阿扎胞苷治疗15例中高危骨髓增生异常综合征的疗效观察  

The effects of Azacitidine treatment in 15 patients with intermediate-2 or high-risk myelodysplastic syndromes

作　　者：高露[1] 王晓燕[1] 鲍立[1] 杨申淼[1] 主鸿鹄[1] 温磊[1] 路瑾[1] 江浩[1] 江倩[1] 黄晓军[1] 赖悦云[1] GAO Lu;WANG Xiaoyan;BAO Li;YANG Shenmiao;ZHU Honghu;WEN Lei;LU Jin;JIANG Hao;JIANG Qian;HUANG Xiaojun;LAI Yueyun(Peking University People's Hospital,Peking University Institute of Hematology,Beijing,100044,China)

机构地区：[1]北京大学人民医院,北京大学血液病研究所,北京100044

出　　处：《临床血液学杂志》2018年第6期854-858,共5页Journal of Clinical Hematology

摘　　要：目的:初步评估阿扎胞苷治疗中高危骨髓增生异常综合征(MDS)的安全性及有效性。方法:对入选的15例MDS患者应用阿扎胞苷75mg·m^(-2)·d^(-1)皮下注射连续7d,原则上每28d为1个疗程,评估治疗期间患者对药物的耐受性及有效性。结果:15例患者总共完成133周期阿扎胞苷治疗。1例患者2个疗程结束出院后不明原因死亡无法评估疗效,1例患者1个疗程获得血液学改善,但因经济原因完成第2个疗程后终止治疗,其余13例患者均完成至少4周期治疗,4～6个疗程后评估疗效总有效率为38.5%(5/13)。其中3例(23.1%)完全缓解,2例(15.4%)骨髓完全缓解,3例疾病稳定,5例治疗失败。阿扎胞苷主要不良反应为血液学毒性,其中5例为3～4级血液学毒性;11例(73.3%)至少出现一次感染性发热;1例注射部位刺激性皮炎;1例间质性肺炎。结论:阿扎胞苷治疗中高危MDS总有效率接近40%,可以推荐用于不能进行造血干细胞移植的中高危MDS患者。Objective:To evaluate the safety and effects of Azacitidine treatment in the patients with intermediate or high-risk myelodysplastic syndromes(MDS).Method:Fifteen selected patients with intermediate-2 or highrisk MDS were treated by subcutaneous injection of 75 mg·m-2·d-1 Azacitidine on days 1 to 7 of a 28-day cycle in principle.Patients were monitored for safety and adverse events and were evaluated for response during treatment.Result:In total,133 cycles of Azacitidine therapy were administered in 15 patients,1 case died of unknown reason after the second cycle of treatment,and the efficacy wasn’t evaluated.Another case achieved a hematological improvement in the first cycle but the treatment was terminated after the second cycle due to economic reasons.All the remaining 13 patients completed at least 4 cycles of treatment.After 4-6 cycles of treatment,the overall response rate was 38.5%(5/13),including 3 cases(23.1%)achieved complete response and 2 cases(15.4%)achieved bone marrow complete response,3 cases maintained stable disease and 5 cases were treatment failure.The main adverse event of Azacitidine was hematological toxicity.Grade Ⅲ to Ⅳof hematological toxicities happened in 5 patients.Infectious complications were seen in 11 out of 15 patients(73.3%)and in 23 cycles out of 133(17.3%),while injection irritant dermatitis and interstitial pneumonia happened in one patient respectively.Conclusion:The overall response rate of Azacitidine treatment in intermediate-2 or high-risk MDS is nearly 40%,it could be recommended to intermediate-risk or high-risk MDS patients who cannot accept hematopoietic stem cell transplantation.

关 键 词：骨髓增生异常综合征 阿扎胞苷 

分 类 号：R733[医药卫生—肿瘤]