Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure.
Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure.
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DOI：
10.1200/jco.2011.35.8135
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发表时间：
2011-08-20
期刊：
影响因子：
--
通讯作者：
Vey N
中科院分区：
文献类型：
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作者：
Prébet T;Gore SD;Esterni B;Gardin C;Itzykson R;Thepot S;Dreyfus F;Rauzy OB;Recher C;Adès L;Quesnel B;Beach CL;Fenaux P;Vey N
阿扎胞苷 (AZA) 是目前治疗高风险（即国际预后评分系统高或中级 2）骨髓增生异常综合征 (MDS) 的标准治疗方法，但大多数患者会经历初次或二次治疗失败。这些患者的结果尚未描述。总体而言，对 435 名患有高危 MDS 和原难治性贫血伴转化中原始细胞过多 (RAEB-T) 的患者进行了 AZA 失败后的结局评估。患者队列包括四个数据集（即 AZA001、J9950 和 J0443 试验以及法国同情使用计划）。 AZA 失败后的中位随访时间为 15 个月。中位总生存期为 5.6 个月，2 年生存概率为 15%。多变量分析显示，年龄增长、男性、高风险细胞遗传学、骨髓母细胞计数较高以及先前对 AZA 缺乏血液学反应与生存率显着较差相关。可获得 270 名患者 AZA 失败后的治疗数据。与传统的临床护理相比，同种异体干细胞移植和研究药物具有更好的结果。 AZA 失败后的结果很差。我们的结果应作为设计该人群二线临床试验的基础。
Azacitidine (AZA) is the current standard of care for high-risk (ie, International Prognostic Scoring System high or intermediate 2) myelodysplastic syndrome (MDS), but most patients will experience primary or secondary treatment failure. The outcome of these patients has not yet been described. Overall, 435 patients with high-risk MDS and former refractory anemia with excess blasts in transformation (RAEB-T) were evaluated for outcome after AZA failure. The cohort of patients included four data sets (ie, AZA001, J9950, and J0443 trials and the French compassionate use program). The median follow-up after AZA failure was 15 months. The median overall survival was 5.6 months, and the 2-year survival probability was 15%. Increasing age, male sex, high-risk cytogenetics, higher bone marrow blast count, and the absence of prior hematologic response to AZA were associated with significantly worse survival in multivariate analysis. Data on treatment administered after AZA failure were available for 270 patients. Allogeneic stem-cell transplantation and investigational agents were associated with a better outcome when compared with conventional clinical care. Outcome after AZA failure is poor. Our results should serve as a basis for designing second-line clinical trials in this population.