Abstract

Background: The VANISH trial (Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hypertrophic Cardiomyopathy) showed valsartan improves LV remodeling in early-stage HCM, but its effects on quality of life (QOL) are unknown. A better understanding of QOL may impact patient wellness. Aims: Describe baseline QOL in early-stage HCM. Assess the impact of valsartan on QOL in early-stage HCM. Methods: We evaluated QOL using age-adjusted versions of the Pediatric Quality of Life Inventory in VANISH participants (ages 8-45): 166 with early-stage HCM, 34 with subclinical HCM (sarcomere variant present but normal LV wall thickness/no HCM diagnosis). Demographic, imaging, CPET, and biomarker correlates of baseline QOL were studied. The impact of valsartan on QOL was analyzed in the early-stage HCM cohort. Results: Participants with early-stage HCM reported good baseline QOL, better than an earlier published healthy validation cohort but diminished compared to subclinical participants (mean overall/physical/psychosocial scores 85/87/84 for early-stage, 90/92/89 subclinical, 82/84/81 earlier validation cohort). Race and ethnicity influenced QOL, with non-white and Hispanic participants reporting lower scores (Table 1). Valsartan improved physical QOL (p=0.03), with suggestion of differential effect by sex and pubertal status, but limited power to detect effect modification (Figure 1). Conclusion: Baseline QOL is lower in patients with early-stage HCM compared to subclinical participants despite NYHA I status. Non-white and Hispanic participants and those with lower baseline peak VO 2 may have lower quality of life. Valsartan treatment improved physical QOL.

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