Mavacamten significantly improves QoL of patients with hypertrophic cardiomyopathy

Mavacamten improved the physical and social function and quality of life in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) compared with placebo, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ). Prof. John Spertus (Saint Luke’s Mid America Heart Institute, MO, USA) presented a health status analysis of the previously published phase 3 EXPLORER-HCM trial results [1–3].

Current guideline-recommended pharmacological management of HCM mainly consists of β-blockers or calcium channel blockers. Mavacamten is a first-in-class myosin inhibitor that directly addresses the pathophysiological mechanism underlying HCM. The EXPLORER-HCM (NCT03470545) trial was a phase 3 randomised, double-blind, placebo-controlled study that compared mavacamten (n=123) with placebo (n=128) in patients with obstructive HCM [2].

Participants (mean age 59 years; 46% women; 5% with diabetes) received a once-daily dose of either mavacamten (starting dose of 5 mg with a 2-step dose titration) or placebo for 30 weeks followed by an 8-week washout period, for a total intervention of 38 weeks. The KCCQ was administered at baseline and again at weeks 6, 12, 18, 30 (end of treatment), and 38 (end of study) [2,3]. The KCCQ assesses symptoms, physical function, social function, and quality of life. Scores range from 0 (i.e. poor score) to 100 (i.e. favourable score).

At week 30, 36% of participants in the mavacamten arm had achieved clinically meaningful improvements in their KCCQ score, as compared with only 15% of participants in the placebo arm. A greater proportion (23%) of patients in the placebo arm showed either no change or a deterioration in their KCCQ score, as compared with only 9% in the mavacamten arm. This benefit was no longer apparent at week 38 (i.e. following the washout period).

Prof. Spertus concluded that mavacamten represents a new potential strategy for improving symptoms, activities of daily living, and patient satisfaction.

1. 
   
   1. Spertus JA. Health Status Benefits of Mavacamten In Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results from the EXPLORER-HCM Randomised Clinical Trial. ACC 2021 Scientific Session, 15–17 May.
   2. Olivotto I, et al. Lancet. 2020;396(10253):759–769.

 

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Posted on 15 May, 202113 July, 2021