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Disease-modifying medical therapy in phenotypically overt hypertrophic cardiomyopathy: a case of too little, too late?
  1. Ashwat Dhillon,
  2. Milind Desai
  1. Department of Cardiovascular Medicine, Heart and Vascular Institute, Cleveland Clinic, Cleveland, Ohio, USA
  1. Correspondence to Dr Ashwat Dhillon, Department of Cardiovascular Medicine, Desk J1-5, Heart and Vascular Institute, Cleveland Clinic, Cleveland, OH 44195, USA; ashwatdhillon{at}gmail.com

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Despite being the most common inherited cardiac disorder, there has been a dearth of randomised studies looking at disease-modifying pharmacological therapy in hypertrophic cardiomyopathy (HCM). This may in part be due to the fact that while it has been established that HCM is a disease of the heart muscle caused by mutations in sarcomeric proteins, there is as yet no consensus on the exact underlying pathophysiological mechanism in this disease process. Therefore, medical therapy in HCM is focused on symptom control and based mainly on observational data, with little research done on pharmacotherapy that may alter the natural history of the disease.1 ,2 A recent review found that since the 1950s when HCM was first described, only 45 pharmacological studies have been done, enrolling approximately 2100 patients, of which only 5 studies were randomised double-blind placebo-controlled trials.3 It was in this background of limited knowledge that the initial small studies to assess the effect of angiotensin receptor blockers (ARBs) in HCM were conducted, with equivocal results in terms of the effect on left ventricular (LV) hypertrophy.4–8

Subsequently, Axelsson et al9 published the original results of the INHERIT trial (INHibition of the renin angiotensin system in HCM and the Effect on hypertrophy—a Randomised Intervention Trial …

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