Bone marrow transplantation for sickle cell disease

N Engl J Med. 1996 Aug 8;335(6):369-76. doi: 10.1056/NEJM199608083350601.


Background: We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.

Methods: Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin.

Results: Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central nervous system hemorrhage or graft-versus-host disease. Kaplan-Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively. Among patients with a history of acute chest syndrome, lung function stabilized; among patients with prior central nervous system vasculopathy who had engraftment, stabilization of cerebrovascular disease was documented by magnetic resonance imaging.

Conclusions: Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.

Publication types

  • Clinical Trial
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / mortality
  • Anemia, Sickle Cell / therapy*
  • Bone Marrow Transplantation* / adverse effects
  • Cerebral Hemorrhage / etiology
  • Cerebral Hemorrhage / mortality
  • Cerebrovascular Disorders / etiology
  • Child
  • Child, Preschool
  • Female
  • Graft Rejection
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / mortality
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Male
  • Survival Analysis
  • Treatment Outcome
  • beta-Thalassemia / complications
  • beta-Thalassemia / therapy