Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
Department of Pediatrics, Peking University People's Hospital, Beijing, China.
Ann Hematol. 2021 Sep;100(9):2215-2228. doi: 10.1007/s00277-021-04544-6. Epub 2021 Jun 5.
To explore the differences in the clinical features, treatment responses, and outcomes among children, adolescents, and adults with chronic myeloid leukemia in the chronic phase (CML-CP) receiving imatinib as first-line therapy. Data from children (0-8 years for girls and 0-10 years for boys), adolescents (9-19 years for girls and 11-19 years for boys), and adults (age ≥ 20 years) with newly diagnosed CML-CP receiving imatinib as first-line therapy between 2006 and 2019 were retrospectively reviewed. In total, 135 children (cohort 1), 189 adolescents (cohort 2), and 658 adults (cohort 3: age 20-39 years, n = 305; cohort 4: age 40-59 years, n = 270; and cohort 5: age 60-83 years, n = 83) were included in this study. When compared with children, adolescents showed a significantly higher white blood cell count (P = 0.033) and basophil percentage in peripheral blood (P = 0.002) and a significantly higher prevalence of splenomegaly (P = 0.004). Both children and adolescents presented with more aggressive clinical features than adults. During median follow-ups of 28 months (range, 3-161 months) in children, 33 months (range, 3-152 months) in adolescents, and 48 months (range, 3-157 months) in adults, multivariate analysis showed that children and adolescents had higher probabilities of achieving complete cytogenetic response, major molecular response, and molecular response. Notably, compared with not only adults (cohort 3 vs. cohort 1: HR = 2.03 [1.03, 3.98], P = 0.040; cohort 4 vs. cohort 1: HR = 2.15 [1.07, 4.33], P = 0.033; cohort 5 vs. cohort 1: HR = 4.22 [1.94, 9.15], P < 0.001) but also adolescents (cohort 2 vs. cohort 1: HR = 2.36 [1.18, 4.72], P = 0.015), children had significantly longer failure-free survival. Age was not associated with progression-free survival or overall survival. Although they exhibited more aggressive clinical features at diagnosis, both children and adolescents achieved superior treatment responses than adults. Adolescents showed even more adverse features and a poor FFS than children.
探讨接受伊马替尼作为一线治疗的慢性髓性白血病慢性期（CML-CP）的儿童、青少年和成人患者的临床特征、治疗反应和结局差异。本研究回顾性分析了 2006 年至 2019 年期间接受伊马替尼作为一线治疗的新诊断为 CML-CP 的儿童（女孩 0-8 岁，男孩 0-10 岁）、青少年（女孩 9-19 岁，男孩 11-19 岁）和成人（年龄≥20 岁）的数据。共纳入 135 名儿童（队列 1）、189 名青少年（队列 2）和 658 名成人（队列 3：年龄 20-39 岁，n=305；队列 4：年龄 40-59 岁，n=270；队列 5：年龄 60-83 岁，n=83）。与儿童相比，青少年外周血白细胞计数（P=0.033）和嗜碱性粒细胞百分比明显升高（P=0.002），脾肿大发生率明显升高（P=0.004）。儿童和青少年的临床特征均比成人更具侵袭性。在儿童中位随访 28 个月（范围 3-161 个月）、青少年中位随访 33 个月（范围 3-152 个月）和成人中位随访 48 个月（范围 3-157 个月）期间，多变量分析显示，儿童和青少年获得完全细胞遗传学缓解、主要分子缓解和分子缓解的可能性更高。值得注意的是，与成人（队列 3 与队列 1：HR=2.03[1.03, 3.98]，P=0.040；队列 4 与队列 1：HR=2.15[1.07, 4.33]，P=0.033；队列 5 与队列 1：HR=4.22[1.94, 9.15]，P<0.001）和青少年（队列 2 与队列 1：HR=2.36[1.18, 4.72]，P=0.015）相比，儿童无失败生存率显著延长。年龄与无进展生存率或总生存率无关。尽管他们在诊断时表现出更具侵袭性的临床特征，但儿童和青少年的治疗反应均优于成人。青少年的无失败生存率甚至比儿童更差。
