中国实用内科杂志

中国实用内科杂志

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减低剂量地西他滨治疗输血依赖的骨髓增生异常综合征难治性血细胞减少伴单系病态造血8例疗效分析

龚亚文，何广胜，吴德沛，孙爱宁，仇惠英，苗瞄，王秀丽   

  1. 作者单位：苏州大学附属第一人民医院 江苏省血液病研究所 卫生部血栓与止血重点实验室，江苏 苏州 215006
  • 出版日期:2014-06-01 发布日期:2014-06-03
  • 通讯作者: 何广胜
  • 基金资助:

    国家科技支撑计划(2008BAI61B02，2008ZX09312-026)；江苏省医学重点人才资助项目(H201126)；高校自然科学研究项目(09KJB320015)；江苏省临床医学中心(ZX201102)

Dose-reduced decitabine treatment of myelodysplastic syndrome-refractory cytopenia with unilineage dysplasia in transfusion dependent patients.

  GONG Ya-wen，HE Guang-sheng,WU De-pei,SUN Ai-ning,QIU Hui-ying,MIAO Miao,WANG Xiu-li,WU Qian,JIN Zheng-ming,TANG Xiao-wen,HAN Yui,FU Zheng-zheng,RUAN Chang-geng   

  1. The First Affiliated Hospital of Suzhou University，Jiangsu Institute of Hematology，Key Laboratory of Thrombosis and Hemostasis of Ministry of Health，Suzhou 215006，China
  • Online:2014-06-01 Published:2014-06-03

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摘要：

目目的 探讨减低剂量地西他滨治疗输血依赖的骨髓增生异常综合征(MDS)-难治性血细胞减少伴单系病态造血(MDS-RCUD)的临床疗效和安全性。方法 评价江苏省血液病研究所2009年11月至2012年5月使用减低剂量地西他滨［20 mg/(m2·d)，连续3 d］治疗的8例MDS-RCUD患者的疗效和不良反应。结果 1例(12.5%)获完全缓解，2例脱离成分血输注，3例达血液学改善，总反应率达75.0%。在4例可行细胞遗传学评估的患者中，1例获部分细胞遗传学缓解。Ⅳ级血液学毒性发生率50.0%，Ⅲ～Ⅳ级感染发生率37.5%，无Ⅲ～Ⅳ级出血、严重恶心呕吐(Ⅲ～Ⅳ级)和肝功能损伤(Ⅲ～Ⅳ级)。中位随访时间386.6 d(108~655 d)，随访期间无患者死亡。结论 减低剂量地西他滨可以改善MDS-RCUD患者的输血依赖，严重血液学毒性和早期病死率的发生率低。

关键词: 骨髓增生异常综合征, 难治性血细胞减少伴单系病态造血, 地西他滨, 血液学毒性, 减低剂量

Abstract:

Abstract：Objective To evaluate the efficacy and safety of dose-reduced decitabine treatment for transfusion dependent patients with myelodysplastic syndrome-refractory cytopenia with unilineage dysplasia (MDS-RCUD).Methods From November 2009 to May 2012，collect the clinical information of 8 cases of MDS-RCUD，treated by dose-reduced decitabine(20 mg/m2 intravenously once daily for 3 days)，and evaluate the efficacy and side-effects of therapy in these patients.Results Of all 8 patients，1 case (12.5%) achieved complete remission (CR)，2 cases (25.0%) achieved transfusion independence，3 cases (37.5%) achieved hematologic improvement (HI) and the overall response rate (ORR) was 75.0%.Of the 4 cases in whom cytogenetic evaluation could be performed，1 case (25.0%) achieved partial cytogenetic remission.Grade Ⅳ hematologic toxicity rate was 50.0%，and grade Ⅲ-Ⅳ infection rate was 37.5%.No grade Ⅲ-Ⅳ bleeding，severe nausea and vomiting (Ⅲ-Ⅳ grade)，liver injury (Ⅲ-Ⅳ) occurred.The median follow-up time was 386.6 days (108-655 days).At the end of following-up，no patients were dead.Conclusion Dose-reduced treatment of decitabine can reduce transfusion dependence in MDS-RCUD patients with low rates of severe hematologic toxicity and early mortality.

Key words: myelodysplastic syndrome, refractory cytopenia with unilineage dysplasia, decitabine, hematologic toxicity, dose-reduced

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