Beta-blockers for preventing aortic dissection in Marfan syndrome

Area of Science:

• Genetics and наследственные заболевания
• Cardiology and cardiovascular diseases
• Pharmacology and drug therapy

Background:

• Marfan syndrome is a genetic connective tissue disorder caused by FBN1 gene mutations.
• It impacts multiple organ systems, with cardiovascular complications like aortic root dilatation being life-threatening.
• Aortic dissection and rupture are major causes of morbidity and mortality in Marfan syndrome.

Purpose of the Study:

• To evaluate the long-term effectiveness and safety of beta-blocker treatment versus placebo or no treatment in Marfan syndrome patients.
• To determine if beta-blockers reduce mortality and major cardiovascular events in this population.

Main Methods:

• A systematic search of multiple databases was conducted up to June 2017.
• Inclusion criteria specified randomized controlled trials (RCTs) of at least one-year duration comparing beta-blocker monotherapy to placebo, no treatment, or surveillance.
• One open-label, single-center RCT with 70 participants was included, comparing propranolol to no treatment.

Main Results:

• The single RCT showed no significant reduction in all-cause mortality or major non-fatal clinical endpoints (aortic dissection, regurgitation, surgery, heart failure) with beta-blocker therapy.
• A statistically significant reduction in aortic dilatation rate was observed with propranolol treatment.
• The study was judged to be at high risk of bias, and the overall quality of evidence was low.

Conclusions:

• The current evidence from one low-quality RCT is insufficient to draw definitive conclusions on the clinical utility of beta-blockers for Marfan syndrome.
• High-quality randomized trials are necessary to assess the long-term efficacy and safety of beta-blocker treatment.
• Future research should prioritize reporting comprehensive clinical endpoints and adverse events to guide clinical practice.