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Bone Marrow Transplantation for Sickle Cell Disease

1996; Massachusetts Medical Society; Volume: 335; Issue: 6 Linguagem: Inglês

10.1056/nejm199608083350601

1533-4406

Mark C. Walters, Melinda Patience, Wendy M. Leisenring, James R. Eckman, J. Paul Scott, William C. Mentzer, Sally C. Davies, Kwaku Ohene‐Frempong, Françoise Bernaudin, Dana C. Matthews, Rainer Storb, Keith M. Sullivan,

Myeloproliferative Neoplasms: Diagnosis and Treatment

We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.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.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.Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.