Curative treatment for severe sickle cell disease: allogeneic transplantation.
Sickle cell disease is an inherited hematologic disorder that in its severe form can result in substantial morbidity and early mortality. Patients with this disorder can suffer from severe pain, lung disease, and strokes, resulting in chronic debilitating conditions, end organ dysfunction, and organ failure. The health care costs of caring for these chronically ill patients are substantial. Allogeneic transplantation is a modality that has the potential to cure these patients. To date, matched sibling donor transplantation is widely accepted as a standard of care for pediatric patients. Utilizing alternative donors for transplant is still under investigation, as is transplant for adult patients with sickle cell disease. This review focuses on the most recent data for hematopoietic cell transplantation for patients with sickle cell disease.
All Children's Hospital, St. Petersburg, Florida.
Benjamin
B
Oshrine
Medical College of Wisconsin, Milwaukee, Wisconsin.
Julie-An
JA
Talano
D000755:Anemia, Sickle Cell / Q000628:therapy*; D036101:Cord Blood Stem Cell Transplantation; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D035781:Siblings; D014019:Tissue Donors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous