Bone marrow transplantation for sickle cell disease.
(Clinical Trial;Journal Article)
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.
Walters, MC; Patience, M; Leisenring, W; Eckman, JR; Scott, JP; Mentzer, WC; Davies, SC; Ohene-Frempong, K; Bernaudin, F; Matthews, DC; Storb, R; Sullivan, KM
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