Bone marrow transplantation for sickle cell disease.
N Engl J Med
; 335(6): 369-76, 1996 Aug 08.
Article
em En
| MEDLINE
| ID: mdl-8663884
ABSTRACT
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.
Buscar no Google
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Medula Óssea
/
Transplante de Células-Tronco Hematopoéticas
/
Anemia Falciforme
Tipo de estudo:
Etiology_studies
Limite:
Adolescent
/
Child
/
Child, preschool
/
Female
/
Humans
/
Male
Idioma:
En
Revista:
N Engl J Med
Ano de publicação:
1996
Tipo de documento:
Article