Long-term outcomes for people with immune TTP in the French national TMA cohort
Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center, TWI-LIGHT
This project uses past medical records to see long-term health outcomes and complications in people in France who had immune thrombotic thrombocytopenic purpura (iTTP).
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1200 (estimated) |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Drugs / interventions | caplacizumab, rituximab |
| Locations | 1 site (Paris) |
| Trial ID | NCT07205861 on ClinicalTrials.gov |
What this trial studies
This is a retrospective, non-interventional analysis coordinated by the French National Reference Center for Thrombotic Microangiopathies (CNR-MAT) using the national registry. It includes clinical, laboratory (including ADAMTS13 activity and troponin), and treatment data (plasma exchange, immunosuppression, caplacizumab) from over 1,200 patients diagnosed between October 1, 2000 and June 1, 2024. The study links acute-phase features and therapies to long-term outcomes such as major adverse cardiovascular events, cognitive impairment, and survival. Patients with cancer-, HIV-, transplant-associated TTP or severe sepsis are excluded from the cohort, and data reuse follows MR-004 non-interventional rules.
Who should consider this trial
Good fit: People diagnosed with autoimmune (immune-mediated) TTP in France who are recorded in the CNR-MAT national registry between October 1, 2000 and June 1, 2024 are the intended population for this analysis.
Not a fit: Patients with cancer-associated, HIV-associated, transplant-associated TTP or severe sepsis were excluded and therefore the results may not apply to them.
Why it matters
Potential benefit: If successful, the findings could identify risk factors and gaps in care that help clinicians reduce long-term complications and improve follow-up strategies for people with iTTP.
How similar studies have performed: Previous observational and registry studies have described acute management and some late outcomes in iTTP, but large national cohorts with extended follow-up remain limited, so this analysis provides important additional evidence.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: -Patients with a diagnosis of immune mediated TTP Exclusion Criteria: * 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
Where this trial is running
Paris
- Service d'Hématologie Hôpital Saint-Antoine — Paris, France (Recruiting)
Study contacts
- Principal investigator: Paul COPPO, MD, PHD — Assistance Publique - Hôpitaux de Paris
- Study coordinator: Paul COPPO, MD, PHD
- Email: aul.coppo@aphp.fr
- Phone: 00 33 1 49 28 34 39
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.