Pharmafile Logo

BHF announces drug combination could delay need for high-risk surgery in patients with Marfan syndrome

An estimated one million people worldwide live with the genetic condition, for which there is currently no cure

BHF

The combination of beta-blockers and angiotensin receptor blockers can delay the need for surgery to correct potentially deadly aortic enlargement in people with Marfan syndrome, according to research funded by the British Heart Foundation (BHF).

Marfan syndrome affects the connective tissues responsible for holding the body’s cells in place, causing patients to have weak blood vessels. One of the most serious complications is weakness of the aorta which can expand over time, increasing the risk that it will tear or burst. If this happens, immediate, lifesaving surgery is required.

People with Marfan syndrome – an estimated one million people worldwide – are monitored closely to keep track of the size of their aorta. If the vessel becomes too large, patients are offered surgery to replace the enlarged part of their aorta, but the surgery is still high-risk and recovery can take months, BHF reports.

Beta-blockers and angiotensin receptor blockers are already prescribed to many patients with Marfan syndrome to try to reduce the rate of progressive aortic root enlargement characteristic of this condition, however the separate and combined effects of the treatments were unknown.

The Marfan Treatment Trialists’ (MTT) Collaboration analysed the results of seven previous clinical trials involving 1,442 people with no prior aortic surgery, aiming to understand more about the effects of two medications.

The MTT found that individually, both medicines helped to slow the growth of the aorta, but a combination of the two was more effective than when they were used alone.

Specifically, the combination therapy may reduce the rate of change by at least half and potentially by much more than this which, if maintained over a sustained period, would be expected to delay the need for surgery substantially, the researchers said.

Professor Sir Nilesh Samani, BHF medical director, said: “This news should be hugely reassuring to people with Marfan syndrome. By slowing the growth of their aortas, this simple combination of two cheaply available medications offers hope that those with the condition will be able to live more of their lives free from the threat of needing risky surgery or experiencing a potentially deadly aortic tear.”

The findings were presented at the European Society of Cardiology Congress in Barcelona and published in The Lancet.

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links