The Rare Diseases Foundation, in partnership with the MCGRE Rare Diseases Health Network and Novo Nordisk, is proud to announce the winner of its research grant in the Humanities and Social Sciences (HSS). This initiative aims to better understand and improve the life course of patients with sickle cell disease or β-thalassemia.
The Winner and His Project
The evaluation committee selected Damien Oudin Doglioni, a researcher at the University of Grenoble Alpes (UGA), for his project entitled Living with beta thalassemia in France: patients' subjective experiences, representation of beta thalassemia, and relationship to treatment (Vivre-BT). Awarded €30,000 over a maximum period of 24 months, this winning project stood out for its relevance and potential impact, particularly its transferability to other hemolytic anemias.
Background of β-Thalassemia
β-thalassemia (BT) is a rare genetic disorder affecting hemoglobin production, caused by more than 500 identified mutations. It affects between 2.5 and 25% of the global population, depending on the region and migration patterns. In Europe, approximately 20,000 people suffer from severe forms of the disease, including nearly 800 in France. Patients with β-thalassemia major (BTM) are dependent on blood transfusions for life. Thanks to recent advances—oral iron chelators, advances in imaging, and multidisciplinary care—life expectancy has improved significantly over the past 20 years. However, daily constraints remain: chronic pain, side effects of treatment, social stigma, and uncertainty about the future, which often complicate treatment adherence and impair quality of life.
Objectives and Methodology of the Vivre-BT Project
Vivre-BT fills a major gap: the lack of French data on patients' subjective experiences. The study will provide a systematic and quantitative analysis of personal experiences, perceptions of the disease, and attitudes toward treatment. It will aim to:
- Conduct a detailed psychological and social assessment.
- Identify factors that impact the quality of life of patients and their loved ones.
- Propose concrete courses of action to optimize the care pathway.
These results, which are expected to be transferable to other similar conditions, will inform practical recommendations for caregivers, associations, and public policy.
A Big Thank You to Our Sponsor
We would like to express our sincere thanks toNovo Nordisk, whose indispensable financial support has made this grant possible. Their exemplary commitment alongside the Rare Diseases Foundation and the MCGRE network strengthens our ability to fund innovative research in the social sciences and humanities, directly benefiting patients and their families.
This call for projects responds to an urgent need for multidisciplinary approaches.
Congratulations to Damien Oudin Doglioni and his team for this recognition! A big thank you to all the candidates for the quality of their proposals, and to the committee experts for their rigorous expertise.