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Comparative efficacy of pharmacological therapies in non-obstructive hypertrophic cardiomyopathy: a frequentist

Bharath Kumar G1, Madhumitha S2, Nidhi S Ibrahimpur2

  • 1Department of Pharmacology, All India Institute of Medical Sciences (AIIMS), Bhubaneswar, India. bharathkumarg24@gmail.com.

European Journal of Clinical Pharmacology
|August 12, 2026
PubMed

Insights

No single drug effectively treated non-obstructive hypertrophic cardiomyopathy (HCM). Different therapies improved specific symptoms, but peak oxygen consumption did not improve. Further head-to-head studies are needed for optimal hypertrophic cardiomyopathy management.

Area of Science:

  • Cardiology
  • Pharmacology
  • Clinical Trials

Background:

  • Symptomatic non-obstructive hypertrophic cardiomyopathy (HCM) management is challenging.
  • Limited evidence directly compares pharmacological therapies for non-obstructive HCM.
  • Network meta-analysis is needed to compare multiple treatments.

Purpose of the Study:

  • To compare the efficacy of available pharmacological treatments for non-obstructive HCM.
  • To identify treatments that improve key physiological outcomes.
  • To guide future clinical trial design and treatment strategies.

Main Methods:

  • Frequentist network meta-analysis following PRISMA-NMA guidelines.
  • Systematic search of RCTs evaluating pharmacological therapies in adult non-obstructive HCM patients.
  • Analysis of continuous outcomes including peak oxygen consumption (peak VO₂), VE/VCO₂ slope, NT-proBNP, E/e' ratio, KCCQ score, and LVEF.

Main Results:

  • Nine RCTs with 1,057 patients were included.
  • No therapy significantly improved peak VO₂ (primary outcome).
  • Mavacamten reduced NT-proBNP; ARNI and ninerafaxstat improved VE/VCO₂ slope; candesartan improved E/e' ratio.

Conclusions:

  • Pharmacological therapies offer domain-specific benefits in non-obstructive HCM.
  • No single therapy consistently improved all outcomes.
  • Heterogeneity in treatment effects necessitates targeting multiple pathways and further head-to-head trials.
Abstract

Keywords:
Cardiopulmonary exercise testingMavacamtenNT-proBNPNetwork meta-analysisNon-obstructive hypertrophic cardiomyopathy

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