Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center, TWI-LIGHT

Status: Recruiting
Location: See location...
Study Type: Observational
SUMMARY

Objectives: 1. Primary: Assess the impact of cardiovascular risk factors (e.g., hypertension, diabetes) and ADAMTS13 activity on life expectancy in iTTP survivors. 2. Secondary: * Evaluate disease burden in underrepresented groups (pregnant/postpartum women, children, elderly patients). * Analyze the influence of new therapies (caplacizumab, rituximab, recombinant ADAMTS13) on care pathways. * Identify prognostic factors and treatment practices. * Characterize neurocognitive outcomes and quality of life post-iTTP. Methodology: *

Design: Non-interventional, retrospective (MR-004 compliance), using data from standard care. * Inclusion: Patients with confirmed iTTP (thrombocytopenia, hemolytic anemia, ADAMTS13 \<10%), diagnosed within the study period, and ≥1 year of follow-up. * Exclusion: Cancer-associated iTTP, severe sepsis, or patient opposition to data reuse. * Data Collection: Clinical, biological, and therapeutic variables from hospital/consultation records, including cardiovascular events, ADAMTS13 activity, and neurocognitive assessments. * Analysis: Kaplan-Meier survival curves and Cox regression models to identify risk factors for non-iTTP-related death. Expected Outcomes: * Prevalence of cardiovascular comorbidities and their correlation with ADAMTS13 activity. * Insights into iTTP subtypes (e.g., gestational, pediatric) and therapeutic efficacy. * Evidence-based strategies for personalized long-term management. Ethical Framework: * AP-HP-sponsored, with oversight from Sorbonne University's ethics committee. * Patients informed of data reuse with opt-out rights; data archived for 15 years. This landmark study will inform clinical guidelines, optimize survivor care, and address unmet needs in iTTP management through comprehensive, real-world data analysis.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Patients with a diagnosis of immune mediated TTP

Locations
Other Locations
France
Service d'Hématologie Hôpital Saint-Antoine
RECRUITING
Paris
Contact Information
Primary
Paul COPPO, MD, PHD
aul.coppo@aphp.fr
00 33 1 49 28 34 39
Time Frame
Start Date: 2024-12-02
Estimated Completion Date: 2028-12
Participants
Target number of participants: 1200
Treatments
iTTP patients
The study is structured around one main cohort of all patients with immune thrombotic thrombocytopenic purpura (iTTP).~Subgroup analyses are planned for specific populations: pregnant and postpartum women, pediatric patients, and elderly patients.~There are no separate intervention or control groups; the design is a single retrospective cohort with multiple subgroup analyses.
Sponsors
Leads: Assistance Publique - Hôpitaux de Paris

This content was sourced from clinicaltrials.gov