Focus On Caring For The Heart In Duchenne Muscular Dystrophy
We are often asked by families what medicines are used to help heart or cardiac function in patients with DMD. This is a difficult question to answer as there have never been any definitive trials looking at what medicines might be best to take, and when to start taking them.
Current medications that have been explored in small numbers of patients potentially include ACE inhibitors, such as Lisinopril; angiotensin receptor blockers (ARBs), such as Losarten, and Eplerenone.
This month a Cochrane review was published in which the focus was on drugs currently used to prevent or treat heart complications in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and X-linked dilated cardiomyopathy
What is a Cochrane review?
Cochrane is a British charity and its aim is to look widely at medical research findings published in recognised and respected scientific journals in order to articulate a consensus view. Cochrane aims to collate and interpret research findings such that they make it much easier and clearer for medical professionals/health organisations (and members of the general public) to make decisions, about health interventions, based on evidence. Cochrane is a volunteer organisation and has input from approximately 30,000 volunteer experts from around the world.
What did the review say?
Based on the available evidence from Randomised Controlled Trials, early treatment with Angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) may help people with DMD. In boys with early heart involvement, the effect of ACE inhibitors and ARBs may be equivalent; however, the evidence is very uncertain. Findings from non-randomised studies, some of which have been long term, have led to the use of these drugs in daily clinical practice. Very low-certainty evidence indicates that adding eplerenone might give additional benefit in DMD when early cardiomyopathy is detected. Cochrane did not find any clinically meaningful effect of growth hormone or idebenone in the studies examined. Additionally, the trials provided only low or very low-certainty evidence on side effects.
Our Director of research, Dr David Bull comments:
“What the review paper is saying is that, of the drugs mentioned in the review, the best evidence for a possible small benefit is associated with ACEIs or ARBs (neither is better than the other). Prescribing of these drugs for DMD is not uncommon. There might be a marginal benefit for the use of eplerenone but there is almost none for the addition of growth hormone or idebenone.”
Please click here to read the full lay version of the paper.
Please do also read chapter 12 of the Family Guide for the new Standards of Care guidelines HERE which recommends starting cardiac medicine before the age of 10.
One of authors of the report is Dr John Bourke. Dr John Bourke is consultant cardiologist at Newcastle Hospital, Duchenne UK is pleased to working with Dr Bourke having granted him £86,460 to carry out a study of Arrhythmias to improve understanding of the causes of heart disease in DMD. Read more here.
We are also funding £82,405 to Dr Lee Borthwick and Professor Derek Mann at Newcastle University to better understand cardiac fibrosis in DMD. Read more here.
Any questions please email [email protected]
NOTES FOR EDITORS
What is Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.
Who are Duchenne UK? Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.
- Direct Debit – Duchenne Direct
- Individual Donation – Donate
- If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
- Take part in one of our fundraising events – Events
- Text DUCH10 £10 to 70070