Efficacy and safety of mavacamten in treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis

Future Sci OA. 2023 Sep 19;9(10):FSO898. doi: 10.2144/fsoa-2023-0059. eCollection 2023 Dec.

Abstract

Aim: This meta-analysis was performed to assess the efficacy and safety of mavacamten in patients with hypertrophic cardiomyopathy.

Methods & materials: A search was conducted using PubMed, Cochrane, and Scopus up to August 2022 for randomized studies reporting our pre-specified outcomes.

Results: It was observed that mavacamten significantly improved New York Heart Association class (p < 0.009), Clinical Summary Score of the Kansas City Cardiomyopathy Questionnaire (p = 0.02), post-exercise left ventricular outflow tract gradient (p < 0.00001), functional end point (p = 0.05), and lowered septal reduction therapy rates (p < 0.00001). However, there were no significant differences in the ≥1 severe adverse events, ≥1 treatment-emergent adverse events, left ventricular volume index, left ventricular filling pressure, left ventricular end-diastolic volume index, and peak oxygen uptake (pVO2).

Conclusion: Future large-scale trials are required to confirm our results and determine the long-term benefits and risks of mavacamten use in these patients.

Keywords: MYK-461; cardiac myosin inhibitor; hypertrophic cardiomyopathy; mavacamten; nonobstructive cardiomyopathy; obstructive cardiomyopathy.

Plain language summary

Mavacamten is a recently introduced medication that relaxes the heart muscle and is indicated for patients with hypertrophic cardiomyopathy (a disease in which parts of the heart become thick and stiff). To determine the effectiveness and safety of this drug, the results of clinical trials were combined in order to produce an overall estimate. Overall, it was observed that mavacamten improved most functional parameters related to the heart and demonstrated no significant increases in the number of side effects. This suggests the effectiveness and safety of mavacamten, although further trials are needed to confirm our results.