来那度胺治疗难治/复发骨髓增生异常综合征伴环形铁粒幼红细胞患者的效果分析

姬姜; 杜亚丽; 阮菁; 黄钰洲; 韩冰

doi:10.13201/j.issn.1004-2806.2020.09.006

来那度胺治疗难治/复发骨髓增生异常综合征伴环形铁粒幼红细胞患者的效果分析

- 中国医学科学院北京协和医学院北京协和医院血液内科(北京, 100730)
基金项目:
北京自然科学基金(No:7192168)

中国医学科学院医学创新基金(No:2016-I2M-3-004)

中国医学科学院中央级公益性科研院所基本科研业务费专项资金(No:2019XK320047)

详细信息

通讯作者: 韩冰,E-mail:hanbing_li@sina.com.cn

中图分类号: R551.3

收稿日期: 2020-07-16

Lenalidomide treatment for refractory/relapsed myelodysplastic syndrome with ring sideroblasts

- Department of Hematology, Peking Union Medical College Hospital, Peking Union Medical College & Chinese Academy of Medical Sciences, Beijing, 100730, China

More Information

Corresponding author: HAN Bing, E-mail: hanbing_li@sina.com.cn

Received Date: 16 July 2020

摘要

摘要: 目的:研究来那度胺治疗难治/复发骨髓增生异常综合征伴环形铁粒幼红细胞(MDS-RS)的有效率及不良反应。方法:回顾性分析我院2018-06-2019-12接受来那度胺治疗的难治/复发MDS-RS患者。所有患者经足疗程促红细胞生成素、雄激素、环孢素等无效/复发后,均接受了来那度胺10 mg qd 21 d/月≥2个疗程的治疗。采集资料完整患者的治疗前和每个疗程结束后的临床相关信息并分析。结果:共纳入12例患者,男性比例50.0%,确诊时中位年龄为62(43~79)岁。IPSS-R低危比例为75.0%,中危比例为25.0%。来那度胺治疗开始时的中位血红蛋白为71(32~81) g/L,中性粒细胞计数为2.36(1.01~4.05)×10⁹/L,血小板计数为216(160~469)×10⁹/L,随访时间为10(5~15)个月。接受来那度胺的中位疗程数为3(2~13),中位起效时间为2(1~3)个月,总反应率为41.7%(5/12),完全缓解率为16.7%(2/12),总有效患者的中位维持时间为10(7~13)个月。随访期间2例(40.0%)患者复发,随访期末总有效率为25.0%,完全缓解率为8.3%;无效患者中1例死亡,12个月总生存率为91.7%。7例(58.3%)患者出现不良反应,其中4例(33.3%)出现中性粒细胞减少。最常见的非血液学不良反应为水肿(16.7%)及头晕(16.7%)。随访期末未发现骨髓纤维化、克隆演变。结论:来那度胺对难治/复发MDS-RS患者有一定疗效,不良反应可耐受。
- 骨髓增生异常综合征伴环形铁粒幼红细胞 /
- 来那度胺 /
- 难治 /
- 疗效 /
- 不良反应
Abstract: Objective: To analyze the effectiveness and safety of lenalidomide in patients with refractory/relapsed myelodysplastic syndrome with ring sideroblasts (MDS-RS).Method: Refractory/relapsed MDS-RS patients who received lenalidomide in our center from June 2018 to December 2019 were analyzed retrospectively.Result: Twelve patients were enrolled.The male proportion was 50.0%.The median age at diagnosis was 62(43-79) years old.The percentage of IPSS-R low-risk patients was 75.0% and the percentage of intermediate-risk patients was 25.0%.The baseline median hemoglobin was 71(32-81) g/L,the median absolute neutrophil count was 2.36(1.01-4.05)×10⁹/L,and the median platelet was 216(160-469)×10⁹/L.The median time of treatment duration was 3(2-13) months.The median follow-up time was 10(5-15) months.The complete response rate of the patients was 16.7% and the overall response rate was 41.7%.The median time to response was 2(1-3) months and the median time of efficacy duration was 10(7-13) months.Two patients (40.0%) relapsed,and the complete response/overall response rate was 8.3%,25.0% at the end of follow-up.One patient died and the 12-month overall survival rate was 91.7%.Seven patients (58.3%) appeared adverse events during treatment,4 cases (33.3%) had neutropenia.Edema (16.7%) and dizziness (16.7%) were the most common non-hematological adverse events.None of the patients experienced bone marrow fibrosis or clonal evolution at the end of follow-up.Conclusion: Lenalidomide is effective and tolerable in patients with refractory/relapsed MDS-RS.
- myelodysplastic syndromes with ring sideroblasts /
- lenalidomide /
- refractory /
- efficacy /
- adverse events

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