Department of Medical Oncology, Dr. BRA Institute Rotary Cancer Hospital, All India Institute of Medical Sciences, AIIMS, New Delhi, India.
Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH.
Clin Lymphoma Myeloma Leuk. 2020 Jul;20(7):e437-e444. doi: 10.1016/j.clml.2020.02.015. Epub 2020 Mar 6.
Chronic myeloid leukemia (CML) is an uncommon malignancy in children in which tyrosine kinase inhibitors (TKIs) have significantly improved outcome in recent years.
We performed a retrospective analysis of CML patients (≤ 18 years old) presenting to our center between January 2005 and December 2018 with respect to baseline demographics, response to imatinib, and real-world management of those with a suboptimal response.
A total of 124 patients were diagnosed with CML with 99 (80%) in the chronic phase. There was a male preponderance (males:females = 3.1:1) with a median age of 13 years. The common presenting clinical features were splenomegaly (90.9%) and fever (51.5%) with a median leukocyte count of 165 × 10/μL. The proportion of patients attaining a complete hematologic response (CHR) at 3 months, a complete cytogenetic response (CCyR) at 12 months, and a major molecular response at 12 months were 79.7%, 54.1%, and 50.9%, respectively. At a median follow-up of 67.4 months, the 5-year overall survival rate and the event-free survival (EFS) rate were 92% ± 3% and 64% ± 6%, respectively. Failure to achieve CCyR at 12 months was associated with poor EFS beyond 1 year (hazard ratio = 2.865, P = .044). Among 15 patients not achieving CHR at 3 months, dose escalation of imatinib resulted in the attainment of CHR in 13 (87%) patients. Seven patients in the cohort had a loss of the established response to imatinib because of documented poor compliance.
Imatinib remains the frontline treatment of choice in CML with a reasonable outcome in children, especially when financial affordability, availability of second-generation TKIs, and poor compliance still remain major challenges in management. Dose escalation of imatinib remains an option in patients with a suboptimal response.
慢性髓性白血病（CML）在儿童中较为罕见，近年来，酪氨酸激酶抑制剂（TKI）的应用显著改善了患者的预后。
我们对 2005 年 1 月至 2018 年 12 月期间在我们中心就诊的 CML 患者（≤18 岁）进行了回顾性分析，内容包括基线人口统计学特征、对伊马替尼的反应以及对那些治疗反应不佳患者的实际管理情况。
共诊断出 124 例 CML 患者，其中 99 例（80%）处于慢性期。男性患者多于女性（男：女=3.1：1），中位年龄为 13 岁。常见的临床表现为脾肿大（90.9%）和发热（51.5%），白细胞计数中位数为 165×10/μL。3 个月时达到完全血液学反应（CHR）、12 个月时达到完全细胞遗传学反应（CCyR）和 12 个月时达到主要分子反应的患者比例分别为 79.7%、54.1%和 50.9%。中位随访 67.4 个月时，5 年总生存率和无事件生存率（EFS）分别为 92%±3%和 64%±6%。12 个月时未达到 CCyR 与 1 年后 EFS 不良相关（风险比=2.865，P=0.044）。在 15 例 3 个月时未达到 CHR 的患者中，伊马替尼剂量增加使 13 例（87%）患者达到 CHR。该队列中有 7 例患者因记录到依从性差而失去了对伊马替尼的既定反应。
伊马替尼仍然是 CML 的一线治疗选择，在儿童中具有合理的疗效，特别是在财务负担能力、第二代 TKI 的可用性和较差的依从性仍然是管理中的主要挑战时。在治疗反应不佳的患者中，伊马替尼剂量增加仍然是一种选择。
