Experimental therapy

Abstract

Bone marrow transplantation, hydroxyurea therapy in children and in patients with sickling disorders other than sickle cell anemia, and prophylactic transfusion for prevention of stroke in children are currently being evaluated as treatments for patients with sickle cell disease. Long-term complications of each of these treatments are incompletely understood. Attempts to inhibit sickling by lowering intracellular hemoglobin concentration are still in progress. Combinations of therapeutic agents with different modes of action, and development of more effective treatment schedules, may further improve the outlook of patients with sickling disorders.