.; Fenaux, P.; Sekeres, M.; Becker, P.S.; Boruchov, A.; Bowen, D.; Hellstrom-Lindberg, E.; Larson, R.A.; Lyons, R.M.; Muus, P.; Shammo, J.; Siegel, R.; Hu, K.; Franklin, J.; Berger, D.P. Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and thrombocytopenia. J. Clin. Oncol., 2010, 28, 437-444.
[337]
Wang, E.S.; Lyons, R.M.; Larson, R.A.; Gandhi, S.; Liu, D.; Matei, C.; Scott, B.; Hu, K.; Yang, A.S. A randomized, double-blind, placebo-controlled phase 2 study evaluating the efficacy and safety of romiplostim treatment of patients with low or intermediate-1 risk myelodysplastic syndrome receiving lenalidomide. J. Hematol. Oncol., 2012, 5, 71.
[338]
Narla, A.; Dutt, S.; McAuley, R.J.; Al-Shahrour, F.; Hurst, S.; McConkey, M.; Neuberg, D.; Ebert, B.L. Dexamethasone and lenalidomide have distinct functional effects on erythropoiesis. Blood, 2011, 118, 2296-2304.
[339]
Komrokji, R.S.; Al Ali, N.H.; Padron, E.; Lee, J.H.; Hillgruber, N.; Tinsley, S.; Lancet, J.E.; List, A. A phase II clinical trial of lenalidomide
and prednisone in low and intermediate-1 IPSS risk,
non-del(5q) MDS patients. Blood, 2013, 122, ASH Meeting Abstract
1506
[340]
Jonasova, A.; Neuwirtova, R.; Polackova, H.; Siskova, M.; Stopka, T.; Cmunt, E.; Belickova, M.; Moudra, A.; Minarik, L.; Fuchs, O.; Michalova, K.; Zemanova, Z. Lenalidomide treatment in lower risk myelodysplastic syndromes-The experience of a Czech hematology center (Positive effect of erythropoietin ±prednisone addition nto lenalidomide in refractory or relapsed patients). Leuk. Res., 2018, 69, 12-17.
[341]
Cmejlova, J.; Dolezalova, L.; Pospisilova, D.; Petrtylova, K.; Petrak, J.; Cmejla, R. Translational efficiency in patients with Diamond-Blackfan anemia. Haematologica, 2006, 91, 1456-1464.
[342]
Pospisilova, D.; Cmejlova, J.; Hak, J.; Adam, T.; Cmejla, R. Successful treatment of a Diamond-Blackfan anemia patient with amino acid leucine. Haematologica, 2007, 92, e66-e67.
[343]
Payne, E.; Virgilio, M.; Narla, A.; Sun, H.; Levine, M.; Paw, B.H.; Berliner, N.; Look, A.T.; Ebert, B.L.; Khanna-Gupta, A. L-leucine improves anemia and developmental defects associated with Diamond-Blackfan anemia and del(5q) MDS by activating the mTOR pathway. Blood, 2012, 120, 2214-2224.
[344]
Yip, B.H.; Pellagatti, A.; Vuppusetty, C.; Giagounidis, A.; Germing, U.; Lamikanra, A.A.; Roberts, D.J.; Fernandez-Mercado, M.; McDonald, E.J.; Killick, S.; Wainscoat, J.S.; Boultwood, J. Effects of L-leucine in 5q- syndrome and other RPS14-deficient erythroblasts. Leukemia, 2012, 26, 2154-2158.
[345]
Heise, C.; Carter, T.; Schafer, P.; Chopra, R. Pleiotropic mechanisms of action of lenalidomide efficacy in del(5q) myelodysplastic syndromes. Expert Rev. Anticancer Ther., 2010, 10, 1663-1672.
[346]
Voutsadakis, I.A.; Cairoli, A. A critical review of the molecular pathophysiology of lenalidomide sensitivity in 5q- myelodysplastic syndromes. Leuk. Lymphoma, 2012, 53, 779-788.
[347]
Wei, S.; Chen, X.; Rocha, K.; Epling-Burnette, P.K.; Djeu, J.Y.; Liu, Q.; Byrd, J.; Sokol, L.; Lawrence, N.; Pireddu, R.; Dewald, G.; Williams, A.; Maciejewski, J.; List, A. A critical role for phosphatase haplodeficiency in the selective suppression of deletion 5q MDS by lenalidomide. Proc. Natl. Acad. Sci. USA, 2009, 106, 12974-12979.
[348]
Moutouh-de Parseval, L.A.; Verhelle, D.; Glezer, E.; Jensen-Pergakes, K.; Ferguson, G.D.; Corral, L.G.; Morris, C.L.; Muller, G.; Brady, H.; Chan, K. Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells. J. Clin. Invest., 2008, 118, 248-258.
[349]
Fuchs, O. Important genes in the pathogenesis of 5q- syndrome and their connection with ribosomal stress and the innate immune system pathway. Leukemia Res. Treat., 2012, 2012, 179402.
[350]
Bursac, S.; Brdovcak, M.C.; Pfannkuchen, M.; Orsolić, I.; Golomb, L.; Zhu, Y.; Katz, C.; Daftuar, L.; Grabušić, K.; Vukelić, I.; Filić, V.; Oren, M.; Prives, C.; Volarevic, S. Mutual protection of ribosomal proteins L5 and L11 from degradation is essential for p53 activation upon ribosomal biogenesis stress. Proc. Natl. Acad. Sci. USA, 2012, 109, 20467-20472.
[351]
Wei, S.; Chen, X.; McGraw, K.; Zhang, L.; Komrokji, R.; Clark, J.; Caceres, G.; Billingsley, D.; Sokol, L.; Lancet, J.; Fortenbery, N.; Zhou, J.; Eksioglu, E.A.; Sallman, D.; Wang, H.; Epling-Burnette, P.K.; Djeu, J.; Sekeres, M.; Maciejewski, J.P.; List, A. Lenalidomide promotes p53 degradation by inhibiting MDM2 autoubiquitination in myelodysplastic syndrome with chromosome 5q deletion. Oncogene, 2013, 32, 1110-1120.
[352]
Escoubet-Lozach, L.; Lin, I.L.; Jensen-Pergakes, K.; Brady, H.A.; Gandhi, A.K.; Schafer, P.H.; Muller, G.W.; Worland, P.J.; Chan, K.W.; Verhelle, D. Pomalidomide and lenalidomide induce p21 WAF-1 expression in both lymphoma and multiple myeloma through a LSD1 mediated epigenetic mechanism. Cancer Res., 2009, 69, 7347-7356.
