Deng Mengyue, Guan Xianmin, Wen Xianhao, Xiao Jianwen, An Xizhou, Yu Jie
Department of Hematology and Oncology; Ministry of Education Key Laboratory of Child Development and Disorders; National Clinical Research Center for Child Health and Disorders; China International Science and Technology Cooperation Base of Child Development and Critical Disorders; Children's Hospital of Chongqing Medical University.
Chongqing Key Laboratory of Pediatrics, Chongqing, P.R China.
Medicine (Baltimore). 2020 Feb;99(7):e19150. doi: 10.1097/MD.0000000000019150.
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.
慢性髓性白血病（CML）在儿童期相对罕见，很少有研究报道伊马替尼（IM）在儿童CML中的临床应用。在本研究中，我们评估了IM在儿童和青少年CML患者中的疗效和耐受性。我们调查了2014年5月至2018年2月期间在重庆医科大学附属儿童医院新诊断为CML并接受IM治疗的21例18岁以下患者。根据欧洲白血病网标准对疾病进行分期，并根据疾病分期确定IM剂量。根据Kaplan-Meier方法估计累积缓解率和生存概率。慢性期慢性髓性白血病（CML-CP）患者3个月时的估计完全血液学缓解率为89.5%。完全细胞遗传学缓解率随时间增加，在6、12和24个月时分别达到47.4%、73.7%和80.3%。12个月和24个月时的累积主要分子缓解率分别为42.1%和76.3%。中位随访时间为33.8个月（范围3.2 - 61.7个月），CML患者的估计2年总生存率（OS）为95.2%（95%置信区间[CI]，70.7% - 99.3%）。没有CML-CP患者进展为加速期或发生急变期。CML-CP队列的2年OS率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%CI，42.1% - 84.2%）。该组患者中没有因药物毒性导致的治疗相关死亡或停用IM的情况，只有1例患者发生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%的患者骨密度降低。在18岁以下的中国CML患者中，IM在整个随访期间均有效且不良反应耐受性良好。
