Actively Recruiting
Auto-immune Thrombotic Thrombocytopenic Purpura Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center TWI-LIGHT
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2025-10-03
1200
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
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.
CONDITIONS
Brief Title
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with a diagnosis of immune mediated TTP
You will not qualify if you...
- Cancer-associated iTTP and HIV-associated iTTP
- Severe sepsis
- Disseminated intravascular coagulation with consumption of coagulation factors
- Transplant-associated TTP
- HIV-associated TTP (AIDS stage)
- Patient not affiliated with a social security scheme
- Patient or parent's objection to the reuse of their healthcare data for research
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person or remote) for data eligibility assessment
Duration - Up to 3 years
Participants' health data from diagnosis through up to 3 years of follow-up are analyzed to assess cardiovascular events, risk factors, ADAMTS13 activity trends, neurocognitive outcomes, relapse rates, and mortality.
Data collected retrospectively from medical records and registries; no additional visits required
Trial Site Locations
Total: 1 location
1
Service d'Hématologie Hôpital Saint-Antoine
Paris, France, 75012
Actively Recruiting
Research Team
P
Paul COPPO, MD, PHD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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