Vascular Ehlers-Danlos syndrome is a rare hereditary connective tissue disorder caused by mutations in the collagen type III gene ( COL3A1 ), which leads to a loss of tissue integrity in many organ systems.

Patients with vascular Ehlers-Danlos syndrome have weakened blood vessels and an increased risk of arterial dissection or rupture that can lead to early death.

The researchers assessed the ability of celiprolol, a ?1-adrenoceptor antagonist with a ?2-adrenoceptor agonist action, to prevent arterial dissections and ruptures in vascular Ehlers-Danlos syndrome.

Patients with clinical vascular Ehlers-Danlos syndrome were randomly assigned to 5 years of treatment with celiprolol or to no treatment.

33 patients were positive for mutation of collagen 3A1 (COL3A1). Celiprolol was uptitrated every 6 months by steps of 100 mg to a maximum of 400 mg twice daily. The primary endpoints were arterial events (rupture or dissection, fatal or not).

Mean duration of follow-up was 47 months, with the trial stopped early for treatment benefit.

The primary endpoints were reached by 20% in the celiprolol group and by 50% controls (hazard ratio [HR] 0·36).

Celiprolol might be the treatment of choice for physicians aiming to prevent major complications in patients with vascular Ehlers-Danlos syndrome.

References: