Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality.
Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality.
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DOI：
10.1182/blood.v48.1.63.63
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发表时间：
1976-07
期刊：
影响因子：
20.3
通讯作者：
B. Camitta;E. Thomas;D. Nathan;G. Santos;E. Gordon-Smith;R. Gale;J. Rappeport;R. Storb
中科院分区：
文献类型：
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作者：
B. Camitta;E. Thomas;D. Nathan;G. Santos;E. Gordon-Smith;R. Gale;J. Rappeport;R. Storb
本研究采用前瞻性随机对照研究方法，比较早期骨髓移植与常规治疗重型再生障碍性贫血的疗效。所有在主要组织相容性区域有同胞匹配的患者都进行了移植。在最初诊断后17-100天（中位数33天）内进行移植。常规治疗包括输血支持，有或没有雄激素。移植组36例患者中有24例在4-20个月（中位数9个月）后存活，完全骨髓重建。只有两种受慢性移植物抗宿主病的限制。相比之下，31例常规治疗的患者中只有12例存活。这些幸存者中有6人有所改善，5人不完全。19例非移植死亡发生在诊断后1-11（中位数3）个月内。与非移植方案相比，早期移植能更有效地恢复正常骨髓功能，降低严重骨髓再生障碍性贫血的急性死亡率（p = 0.006）。在长期随访中，早期骨髓移植似乎是治疗重型再生障碍性贫血最有效的方法。
A prospective randomized trial of therapy for severe aplastic anemia was designed to compare early bone marrow transplantation with conventional treatments. All patients with a sibling matched at the major histocompatibility region were transplanted. Transplantation was performed with 17-100 (median 33) days of original diagnosis. Conventional treatments included transfusion support with or without androgens. Twenty-four of 36 patients intered on the transplant arm are alive after 4-20 (median 9) mo with full marrow reconstitution. Only two are limited by chronic graft-versus-host disease. In contrast only 12 of 31 conventionally treated patients are alive. Six of these survivors have improved, five incompletely. The 19 nontransplant deaths have occurred within 1-11 (median 3) mo of diagnosis. Compared to nontransplant regimens, early transplantation more effectively restores normal marrow function and decreases the acute mortality of severe marrow aplasia (p = 0.006). Pending longer follow-up, early marrow transplantation appears to be the most effective available treatment for severe aplastic anemia.