Real-world experience of imatinib in pediatric chronic phase chronic myeloid leukemia: a single-center experience from India

Abstract

Introduction Chronic myeloid leukemia (CML) is an uncommon malignancy in children in which tyrosine kinase inhibitors (TKIs) have significantly improved outcome in recent years. Patients and Methods 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. Results 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 × 103/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. Conclusion 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.