Abstract Rationale Difficult-to-treat asthma remains a significant burden, especially among patients taking medium/high-dose ICS-LABA. Limited real-world data exist on longitudinal outcomes in this group, particularly on disease progression, symptoms impact, and healthcare resource utilization (HCRU). Methods This retrospective, longitudinal analysis used NOVELTY cohort data from 18 countries. Patients (N = 1,102) aged ≥12 years with physician-assigned diagnosis of asthma and receiving medium/high-dose ICS-LABA were stratified by baseline asthma control status: uncontrolled (Asthma Control Test ACT 20 and/or ≥1 severe exacerbation in the previous year, ie, Global Initiative for Asthma GINA-defined “difficult-to-treat” asthma) or controlled. Clinical, symptoms, and HCRU outcomes were assessed at baseline and follow-up years 1, 2, and 3. Results At baseline, patients with difficult-to-treat asthma (n = 610, 55%) were younger (mean age 51 vs 54 years), more often female (66.9% vs 57.5%), had higher BMI (mean 29.0 vs 26.5 kg/m²), and higher prevalence of obesity, GERD, and type 2 diabetes than patients with controlled asthma on medium/high-dose ICS-LABA (n = 492, 45%). Mean baseline blood eosinophil counts were 239 and 212 cells/μL in patients with difficult-to-treat and controlled asthma, respectively; GINA step 5 treatment was taken by 43.6% and 36.6% of patients, respectively. Clinical and economic outcomes at baseline and over three years are presented in the Table. Patients with difficult-to-treat versus controlled asthma exhibited persistently higher severe exacerbation rates (mean 0.82, 0.45, 0.40, and 0.34 vs 0.00, 0.14, 0.18, and 0.12 at baseline, year 1, 2, and 3, respectively), greater lung function impairment (mean post-bronchodilator FEV1 86%, 84%, 85%, and 83% vs 90% predicted normal value by year 3), and worse quality of life over time (mean Chronic Airways Assessment Test CAAT 18, 15, 15, and 14 vs 9, 9, 9, and 8; EQ-5D VAS 69, 71, 71, and 73 vs 80, 80, 79, and 80). Economic burden remained high for patients with difficult-to-treat asthma at baseline and over three years, with more asthma-related general practitioner visits (mean 1.75, 1.48, 1.26, and 1.33 at baseline, year 1, 2, and 3, respectively) and more unscheduled visits for exacerbations (19.7%, 10.8%, 8.8%, and 7.5% with ≥1 visit). These patterns were generally consistent across difficult-to-treat asthma subgroups (ACT 20 only, ≥1 exacerbation only, and both). Conclusions In this large, multinational cohort, patients with difficult-to-treat asthma had substantial and persistent clinical burden and increased HCRU at baseline and over three years of follow-up. The findings underscore an ongoing unmet need and support consideration of alternative treatment options. This abstract is funded by: AstraZeneca
Papi et al. (Fri,) studied this question.