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Volume 335:369-376 August 8, 1996 Number 6 Next

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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.

Source Information

From the Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle (M.C.W., M.P., W.L., D.C.M., R.S., K.M.S.); the Departments of Pediatrics (M.C.W., D.C.M.) and Medicine (R.S., K.M.S.), University of Washington, Seattle; Emory University, Atlanta (J.R.E.); the Medical College of Wisconsin, Milwaukee (J.P.S.); the University of California, San Francisco (W.C.M.); Central Middlesex Hospital National Health Service Trust, London (S.C.D.); the University of Pennsylvania, Philadelphia (K.O.-F.); and Hôpital Henri Mondor, Creteil, France (F.B.). Investigators and centers participating in this study are listed in the Appendix.Other contributing authors were George R. Buchanan, M.D., University of Texas Southwestern Medical Center, Dallas; Philip J. Darbyshire, M.B., Ch.B., Birmingham Children's Hospital National Health Service Trust, Birmingham, United Kingdom; Robertson Parkman, M.D., University of Southern California, Los Angeles; Roswitha Dickerhoff, M.D., University of Bonn, Augustin, Germany; Franklin O. Smith, M.D., Indiana University School of Medicine, Indianapolis; and Jean E. Sanders, M.D., Fred Hutchinson Cancer Research Center, Seattle.

Address reprint requests to Dr. Sullivan at the Fred Hutchinson Cancer Research Center, Division of Clinical Research, FB600, 1124 Columbia St., Seattle, WA 98104.

Full Text of this Article

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N Engl J Med 1996; 335:1845-1846, Dec 12, 1996. Correspondence

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