Sickle cell disease

Sickle cell disease (SCD) is the most common inherited disorder of hemoglobin in the United States. The severity of SCD varies between individuals and between different locations of geographic origin. The most severe manifestations are found in patients with hemoglobin (Hb) SS and Sp° thalassemia and progress with age. Interventions such as penicillin prophylaxis, red cell transfusions, erythrocytapheresis, iron chelation, and hydroxyurea have revolutionized treatment and increased survival especially in children. The majority of transplants for SCD have been performed in children with human leukocyte antigen (HLA)-matched sibling donors (MSD) except for the NIH trial (SCURT - BMT CTN 0601). Adult transplant attempts from alternate donors have been otherwise limited by toxicity likely due to pre-existing disease related organ damage. Electrolyte imbalance due to renal tubular dysfunction can be expected to worsen with age and SCD related renal damage requiring close follow up and supplementation as needed.