目的探讨超小剂量地西他滨方案治疗骨髓增生异常综合征(MDS)的临床疗效及安全性。方法收集2014年1月至2015年10月江苏省人民医院血液科收治接受地西他滨治疗可评价的MDS患者37例。皮下注射地西他滨(每日5~7 mg/m2,治疗第1~3天,第8、15、22天),每4周为1个疗程,连续4~6个疗程。比较不同预后分组间疗效差异,评价可能与疗效相关的参数。结果 20例(54%)获得了临床反应,其中2例(5.4%)完全缓解,骨髓完全缓解无血液学改善1例(2.7%),骨髓完全缓解伴血液学改善1例(2.7%),血液学改善14例(37.8%),脱离输血2例(5.4%);疾病稳定14例(37.8%),疾病进展3例(8.1%)。按WPSS预后分层分组,较低危组与较高危组之间总反应率差异有统计学意义(70.0%对35.3%,χ~2=4.457,P=0.035);按IPSS-R细胞遗传学分层,预后良好组与中危组、高危组之间总反应率差异有统计学意义(60%对25%,χ~2=6.036,P=0.014);根据查尔森合并症指数(CCI)分组,各组间疗效差异无统计学意义。Ⅲ~Ⅳ度骨髓抑制13例(35.1%);在粒细胞缺乏期9例(24.3%)(Ⅱ~Ⅳ度)发生感染性发热;没有因血液学毒性而死亡;未观察到Ⅲ~Ⅳ级胃肠道及心、肝、肾等毒副反应。骨髓抑制、发热等不良反应根据CCI危险分层比较,低危、中危及高危之间不良反应发生率差异无统计学意义(P>0.05)。结论超小剂量地西他滨方案治疗MDS疗效佳,且耐受性良好,低、中危组MDS疗效好于高危组。有合并症不影响疗效。 Objective To investigate the clinical efficacy and safety of ultra-low dose decitabine in the treatment of myelodysplastic syndrome (MDS). Methods Thirty-seven patients with MDS who were treated with decitabine in Department of Hematology, Jiangsu Provincial People’s Hospital from January 2014 to October 2015 were collected. Subcutaneous injection of decitabine (daily 5 ~ 7 mg / m2, treatment days 1 to 3 days 8,15,15 days), every 4 weeks for a course of treatment for 4 to 6 courses. Differences in the efficacy of different prognostic groups were compared to assess parameters that may be related to efficacy. Results The clinical response was obtained in 20 patients (54%), of which 2 patients (5.4%) were completely relieved, 1 patient (2.7%) had complete remission without hematological improvement, 1 patient (2.7% Hematology improved in 14 cases (37.8%), 2 cases (5.4%) were separated from transfusion; 14 cases (37.8%) were stable and 3 cases (8.1%) were advanced in disease. According to the WPSS prognosis, the total response rate was significantly lower in the lower-risk group and the higher-risk group (70.0% vs 35.3%, χ ~ 2 = 4.457, P = 0.035); according to the IPSS-R cytogenetics According to the Charlson’s comorbidity index (CCI), the overall response rate between stratified, good prognosis group and moderate risk group and high risk group was statistically significant (60% vs. 25%, χ ~ 2 = 6.036, P = 0.014) ) Group, no significant difference between the efficacy of each group. Thirteen cases (35.1%) had myelosuppression Ⅲ ~ Ⅳ, and infective fever occurred in 9 cases (24.3%) (Ⅱ ~ Ⅳ degrees) in agranulocytosis; they did not die from hematological toxicity; Ⅲ ~ Ⅳ were not observed Gastrointestinal tract and heart, liver, kidney toxicity. Bone marrow suppression, fever and other adverse reactions According to the CCI risk stratification, there was no significant difference in the incidence of adverse reactions between low-risk, intermediate-risk and high-risk (P> 0.05). Conclusion Ultra-low-dose decitabine regimen has good curative effect and good tolerance to MDS. The efficacy of MDS in low and intermediate risk groups is better than that in high-risk groups. A complication does not affect the efficacy.