[353]
Fisher, J.W. Erythropoietin: physiology and pharmacology update. Exp. Biol. Med. (Maywood), 2003, 228, 1-14.
[354]
Perreault, A.A.; Venters, B.J. Integrative view on how erythropoietin signaling controls transcription patterns in erythroid cells. Curr. Opin. Hematol., 2018, 25, 189-195.
[355]
Hennighausen, L.; Robinson, G.W. Interpretation of cytokine signaling through in the transcription factors STAT5A and STAT5B. Genes Dev., 2008, 22, 711-721.
[356]
List, A.; Estes, M.; Williams, A.; Sekharam, M.; Ozawa, U.; Gao, G.; Wu, J.; Gao, G.; Sokol, L. Lenalidomide (CC-5013; Revlimid)
promotes erythropoiesis in myelodysplastic syndromes by CD45
protein tyrosine phosphatase inhibition. Blood, 2006, 108, ASH
Meeting Abstract 1360.
[357]
Warlick, E.D.; Miller, J.S. Myelodysplastic syndromes: the role of the immune system in pathogenesis. Leuk. Lymphoma, 2011, 52, 2045-2049.
[358]
Braun, T.; Fenaux, R. Myelodysplastic syndromes (MDS) and autoimmune disorders (AD): cause or consequence? Best Pract. Res. Clin. Haematol., 2013, 26, 327-336.
[359]
Gañán-Gómez, I.; Wei, Y.; Starczynowski, D.T.; Colla, S.; Yang, H.; Cabrero-Calvo, M.; Bohannan, Z.S.; Verma, A.; Steidl, U.; Garcia-Manero, G. Deregulation of innate immune and inflammatory signaling in myelodysplastic syndromes. Leukemia, 2015, 29, 1458-1469.
[360]
Varney, M.E.; Melgar, K.; Niederkorn, M.; Smith, M.; Barreyro, L.; Starczynowski, D.T. Deconstructing innate immune signaling in myelodysplastic syndromes. Exp. Hematol., 2015, 43, 587-598.
[361]
Wolach, O.; Stone, R. Autoimmunity and inflammation in myelodysplastic syndromes. Acta Haematol., 2016, 136, 108-117.
[362]
Fuchs, O. The immune mechanisms involved in the pathogenesis
and pathophysiology of myelodysplastic syndromes and immunotherapeutic
strategies. J. Hematol. Hemother., 2016, 1, 001.
[363]
Fozza, C. The burden of autoimmunity in myelodysplastic syndromes. Hematol. Oncol., 2018, 36, 15-23.
[364]
Epling-Burnette, P.K.; List, A.F. Advancements in the molecular pathogenesis of myelodysplastic syndrome. Curr. Opin. Hematol., 2009, 16, 70-76.
[365]
Epling-Burnette, P.K.; Painter, J.S.; Rollison, D.E.; Ku, E.; Vendron, D.; Widen, R.; Boulware, D.; Zou, J.X.; Bai, F.; List, A.F. Prevalence and clinical association of clonal T cell expansions in myelodysplastic syndrome. Leukemia, 2007, 21, 659-667.
[366]
Pellagatti, A.; Boultwood, J. Recent advances in the 5q- syndrome. Mediterr. J. Hematol. Infect. Dis., 2015, 7, e2015037.
[367]
Schafer, P.H.; Gandhi, A.K.; Loveland, M.A.; Chen, R.S.; Man, H.W.; Schnetkamp, P.P.; Wolbring, G.; Govinda, S.; Corral, L.G.; Payvandi, F.; Muller, G.W.; Stirling, D.I. Enhancement of cytokine production and AP-1 transcriptional activity in T cells by thalidomide-related immunomodulatory drugs. J. Pharmacol. Exp. Ther., 2003, 305, 1663-1672.
[368]
Zhu, D.; Corral, L.G.; Fleming, Y.W.; Stein, B. Immunomodulatory drugs Revlimid (Lenalidomide) and CC-4047 induce apoptosis of both hematological and solid tumor cells through NK cell activation. Cancer Immunol. Immunother., 2008, 57, 1849-1859.
[369]
Chang, D.H.; Liu, N.; Klimek, V.; Hassoun, H.; Mazumder, A.; Nimer, S.D.; Jagannath, S.; Dhodapkar, M.V. Enhancement of ligand-dependent activation of human natural killer T cells by lenalidomide: therapeutic implications. Blood, 2006, 108, 618-621.
[370]
Starczynowski, D.T.; Kuchenbauer, F.; Argiropoulos, B.; Sung, S.; Morin, R.; Muranyi, A.; Hirst, M.; Hogge, D.; Marra, M.; Wells, R.A.; Buckstein, R.; Lam, W.; Humphries, R.K.; Karsan, A. Identification of miR-145 and miR-146a as mediators of the 5q- syndrome phenotype. Nat. Med., 2010, 16, 49-58.
[371]
Starczynowski, D.T.; Karsan, A. Deregulation of innate immune signaling in myelodysplastic syndromes is associated with deletion of chromosome arm 5q. Cell Cycle, 2010, 9, 855-856.
[372]
Starczynowski, D.T.; Karsan, A. Innate immune signaling in the myelodysplastic syndromes. Hematol. Oncol. Clin. North Am., 2010, 24, 343-359.
[373]
Rhyasen, G.W.; Starczynowski, D.T. Deregulation of microRNAs in myelodysplastic syndrome. Leukemia, 2012, 26, 13-22.
[374]
Fang, J.; Varney, M.; Starczynowski, D.T. Implication of microRNAs in the pathogenesis of MDS. Curr. Pharm. Des., 2012, 18, 3170-3179.
[375]
Fang, J.; Barker, B.; Bolanos, L.; Liu, X.; Jerez, A.; Makishima, H.; Christie, S.; Chen, X.; Rao, D.S.; Grimes, H.L.; Komurov, K.; Weirauch, M.T.; Cancelas, J.A.; Maciejewski, J.P.; Starczynowski, D.T. Myeloid malignancies with chromosome 5q deletions acquire a dependency on an intrachromosomal NF-κB gene network. Cell Reports, 2014, 8, 1328-1338.
[376]
Merkerova, M.D.; Krejcik, Z.; Belickova, M.; Hrustincova, A.; Klema, J.; Stara, E.; Zemanova, Z.; Michalova, K.; Cermak, J.; Jonasova, A. Genome-wide miRNA profiling in myelodysplastic syndrome with del(5q) treated with lenalidomide. Eur. J. Haematol., 2015, 95, 35-43.
[377]
Varney, M.E.; Niederkorn, M.; Konno, H.; Matsumura, T.; Gohda, J.; Yoshida, N.; Akiyama, T.; Christie, S.; Fang, J.; Miller, D.; Jerez, A.; Karsan, A.; Maciejewski, J.P.; Meetei, R.A.; Inoue, J.; Starczynowski, D.T. Loss of Tifab, a del(5q) MDS gene, alters hematopoiesis through derepression of Toll-like receptor-TRAF6 signaling. J. Exp. Med., 2015, 212, 1967-1985.
[378]
Oliva, E.N.; Cuzzola, M.; Nobile, F.; Ronco, F.; D’Errigo, M.G.; Laganà, C.; Morabito, F.; Galimberti, S.; Cortelezzi, A.; Aloe-Spiriti, M.A.; Specchia, G.; Poloni, A.; Breccia, M.; Ghio, R.; Finelli, C.; Iacopino, P.; Alimena, G.; Latagliata, R. Changes in RPS 14 expression levels during lenalidomide treatment in low and intermediate-1-risk myelodysplastic syndromes with chromosome 5q deletion. Eur. J. Haematol., 2010, 85, 231-235.
[379]
Oliva, E.N.; Cuzzola, M.; Aloe-Spiriti, M.A.; Poloni, A.; Laganà, C.; Rigolino, C.; Morabito, F.; Galimberti, S.; Ghio, R.; Cortelezzi, A.; Palumbo, G.A.; Sanpaolo, G.; Finelli, C.; Ricco, A.; Volpe, A.; Rodà, F.; Breccia, M.; Alimena, G.; Nobile, F.; Latagliata, R. Biological activity of lenalidomide in myelodysplastic syndromes with del5q: results of gene expression profiling from a multicenter phase II study. Ann. Hematol., 2013, 92, 25-32.
[380]
Venner, C.P.; Wegrzyn-Woltosz, J.; Nevill, T.J.; Deeg, H.J.; Caceres, G.; Platzbecker, U.; Scott, B.L.; Sokol, L.; Sung, S.; List, A.F.; Karsan, A. Correlation of clinical response and response duration with miR-145 induction by lenalidomide in CD34+ cells from patients with del(5q) myelodysplastic syndrome. Haematologica, 2013, 98, 409-413.
[381]
Min, Y.; Wi, S.M.; Kang, J.A.; Yang, T.; Park, C.S.; Park, S.G.; Chung, S.; Shim, J.H.; Chun, E.; Lee, K.Y. Cereblon negatively regulates TLR4 signaling through the attenuation of ubiquitination of TRAF6. Cell Death Dis., 2016, 7, e2313.
[382]
Messingerova, L.; Jonasova, A.; Barancik, M.; Poleková, L.; Šereš, M.; Gibalová, L.; Breier, A.; Sulová, Z. Lenalidomide treatment induced the normalization of marker protein levels in blood plasma of patients with 5q-myelodysplastic syndrome. Gen. Physiol. Biophys., 2015, 34, 399-406.
[383]
Fang, J.; Liu, X.; Bolanos, L.; Barker, B.; Rigolino, C.; Cortelezzi, A.; Oliva, E.N.; Cuzzola, M.; Grimes, H.L.; Fontanillo, C.; Komurov, K.; MacBeth, K.; Starczynowski, D.T. A calcium and calpain-dependent pathway determines the response to lenalidomide in myelodysplastic syndromes. Nat. Med., 2016, 22, 727-734.
[384]
Schecter, J.; Galili, N.; Raza, A. MDS: Refining existing therapy through improved biologic insights. Blood Rev., 2012, 26, 73-80.
[385]
Savic, A.; Cemerikic-Martinovic, V.; Dovat, S.; Rajic, N.; Urosevic, I.; Sekulic, B.; Kvrgic, V.; Popovic, S. Angiogenesis and survival in patients with myelodysplastic syndrome. Pathol. Oncol. Res., 2012, 18, 681-690.
[386]
Pardanani, A.; Finke, C.; Lasho, T.L.; Al-Kali, A.; Begna, K.H.; Hanson, C.A.; Tefferi, A. IPSS-independent prognostic value of plasma CXCL10, IL-7 and IL-6 levels in myelodysplastic syndromes. Lekemia, 2012, 26, 693-699.
[387]
Kim, C.K.; Han, D.H.; Ji, Y.S.; Lee, M.S.; Min, C.W.; Park, S.K.; Kim, S.H.; Yun, J.; Kim, H.J.; Kim, K.H.; Lee, K.T.; Won, J.H.; Hong, D.S.; Kim, H.K. Biomarkers of angiogenesis as prognostic factors in myelodysplastic syndrome patients treated with hypomethylating agents. Leuk. Res., 2016, 50, 21-28.
[388]
Invernizzi, R.; Travaglino, E.; Della Porta, M.G. Vascular endothelial growth factor overexpression in myelodysplastic syndrome bone marrow cells: biological and clinical implications. Leuk. Lymphoma, 2017, 58, 1711-1720.
[389]
Dredge, K.; Maarriott, J.B.; Macdonald, C.D.; Man, H.W.; Chen, R.; Muller, G.W.; Stirling, D.; Dalgleish, A.G. Novel thalidomide analogues display anti-angiogenic activity independently of immunomodulatory effects. Br. J. Cancer, 2002, 87, 1166-1172.
[390]
Dredge, K.; Horsfall, R.; Robinson, S.P.; Zhang, L.H.; Lu, L.; Tang, Y.; Shirley, M.A.; Muller, G.; Schafer, P.; Stirling, D.; Dalgleish, A.G.; Bartlett, J.B. Orally administered lenalidomide (CC-5013) is anti-angiogenic in vivo and inhibits endothelial cell migration and Akt phosphorylation in vitro. Microvasc. Res., 2005, 69, 56-63.
[391]
Gandhi, A.K.; Kang, J.; Naziruddin, S.; Parton, A.; Schafer, P.H.; Stirling, D.I. Lenalidomide inhibits proliferation of Namalwa CSN.70 cells and interferes with Gab1 phosphorylation and adaptor protein complex assembly. Leuk. Res., 2006, 30, 849-858.
