Antithymocyte globulin and cyclosporine for severe aplastic anemia - Association between hematologic response and long-term outcome
Antithymocyte globulin and cyclosporine for severe aplastic anemia - Association between hematologic response and long-term outcome
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DOI：
10.1001/jama.289.9.1130
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发表时间：
2003-03-05
影响因子：
120.7
通讯作者：
Young, NS
中科院分区：
文献类型：
--
作者：
Rosenfeld, S;Follmann, D;Young, NS
在大多数患者中，再生障碍性贫血是由t细胞介导的骨髓免疫破坏引起的。再生障碍性贫血可通过干细胞移植或免疫抑制有效治疗。目的评价免疫抑制治疗后的远期疗效。设计、环境和患者：根据骨髓细胞和血细胞计数标准，122例患有严重再生障碍性贫血的患者（31例小于或等于18岁，91例大于或等于18岁）于1991年至1999年在一家联邦政府研究医院纳入单臂介入研究方案。干预措施：抗胸腺细胞球蛋白每天40mg /kg，持续4天；环孢素每天10 - 12mg /kg，持续6个月（根据血液水平调整）；皮质类固醇短期疗程（1 mg/d强的松龙，持续约2周）。主要观察指标：生存、全血细胞减少和输血独立性的改善、复发和向其他血液病的演变。结果治疗后3个月有效率为60%，6个月有效率为61%，1年有效率为58%。有反应的患者的血细胞计数不再满足严重程度标准，他们是输血独立的。7年的总精算生存率为55%。生存率与早期反应标准满意度相关（86% vs 40%）； P50 x 10(3)/muL预测恢复不太强劲的患者的5年生存率为90% [64/71]vs 42% [12/34]
Context In most patients, aplastic anemia results from T-cell-mediated immune destruction of bone marrow. Aplastic anemia can be effectively treated by stem cell transplantation or immunosuppression.Objective To assess long-term outcomes after immunosuppressive therapy.Design, Setting, and Patients Cohort of 122 patients (31 were less than or equal to18 years and 91 were > 18 years) with severe aplastic anemia, as determined by bone marrow cellularity and blood cell count criteria, were enrolled in a single-arm interventional research protocol from 1991 to 1999 at a federal government research hospital.Interventions A dose of 40 mg/kg per day of antithymocyte globulin administered for 4 days, 10 to 12 mg/kg per day of cyclosporine for 6 months (adjusted for blood levels), and a short course of corticosteroids (1 mg/d of m ethyl prednisolone for about 2 weeks).Main Outcome Measures Survival, improvement of pancytopenia and transfusion-independence, relapse, and evolution to other hematologic diseases.Results Response rates were 60% at 3 months after initiation of treatment, 61% at 6 months, and 58% at 1 year. The blood cell counts of patients who responded no longer satisfied severity criteria and they were transfusion-independent. Overall actuarial survival at 7 years was 55%. Survival was associated with early satisfaction of response criteria (86% vs 40% at 5 years; P50 x 10(3)/muL predicted survival at 5 years of 90% [64/71] vs 42% [12/34] for patients with less robust recovery [P