Asian Hematology Research Journal

Objective: This study aimed to evaluate the use, efficacy, and safety of ponatinib in patients with chronic myeloid leukaemia (CML) treated outside clinical-trial settings in Bangladesh, where trial evidence may not fully reflect routine practice.

Methods: A retrospective, multicentre observational study was conducted among patients with CML treated with ponatinib at four tertiary hospitals between March 2022 and June 2023. Demographic, clinical, treatment, response, and safety data were collected and analysed.

Results: Among 60 patients, the median age was 42 years (range, 22–66 years), which was younger than that reported in major clinical trials. Thirty-six patients (60.0%) had advanced-phase disease (accelerated phase or blast crisis). All patients had previously received tyrosine kinase inhibitors (TKIs), and 56 (93.3%) switched because of treatment failure or a suboptimal response. The T315I mutation was detected in 15 patients (25.0%). Among 59 patients with evaluable response data, the overall response rate was 78.0% (46/59), and 26 patients (43.3%) achieved at least a major molecular response. Dose reduction from 45 mg to 30 mg once daily occurred in 28 patients (46.7%) because of adverse events, including vascular complications. Median failure-free survival and overall survival were 18 and 24 months, respectively.

Conclusion: In routine clinical practice in Bangladesh, ponatinib was used mainly as salvage therapy in a relatively young cohort with multi-TKI failure, irrespective of T315I status. Despite frequent dose reductions for safety, clinically meaningful responses were observed in this high-risk population.