[392]
Lu, L.; Payvandi, F.; Wu, L.; Zhang, L.H.; Hariri, R.J.; Man, H.W.; Chen, R.S.; Muller, G.W.; Hughes, C.C.; Stirling, D.I.; Schafer, P.H.; Bartlett, J.B. The anti-cancer drug lenalidomide inhibits angiogenesis and metastasis via multiple inhibitory effects on endothelial cell function in normoxic and hypoxic conditions. Microvasc. Res., 2009, 77, 78-86.
[393]
Buesche, G.; Dieck, S.; Giagounidis, A.; Göhring, G.; Schlegelberger, B.; Knight, R.; Aul, C.; Kreipe, H. Anti-angiogenic in-vivo
effect of lenalidomide and its impact on neoplastic and nonneoplastic
hematopoiesis in MDS with del(5q) chromosome abnormality.
Blood, 2009, 114, ASH Meeting Abstract 3800.
[394]
Goode, B.; Eck, M.J. Mechanism and function of formins in the control of actin assembly. Annu. Rev. Biochem., 2007, 76, 593-627.
[395]
Aspenström, P. Formin-binding proteins: modulators of formin-dependent actin polymerization. Biochim. Biophys. Acta, 2010, 1803, 174-182.
[396]
Peng, J.; Kitchen, S.M.; West, R.A.; Sigler, R.; Eisenmann, K.M.; Alberts, A.S. Myeloproliferative defects following targeting of the Drf1 gene encoding the mammalian diaphanous related formin mDia1. Cancer Res., 2007, 67, 7565-7571.
[397]
Eisenmann, K.M.; West, R.A.; Hildebrand, K.; Kitchen, S.M.; Peng, J.; Sigler, R.; Zhang, J.; Siminovitch, K.A.; Alberts, A.S. T cell responses in mammalian diaphanous-related formin mDia1 knock.out mice. J. Biol. Chem., 2007, 282, 25152-25158.
[398]
DeWard, A.D.; Leali, K.; West, R.A.; Prendergast, G.C.; Alberts, A.S. Loss of RhoB expression enhances the myelodysplastic phenotype of mammalian diaphanous-related formin mDia1 knockout mice. PLoS One, 2009, 4, e7102.
[399]
Ximeri, M.; Galanopoulos, A.; Klaus, M.; Parcharidou, A.; Giannikou, K.; Psyllaki, M.; Symeonidis, A.; Pappa, V.; Kartasis, Z.; Liapi, D.; Hatzimichael, E.; Kokoris, S.; Korkolopoulou, P.; Sambani, C.; Pontikoglou, C.; Papadaki, H.A. Hellenic MDS Study Group. Effect of lenalidomide therapy on hematopoiesis of patients with myelodysplastic syndrome associated with chromosome 5q deletion. Haematologica, 2010, 95, 406-414.
[400]
Sardnal, V.; Rouquette, A.; Kaltenbach, S.; Bally, C.; Chesnais, V.; Leschi, C.; Ades, L.; Santini, V.; Park, S.; Toma, A.; Fenaux, P.; Dreyfus, F.; Fontenay, M.; Kosmider, O.A. G polymorphism in the CRBN gene acts as a biomarker of response to treatment with lenalidomide in low/int-1 risk MDS without del(5q). Leukemia, 2013, 27, 1610-1613.
[401]
Jerez, A.; Gondek, L.P.; Jankowska, A.M.; Makishima, H.; Przychodzen, B.; Tiu, R.V.; O’Keefe, C.L.; Mohamedali, A.M.; Batista, D.; Sekeres, M.A.; McDevitt, M.A.; Mufti, G.J.; Maciejewski, J.P. Topography, clinical, and genomic correlates of 5q myeloid malignancies revisited. J. Clin. Oncol., 2012, 30, 1343-1349.
[402]
Brezinova, J.; Zemanova, Z.; Bystricka, D.; Sarova, I.; Lizcova, L.; Malinova, E.; Izakova, S.; Sajdova, J.; Sponerova, D.; Jonasova, A.; Cermak, J.; Michalova, K. Deletion of the long arm nut not the 5q31 region of chromosome 5 in myeloid malignancies. Leuk. Res., 2012, 36, e43-e45.
[403]
Mallo, M.; del Rey, M.; Ibánez, M.; Calasanz, M.J.; Arenillas, L.; Larráyoz, M.J.; Pedro, C.; Jerez, A.; Maciejewski, J.; Costa, D.; Nomdedeu, M.; Diez-Campelo, M.; Lumbreras, E.; González-Martínez, T.; Marugán, I.; Such, E.; Cervera, J.; Cigudosa, J.C.; Alvarez, S.; Florensa, L.; Hernández, J.M.; Solé, F. Response to lenalidomide in myelodysplastic syndromes with del(5q): influence of cytogenetics and mutations. Br. J. Haematol., 2013, 162, 74-86.
[404]
Zemanova, Z.; Michalova, K.; Buryova, H.; Brezinova, J.; Kostylkova, K.; Bystricka, D.; Novakova, M.; Sarova, I.; Izakova, S.; Lizcova, L.; Ransdorfova, S.; Krejcik, Z.; Merkerova, M.D.; Dohnalova, A.; Siskova, M.; Jonasova, A.; Neuwirtova, R.; Cermak, J. Involvement of deleted chromosome 5 in complex chromosomal aberrations in newly diagnosed myelodysplastic syndromes (MDS) is correlated with extremely adverse prognosis. Leuk. Res., 2014, 38, 537-544.
[405]
Volkert, S.; Kohlmann, A.; Schnittger, S.; Kern, W.; Haferlach, T.; Haferlach, C. Association of the type of 5q loss with complex karyotype, clonal evolution, TP53 mutation status, and prognosis in acute myeloid leukemia and myelodysplastic syndrome. Genes Chromosomes Cancer, 2014, 53, 402-410.
[406]
Schneider, R.K.; Ademà, V.; Heckl, D.; Järås, M.; Mallo, M.; Lord, A.M.; Chu, L.P.; McConkey, M.E.; Kramann, R.; Mullally, A.; Bejar, R.; Solé, F.; Ebert, B.L. Role of casein kinase 1A1 in the biology and targeted therapy of del(5q) MDS. Cancer Cell, 2014, 26, 509-520.
