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

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