Bone marrow transplantation for sickle cell disease

被引:434
作者
Walters, MC
Patience, M
Leisenring, W
Eckman, JR
Scott, JP
Mentzer, WC
Davies, SC
OheneFrempong, K
Bernaudin, F
Matthews, DC
Storb, R
Sullivan, KM
机构
[1] FRED HUTCHINSON CANC RES CTR, DIV CLIN RES, SEATTLE, WA 98104 USA
[2] UNIV WASHINGTON, DEPT PEDIAT, SEATTLE, WA 98195 USA
[3] UNIV WASHINGTON, DEPT MED, SEATTLE, WA 98195 USA
[4] EMORY UNIV, ATLANTA, GA 30322 USA
[5] MED COLL WISCONSIN, MILWAUKEE, WI 53226 USA
[6] UNIV CALIF SAN FRANCISCO, SAN FRANCISCO, CA 94143 USA
[7] CENT MIDDLESEX HOSP NATL HLTH SERV TRUST, LONDON, ENGLAND
[8] UNIV PENN, PHILADELPHIA, PA 19104 USA
[9] HOP HENRI MONDOR, F-94010 CRETEIL, FRANCE
关键词
D O I
10.1056/NEJM199608083350601
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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 engraft ment, there was stable mixed chimerism. Two patients died of central nervous system hemorrhage dor 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. (C) 1996, Massachusetts Medical Society.
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页码:369 / 376
页数:8
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