Title:Pharmacotherapy of Sickle Cell Disease in Children
Volume: 21
Issue: 39
Author(s): Kathleen A. Neville and Julie A. Panepinto
Affiliation:
Keywords:
Sickle cell disease, therapeutics, treatment, children.
Abstract: Sickle cell disease (SCD) is a potentially devastating and life threatening condition that is caused by an
autosomal recessive inherited hemoglobinopathy which results in vaso-occlusive phenomena and hemolysis. The
severity of this disorder is widely variable, but overall mortality is increased and life expectancy decreased when
compared to the general population.
Care of patients with sickle cell disease is largely supportive. In fact, hydroxyurea is the only drug used that modifies
disease pathogenesis. Painful vaso-occlusive events are the most common complication experienced by both
children and adults with sickle cell disease and hydroxyurea is the only treatment option available to prevent the
development of these events. Most events are managed with traditional supportive care measures (i.e. aggressive hydration, antiinflammatory
and narcotic analgesics) that have not changed in decades. As such, there is an overwhelming need for both the development
of new agents and new approaches to treatment with existing modalities for patients with sickle cell disease.
