Cardiomyopathy

Top 10 priorities

The most important questions

1. What are the emotional and psychological impacts of living with cardiomyopathy? How are these best treated and managed?
2. How often should family members at risk of developing cardiomyopathy be screened and which are the best tests to use? When is it safe to stop screening?
3. Should treatment for cardiomyopathy be tailored to the individual, e.g. based on their specific gene variant, age or gender?
4. What triggers the start of cardiomyopathy (e.g. age, stress, pregnancy, other health conditions)? How do these triggers work and can they be blocked?
5. Are there treatments which can prevent cardiomyopathy developing in people at risk? Are there treatments to stop it getting worse in people with symptoms?
6. What are the biological mechanisms that change heart muscle cells in cardiomyopathy? Could this understanding lead to new treatments?
7. Why are people with the same genetic variant affected differently? Why do some people with a genetic variant never develop cardiomyopathy? Could this understanding lead to new treatments?
8. Do people with cardiomyopathy experience better outcomes if they are treated at a specialist clinic rather than a general clinic?
9. What does ongoing monitoring and long-term care for people with cardiomyopathy need to include?
10. What are the best approaches to cardiac rehabilitation for people with cardiomyopathy?

The following questions were also discussed and put in order of priority at the workshop:

11. Are there tests which can predict the risk of getting worse, heart failure and/or sudden death in people with cardiomyopathy? How do people with different levels of risk need to be treated differently?
12. Can gene therapy be used to prevent cardiomyopathy developing in people at risk or to treat people with symptoms?
13. What is a safe and beneficial level of exercise for adults and children with a genetic risk of cardiomyopathy?
14. Which people with cardiomyopathy benefit most from an ICD (implantable cardioverter defibrillator)?
15. What is a safe and beneficial level of physical activity or exercise for people with cardiomyopathy?
16. What genetic and environmental factors influence the risk of getting worse, heart failure and/ or sudden death in people with cardiomyopathy?
17. What are the best ways for people with cardiomyopathy to monitor their symptoms at home and to know when to seek medical help?
18. What are the long-term side-effects of treatment for cardiomyopathy? Can drugs with fewer side-effects be developed?
19. What happens to people with cardiomyopathy as they get older? How does the condition change over time?
20. Can drug treatment reverse changes to the heart to a point where people with cardiomyopathy can safely stop their medication?
21. Can stem cells be used to repair or restore damaged heart muscle in people with cardiomyopathy?
22. How is cardiomyopathy linked to atrial fibrillation (an irregular and fast heartbeat)?
23. What are the best ways to treat heart failure in people with cardiomyopathy?
24. What causes fatigue in people with cardiomyopathy and how is it best treated and managed?
25. What lifestyle changes (e.g. diet, avoiding alcohol) help people with cardiomyopathy to avoid getting worse, and/or reduce their risk of heart failure and sudden death?
26. What are the best ways to treat and manage breathlessness in people with cardiomyopathy?

Key documents

These documents set out the aims, objectives and commitments of the PSP.

Cardiomyopathy PSP protocol

Cardiomyopathy PSP Steering Group terms of reference

Cardiomyopathy PSP question verification form

Cardiomyopathy PSP engagement summary