Department of Leukemia, University of Texas M D Anderson Cancer Center, Houston, TX 77030, USA.
Br J Haematol. 2012 May;157(3):312-20. doi: 10.1111/j.1365-2141.2012.09064.x. Epub 2012 Feb 24.
This report describes the final results of a Phase II clinical trial investigating the efficacy of rabbit antithymocyte globulin (rATG), ciclosporin, steroids, and granulocyte colony-stimulating factor (GCSF) in patients with untreated aplastic anaemia (AA), or low to intermediate-risk and hypocellular myelodysplastic syndrome (MDS). We treated 24 patients each with AA and MDS with rATG (3·5 mg/kg/d × 5; reduced to 2·5 mg/kg/d × 5 in patients with MDS ≥ 55 years), ciclosporin (5 mg/kg orally daily × 6 months), steroids (1 mg/kg daily, tapered off over 1 month), and GCSF. The overall response rate in AA patients was 64% compared to 25% in MDS patients. The median time to response was 3 months in AA patients and 4 months in MDS patients. Pretreatment clinical characteristics, such as age, sex, blood counts, cellularity, cytogenetics, or HLA-DR15 status, did not predict for response. Response to therapy, however, predicted for improved overall survival (OS), with a 3-year OS of 89% vs. 43% in responders versus non-responders, respectively (P < 0·001). Infusion reactions occurred in about half the patients and were manageable. Myelosuppression, elevation in liver enzymes, and infections were common. The early mortality in MDS patients was 13% vs. 0% in AA patients.
本报告描述了一项 II 期临床试验的最终结果，该试验研究了兔抗胸腺细胞球蛋白（rATG）、环孢素、类固醇和粒细胞集落刺激因子（GCSF）在未经治疗的再生障碍性贫血（AA）或低危至中危和低细胞性骨髓增生异常综合征（MDS）患者中的疗效。我们用 rATG（3.5mg/kg/d×5；MDS 患者年龄≥55 岁时减至 2.5mg/kg/d×5）、环孢素（5mg/kg 口服每日×6 个月）、类固醇（1mg/kg 每日，1 个月内逐渐减量）和 GCSF 治疗了 24 例 AA 患者和 24 例 MDS 患者。AA 患者的总体缓解率为 64%，而 MDS 患者为 25%。AA 患者的中位缓解时间为 3 个月，MDS 患者为 4 个月。治疗前的临床特征，如年龄、性别、血细胞计数、细胞数、细胞遗传学或 HLA-DR15 状态，不能预测缓解。然而，对治疗的反应预测了总体生存（OS）的改善，缓解者的 3 年 OS 为 89%，而无缓解者为 43%（P<0.001）。约一半的患者出现输注反应，且可管理。骨髓抑制、肝酶升高和感染很常见。MDS 患者的早期死亡率为 13%，而 AA 患者为 0%。