[407]
Woll, P.S.; Kjallquist, U.; Chowdhury, O.; Doolittle, H.; Wedge, D.C.; Thongjuea, S.; Erlandsson, R.; Ngara, M.; Anderson, K.; Deng, Q.; Mead, A.J.; Stenson, L.; Giustacchini, A.; Duarte, S.; Giannoulatou, E.; Taylor, S.; Karimi, M.; Scharenberg, C.; Mortera-Blanco, T.; Macaulay, I.C.; Clark, S.A.; Dybedal, I.; Josefsen, D.; Fenaux, P.; Hokland, P.; Holm, M.S.; Cazzola, M.; Malcovati, L.; Tauro, S.; Bowen, D.; Boultwood, J.; Pellagatti, A.; Pimanda, J.E.; Unnikrishnan, A.; Vyas, P.; Göhring, G.; Schlegelberger, B.; Tobiasson, M.; Kvalheim, G.; Constantinescu, S.N.; Nerlov, C.; Nilsson, L.; Campbell, P.J.; Sandberg, R.; Papaemmanuil, E.; Hellström-Lindberg, E.; Linnarsson, S.; Jacobsen, S.E. Myelodysplastic syndromes are propagated by rare and distinct human cancer stem cells in vivo. Cancer Cell, 2014, 25, 794-808.
[408]
Heuser, M.; Meggendorfer, M.; Cruz, M.M.A.; Fabisch, J.; Klesse, S.; Köhler, L.; Göhring, G.; Ganster, C.; Shirneshan, K.; Gutermuth, A.; Cerny-Reiterer, S.; Krönke, J.; Panagiota, V.; Haferlach, C.; Koenecke, C.; Platzbecker, U.; Thiede, C.; Schroeder, T.; Kobbe, G.; Ehrlich, S.; Stamer, K.; Döhner, K.; Valent, P.; Schlegelberger, B.; Kroeger, N.; Ganser, A.; Haase, D.; Haferlach, T.; Thol, F. Frequency and prognostic impact of casein kinase 1A1 mutations in MDS patients with deletion of chromosome 5q. Leukemia, 2015, 29, 1942-1945.
[409]
Bello, E.; Pellagatti, A.; Shaw, J.; Mecucci, C.; Kušec, R.; Killick, S.; Giagounidis, A.; Raynaud, S.; Calasanz, M.J.; Fenaux, P.; Boultwood, J. CSNK1A1 mutations and gene expression analysis in myelodysplastic syndromes with del(5q). Br. J. Haematol., 2015, 171, 210-214.
[410]
Saft, L.; Karimi, M.; Ghaderi, M.; Matolcsy, A.; Mufti, G.J.; Kulasekararaj, A.; Göhring, G.; Giagounidis, A.; Selleslag, D.; Muus, P.; Sanz, G.; Mittelman, M.; Bowen, D.; Porwit, A.; Fu, T.; Backstrom, J.; Fenaux, P.; MacBeth, K.J.; Hellström-Lindberg, E. p53 protein expression independently predicts outcome in patients with lower-risk myelodysplastic syndromes with del(5q). Haematologica, 2014, 99, 1041-1049.
[411]
Bally, C.; Renneville, A.; Preudhomme, C.; Legrand, M.; Adès, L.; de Thé, H.; Fenaux, P.; Lehmann-Che, J. Comparison of TP53 mutations screening by functional assay of separated allele in yeast and next-generation sequencing in myelodysplastic syndromes. Leuk. Res., 2015, 39, 1214-1219.
[412]
McGraw, K.L.; Zhang, L.M.; Rollison, D.E.; Basiorka, A.A.; Fulp, W.; Rawal, B.; Jerez, A.; Billingsley, D.L.; Lin, H.Y.; Kurtin, S.E.; Yoder, S.; Zhang, Y.; Guinta, K.; Mallo, M.; Solé, F.; Calasanz, M.J.; Cervera, J.; Such, E.; González, T.; Nevill, T.J.; Haferlach, T.; Smith, A.E.; Kulasekararaj, A.; Mufti, G.; Karsan, A.; Maciejewski, J.P.; Sokol, L.; Epling-Burnette, P.K.; Wei, S.; List, A.F. The relationship of TP53 R72P polymorphism to disease outcome and TP53 mutation in myelodysplastic syndromes. Blood Cancer J., 2015, 5, e291.
[413]
Loghavi, S.; Al-Ibrahaemi, A.; Zuo, Z.; Garcia-Manero, G.; Yabe, M.; Wang, S.A.; Kantarjian, H.M.; Yin, C.C.; Miranda, R.N.; Luthra, R.; Medeiros, L.J.; Bueso-Ramos, C.E.; Khoury, J.D. TP53 overexpression is an independent adverse prognostic factor in de novo myelodysplastic syndromes with fibrosis. Br. J. Haematol., 2015, 171, 91-99.
[414]
Zhang, L.; McGraw, K.L.; Sallman, D.A.; List, A.F. The role of p53 in myelodysplastic syndromes and acute myeloid leukemia: molecular aspects and clinical implications. Leuk. Lymphoma, 2017, 58, 1777-1790.
[415]
Prebet, T.; Cluzeau, T.; Park, S.; Sekeres, M.A.; Germing, U.; Ades, L.; Platzbecker, U.; Gotze, K.; Vey, N.; Oliva, E.; Sugrue, M.M.; Bally, C.; Kelaidi, C.; Al Ali, N.; Fenaux, P.; Gore, S.D.; Komrokji, R. Outcome of patients treated for myelodysplastic syndromes with 5q deletion after failure of lenalidomide therapy. Oncotarget, 2017, 8, 81926-81935.
[416]
Mallo, M.; Cervera, J.; Schanz, J.; Such, E.; García-Manero, G.; Luño, E.; Steidl, C.; Espinet, B.; Vallespí, T.; Germing, U.; Blum, S.; Ohyashiki, K.; Grau, J.; Pfeilstöcker, M.; Hernández, J.M.; Noesslinger, T.; Giagounidis, A.; Aul, C.; Calasanz, M.J.; Martín, M.L.; Valent, P.; Collado, R.; Haferlach, C.; Fonatsch, C.; Lübbert, M.; Stauder, R.; Hildebrandt, B.; Krieger, O.; Pedro, C.; Arenillas, L.; Sanz, M.Á.; Valencia, A.; Florensa, L.; Sanz, G.F.; Haase, D.; Solé, F. Impact of adjunct cytogenetic abnormalities for prognostic stratification in patients with myelodysplastic syndrome and deletion 5q. Leukemia, 2011, 25, 110-120.
