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50例接受骨髓移植或免疫抑制治疗的多次输血的重型再生障碍性贫血患者的临床结果。

Clinical results in 50 multiply transfused patients with severe aplastic anemia treated with bone marrow transplantation or immunosuppressive therapy.

作者信息

Arranz R, Otero M J, Ramos R, Steegmann J L, Lamana M L, Tomás J F, de la Cámara R, Figuera A, Vázquez L, Fernádez-Rañada J M

机构信息

Department of Haematology, Hospital de la Princesa, Madrid, Spain.

出版信息

Bone Marrow Transplant. 1994 Apr;13(4):383-7.

PMID:8019461
Abstract

Fifty patients with aplastic anemia (AA) were treated with BMT or immunosuppressive therapy (IST). Twenty-one patients underwent BMT using cyclophosphamide (CY) and 7 Gy total lymphoid irradiation (TLI) and cyclosporin A (CsA) plus methotrexate (MTX). Actuarial survival is 71% at 5.3 years with an incidence of graft failure of 0% and of acute GVHD of 38.9%. Univariate analysis of variables influencing survival showed a trend for a poorer outcome in patients who received > 30 transfusions prior to BMT and in male recipients from female donors. Twenty-nine patients > 40 years of age or without matched siblings received antithymocyte/antilymphocyte globulin (ATG/ALG). Response rate to the first course of treatment was 46.4%. Subsequent courses of IST rescued 33% of patients who relapsed or had not responded. Actuarial survival is 62% at 8.6 years. In our experience both treatment strategies have given encouraging results although overall morbidity is higher in the IST group because 25% of patients are therapy or transfusion-dependent. The role of irradiation in the conditioning regimen of BMT patients, recently challenged, is discussed.

摘要

五十例再生障碍性贫血(AA)患者接受了骨髓移植(BMT)或免疫抑制治疗(IST)。21例患者采用环磷酰胺(CY)、7Gy全身淋巴照射(TLI)、环孢素A(CsA)加甲氨蝶呤(MTX)进行骨髓移植。5.3年时的实际生存率为71%,移植失败发生率为0%,急性移植物抗宿主病(GVHD)发生率为38.9%。对影响生存的变量进行单因素分析显示,在骨髓移植前接受超过30次输血的患者以及女性供体的男性受者中,预后较差的趋势明显。29例年龄大于40岁或无匹配同胞的患者接受了抗胸腺细胞/抗淋巴细胞球蛋白(ATG/ALG)治疗。第一疗程治疗的缓解率为46.4%。后续的免疫抑制治疗疗程挽救了33%复发或未缓解的患者。8.6年时的实际生存率为62%。根据我们的经验,尽管免疫抑制治疗组的总体发病率较高,因为25%的患者依赖治疗或输血,但两种治疗策略都取得了令人鼓舞的结果。本文还讨论了放疗在骨髓移植患者预处理方案中的作用,该作用最近受到了挑战。

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First-line allogeneic hematopoietic stem cell transplantation of HLA-matched sibling donors compared with first-line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia.与一线使用环孢素和/或抗胸腺细胞或抗淋巴细胞球蛋白治疗获得性重型再生障碍性贫血相比,HLA匹配的同胞供者进行一线异基因造血干细胞移植的情况。
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