Beyond disease control in chronic myeloid leukemia: Real-world quality of life across TKIs and treatment-free remission

Abstract

Tyrosine kinase inhibitors (TKIs) have transformed chronic myeloid leukemia (CML) into a chronic condition with near-normal life expectancy. As patients remain on therapy for years, health-related quality of life (HRQoL) and patient-reported outcomes (PROs) are increasingly important in therapeutic decision-making. However, real-world data comparing HRQoL across different TKIs, including asciminib, and in patients achieving treatment-free remission (TFR) remain limited. We conducted a nationwide, cross-sectional survey in collaboration with the Israeli CML Patients’ Organization and the Institute of Hematology at the Davidoff Cancer Center, Rabin Medical Center. Adults with CML who had received TKI therapy for ≥ 3 months or discontinued therapy ≥ 3 months earlier were eligible. Participants completed validated Hebrew versions of the EORTC QLQ-C30 and QLQ-CML24 questionnaires. Patients were grouped by treatment status: TFR, first-, second-, or third-generation TKIs, and asciminib. A total of 233 patients participated. Global HRQoL was generally preserved across groups. However, symptom burden differed by treatment status. Patients receiving second-generation TKIs reported a greater symptom burden in selected domains, including fatigue and gastrointestinal symptoms, compared with patients in TFR and other treatment groups. Mood disturbance differed significantly across treatment groups, with numerically higher scores among patients receiving second-generation TKIs. In contrast, patients in TFR and those receiving asciminib consistently reported lower symptom burden and more favorable HRQoL profiles. Treatment satisfaction and social functioning remained high overall. In this nationwide real-world cohort, overall HRQoL was maintained, yet clinically meaningful differences in symptom burden were observed across treatment contexts. Patients in TFR and those receiving asciminib demonstrated consistently lower symptom burden, underscoring the importance of integrating PROs into individualized therapeutic decision-making in modern CML care.