达沙替尼二线治疗慢性髓系白血病慢性期儿童青少年患者的疗效及安全性分析

Efficacy and safety analysis of domestic dasatinib as second-line treatment for pediatric adolescents with chronic myeloid leukemia in chronic phase

目的:探讨国产达沙替尼二线治疗慢性髓系白血病(chronic myeloid leukemia,CML)慢性期儿童青少年患者的疗效及安全性。方法:回顾性分析2016年8月至2021年9月我院收治的国产达沙替尼二线治疗CML慢性期儿童青少年患者的病例资料,统计治疗3、6、12个月时最佳反应率、累积完全细胞遗传学反应率、累积主要分子学反应率、无进展生存率、无事件生存率及不良反应发生情况。结果:共纳入29例患者,中位随访时间为26(12~75)个月,治疗3、6、12个月时最佳反应率分别为82.8%(24/29)、93.1%(27/29)、86.2%(25/29),至随访截止时间,累积完全细胞遗传学反应率、累积主要分子学反应率分别为86.2%(25/29)和65.5%(19/29)。随访时间内,无进展生存率为100.0%(29/29),无事件生存率为82.8%(24/29)。最常见的非血液学不良反应为肝酶升高(10.3%)、皮疹(10.3%),血液学不良反应主要有贫血(24.1%)、血小板减少(6.9%)。结论:国产达沙替尼二线治疗CML慢性期儿童青少年患者具有较好的疗效与安全性,可作为CML慢性期儿童青少年患者的二线用药选择之一。

Objective: To investigate the efficacy and safety of domestic dasatinib as second-line treatment for pediatric adolescents with chronic myeloid leukemia in chronic phase(CML-CP).Methods: A retrospective analysis of clinical data of CML-CP pediatric adolescents who received domestic dasatinib as second-line treatment in the Henan Tumor Hospital from August 2016 to September 2021 was performed.The optimal response rate at 3,6 and 12 months,the cumulative rates of complete cytogenetic response and major molecular responses,the rates of progression free survival and event free survival,adverse events of the patients were assessed.Results: A total of 29 patients were enrolled in this study.The median follow-up time was 26(12-75)months.The optimal response rates at 3,6 and 12 months were 82.8%(24/29),93.1%(27/29),86.2%(25/29),respectively.By the end of follow-up,the cumulative rates of complete cytogenetic response and major molecular response were 86.2%(25/29)and 65.5%(19/29),respectively.During follow-up,the rates of progression free survival and event free survival were 100.0%(29/29)and 82.8%(24/29),respectively.The most common non-hematological adverse events of dasatinib were elevated transaminase level(10.3%)and rash(10.3%).The common hematological adverse events of dasatinib were anemia(24.1%)and thrombocytopenia(6.9%).Conclusion: Domestic dasatinib is effective and safe as the second-line treatment of CML-CP pediatric adolescents and can be a good choice.