[417]
Jonasova, A.; Cermak, J.; Vondrakova, J.; Siskova, M.; Hochova, I.; Kadlckova, E.; Cerna, O.; Sykora, M.; Vozobulova, V.; Seifertova, N.; Michalova, K.; Zemanova, Z.; Brezinova, J.; Belohlavkova, P.; Kostecka, A.; Neuwirtova, R. Thrombocytopenia at diagnosis as an important negative prognostic marker in isolated 5q- MDS (IPSS low and intermediate-1). Leuk. Res., 2012, 36, e222-e224.
[418]
Sugimoto, Y.; Sekeres, M.A.; Makishima, H.; Traina, F.; Visconte, V.; Jankowska, A.; Jerez, A.; Szpurka, H.; O’Keefe, C.L.; Guinta, K.; Afable, M.; Tiu, R.; McGraw, K.L.; List, A.F.; Maciejewski, J. Cytogenetic and molecular predictors of response in patients with myeloid malignancies without del(5q) treated with lenalidomide. J. Hematol. Oncol., 2012, 5, 4.
[419]
Chesnais, V.; Renneville, A.; Toma, A.; Lambert, J.; Passet, M.; Dumont, F.; Chevret, S.; Lejeune, J.; Raimbault, A.; Stamatoullas, A.; Rose, C.; Beyne-Rauzy, O.; Delaunay, J.; Solary, E.; Fenaux, P.; Dreyfus, F.; Preudhomme, C.; Kosmider, O.; Fontenay, M. Groupe Francophone des Myélodysplasies. Effect of lenalidomide treatment on clonal architecture of myelodysplastic syndromes without 5q deletion. Blood, 2016, 127, 749-760.
[420]
Giagounidis, A.A. Where Does Lenalidomide Fit in Non-del(5q) MDS? Curr. Hematol. Malig. Rep., 2015, 10, 303-308.
[421]
Fehninger, T.A.; Byrd, J.C.; Marcucci, G.; Abboud, C.N.; Kefauver, C.; Payton, J.E.; Vij, R.; Blum, W. Single-agent lenalidomide induces complete remission of acute myeloid leukemia in patients with isolated trisomy 13. Blood, 2009, 113, 1002-1005.
[422]
Blum, W.; Klisovic, R.B.; Becker, H.; Yang, X.; Rozewski, D.M.; Phelps, M.A.; Garzon, R.; Walker, A.; Chandler, J.C.; Whitman, S.P.; Curfman, J.; Liu, S.; Schaaf, L.; Mickle, J.; Kefauver, C.; Devine, S.M.; Grever, M.R.; Marcucci, G.; Byrd, J.C. Dose escalation of lenalidomide in relapsed or refractory acute leukemias. J. Clin. Oncol., 2010, 28, 4919-4925.
[423]
Fehninger, T.A.; Uy, G.L.; Trinkaus, K.; Nelson, A.D.; Demland, J.; Abboud, C.N.; Cashen, A.F.; Stockerl-Goldstein, K.E.; Westervelt, P.; DiPersio, J.F.; Vij, R. A phase 2 study of high-dose lenalidomide as initial therapy for older patients with acute myeloid leukemia. Blood, 2011, 117, 1828-1833.
[424]
Sekeres, M.A.; Gundacker, H.; Lancet, J.; Advani, A.; Petersdorf, S.; Liesveld, J.; Mulford, D.; Norwood, T.; Willman, C.L.; Appelbaum, F.R.; List, A.F. A phase 2 study of lenalidomide monotherapy in patients with deletion 5q acute myeloid leukemia: SWOG Study S0605. Blood, 2011, 118, 523-528.
[425]
Chen, Y.; Kantarjian, H.; Estrov, Z.; Faderl, S.; Ravandi, F.; Rey, K.; Cortes, J.; Borthakur, G. A phase II study of lenalidomide alone in relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes with chromosome 5 abnormalities. Clin. Lymphoma Myeloma Leuk., 2012, 12, 341-344.
[426]
Steensma, D.P.; Stone, R.M. Lenalidomide in AML: Del(5q) or who? Blood, 2011, 118, 481-482.
[427]
Ades, L.; Prebet, T.; Stamatoullas, A.; Recher, C.; Guieze, R.; Raffoux, E.; Bouabdallah, K.; Hunault, M.; Wattel, E.; Stalnikiewicz, L.; Toma, A.; Dombret, H.; Vey, N.; Sebert, M.; Gardin, C.; Chaffaut, C.; Chevret, S.; Fenaux, P. Lenalidomide (LEN) combined to intensive chemotherapy (IC) in AML and higher risk MDS with del 5q. Results of a phase II study of the Groupe Francophone des Myelodysplasies. Haematologica, 2017, 102, 728-735.
[428]
DeAngelo, D.J.; Brunner, A.M.; Werner, L.; Avigan, D.; Fathi, A.T.; Sperling, A.S.; Washington, A.; Stroopinsky, D.; Rosenblatt, J.; McMasters, M.; Luptakova, K.; Wadleigh, M.; Steensma, D.P.; Hobbs, G.S.; Attar, E.C.; Amrein, P.C.; Ebert, B.L.; Stone, R.M.; Ballen, K.K. A phase I study of lenalidomide plus chemotherapy with mitoxantrone, etoposide, and cytarabine for the reinduction of patients with acute myeloid leukemia. Am. J. Hematol., 2018, 93, 254-261.
[429]
Higgins, J.J.; Rosen, D.R.; Loveless, J.M.; Clyman, J.C.; Grau, M.J. A gene for nonsyndromic mental retardation maps to chromosome 3p25-pter. Neurology, 2000, 55, 335-340.
[430]
Higgins, J.J.; Pucilowska, J.; Lombardi, R.Q.; Rooney, J.P. A mutation in a novel ATP-dependent Lon protease gene in a kindred with mild mental retardation. Neurology, 2004, 63, 1927-1931.
[431]
Carling, D. The AMP-activated protein kinase cascade-a unifying system for energy control. Trends Biochem. Sci., 2004, 29, 18-24.
[432]
Lage, R.; Diéguez, C.; Vidal-Puig, A.; López, M. AMPK: a metabolic gauge regulating whole-body energy homeostasis. Trends Mol. Med., 2008, 14, 539-549.
[433]
Hardie, D.G.; Ashford, M.L. AMPK: Regulating energy balance at the cellular and whole body levels. Physiology (Bethesda), 2014, 29, 99-107.
