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Lung drug trial offers new hope for heart failure patients

An early phase trial of a drug currently used to treat lung fibrosis has shown it may also help patients with a common form of heart failure. The trial, funded by the National Institute for Health Research (NIHR), found that pirfenidone could offer a viable treatment for heart failure with preserved ejection fraction.

The study was led by Dr Christopher Miller, NIHR Clinician Scientist at The University of Manchester and Consultant Cardiologist at Manchester University NHS Foundation Trust. The findings are published in Nature Medicine.

The trial involved 94 patients, randomised into two equal groups to take pirfenidone or a placebo daily. MRI scans and blood tests were used to measure scarring and fluid retention respectively. A year into the trial both measures showed significant improvement in the group taking pirfenidone compared with the group receiving placebo.

Whilst the results are promising for patients with heart failure, larger scale trials will be needed to confirm the findings for pirfenidone before it could be licensed for use in the NHS.

Around a million people in the UK live with heart failure, and up to three in 10 die within a year of diagnosis. Heart failure causes shortness of breath, swelling and extreme fatigue when the heart can no longer pump blood around the body properly.

In about half of affected patients, the forward pumping function of the heart is normal. This is called heart failure with preserved ejection fraction (HFpEF). Scarring or fibrosis of the heart muscle is thought to be a causal factor in HFpEF and is associated with adverse outcomes.

Dr Miller said: “Heart failure is as devastating an illness as some of the most common cancers, however its profile is much lower and treatment options for HFpEF are very limited.

“Based on data from previous studies, this reduction in heart scarring could translate into a substantial reduction in rates of death and admission to hospital for heart failure. The associated improvement in fluid retention provides support for heart scarring having a causal role in heart failure and being an effective treatment target.

“Although further investigation is required, the findings are exciting and suggest that pirfenidone could benefit patients with this common form of heart failure.”

The paper Pirfenidone in heart failure with preserved ejection fraction: a randomized phase 2 trial is published in Nature Medicine.