Long-Term Study of Immune Thrombotic Thrombocytopenic Purpura Patients in the French National TMA Cohort Evaluating Cardiovascular Risks, Treatment Effects, and Outcomes

What this Trial Is About

Researchers are investigating immune thrombotic thrombocytopenic purpura iTTP, a rare and life-threatening disorder caused by a severe deficiency of the enzyme ADAMTS13, leading to clotting in small blood vessels and organ damage. This condition often results in serious symptoms like neurological problems and heart events, with very high mortality if untreated. The study focuses on analyzing long-term outcomes, including the impact of cardiovascular risk factors and enzyme activity on life expectancy in over 1,200 patients diagnosed between 2000 and 2024. This research uses a retrospective, observational design based on data collected from routine care stored in the French National Reference Center for Thrombotic Microangiopathies registry. It includes clinical, biological, and treatment information from diagnosis through follow-up, covering cardiovascular events, enzyme activity trends, neurocognitive outcomes, and therapies such as plasma exchange, immunosuppressants, and caplacizumab. Special attention is given to underrepresented groups like pregnant women, children, and elderly patients. Participants data are collected securely and pseudonymized, with analyses to identify risk factors linked to survival and treatment effects. Researchers assess cardiovascular events, quality of life, cognitive function, and mortality over up to three years of follow-up. The study aims to inform personalized care, improve management guidelines, and address long-term risks for iTTP survivors while ensuring ethical oversight and patient data protection.

Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center