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来那度胺治疗失败后未发生5q缺失的骨髓增生异常综合征患者的治疗结果

Outcome of patients treated for myelodysplastic syndromes without deletion 5q after failure of lenalidomide therapy.

作者信息

Prebet Thomas, Toma Andrea, Cluzeau Thomas, Sekeres Mikkael A, Vey Norbert, Park Sophie, Al Ali Najla, Sugrue Marie M, Komrokji Rami, Fenaux Pierre, Gore Steven D

机构信息

Section of Hematology, Department of Internal Medicine, Yale University, New Haven, CT, USA.

Groupe Francophone des Myelodysplasies, Hopital Saint Louis, Paris, France.

出版信息

Oncotarget. 2017 Jun 6;8(23):37866-37874. doi: 10.18632/oncotarget.15200.

Abstract

Anemia is a key survival prognostic factor in lower-risk myelodysplastic syndromes (MDS). Lenalidomide (LEN) can correct anemia in 25% of MDS patients without deletion 5q (del5q). As this therapy will inevitably fail, understanding the outcome of these patients will facilitate development of subsequent treatment strategies. To answer this question, an international retrospective study focused on LEN-treated lower-risk, non-del5q, MDS patients was performed. We analyzed the overall survival after LEN failure, its prognostic factors and the impact of post LEN treatment options. We included a total of 384 patients. The median overall survival after failure of LEN was 43 months. In multivariate analysis, adverse cytogenetics, excess of blasts at the initiation of LEN, and the type of failure (classified as stable disease, relapse, intolerance, or progression) were the main determinants of outcome. Subsequent therapy with hypomethylating agents was associated with a prolonged survival compared to BSC (median OS= 51m vs. 36m, p=0.01). In conclusion, the survival for non-del5q MDS patients after failure of LEN remains relatively prolonged, though with a wide range. Clinical trial participation remains the recommendation for these patients even if options such as hypomethylating agents may be considered.

摘要

贫血是低危骨髓增生异常综合征(MDS)患者生存预后的关键因素。来那度胺(LEN)可使25%无5号染色体缺失(del5q)的MDS患者贫血得到纠正。由于这种治疗不可避免会失败,了解这些患者的预后将有助于制定后续治疗策略。为回答这一问题,开展了一项针对接受LEN治疗的低危、非del5q MDS患者的国际回顾性研究。我们分析了LEN治疗失败后的总生存期、预后因素以及LEN治疗后选择的影响。我们共纳入了384例患者。LEN治疗失败后的中位总生存期为43个月。多因素分析显示,不良细胞遗传学、LEN治疗开始时原始细胞增多以及失败类型(分为疾病稳定、复发、不耐受或进展)是预后的主要决定因素。与最佳支持治疗(BSC)相比,使用去甲基化药物进行后续治疗可延长生存期(中位总生存期=51个月对36个月,p=0.01)。总之,LEN治疗失败后,非del5q MDS患者的生存期仍相对延长,尽管差异较大。即使可以考虑使用去甲基化药物等治疗选择,仍建议这些患者参加临床试验。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/32c4/5514957/01b707647a28/oncotarget-08-37866-g001.jpg

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