The focus was on vascular liver disease, organized in various edge-cutting sessions:
Session 1: Definition of the needs / position papers / guidelines
Session 2: Research strategies in VLD
Session 3: ERN RARE-LIVER / VALDIG organization: Communication.
Session 4: Scientific research projects proposals
- The first part of the workshop summarized these questions and the current unmet needs in these diseases.
- The second part identified and clarified the existing databases and the advances for Castor database.
- In a third part ongoing calls were presented that have been accepted in VLD and future proposals.
- Collaborative scientific proposals were discussed to be launched on a European scale.
- Future patients’ webinars were planned as well as
- Systematic easy to understand summaries to patients of all studies on vascular liver diseases.
The input from patient’s associations was invaluable in recognizing the need for greater collaboration and involving them at the earliest stages of decision-making processes.
Potential impact on rare liver disease patient care:
Although recent studies from our consortium have provided a marked impetus to the field of clinical research on vascular liver diseases, crucial information is still lacking, particularly in the areas of etiology, and treatment. Etiology is still unknown in 40% of the patients; NGS seems to have a clear impact on diagnosis and prognosis, similarly prothrombotic markers may play a role in treatment monitoring. Therapeutic trials, although badly needed have scarcely been performed, so that recommendations for treatments (with potentially fatal side effects in these high bleeding risk population) rely on limited data. The workshop helped launch collaborative projects to answer these questions that have a clear impact on patients care.