Clinical Outcomes and Patient Experiences With Celiprolol Therapy in Vascular Ehlers-Danlos Syndrome: The First Non-European Cohort

Heterozygous pathogenic COL3A1 variants → genetically confirmed vEDS; study of celiprolol efficacy and patient experience in a non-European (Japanese) cohort

In the first non-European cohort of vascular Ehlers-Danlos syndrome (vEDS) treated with celiprolol, 26 Japanese patients (mean follow-up: 96.6 months) were included. One patient died of hepatic artery rupture, 11 had vascular events, and 14 remained event-free. No significant association was found between COL3A1 genotype and vascular event occurrence. Interviews with 11 patients reveal that celiprolol provides substantial psychological benefit, promoting a proactive approach to disease management.

Synthesis written by Geno'X. For the full original abstract, please refer to the source publication.

This study confirms that celiprolol is used in real-world practice in an Asian population with acceptable tolerability, but the data remain insufficient to conclude significant efficacy in this population. The lack of genotype-event correlation is a reminder of the difficulty in predicting individual course in vEDS. The qualitative contribution on patient experience is original and clinically relevant for counseling.

Clinical impact: 2/3 · Evidence strength: 2/3 · Novelty: 0/2 · Sample size: 1/1 · Journal quality: 1/1 → Total: 6/10