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Comment & Response
June 12, 2019

Hypertrophic Cardiomyopathy—Need for Gene-Specific Treatment?—Reply

Author Affiliations
  • 1UCL Institute of Cardiovascular Science, UCL Faculty of Population Health Sciences, University College London, London, United Kingdom
  • 2Queen Elizabeth University Hospital, Glasgow, United Kingdom
  • 3University of East Anglia, Norwich Research Park, Norwich, United Kingdom
  • 4Barts Heart Centre, Barts Health NHS Trust, London, United Kingdom
JAMA Cardiol. 2019;4(8):831-832. doi:10.1001/jamacardio.2019.1766

In Reply We thank van Driel and van der Velden for their interest in our randomized clinical trial of trimetazidine in nonobstructive hypertrophic cardiomyopathy (HCM).1 They raise pertinent questions about characteristics of the study design that are relevant to future randomized clinical trials in HCM.

Genetic testing was not a mandatory part of the trial protocol. However, of the 51 patients enrolled, 28 had undergone genetic testing at the time of manuscript preparation; 13 (46%) had no pathogenic variant, 8 (28%) had a pathogenic or likely pathogenic variant, and 7 (25%) had a variant of uncertain significance. The sample size is too small to investigate an association of genetic status with efficacy of trimetazidine, but post hoc exploratory analysis did not reveal any obvious differences.