Rosenfeld Stephen, Follmann Dean, Nunez Olga, Young Neal S
Hematology Branch, Bldg 10, National Heart, Lung, and Blood Institute, Bethesda, Md, USA.
JAMA. 2003 Mar 5;289(9):1130-5. doi: 10.1001/jama.289.9.1130.
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.
To assess long-term outcomes after immunosuppressive therapy.
DESIGN, SETTING, AND PATIENTS: Cohort of 122 patients (31 were < or =18 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 1998 at a federal government research hospital.
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 methylprednisolone for about 2 weeks).
Survival, improvement of pancytopenia and transfusion-independence, relapse, and evolution to other hematologic diseases.
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; P<.001) and by blood counts at 3 months (reticulocyte count or platelet count of >50 x 10(3)/ microL predicted survival at 5 years of 90% [64/71] vs 42% [12/34] for patients with less robust recovery [P<.001 by log-rank test]). There were no deaths among responders more than 3 years after treatment. Relapse was common, but severe pancytopenia usually did not recur. Relapse did not influence survival. Thirteen patients showed evolution to other hematologic diseases, including monosomy 7.
Approximately half of patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine have durable recovery and excellent long-term survival. These outcomes were related to the quality of hematologic recovery.
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所致。再生障碍性贫血可通过干细胞移植或免疫抑制得到有效治疗。
评估免疫抑制治疗后的长期疗效。
设计、地点和患者：根据骨髓细胞计数和血细胞计数标准确定为严重再生障碍性贫血的122例患者队列（31例年龄≤18岁，91例年龄＞18岁），于1991年至1998年在一家联邦政府研究医院纳入一项单臂干预研究方案。
每天给予40mg/kg抗胸腺细胞球蛋白，共4天；每天给予10至12mg/kg环孢素，共6个月（根据血药浓度调整）；以及短期使用皮质类固醇（每天1mg甲泼尼龙，约2周）。
生存率、全血细胞减少的改善情况、输血独立性、复发情况以及向其他血液系统疾病的演变。
治疗开始后3个月的缓解率为60%，6个月时为61%，1年时为58%。有反应的患者血细胞计数不再满足严重程度标准，且不再依赖输血。7年时的总体精算生存率为55%。生存率与早期达到反应标准有关（5年时为86%对40%；P＜0.001），也与3个月时的血细胞计数有关（网织红细胞计数或血小板计数＞50×10³/μL预测5年生存率为90%[64/71]，而恢复较差的患者为42%[12/34] [对数秩检验P＜0.001]）。治疗后3年以上有反应的患者无死亡。复发很常见，但严重全血细胞减少通常不再复发。复发不影响生存率。13例患者出现向其他血液系统疾病的演变，包括7号染色体单体。
接受抗胸腺细胞球蛋白和环孢素治疗的严重再生障碍性贫血患者中，约一半有持久缓解和良好的长期生存。这些结果与血液学恢复的质量有关。
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