Marfan Sartan: a randomized, double-blind, placebo-controlled trial

Area of Science:

• Cardiology
• Genetics
• Pharmacology

Background:

• Marfan syndrome (MFS) is a genetic disorder affecting connective tissue, often leading to aortic dilatation and dissection.
• Current management of MFS primarily focuses on preventing aortic complications, with beta-blockers as standard first-line therapy.

Purpose of the Study:

• To investigate the efficacy of Losartan as an add-on therapy to baseline treatment in patients diagnosed with Marfan syndrome.
• To assess the impact of Losartan on aortic diameter progression and blood pressure control in MFS patients.

Main Methods:

• A double-blind, randomized, multi-center, placebo-controlled trial involving 303 patients with Marfan syndrome (age >10 years).
• Patients received either Losartan or a placebo in addition to their standard therapy, with a median follow-up of 3.5 years.
• Aortic diameter at the sinuses of Valsalva and blood pressure were primary outcome measures.

Main Results:

• Losartan did not significantly alter the rate of aortic diameter increase compared to placebo (0.44 mm/year vs. 0.51 mm/year).
• Patients on Losartan experienced a modest but significant reduction in systolic and diastolic blood pressure (5 mmHg).
• Rates of aortic surgery and mortality were similar between the Losartan and placebo groups.

Conclusions:

• Losartan effectively lowers blood pressure in Marfan syndrome patients but does not inhibit aortic dilatation over a three-year period.
• Beta-blocker therapy should continue as the primary treatment for Marfan syndrome patients.
• Further research may explore long-term effects or different therapeutic strategies for aortic root growth in MFS.