国产阿扎胞苷治疗骨髓增生异常综合征效果及安全性

EFFICACY AND SAFETY OF DOMESTIC AZACITIDINE IN TREATMENT OF MYELODYSPLASTIC SYNDROME

目的探讨国产阿扎胞苷治疗中、高危骨髓增生异常综合征(MDS)病人有效性及安全性。方法分析2014年1月—2015年12月在我科诊断为中、高危MDS病人20例临床资料,均采用国产阿扎胞苷75 mg/(m 2•d)皮下注射治疗,连续使用7 d,每28 d为1疗程。观察治疗有效率(ORR)、中位生存时间、不良反应和病人耐受情况。结果20例病人共接受104个疗程治疗,中位疗程为6(3~11)个;随访至2018年11月,中位生存期为11个月;有11例获得缓解,ORR为55%,完全缓解(CR)1例(5%),骨髓缓解(mCR)8例(40%),其中mCR伴血液学改善(HI)7例,单纯HI者2例(10%)。第1疗程后ORR为5%,第3疗程后ORR为35%,第6疗程后ORR为45%。不良事件主要为骨髓抑制、血细胞减少相关的感染和胃肠道反应。治疗过程中,20例病人均出现Ⅲ~Ⅳ级血液学不良反应,辅以对症支持治疗,病人可耐受;13例病人出现发热,其中8例出现感染症状,予以积极抗感染治疗后好转;15例病人出现了恶心、呕吐等胃肠道反应;3例病人出现肝功能异常;2例病人出现注射部位红肿、疼痛。给予对症治疗后均好转,未影响治疗。病人无心脏、肾功能损害发生。结论国产阿扎胞苷治疗中、高危MDS病人具有较好的效果和安全性。

Objective To investigate the efficacy and safety of domestic azacitidine in the treatment of moderate-or high-risk myelodysplastic syndrome(MDS).Methods A retrospective analysis was performed for the clinical data of 20 patients who were diagnosed with moderate-or high-risk MDS in our department from January 2014 to December 2015,and all patients were treated with subcutaneous injection of domestic azacitidine at a dose of 75 mg/(m 2•d)for 7 consecutive days,with 28 d as one course of treatment.Overall response rate(ORR),median survival time,adverse reactions,and tolerance of patients were observed.Results All 20 patients received 104 courses of treatment in total,with a median of 6(3-11)courses per patient.The patients were followed up to November 2018,and the median survival time was 11 months.Of all 20 patients,11 achieved remission,resulting in an ORR of 55%;1(5%)achieved complete remission and 8(40%)achieved marrow complete remission(mCR),among whom 7 achieved mCR and hematological improvement(HI)and 2(10%)achieved HI alone.The ORR was 5%after the first course,35%after the third course,and 45%after the sixth course.Adverse reactions mainly included myelosuppression,hemocytopenia-related infections,and gastrointestinal reactions.During treatment,all 20 patients experienced gradeⅢ-Ⅳhematological adverse reactions,which were tolerable after symptomatic/supportive treatment;13 patients experienced pyrexia,among whom 8 had infection symptoms and were improved after anti-infective therapy;15 patients experienced gastrointestinal reactions including nausea and vomiting;3 patients experienced abnormal liver function;2 patients experienced swelling and pain at the injection site,which were improved after symptomatic treatment and did not affect the treatment.No heart or renal dysfunction was observed.Conclusion Domestic azacitidine has good efficacy and safety in the treatment of patients with moderate-or high-risk MDS.