title: Stem cell transplantation for sickle cell disease: can we reduce the toxicity?
abstract: Since the genetic basis of sickle cell anemia was discovered over 50 years ago, many therapies have been developed for the treatment of this disorder. Hematopoietic cell transplantation offers curative potential, but it is associated with a 5-10% risk of dying. Patients who undergo allografting but develop stable donor-host hematopoietic chimerism appear to experience a significant clinical benefit. Our paper discusses the risks and benefits of hematopoietic cell transplantation in patients with sickle cell disease and summarizes the outcome of 147 patients who received allografts for sickle cell disease. We also review the development of new approaches to establish stable mixed chimerism after transplantation for sickle cell disease.
authors.affiliation: Department of Hematology and Oncology, Program in Blood and Marrow Transplantation, Children's Hospital Oakland, 747 Fifty-Second Street, Oakland, CA 94609-1809, USA.
authors.forename: J
authors.initials: J
authors.lastname: Fixler
authors.forename: E
authors.initials: E
authors.lastname: Vichinsky
authors.forename: M C
authors.initials: MC
authors.lastname: Walters
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000628:therapy*; D000818:Animals; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000401:mortality; D002543:Cerebral Hemorrhage / Q000209:etiology; D002648:Child; D004285:Dogs; D005260:Female; D005319:Fetal Hemoglobin / Q000096:biosynthesis; D005544:Forecasting; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality; D006801:Humans; D007246:Infertility / Q000209:etiology; D008297:Male; D015337:Multicenter Studies as Topic; D012306:Risk; D012640:Seizures / Q000209:etiology; D020521:Stroke / Q000517:prevention & control; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D005319:Fetal Hemoglobin