RiTA

WHY?

Porto-sinusoidal vascular disorder (PSVD) is a cluster of diseases that primarily affect the intrahepatic vasculature, resulting in portal hypertension (PH)and its associated life-threatening complications. The exact cause of this damage is still uncertain, and unfortunately, there are no targeted treatments available at present. Patients are only able to receive symptomatic care.

Additionally, the absence of prognostic scores and biomarkers makes it difficult to identify individuals who are at a heightened risk of experiencing adverse outcomes.

PSVD predominantly impacts young individuals and is frequently prone to misdiagnosis. Consequently, it leads to disability, a significant reduction in quality of life, and places a considerable burden on the healthcare system. This burden is especially pronounced for individuals in their childbearing years and those economically active, resulting in substantial costs.

WHAT?

The primary goal of the RiTA project is to investigate the natural history and progression of PSVD in order to develop tools that can predict patient prognosis and identify new therapeutic targets.

Our strategy to achieve these objectives relies on a scientifically strong and multidisciplinary consortium including patient associations. This collaboration will allow for the inclusion of a large number of patients and the pooling of diverse expertise, enabling groundbreaking research using state-of-the-art technology.

Expected Impact

• Enabling the identification of high-risk patients prone to decompensation or mortality through prognostic scores or biomarkers.
• Increasing awareness of this often-overlooked disorder, leading to more timely and accurate diagnoses and reducing instances of misdiagnosis.
• Initiating the discovery process for new treatments
• Empowering patients through active involvement and participation in their own healthcare decisions.
• Quantifying the impact of PSVD on quality of life and identifying relevant PROs that can potentially serve as surrogate endpoints in future studies.