减低剂量地西他滨治疗骨髓增生异常综合征伴单系发育不良患者的疗效观察
Clinical observation on the treatment of myelodysplastic syndrome with single lineage dysplasia by dose-reduced decitabine

龚辉.减低剂量地西他滨治疗骨髓增生异常综合征伴单系发育不良患者的疗效观察[J].内科急危重症杂志,2019,25(2):107-109

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DOI：10.11768/nkjwzzzz20190205

中文关键词: 骨髓增生异常综合征伴单系病态造血地西他滨减低剂量临床效果血液学毒性

英文关键词:

基金项目:深科技创新2017（No:131）

作者	单位	E-mail
龚辉	深圳罗湖区人民医院	334656273@qq.com

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中文摘要:

目的: 探讨减低剂量地西他滨治疗骨髓增生异常综合征伴单系发育不良（MDS-SLD）患者的临床疗效和安全性。方法: 使用减低剂量地西他滨\[15mg/(m2•d),连续5d\]治疗12例MDS-SLD患者,观察其疗效和不良反应。结果: 2例（16.6%）获完全缓解,1例（8.3%）获部分缓解,5例（41.6％）达血液学改善，总反应率达66.6％。在5例可行细胞遗传学评估的患者中,1例(20.0%)获PR。Ⅳ级血液学毒性发生率 50.0%， Ⅲ～Ⅳ级感染发生率33.3%，无Ⅲ～Ⅳ级出血，无Ⅲ～Ⅳ级恶心呕吐和Ⅲ～Ⅳ级肝功能损伤。中位随访时间16(2～32)个月，随访期间无患者死亡。结论: 减低剂量地西他滨治疗骨髓增生异常综合征完全缓解及部分缓解率高，药物不良反应发生率低。

英文摘要:

Objective: To observe the clinical effect of dose-reduced decitabine in the treatment of patients with myelodysplastic syndrome with single lineage dysplasia (MDS-SLD). Methods: Clinical data of 12 cases of MDS-SLD were collected, and all patients were treated by dose-reduced decitabine (15mg/m2 intravenously once daily for 5 days). The efficacy and side-effects of therapy in these patients were evaluated. Results: Of all 12 patients, 2 cases (16.6%) achieved complete remission (CR), 1 case achieved partial remission (8.3%), 5 cases (41.6%) achieved hematologic improvement (HI), and the overall response rate (ORR) was 66.6%. Of the 5 cases in whom cytogenetic evaluation could be performed, 1 case (20.0%) achieved partial cytogenetic remission. Grade Ⅳ hematologic toxicity rate was 50.0%, and grade Ⅲ-Ⅳ infection rate was 33.3%. No grade Ⅲ-Ⅳ bleeding, severe nausea and vomiting (grade Ⅲ-Ⅳ) and liver injury (Ⅲ-Ⅳ) occurred. The median follow-up time was 16 months (2-32 months). At the end of follow-up, no patients were dead. Conclusion: In the treatment of MDS, the rate of CR and PR was high, and the incidence of adverse drug reactions was low.