Real-world burden of hypertrophic cardiomyopathy on healthcare use and health-related quality of life in Italy, Spain, and the United States.

Abstract

Background and aims: Hypertrophic cardiomyopathy (HCM) is a chronic, progressive, genetic heart disease characterized by hypercontractility and hypertrophy. As treatment options evolve, characterizing the existing healthcare needs and health-related quality of life (HRQoL) for patients with HCM is important.

Methods: Data were derived from the Adelphi Real World HCM Disease Specific Programme, a cross-sectional survey in Italy, Spain, and the United States. Physicians reported demographic and clinical characteristics; patients completed the Kansas City Cardiomyopathy Questionnaire (KCCQ-23), EQ-5D-5L and EQ-VAS, and the Work Productivity and Activity Impairment (WPAI) questionnaire. Outcomes were statistically compared by New York Heart Association (NYHA) class or KCCQ clinical summary score (KCCQ-CSS) to assess impact of disease severity.

Results: Patient mean age was 56.5 ± 14.8 years; 60.7% were male, and 78.3% had NYHA class II disease. Patients most commonly received beta blockers (88.4%) and diuretics (31.8%). HCM-related hospitalizations were reported for 25.8% of patients, increasing with higher NYHA or lower KCCQ-CSS. Mean EQ-5D-5L was 0.85, while EQ-VAS was 75.0, both decreasing with higher NYHA class or lower KCCQ-CSS. Overall work impairment and activity impairment (WPAI) were 16.1% and 26.6%, respectively, increasing with higher NYHA class or lower KCCQ-CSS.

Conclusion: Our data show a significant remaining symptom burden and healthcare resource utilization (HCRU), as well as impact on HRQoL, work productivity, and daily activities, despite treatment intervention across a commonly applied measure of disease severity in HCM. These findings underscore the need for new treatments that can reduce HCRU and improve HRQoL of patients with HCM.