Poor Outcome of Patients With Myelodysplastic Syndrome After Azacitidine Treatment Failure
Poor Outcome of Patients With Myelodysplastic Syndrome After Azacitidine Treatment Failure
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DOI：
10.1016/j.clml.2013.07.007
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发表时间：
2013-12-01
影响因子：
2.7
通讯作者：
Komrokji, Rami S.
中科院分区：
文献类型：
--
作者：
Duong, Vu H.;Lin, Karen;Komrokji, Rami S.
对于患有高危骨髓增生异常综合征 (MDS) 的患者，阿扎胞苷治疗可带来有意义的生存获益。然而，大多数反应者在 2 年内经历疾病进展。我们对 59 名阿扎胞苷治疗失败的患者进行回顾性分析，结果很差。随后采用强化化疗或地西他滨治疗导致的反应率令人失望，这强调了优先考虑临床试验的重要性。背景：描述氮核苷治疗失败的 MDS 患者的结果和预后的数据有限。我们报告了我们对阿扎胞苷治疗失败的高危 MDS 患者的单机构经验。患者和方法：这是一项对在莫菲特癌症中心接受阿扎胞苷治疗方案失败的 MDS 患者进行的回顾性研究。通过莫菲特数据库识别患者，并提取临床数据。阿扎胞苷失败定义为至少 4 个治疗周期后未能实现血液学改善或更好、治疗期间失去反应或疾病进展。目的是描述阿扎胞苷失败后对挽救治疗的反应并估计总体生存率。所有反应均根据国际工作组 2006 年标准定义，并使用 Kaplan-Meier 方法估计生存率。结果：总共确定了 59 名阿扎胞苷治疗失败的患者。治疗失败的中位年龄为 68 岁，大多数是白人男性患者。 13 名患者接受了强化化疗，总体缓解率为 31%。六名患者接受了地西他滨治疗，但没有任何反应。阿扎胞苷失败后整个队列的中位总生存期为 5.8 个月（95% 置信区间，1.3-10.3 个月），估计 12 个月生存率为 17%。结论：阿扎胞苷治疗失败的高危MDS患者预后较差，对挽救治疗产生反应的可能性较低。氮杂核苷失败后的护理标准应该是参加临床试验。
For patients with higher-risk myelodysplastic syndrome (MDS), treatment with azacitidine yields a meaningful survival benefit. However, most responders experience disease progression within 2 years. In our retrospective review of 59 patients in whom azacitidine therapy had failed, outcome was poor. Subsequent treatment with intensive chemotherapy or decitabine resulted in disappointing response rates, emphasizing the importance of prioritizing consideration for clinical trials.Background: Limited data have been reported describing the outcome and prognosis of patients with MDS in whom treatment with azanucleosides has failed. We report our single-institutional experience of patients with higher-risk MDS in whom therapy with azacitidine has failed. Patients and Methods: This was a retrospective study of MDS patients treated at the Moffitt Cancer Center in whom azacitidine treatment regimens had failed. Patients were identified through the Moffitt database, and clinical data were extracted. Azacitidine failure was defined as failure to achieve hematologic improvement or better after at least 4 cycles of therapy, loss of response, or disease progression during therapy. The objectives were to characterize response to salvage therapies after azacitidine failure and to estimate the overall survival. All responses were defined according to the International Working Group 2006 criteria, and survival was estimated using the Kaplan-Meier method. Results: A total of 59 patients in whom azacitidine treatment had failed were identified. The median age at treatment failure was 68 years, and most were Caucasian male patients. Thirteen patients received intensive chemotherapy with an overall response rate of 31%. Six patients were treated with decitabine, and none responded. Median overall survival of the entire cohort after azacitidine failure was 5.8 months (95% confidence interval, 1.3-10.3 months), with an estimated 12-month survival of 17%. Conclusion: Patients with higher-risk MDS in whom azacitidine treatment has failed have a poor prognosis and low probability of response to salvage treatments. The standard of care after azanucleoside failure should be enrollment in clinical trials.