Jabbour Elias, Strati Paolo, Cabrero Monica, O'Brien Susan, Ravandi Farhad, Bueso-Ramos Carlos, Wei Qiao, Hu Jianhua, Abi Aad Simon, Short Nicholas J, Dinardo Courtney, Daver Naval, Kadia Tapan, Wierda William, Wei Yue, Colla Simona, Borthakur Gautam, Cortes Jorge, Estrov Zeev, Kantarjian Hagop, Garcia-Manero Guillermo
Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
Department of Hematopathology, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
Am J Hematol. 2017 Apr;92(4):351-358. doi: 10.1002/ajh.24650. Epub 2017 Feb 13.
Two hundred and sixteen consecutive patients with MDS and abnormal karyotype treated with hypomethylating agents between 4/04 and 10/12 were reviewed. Median follow-up was 17 months. Using IWG criteria, best responses were complete response (CR) in 79 patients (37%), partial response (PR) in 4 (2%), and hematologic improvement (HI) in 10 (5%). Cytogenetic response (CyR) was achieved in 78 patients (36%): complete (CCyR) in 62 (29%) and partial in 16 (7%). CyR was achieved in 48 of 79 patients (61%) with CR, 1 of 14 (7%) with PR/HI, and in 29 of the 123 (24%) with no morphologic response. Median overall survival (OS) and leukemia-free survival (LFS) for patients with and without CCyR were 21 and 13 months (P = .007), and 16 and 9 months (P = .001), respectively. By multivariate analysis, the achievement of CCyR was predictive for better OS (HR = 2.1; P < .001). In conclusion, CyR occurs at a rate of 36% (complete in 29%) in patients with MDS treated with HMA and is not always associated with morphological response. The achievement of CCyR is associated with survival improvement and constitutes a major predictive factor for outcome particularly in patients without morphologic response. Therefore, the achievement of CCyR should be considered a milestone in the management of patients with MDS.
对2004年4月至2012年10月期间接受去甲基化药物治疗的216例连续的骨髓增生异常综合征（MDS）且核型异常的患者进行了回顾性研究。中位随访时间为17个月。根据国际工作组（IWG）标准，最佳反应为79例患者（37%）达到完全缓解（CR），4例（2%）达到部分缓解（PR），10例（5%）达到血液学改善（HI）。78例患者（36%）实现了细胞遗传学反应（CyR）：62例（29%）为完全细胞遗传学缓解（CCyR），16例（7%）为部分缓解。79例CR患者中有48例（61%）实现了CyR，14例PR/HI患者中有1例（7%）实现了CyR，123例无形态学反应的患者中有29例（24%）实现了CyR。有和没有CCyR的患者的中位总生存期（OS）分别为21个月和13个月（P = 0.007），无白血病生存期（LFS）分别为16个月和9个月（P = 0.001）。多因素分析显示，实现CCyR可预测更好的OS（HR = 2.1；P < 0.001）。总之，接受HMA治疗的MDS患者中CyR发生率为36%（完全缓解率为29%），且并不总是与形态学反应相关。实现CCyR与生存改善相关，是结局的主要预测因素，尤其是在无形态学反应的患者中。因此，实现CCyR应被视为MDS患者管理中的一个里程碑。