[434]
Steinberg, G.R.; Kemp, B.E. AMPK in health and disease. Physiol. Rev., 2009, 89, 1025-1078.
[435]
Lee, K.M.; Jo, S.; Kim, H.; Lee, J.; Park, C.S. Functional modulation of AMP-activated protein kinase by cereblon. Biochim. Biophys. Acta, 2011, 1813, 448-455.
[436]
Lee, K.M.; Yang, S.J.; Kim, Y.D.; Choi, Y.D.; Nam, J.H.; Choi, C.S.; Choi, H.S.; Park, C.S. Disruption of the cereblon gene enhances hepatic AMPK activity and prevents high-fat diet-induced obesity and insulin resistance in mice. Diabetes, 2013, 62, 1855-1864.
[437]
Lee, K.M.; Yang, S.J.; Choi, J.H.; Park, C.S. Functional effects of a pathogenic mutation in Cereblon (CRBN) on the regulation of protein synthesis via the AMPK-mTOR cascade. J. Biol. Chem., 2014, 289, 23343-23352.
[438]
Bavley, C.C.; Rice, R.C.; Fischer, D.K.; Fakira, A.K.; Byrne, M.; Kosovsky, M.; Rizzo, B.K.; Del Prete, D.; Alaedini, A.; Morón, J.A.; Higgins, J.J.; D’Adamio, L.; Rajadhyaksha, A.M. Rescue of Learning and Memory Deficits in the Human Nonsyndromic Intellectual DisabilityCereblon Knock-Out Mouse Model by Targeting the AMP-Activated Protein Kinase-mTORC1 Translational Pathway. J. Neurosci., 2018, 38, 2780-2795.
[439]
Sawamura, N.; Yamada, M.; Fujiwara, M.; Yamada, H.; Hayashi, H.; Takagi, N.; Asahi, T. The Neuroprotective Effect of Thalidomide against Ischemia through the Cereblon-mediated Repression of AMPK Activity. Sci. Rep., 2018, 8, 2459.
[440]
Gil, M.; Kim, Y.K.; Kim, H.Y.; Pak, H.K.; Park, C.S.; Lee, K.J. Cereblon deficiency confers resistance against polymicrobial sepsis by the activation of AMP activated protein kinase and heme-oxygenase-1. Biochem. Biophys. Res. Commun., 2018, 495, 976-981.
[441]
Koirala, S.; Potts, P.R. An Acetyldegron Triggers CRBN to Take Down the “Q”. Mol. Cell, 2016, 61, 795-796.
[442]
Nguyen, T.V.; Lee, J.E.; Sweredoski, M.; Yang, S.J.; Jeon, S.J.; Harrison, J.S.; Yim, J.H.; Lee, S.G.; Handa, H.; Kuhlman, B.; Jeong, J.S.; Reitsma, J.M.; Park, C.S.; Hess, S.; Deshaies, R.J. Glutamine Triggers Acetylation-Dependent Degradation of Glutamine Synthetase via the Thalidomide Receptor Cereblon. Mol. Cell, 2016, 61, 809-820.
[443]
Ross, C.A.; Poirier, M.A. Protein aggregation and neurodegenerative disease. Nat. Med., 2004, 10, S10-S17.
[444]
Ciechanover, A.; Kwon, Y.T. Degradation of misfolded proteins in neurodegenerative diseases: therapeutic targets and strategies. Exp. Mol. Med., 2015, 47, e147.
[445]
Zhou, L.; Hao, Z.; Wang, G.; Xu, G. Cereblon suppresses the formation of pathogenic protein aggregates in a p62-dependent manner. Hum. Mol. Genet., 2018, 27, 667-678.
[446]
Hagner, P.R.; Man, H.W.; Fontanillo, C.; Wang, M.; Couto, S.; Breider, M.; Bjorklund, C.; Havens, C.G.; Lu, G.; Rychak, E.; Raymon, H.; Narla, R.K.; Barnes, L.; Khambatta, G.; Chiu, H.; Kosek, J.; Kang, J.; Amantangelo, M.D.; Waldman, M.; Lopez-Girona, A.; Cai, T.; Pourdehnad, M.; Trotter, M.; Daniel, T.O.; Schafer, P.H.; Klippel, A.; Thakurta, A.; Chopra, R.; Gandhi, A.K. CC-122, a pleiotropic pathway modifier, mimics an interferon response and has antitumor activity in DLBCL. Blood, 2015, 126, 779-789.
[447]
Cubillos-Zapata, C.; Cordoba, R.; Avendaño-Ortiz, J.; Arribas-Jiménez, C.; Hernández-Jiménez, E.; Toledano, V.; Villaescusa, T.; Moreno, V.; López-Collazo, E. CC-122 immunomodulatory effects in refractory patients with diffuse large B-cell lymphoma. OncoImmunology, 2016, 5, e1231290.
[448]
Nakayama, Y.; Kosek, J.; Capone, L.; Hur, E.M.; Schafer, P.H.; Ringheim, G.E. Aiolos Overexpression in Systemic Lupus Erythematosus B Cell Subtypes and BAFF-Induced Memory B Cell Differentiation Are Reduced by CC-220 Modulation of Cereblon Activity. J. Immunol., 2017, 199, 2388-2407.
[449]
Matyskiela, M.E.; Zhang, W.; Man, H.W.; Muller, G.; Khambatta, G.; Baculi, F.; Hickman, M.; LeBrun, L.; Pagarigan, B.; Carmel, G.; Lu, C.C.; Lu, G.; Riley, M.; Satoh, Y.; Schafer, P. Daniel. T.O.; Carmichael, J.; Cathers, B.E.; Chamberlain, P.P. A Cereblon Modulator (CC-220) with Improved Degradation of Ikaros and Aiolos. J. Med. Chem., 2018, 61, 535-542.
[450]
Matyskiela, M.E.; Lu, G.; Ito, T.; Pagarigan, B.; Lu, C.C.; Miller, K.; Fang, W.; Wang, N.Y.; Nguyen, D.; Houston, J.; Carmel, G.; Tran, T.; Riley, M.; Nosaka, L.; Lander, G.C.; Gaidarova, S.; Xu, S.; Ruchelman, A.L.; Handa, H.; Carmichael, J.; Daniel, T.O.; Cathers, B.E.; Lopez-Girona, A.; Chamberlain, P.P. A novel cereblon modulator recruits GSPT1 to the CRL4(CRBN) ubiquitin ligase. Nature, 2016, 535, 252-257.
