RiTA

PortoSinusoidal Vascular Disorder: Risk stratification & Therapeutic Approaches (RiTA)

RiTA is a new project which objective is to enhance our understanding of the natural history and progression of PSVD (Portosinusoidal Vascular Disorder) and to define targets for future interventions through a comprehensive four-step approach: 

 

  1. Characterization of heterogeneity and identification of prognostic scores based on clinical data
  2. Identification of patient-reported outcomes (PROs) and quality of life (QoL) measures that may become relevant endpoints 
  3. Development of mechanistic biomarkers with prognostic value 
  4. Identification of actionable therapeutic targets

 

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. 

 

HOW?

To achieve these goals, our project brings together partners from diverse disciplines, including: 

  • Clinicians
  • Experts in social sciences and humanities 
  • Basic researchers
  • Representatives of patients

This collaborative effort is supported by a well-balanced workplan involving participants from five EU countries: France, Italy, Germany, Israel and Spain.

 

ABOUT

The RiTA project involves five European countries through the close collaboration of a group of clinicians, academic researchers, and patient organizations. See below who are the clinicians and hospitals involved in the projects, as well as the academic researchers and their research organizations, and how the project is funded.

 

WORK PACKAGE LEADERS

 

Virginia Hernández-Gea 

Fundació de Recerca Clínic Barcelona-Institut d’Investigacions Biomèdiques August Pi i Sunyer (FRCB-IDIBAPS), Hepatic Hemodynamic Unit 

COORDINATOR of the project

Pierre Emmanuel Rautou

ASSISTANCE PUBLIQUE HOPITAUX DE PARIS (APHP), Service d’Hépatologie; Hôpital Beaujon 

WP1 leader: Characterization of PSVD heterogeneity and development of prognostic scores using readily available data 

Agnes Dumas

INSERM (National Institute for  Health and Medical Research)

WP 2 Leader: Definition of reliable patient reported outcomes (PROs) for PSVD patients

Mathias Heikenwaelder

DEUTSCHES KREBSFORSCHUNGSZENTRUM HEIDELBERG (DKFZ), Chronic Inflammation and Cancer Group

WP 3 Leader: Understand vascular changes associated with PSVD to base the development of future accurate mechanistic biomarkers with prognostic value

 Alberto Zanetto

University of Padova (UPadova), Multivisceral Transplant Unit, Department of Surgery Oncology and Gastroenterology

Early Career Researcher and co-leader of WP3: Understand vascular changes associated with PSVD to base the development of future accurate mechanistic biomarkers with prognostic value

 Shalev Itzkovitz

WEIZMANN INSTITUTE OF SCIENCE, Department of Molecular Cell Biology

WP 4 Leader: Understand the mechanisms involved in PSVD to identify of therapeutic targets 

 

PATIENT ORGANISATIONS

Asociación Española de Enfermos Vasculares Hepáticos (AEVH)

Jose Francisco Tomé Atencia (president) – Spain

Association des maladies vasculaires du foie (AMVF)

Liliane Veniat (president) – France

 

CONSORTIUM COLLABORATORS

 

FUNDING

The RiTA (PortoSinusoidal Vascular Disease: Risk stratification & Therapeutic Approaches) has received funding from (French National Research Agency (ANR), National Institute of Health Carlos III (ISCIII), Chief Scientist office, Ministry of Health (CSO-MOH), German Federal Ministry for Education and Research (BMBF) and Fondazione Telethon (FTELE)) partners of the EJP RD. The EJP RD initiative has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575”

 

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