Updated: Dec 30, 2021
The April 2019 study published in the Journal of American College of Cardiology is mostly good news for vEDS patients; however, it leaves some major questions unanswered.
In April 2019, an observational study of vascular EDS was published in the Journal of American College of Cardiology. The good news is that the multi-pronged approach that researchers are using at Hôpital Européen Georges Pompidou in Paris is having a meaningful impact on life expectancy for vEDS patients. The hospital, which contains the French National Referral Center for Rare Vascular Diseases, has been treating vEDS patients with the following approach:
Regular follow-up with knowledgeable physicians: Patients in the program had follow-up visits every 6 to 9 months. If the patient was making changes to medication, the follow-ups were made even more frequently
Periodic imaging: All patients received imaging at the time of diagnosis to establish a baseline (i.e., ultrasound, magnetic resonance angiography (MRA), or computed tomography angiogram (CTA)). Patients received a repeat scan at least every 12-18 months
Medical management: Patients were given the beta-blocker celiprolol. At the start of the study, only 33% of patients were taking celiprolol and by the end of the study 90% of patients were taking celiprolol either alone or in combination with another blood pressure medication
Coordinated emergency care: Patients and local physicians had access to a “hotline” that allowed them to contact the main center of excellence in Paris in the event an emergency occurred, giving them access to vEDS experts wherever they were
Photo: Hôpital Européen Georges Pompidou in Paris
During the 17 years studied, the researchers saw excellent outcomes in their vEDS population. In total, their 1-year overall survival was 99.3% and their 5-year overall survival was 89.9%. We believe this is a positive for vEDS patients and reminds us of the progress that Marfan Syndrome achieved between the 1970s-1990s, which FIGHT vEDS posted about in November 2018.
These results are significant and those of us outside of Europe should certainly learn from this approach. What we can’t conclude is how much of a role celiprolol played in the results. The researchers were direct regarding the limitation of the study, saying:
“It is difficult to formally assess this beneficial effect (the benefit of celiprolol on survival) in the absence of a placebo-controlled prospective trial, because other confounders might have influenced this observation”.
Dr. Julie de Backer, a vEDS researcher not involved in the study, wrote in an editorial:
"We appreciate this report very much, especially because its main message is that patients with this rare disorder do benefit from a structured multi-disciplinary follow-up in a strong reference center. Whether the systematic treatment with celiprolol has an additional genuine pharmacological beneficial effect or helps ensure better follow up cannot be answered with this study. The only way to determine if it is celiprolol contributing to the better outcome is to conduct a randomized prospective trial comparing celiprolol to another beta-blocker in patients with molecularly confirmed vEDS."
While conclusions on celiprolol cannot be made based on this study alone, we should still be excited by this promising research! This information helps us understand vEDS patients better and may help guide us in the direction of a better approach to treatment and management.
For a detailed write-up of the entire study, please visit the FIGHT vEDS Research page.