Factors implicated in underutilization and poor persistence with hypomethylating agent therapy in myelodysplastic syndromes and chronic myelomonocytic leukaemia in Pakistan

Abstract

Objective: To assess the clinical and non-clinical factors leading to non-compliance or discontinuation of hypomethylating agent therapy in patients of myelodysplastic syndromes and chronic myelomonocytic leukaemia.

Method: The prospective, observational, cohort study was conducted at the National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi, from January 2022 to January 2024, and comprised patients diagnosed with myelodysplastic syndromes or chronic myelomonocytic leukaemia who were started on a monthly cycle of hypomethylating agent therapy, but either discontinued treatment or suffered from treatment interruption of 60 days or more. Demographics and clinical data was collected from the medical records and a questionnaire-based survey via direct interview with the patients. Data was analysed using SPSS 25.

Results: Of the 39 patients with median age 60 years (interquartile range: 53-70 years), 25(64.1%) were male, 23(59%) had myelodysplastic syndromes, and 16(41%) had chronic myelomonocytic leukaemia. The median number of hypomethylating agent cycles received was 3 (interquartile range: 01-11). Common clinical factors influencing poor persistence to therapy included comorbidities 16(41.0%) and infections 15(38.4%). Among the non-clinical factors, affordability, patients’ perception of disease, weak family support, and drug unavailability were commonly associated with non-persistence to therapy, with treatment affordability showing a statistically significant association (p=0.037).

Conclusion: The factors leading to poor compliance related to hypomethylating agent therapy among patients with myelodysplastic syndromes and chronic myelomonocytic leukaemia could be predicted beforehand and addressed to a certain degree through collaborative efforts.

Key Words: Myelodysplastic syndromes, Chronic myelomonocytic leukaemia, Hypomethylating agents.