Dataset Information

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

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.

SUBMITTER: Memon A

PROVIDER: S-EPMC10518811 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature

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Publications

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

Memon Areeba A Larik Muhammad Omar MO Khan Zoha Z Urooj Maryam M Irfan Areeka A Kumari Beena B Faisal Mehak M Siddiqui Rida R Tehrim Moniba M Hameed Ishaque I

Future science OA 20230919 10

<h4>Aim</h4>This meta-analysis was performed to assess the efficacy and safety of mavacamten in patients with hypertrophic cardiomyopathy.<h4>Methods & materials</h4>A search was conducted using PubMed, Cochrane, and Scopus up to August 2022 for randomized studies reporting our pre-specified outcomes.<h4>Results</h4>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) ...[more]

PMID: 37753355

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Project description:BackgroundUnlike other suggested therapies, myosin inhibitors have been shown to change the course of hypertrophic cardiomyopathy by altering the contractile mechanics of cardiomyocytes. This meta-analysis sought to determine the efficacy of mavacamten and aficamten in hypertrophic cardiomyopathy.MethodsThe online databases were searched from inception to July 2024, including the Cochrane Central Register of Controlled Trials (CENTRAL), PubMed, ClinicalTrials.gov. The meta-analytical data were pooled using risk ratios (RRs) with 95% CI, standard mean difference (SMD) and SE.ResultsA total of 6 randomised controlled trials with 826 hypertrophic cardiomyopathy patients (mean age±SD up to 59.8±14.2 years in intervention vs 60.9±10.5 years in placebo) were included in our study. Of these, 443 received a cardiac myosin inhibitor and 383 received a placebo. The resting left ventricular outflow tract (LVOT) gradient between the two groups was considerably improved by cardiac myosin inhibitors (MD -57.27; 95% CI -63.05 to -51.49). Significant differences were also observed in the post-Valsalva LVOT gradient between the two groups (MD -55.86; 95% CI -65.55 to -46.18). Significantly decreased left ventricle ejection fraction (LVEF) was also seen (MD -4.74; 95% CI -7.22 to -2.26). The New York Health Association (NYHA) class improvement between the two groups also changed significantly (RR 2.21; 95% CI 1.75 to 2.80). Cardiac myosin inhibitors also caused significant improvement in the Kansas City Cardiomyopathy Questionnaire in a Clinical Summary Score between the two groups (MD 7.71; 95% CI 5.37 to 10.05) and significant reduction in the N-terminal pro-B-type natriuretic peptide (SMD -13.27; 95% CI -17.51 to -9.03) and the cardiac troponin I (SMD -11.90; 95% CI -15.07 to -8.72).ConclusionAccording to our meta-analysis, cardiac myosin inhibitors significantly improve the resting and post-Valsalva LVOT gradient, reduce the LVEF and improve the NYHA class and cardiac biomarkers when compared with the placebo.Prospero registration numberCRD52024586161.

Project description:BackgroundThe phase 2 PIONEER-HCM (Phase 2 Open-label Pilot Study Evaluating Mavacamten in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction) study showed that mavacamten improved left ventricular outflow tract gradients, exercise capacity, and symptoms in patients with obstructive hypertrophic cardiomyopathy (HCM), but the results of longer-term treatment are less well described. We report interim results from the PIONEER-OLE (PIONEER Open-Label Extension) study, the longest-term study of mavacamten in patients with symptomatic obstructive HCM.Methods and resultsPatients who previously completed PIONEER-HCM (n=20) were eligible to enroll in PIONEER-OLE. Patients received oral mavacamten, 5 mg once daily (starting dose), with individualized dose titration at week 6. Evaluations included serial monitoring of safety, echocardiography, Kansas City Cardiomyopathy Questionnaire-Overall Summary Score, and serum NT-proBNP (N-terminal pro-B-type natriuretic peptide) levels. Thirteen patients enrolled and received mavacamten (median study duration at data cutoff, 201 weeks). Most patients (92.3%) received β-blockers concomitantly. Treatment-emergent adverse events were predominantly mild/moderate. One patient had an isolated reduction in left ventricular ejection fraction to 47%, which recovered and remained normal with continued treatment at a reduced dose. At week 180, mavacamten was associated with New York Heart Association class improvements from baseline (class II to I, n=9; class III to II, n=1; and unchanged, n=2), sustained reductions in left ventricular outflow tract gradients (mean [SD] change from baseline: resting, -50 [55] mm Hg; Valsalva, -70 [41] mm Hg), and serum NT-proBNP levels (median [interquartile range] change from baseline: -498 [-2184 to -76] ng/L), and improved Kansas City Cardiomyopathy Questionnaire-Overall Summary Score (mean [SD] change from baseline: +17 [16]).ConclusionsThis long-term analysis supports the continued safety and effectiveness of mavacamten for >3 years in obstructive HCM.RegistrationURL: https://www.clinicaltrials.gov; Unique identifier: NCT03496168.

Dataset Information

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

Aim

Methods & materials

Results

Conclusion

Publications

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

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