[451]
Jelinek, T.; Hajek, R. PD-1/PD-L1 inhibitors in multiple myeloma: The present and the future. OncoImmunology, 2016, 5, e1254856.
[452]
Chung, C. Role of Immunotherapy in Targeting the Bone Marrow Microenvironment in Multiple Myeloma: An Evolving Therapeutic Strategy. Pharmacotherapy, 2017, 37, 129-143.
[453]
Gay, F.; D’Agostino, M.; Giaccone, L.; Genuardi, M.; Festuccia, M.; Boccadoro, M.; Bruno, B. Immuno-oncologic approaches: CAR-T cells and checkpoint inhibitors. Clin. Lymphoma Myeloma Leuk., 2017, 17, 471-478.
[454]
Nguyen-Pham, T.N.; Jung, S.H.; Vo, M.C.; Thanh-Tran, H.T.; Lee, Y.K.; Lee, H.J.; Choi, N.R.; Hoang, M.D.; Kim, H.J.; Lee, J.J. Lenalidomide Synergistically Enhances the Effect of Dendritic Cell Vaccination in a Model of Murine Multiple Myeloma. J. Immunother., 2015, 38, 330-339.
[455]
Görgűn, G.; Samur, M.K.; Cowens, K.B.; Paula, S.; Bianchi, G.; Anderson, J.E.; White, R.E.; Singh, A.; Ohguchi, H.; Suzuki, R.; Kikuchi, S.; Harada, T.; Hideshima, T.; Tai, Y.T.; Laubach, J.P.; Raje, N.; Magrangeas, F.; Minvielle, S.; Avet-Loiseau, H.; Munshi, N.C.; Dorfman, D.M.; Richardson, P.G.; Anderson, K.C. Lenalidomide enhances immune checkpoint blockade-induced immune response in multiple myeloma. Clin. Cancer Res., 2015, 21, 4607-4618.
[456]
Tremblay-LeMay, R.; Rastgoo, N.; Chang, H. Modulating PD-L1 expression in multiple myeloma: an alternative strategy to target the PD-1/PD-L1 pathway. J. Hematol. Oncol., 2018, 11, 46.
[457]
Wang, X.; Walter, M.; Urak, R.; Weng, L.; Huynh, C.; Lim, L.; Wong, C.W.; Chang, W.C.; Thomas, S.H.; Sanchez, J.F.; Yang, L.; Brown, C.E.; Pichiorri, F. Htut. M.; Krishnan, A.Y.; Forman, S.J. Lenalidomide Enhances the Function of CS1 Chimeric Antigen Receptor-Redirected T Cells Against Multiple Myeloma. Clin. Cancer Res., 2018, 24, 106-119.
[459]
Tan, M.; Fong, R.; Lo, M.; Young, R. Lenalidomide and secondary acute lymphoblastic leukemia: a case series. Hematol. Oncol., 2017, 35, 130-134.
[460]
Ying, L. YinHui, T.; Yunliang, Z.; Sun, H. Lenalidomide and the risk of serious infection in patients with multiple myeloma: a systematic review and meta-analysis. Oncotarget, 2017, 8, 46593-46600.
[461]
Chen, M.; Zhao, Y.; Xu, C.; Wang, X.; Zhang, X.; Mao, B. Immunomodulatory drugs and the risk of serious infection in multiple myeloma: systematic review and meta-analysis of randomized and observational studies. Ann. Hematol., 2018, 97(6), 925-944.
[462]
Mauro, F.R.; Foà, R. Gene mutations in lenalidomide-treated CLL. Gene mutations in lenalidomide-treated CLL. Blood, 2018, 131, 1769-1771.
[463]
Takahashi, K.; Hu, B.; Wang, F.; Yan, Y.; Kim, E.; Vitale, C.; Patel, K.P.; Strati, P.; Gumbs, C.; Little, L.; Tippen, S.; Song, X.; Zhang, J.; Jain, N.; Thompson, P.; Garcia-Manero, G.; Kantarjian, H.; Estrov, Z.; Do, K.A.; Keating, M.; Burger, J.A.; Wierda, W.G.; Futreal, P.A.; Ferrajoli, A. Clinical implications of cancer gene mutations in patients with chronic lymphocytic leukemia treated with lenalidomide. Blood, 2018, 131, 1820-1832.
[464]
Ren, Y.; Wang, M.; Couto, S.; Hansel, D.E.; Miller, K.; Lopez-Girona, A.; Bjorklund, C.C.; Gandhi, A.K.; Thakurta, A.; Chopra, R.; Breider, M. A dual color immunohistochemistry assay for measurement of cereblon in multiple myeloma patient samples. Appl. Immunohistochem. Mol. Morphol., 2016, 24, 695-702.
[465]
Blommestein, H.M.; Armstrong, N.; Ryder, S.; Deshpande, S.; Worthy, G.; Noake, C.; Riemsma, R.; Kleijnen, J.; Severens, J.L.; Al, M.J. Lenalidomide for the treatnent of low- or intermediate-1-risk myelodysplastic syndromes associated with deletion 5q cytogenetic abnormality: an evidence review of the NICE submission from Celgene. Pharmacoeconomics, 2016, 34, 23-31.
[466]
Rajkumar, S.V.; Harousseau, J.L. Next-generation multiple myeloma treatment: a pharmacoeconomic perspective. Blood, 2016, 128, 2757-2764.
[467]
Santini, V.; Almeida, A.; Giagounidis, A.; Platzbecker, U.; Buckstein, R.; Beach, C.L.; Guo, S.; Altincatal, A.; Wu, C.; Fenaux, P. The effect of lenalidomide on health-related quality of life in patients with lower-risk non-del(5q) myelodysplastic syndromes: results from the MDS-005 study. Clin. Lymphoma Myeloma Leuk., 2018, 18, 136-144.
[468]
Almeida, A.; Fenaux, P.; Garcia-Manero, G.; Goldberg, S.L.; Gröpper, S.; Jonasova, A.; Vey, N.; Castaneda, C.; Zhong, J.; Beach, C.L.; Santini, V. Safety profile of lenalidomide in patients
with lower-risk myelodysplastic syndromes without del(5q): results
of a phase 3 trial. Leuk. Lymphoma, 2018, doi: 10.1080/10428194.
2017.1421758. [Epub ahead of print]
