RaDiCo - ECYSCO

Cystinosis is a rare multisystemic disease caused by cystine accumulation, with an incidence of 1:180,000 births.
Specific treatment with cysteamine slows down renal and extra-renal complications and improves life expectancy. However, knowledge gaps remain concerning the long-term impact of therapy and follow-up.

Recently, new treatments have become available in Europe, including extended-release cysteamine and a new ophthalmic solution. Moreover, stem cell transplantation has emerged as a promising therapeutic option.

The ECYSCO cohort is a multicenter, longitudinal, non-interventional European study designed to collect homogeneous clinical and therapeutic data. It involves 27 centers in France and 4 in other European countries.

The migration and integration of the ECYSCO database into the France Cohortes information system will also make it possible to ensure interoperability with a European registry, enabling broader data sharing for research.

This well-characterized cohort is a major tool for:

• Setting up clinical trials with innovative therapeutic strategies currently under development for cystinosis
• Identifying eligible patients
• Serving as a control group
• Evaluating the efficacy and safety of treatments on disease complications

Scientific and Technical Leads:

• Dr. Aude SERVAIS – Hôpital Necker, AP-HP, Paris
• Prof. Patrick NIAUDET – Hôpital Necker, AP-HP, Paris
  European Reference Network (ERN) ERKNet

This data management initiative, provided by France Cohortes, is supported by the French government through the National Research Agency (ANR) under the France 2030 program, reference ANR-21-PMRB-0012.

• Click here to access to the RaDiCo Program main page.