Despite current treatment and intervention, there is still an unmet need in hypertrophic cardiomyopathy (HCM)1

  • Current pharmacological treatments for HCM are based on a small number of studies, few of which were randomized, controlled prospective trials1
  • These treatments, as well as septal reduction therapies, do not target the underlying pathophysiology of the disease1,2

Overview of current treatment options

Type of
treatment option		 Description
and limitations
Pharmacological

Use of beta blockers, calcium-channel blockers, sodium-channel blockers, antiarrhythmic and anticoagulant medications (i.e., disopyramide, amiodarone, blood thinners) for obstructive HCM manage the symptoms1-3

  • The American Heart Association (AHA)/American College of Cardiology (ACC) and European Society of Cardiology (ESC) guidelines for pharmacologic therapy to reduce HCM symptoms are solely observation based1,4
  • None of these options treat the underlying disease; instead, they focus on reducing symptoms1
Septal reduction therapies
  • For those still seeking treatment, surgical septal myectomy and septal alcohol ablation are additional options for patients with inadequate response to pharmacological treatment1,2
  • Surgical options may be considered to reduce symptoms in highly selected patients when experienced surgeons are available at comprehensive HCM centres*
  • Patients who have undergone these therapies
    • can still have symptoms that require pharmacological management; and
    • may require further septal reduction therapy, which is associated with greater risk of later requiring a permanent pacemaker1,2
Type of option Pharmacological
Treatment limitations
  • Use of beta blockers, calcium-channel blockers, sodium channel blockers, antiarrhythmic and anticoagulant medications (i.e., disopyramide, amiodarone, blood thinners) for obstructive HCM manage the symptoms1-3
  • The American Heart Association (AHA)/American College of Cardiology (ACC) and European Society of Cardiology (ESC) guidelines for pharmacologic therapy to reduce HCM symptoms are solely observation based1,4
  • None of these options treat the underlying disease; instead, they focus on reducing symptoms1
Type of option Septal reduction therapies
Treatment limitations
  • For those still seeking treatment, surgical septal myectomy and septal alcohol ablation are additional options for patients with inadequate response to pharmacological treatment1,2
  • Surgical options may be considered to reduce symptoms in highly selected patients when experienced surgeons are available at comprehensive HCM centres*
  • Patients who have undergone these therapies
    • can still have symptoms that require pharmacological management; and
    • may require further septal reduction therapy, which is associated with greater risk of later requiring a permanent pacemaker1,2

* As stated in the 2020 AHA/ACC Guideline for the Diagnosis and Treatment of Patients with HCM: “Similar to the recommendations regarding surgery for patients with asymptomatic mitral valve disease, earlier surgery in patients with HCM should be limited to those comprehensive HCM centers with documented evidence of the highest success rates and lowest complication rates (i.e., durable success is >90% with an expected mortality rate <1%).”1

For specific guideline recommendations and management algorithms, please refer to the 2020 AHA/ACC Guideline for the Diagnosis and Treatment of Patients with HCM.1 These guidelines refer to one of the most commonly used clinical classification systems, the New York Heart Association (NYHA) Functional Classification, which places patients in one of four categories based on how much they are limited during physical activity.5

Another resource that may be helpful is the Kansas City Cardiomyopathy Questionnaire (KCCQ), which has been shown in clinical studies to serve as a relevant endpoint in clinical trials of obstructive HCM therapy and useful in the obstructive HCM patients’ clinical care. A recent study found the KCCQ is also well understood by patients and has strong evidence of good psychometric performance.6-8

Monitoring & treatment evaluation

References

Hide references
  1. Ommen SR, et al. 2020 AHA/ACC guideline for the diagnosis and treatment of patients with hypertrophic cardiomyopathy: a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. Circulation. 2020;142(25):e558-e631.
  2. Naidu, Srihari S, editor. Hypertrophic Cardiomyopathy. Second ed., Springer International Publishing, 2019.
  3. Data on file. 80-question, web-based survey. Individual patient survey results (as published January 22, 2016).
  4. Elliott PM, et al. 2014 ESC Guidelines on diagnosis and management of hypertrophic cardiomyopathy: the Task Force for the Diagnosis and Management of Hypertrophic Cardiomyopathy of the European Society of Cardiology (ESC). Eur Heart J. 2014;35(39):2733-79.
  5. American Heart Association editorial staff and reviewed by science and medicine advisers (last reviewed: May 31, 2017). Classes of heart failure. www.heart.org. Retrieved October 3, 2022, from https://www.heart.org/en/health-topics/heart-failure/what-is-heart-failure/classes-of-heart-failure.
  6. Rowin EJ, Maron MS. Refining Endpoint Measures in Clinical Trials for Hypertrophic Cardiomyopathy: The Emerging Role of Patient-Reported Outcomes. JACC Heart Fail. 2022;10(8):540-2.
  7. Hejjaji V, et al. Psychometric Evaluation of the Kansas City Cardiomyopathy Questionnaire in Men and Women With Heart Failure. Circ Heart Fail. 2021;14(9):e008284.
  8. Nassif, M. et al. Validation of the Kansas City Cardiomyopathy Questionnaire in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Fail. 2022;10:531-9.