Health-related Quality of Life and Health Utility Among Brazilian Patients With Chronic-phase Chronic Myeloid Leukemia Receiving Tyrosine Kinase Inhibitors: Real-world Evidence From Brazil
Year: 2026
Type: article
Study design: Observational Study
Abstract: Background Advances in tyrosine kinase inhibitor (TKI) therapy have substantially improved survival outcomes for patients with chronic myeloid leukemia (CML). As patients increasingly achieve long-term disease control, health-related quality of life (HRQoL) has become an important treatment outcome. Aim To characterize HRQoL and health utility scores among Brazilian patients with chronic-phase CML receiving TKI and to explore associations between treatment line and comorbidity burden on patient-reported outcomes. Materials and methods This prospective, cross-sectional, non-interventional study enrolled Brazilian adults (≥ 18 years) with confirmed chronic-phase CML receiving an approved TKI for at least 1 month. Participants were recruited through the Brazilian Blood Cancer Association (ABRALE). Following ethics committee approval, eligible participants provided electronic informed consent and completed structured self-administered digital questionnaires through an electronic platform. HRQoL was assessed using the EuroQol 5-Dimension 3-Level questionnaire (EQ-5D-3L) and the EQ Visual Analogue Scale (EQ-VAS). EQ-5D-3L utility scores were derived using the Brazilian time trade-off (TTO) value set developed by Santos et al. Patients unwilling or unable to provide informed consent or currently participating in an interventional clinical trial were excluded. Results Of 216 respondents, 137 were eligible for inclusion after excluding patients with non–chronic-phase CML (n = 4), participation in or uncertainty regarding participation in an interventional clinical trial (n = 22), no current TKI treatment (n = 46), or age < 18 lt; 18 years (n = 7). Of these, 99 completed the EQ-5D-3L and were included in the HRQoL analysis. Participants were predominantly female (61.6%), aged 40–59 years (46.5%), White (65.7%), residing in Southeastern Brazil (52.5%), and had a university degree (41.4%); 52.5% received care through private health insurance. Regarding treatment history, 40 (40.4%) patients were receiving first-line (1L) TKI therapy, 35 (35.4%) second-line (2L) therapy, and 24 (24.2%) third-line or later (≥ 3L) therapy. Overall, 45.5% of patients reported no comorbidities, 32.3% reported one comorbidity, and 22.2% reported two or more comorbidities. The mean EQ-5D-3L utility score was 0.748 (95% CI, 0.716–0.780), and the mean EQ-VAS score was 73.5 (95% CI, 69.9–77.0). Mean EQ-5D-3L utility scores were 0.766 (95% CI: 0.720–0.812), 0.762 (95% CI: 0.715–0.810), and 0.696 (95% CI: 0.615–0.777) among patients receiving 1L, 2L, and ≥ 3L therapy, respectively. HRQoL decreased progressively with increasing comorbidity burden, with mean EQ-5D-3L utility scores of 0.826 (95% CI, 0.784–0.869) among patients without comorbidities, 0.706 (95% CI, 0.659–0.753) among those with one comorbidity, and 0.648 (95% CI, 0.582–0.714) among those with ≥ 2 comorbidities (Kruskal–Wallis p < 0.001). Conclusion This study provides one of the first real-world assessments of HRQoL and health utility among Brazilian patients with chronic-phase CML receiving TKI therapy. Overall, HRQoL was favorable; however, lower utility scores were observed among patients receiving third-line or later treatment and among those with a higher comorbidity burden. The progressive decline in HRQoL with increasing comorbidity burden highlights the impact of comorbid conditions on patient well-being beyond leukemia control alone.
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: diamond
OpenAlex ID: W7223089891