MTT: Marfan Treatment Trialists' Collaboration

Marfan syndrome is a genetic disorder of connective tissue which is associated with a substantial risk of serious cardiovascular complications. A number of trials of novel therapies have been established, all of which aim to study the benefits (or harms) of these treatments in patients with Marfan syndrome.

In 2012, in collaboration with Dr Alex Pitcher, the CTSU established the Marfan Treatment Trialists’ (MTT) Collaboration, involving trialists, meta-analysts, statisticians, clinicians and basic scientists, with the aim of conducting a collaborative overview (or meta-analysis) of individual patient data from all trials of angiotensin receptor blockers in Marfan syndrome worldwide. A protocol for the meta-analysis was written by the group prior to the results of the main trials being known, and was published in the American Heart Journal.

The meta-analysis, which includes individual patient data from around 1442 participants, was published in The Lancet in 2022. It showed that Angiotensin Receptor Blockers (ARBs) and beta-blockers have similar, substantial and independent effects on reducing the rate of enlargement of the aortic root and that combination drug therapy with ARBs and beta-blockers may delay the need for surgery to correct aortic enlargement in people with Marfan syndrome.