Molecular Response to TKI and Exploring the Factors of Treatment Compliance in CML Patients from Assam, India

Achievement of molecular response in chronic myeloid leukemia (CML) depends on compliance with tyrosine kinase inhibitors (TKIs) therapy. Despite the high efficacy of TKIs shown in studies from western countries, suboptimal adherence remains one of themajor barriers to optimal treatment outcomes in the Indian setting. Despite its clinical significance, there are no published long-term follow-up MMR data on major molecular response (MMR) from this region. The present study aimed to determine the molecular response to TKI therapy and identify factors associated with treatment compliance in patients with CML from Assam, India. This was a 3-year hospital-based prospective-cohort study, conducted in patients with confirmed CML having molecular detection of BCR-ABL1 transcript. Treatment response was evaluated in accordance with the European Leukemia Net (ELN) 2020 recommendations. MMR was assessed by measuring Breakpoint Cluster Region gene-Abelson Murine Leukemia Viral Oncogene Homolog 1 gene (BCR-ABL1) transcript copy numbers using quantitative real-time polymerase chain reaction and expressing the results on the International Scale – -Normalized Copy Number level level. A total 55 CML participants were enrolled, and the mean age was 41 years. The chronic phase of CML was observed in 83.37% of participants. Regular TKI consumption was reported by 74.5% of participants during the study period. MMR was achieved in 38.1% of participants, while 14.54% achieved deep molecular response (DMR) after achieving MMR. The median time to achieve MMR was 7.9 months. True MMR after excluding 25% non-compliant group increased to 69.04%. In our cohort, non-compliance at the 6-month assessment was initially high (57%), then dropped down to 43% in 1 year and in 3 years it further dropped to 25% as a result of structured intervention. Addressing socio-economic, psychological barriers by patient education, financial support for molecular monitoring, and decentralized drug distribution may substantially improve TKI compliance and response.