摘要
背景儿童费城染色体阳性急性淋巴细胞白血病(Ph+ALL)复发后预后差,治疗棘手。奥雷巴替尼在Ph+ALL成人患者中显示出良好的疗效,但对儿童患者的疗效和安全性尚不明确。目的评估奥雷巴替尼在复发Ph+ALL患儿中的有效性和安全性。设计病例系列报告。方法回顾性分析2022年1月至2024年7月在北京大学人民医院儿科住院的复发Ph+ALL患儿应用奥雷巴替尼治疗的效果、安全性和转归情况。主要结局指标完全缓解(CR)、微小残留病(MRD)阴性、主要分子学反应(MMR)和完全分子学反应(CMR)。结果8例复发Ph+ALL患儿纳入分析,男6例,女2例,中位年龄9岁,应用奥雷巴替尼前血液学复发3例、分子学复发5例。所有患儿在应用奥雷巴替尼单药或联合化疗治疗1个周期后均为CR,血液学复发的3例患儿MRD阴性获得率100%、MMR获得率66%。分子学复发的5例患儿MRD阴性获得率、MMR获得率和CMR获得率均为100%。中位随访时间13个月,8例患儿均持续为CR,MRD阴性获得率和MMR获得率均为100%,其中CMR获得率75%,随访期间所有患儿均存活。其中,1例患儿为中枢神经系统白血病(CNSL)复发,经过奥雷巴替尼单药及鞘内注射化疗药物1次后,脑脊液MRD阴性并持续为阴性。治疗期间的相关不良反应主要与联合使用的化疗药物有关,单药奥雷巴替尼治疗未出现明显不适,1例患儿因药物不良反应停用奥雷巴替尼。结论对于复发Ph+ALL患儿,奥雷巴替尼单药或联合化疗治疗具有良好的疗效和较高的安全性。
Background Children with Philadelphia chromosome positive acute lymphoblastic leukemia(Ph+ALL)have a poor prognosis after relapse,and treatment is challenging.Olverembatinib has shown good efficacy in adult patients with relapsed Ph+ALL,but its efficacy and safety in children are not well defined.Objective To evaluate the efficacy and safety of olverembatinib in children with Ph+ALL.Design Case series report.Methods A retrospective analysis was conducted on children with Ph+ALL admitted to the department of pediatrics of Peking University People's Hospital from January 2022 to July 2024,who were treated with olverembatinib.The effects,side effects,and outcomes of the treatment were analyzed.Main outcome measures Complete remission(CR),minimal residual disease(MRD)negativity,major molecular response(MMR),and complete molecular response(CMR).Results A total of eight pediatric patients with relapsed Ph+ALL were included in the analysis,comprising six males and two females,with a median age of 9 years.Among them,three had hematologic relapse,while five had molecular relapse before receiving olverembatinib.All patients achieved CR after one treatment cycle of olverembatinib,either as monotherapy or in combination with chemotherapy.Among the three patients with hematologic relapse,the MRD negativity rate was 100%,and the MMR rate was 66%.Among the five patients with molecular relapse,the MRD negativity rate,MMR rate,and CMR rate were all 100%.The median follow-up duration was 13 months,during which all eight patients maintained CR.The MRD negativity and MMR rates remained 100%,while the CMR rate was 75%.All patients survived throughout the follow up period.Notably,one patient experienced a relapse of central nervous system leukemia(CNSL)but achieved sustained cerebrospinal fluid MRD negativity after receiving olverembatinib monotherapy combined with a single intrathecal chemotherapy injection.The adverse effects observed during treatment were mainly attributed to the combined use of chemotherapy drugs.Olverembatinib monotherapy did not cause significant discomfort in most patients;however,one patient discontinued olverembatinib due to drug-related adverse reactions.Conclusion For the children with relapse of Ph+ALL,olverembatinib monotherapy or in combination with chemotherapy shows good efficacy and safety.
作者
郑方圆
丁明明
陆爱东
贾月萍
曾慧敏
张乐萍
ZHENG Fangyuan;DING Mingming;LU Aidong;JIA Yueping;ZENG Huimin;ZHANG Leping(Department of Pediatrics,Peking University People's Hospital,Beijing 100044,China)
出处
《中国循证儿科杂志》
北大核心
2025年第1期22-26,共5页
Chinese Journal of Evidence Based Pediatrics
关键词
奥雷巴替尼
复发
费城染色体阳性
急性淋巴细胞白血病
儿童
Olverembatinib
Relapsed
Philadelphia chromosome positive
Acute lymphoblastic leukemia
Children
