Clinical efficacy and safety of imatinib treatment in children and adolescents with chronic myeloid leukemia: A single-center experience in China
Clinical efficacy and safety of imatinib treatment in children and adolescents with chronic myeloid leukemia: A single-center experience in China
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伊马替尼治疗儿童青少年慢性粒细胞白血病的临床疗效和安全性
DOI：
10.1097/md.0000000000019150
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发表时间：
2020-02-01
期刊：
影响因子：
1.6
通讯作者：
Yu, Jie
中科院分区：
文献类型：
--
作者：
Deng, Mengyue;Guan, Xianmin;Yu, Jie
摘要慢性粒细胞白血病(CML)在儿童时期相对少见，伊马替尼(IM)在儿童CML中的临床应用研究较少。在这项研究中，我们评估了IM在慢性粒细胞白血病儿童和青少年中的疗效和耐受性。我们于2014年5月至2018年2月在重庆医科大学儿童医院调查了21例18岁以下初诊慢性粒细胞白血病并接受IM治疗的患者。根据欧洲白血病网络标准对疾病进行分期，并根据疾病阶段确定IM剂量。根据Kaplan-Meier方法估计累积反应和生存概率。慢性期慢性粒细胞白血病(CML-CP)3个月的血液学完全缓解率为89.5%。细胞遗传学完全应答率随时间延长而增加，6、12、24个月分别达到47.4%、73.7%、80.3%。12个月和24个月的累积主要分子缓解率分别为42.1%和76.3%。中位随访期为33.8个月(3.2-61.7个月)，CML的2年总生存率为95.2%(95%可信区间，70.7%-99.3%)。CML-CP患者中无一例进展到加速期或出现爆发性危象。CML-CP队列的2年OS和无进展生存率均为100%，而估计的2年无事件生存率为68%(95%可信区间为42.1%-84.2%)。在这组患者中，没有患者因药物毒性而出现与治疗相关的死亡或肌注中断，只有1名患者出现III-IV级非血液学不良事件。总体而言，贫血是最常见的不良反应，42.9%的患者骨密度下降。在中国18岁以下的慢性粒细胞白血病患者的随访期内，肌注有效，不良反应耐受性良好。
Abstract Chronic myeloid leukemia (CML) is relatively rare in childhood and few studies have reported the clinical use of imatinib (IM) in pediatric CML. In this study, we evaluated the efficacy and tolerability of IM in children and adolescents with CML. We investigated 21 patients under 18 years of age with newly diagnosed CML and treated with IM in Children's Hospital of Chongqing Medical University between May 2014 and February 2018. The disease was staged according to the European LeukemiaNet criteria and the IM dose was determined based on the disease stage. Cumulative responses and survival probabilities were estimated according to the Kaplan–Meier method. The estimated complete hematologic response rate of chronic phase-chronic myeloid leukemia (CML-CP) was 89.5% at 3 months. The complete cytogenetic response rates increased with time, reaching 47.4%, 73.7%, and 80.3% at 6, 12, and 24 months, respectively. The cumulative major molecular response rates were 42.1% and 76.3% at 12 and 24 months, respectively. With a median follow-up time of 33.8 months (range, 3.2–61.7 months), the estimated 2-year overall survival (OS) rate for CML was 95.2% (95% confidence interval [CI], 70.7%–99.3%). None of the CML-CP patients progressed to the accelerated phase or had a blast crisis. The 2-year OS and progression-free survival rates for the CML-CP cohort were both 100%, while the estimated 2-year event-free survival rate was 68% (95% CI, 42.1%–84.2%). None of the patients in this group had treatment-related deaths or IM discontinuation due to drug toxicities, and only 1 patient had a grade III–IV nonhematologic adverse event. Overall, anemia was the most common adverse effect and 42.9% of patients had a decrease in bone mineral density. IM was effective and the adverse effects were well-tolerated throughout the follow-up period in Chinese CML patients under 18 years of age.