High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up

被引：88

作者：

Brodsky, Robert A. ^{[1
,2
]}

Chen, Allen R. ^{[2
]}

Dorr, Donna ^{[1
]}

Fuchs, Ephraim J. ^{[2
]}

Huff, Carol Ann ^{[2
]}

Luznik, Leo ^{[2
]}

Smith, B. Douglas ^{[2
]}

Matsui, William H. ^{[2
]}

Goodman, Steven N. ^{[2
]}

Ambinder, Richard F. ^{[2
]}

Jones, Richard J. ^{[2
]}

机构：

[1] Johns Hopkins Univ, Sch Med, Dept Med, Div Hematol, Baltimore, MD 21205 USA

[2] Johns Hopkins Univ, Sch Med, Sidney Kimmel Comprehens Canc Ctr Johns Hopkins, Baltimore, MD 21205 USA

来源：

BLOOD | 2010年 / 115卷 / 11期

基金：

美国国家卫生研究院;

关键词：

BONE-MARROW-TRANSPLANTATION; COLONY-STIMULATING FACTOR; ANTITHYMOCYTE GLOBULIN; ANTILYMPHOCYTE GLOBULIN; REFRACTORY MYASTHENIA; CYCLOSPORINE; THERAPY; IMMUNOSUPPRESSION; REMISSION; CHILDREN;

D O I：

10.1182/blood-2009-06-225375

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder that can be treated with bone marrow transplantation, immunosuppressive therapy, and high-dose cyclophosphamide. Here, we report long-term follow-up on 67 SAA patients (44 treatment-naive and 23 refractory) treated with high-dose cyclophosphamide. At 10 years, the overall actuarial survival was 88%, the response rate was 71% with the majority being complete, and the actuarial event-free survival was 58% in 44 treatment-naive SAA patients. Patients with refractory SAA fared less well after high-dose cyclophosphamide therapy; at 10 years, overall actuarial survival, response, and actuarial event-free survival rates were 62%, 48%, and 27%, respectively. High-dose cyclophos-phamide is highly effective therapy for severe aplastic anemia. Large randomized controlled trials will be necessary to establish how results of high-dose cyclophosphamide compare with either bone marrow transplantation or standard immunosuppressive regimens, such as antithymocyte globulin and cyclosporine. (Blood. 2010;115:2136-2141)

引用

页码：2136 / 2141

页数：6

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