Non-optimal treatment with first tyrosine kinase inhibitor and associated economic burden in chronic myeloid leukemia

Although adenosine triphosphate (ATP)-competitive tyrosine kinase inhibitors (TKI) approved by the Food and Drug Administration have demonstrated effectiveness in treating chronic myeloid leukemia (CML), patients often experience intolerance or resistance, leading to non-optimal treatment (NOPT). This study assessed the treatment patterns, as well as NOPT and its associated economic burden, in newly diagnosed patients with CML receiving first TKI treatment. This retrospective study identified adult patients with ≥2 CML diagnoses who initiated first TKI treatment (imatinib, dasatinib, nilotinib, or bosutinib) in 2012 or later from United States administrative health claims databases (01 January 2007–30 June 2022). Treatment sequence and time to treatment discontinuation/switch were assessed. NOPT, identified based on treatment modification/adherence criteria, and its associated incremental healthcare resource utilization and medical costs were evaluated. Nearly a quarter of patients experienced NOPT, as indicated by early treatment modifications or low treatment adherence. NOPT was associated with significant incremental healthcare resource utilization (80% more inpatient admissions; twice as many inpatient days; 30% more outpatient visits) and medical costs (adjusted mean cost difference = $13,551 per-patient-per-year). Given the lack of information on reasons of treatment modification in health claims data, NOPT was identified based on indicators of management of intolerance and resistance to TKI. These findings highlight that unmet clinical needs and significant economic burden still persist in this population and that patients with CML may benefit from using therapeutic options with better efficacy and tolerability profiles as first treatment.