摘要
目的:评价维奈克拉(venetoclax,VEN)快速剂量递增、最长治疗时间为 14天,联合低剂量阿糖胞苷(low-dose cytarabine,LDAC)方案挽救治疗复发/难治性急性髓系白血病(relapsed/refractory acute myeloid leukemia,R/RAML)的安全性和有效性.方法:回顾性分析 2018年 10月至 2023年 11月于江苏大学附属医院接受VEN+LDAC方案挽救治疗的 16例R/R AML患者,所有患者既往均未接受过含VEN方案治疗.该方案VEN的剂量第 1天为 200 mg,其后均为 400 mg固定剂量;LDAC 20 mg/m2/d皮下注射.患者在治疗第 8天复查骨髓,根据骨髓增生情况决定总疗程为 10天还是 14天.所有患者均不给予VEN单药治疗.有治疗反应的患者采用相同方案维持直到疾病进展或移植.结果:本研究纳入的R/R AML患者,中位随诊时间为 27.5 个月.治疗期间未发生有临床表现的肿瘤溶解综合症(tumor lysis syndrome,TLS).治疗后总反应率(overall response rate,ORR)为68.75%,其中 4例达完全缓解(complete response,CR),1例达血液学未恢复的完全缓解(CR with incomplete hematologic re-covery,CRi),6例达部分缓解(partial response,PR).达最佳疗效的治疗周期中位数为 1个周期.中位总生存期(overall sur-vival,OS)为 5.8(0.5～47.2)个月,中位无进展生存期(progression-free survival,PFS)为 22.2(7.3～42.9)个月.发生的不良反应主要为 3～4级的血液学不良事件和感染.结论:本研究根据治疗第 8天骨髓复查结果调整用药天数的VEN+LDAC方案,对于既往没有接受过含VEN方案治疗的R/R AML患者有较好的安全性和有效率.即使14天的VEN+LDAC治疗也是安全的.
Abstract
Objective:To explored the feasibility and efficacy of a rapid ramp-up,2-week maximum regimen of venetoclax(VEN)plus low-dose cytarabine(LDAC)for treating relapsed/refractory acute myeloid leukemia(R/R AML).Methods:We retrospectively analyzed patients with venetoclax-naïve R/R AML treated with VEN+LDAC between October 2018 and November 2023.On the first day,patients received 200 mg of VEN,and the dose was quickly increased to 400 mg for the rest of the treatment;cytarabine was administered subcutaneously at a low dose of 20 mg/m2/day.The treatment duration was 10 or 14 days,depending on the condition of bone marrow hyperplasia determined on the 8th day of treatment.No patients received venetoclax monotherapy.All patients responding to salvage therapy received VEN+LDAC until disease progression or transplantation.Results:Among the patients,the median follow-up duration was 27.5 months.No clinical mani-festations of tumor lysis syndrome(TLS)occurred during the treatment.The overall response rate(ORR)was 68.75%,including four com-plete responses(CR),one complete remission with incomplete hematologic recovery(CRi),and six partial responses(PR).The median num-ber of best treatment result cycles was one cycle.The median overall survival(OS)in the whole cohort was 5.8(0.5-47.2)months;the medi-an progression-free survival(PFS)was 22.2(7.3-42.9)months.The major adverse events were grade 3-4 hematologic adverse events and in-fections.Conclusions:The 8th-day myelosuppression-adjusted VEN+LDAC regimen is a feasible salvage option with a reasonable safety pro-file in patients with venetoclax-naïve R/R AML.Most patients tolerated the 14-day treatment;the response was generally rapid in the re-sponding patients.
基金项目
江苏省社会发展重点项目(临床前沿技术)课题(BE2020681)
镇江市重点研发计划(社会发展)项目(SH2022038)
NETL
NSTL
万方数据