NCT05937828

OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

"French National Cohort of Patients With Pediatric-onset of Autoimmune Cytopenia (OBS'CEREVANCE Cohort)"

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 3,500 (estimated)
• Sponsor: University Hospital, Bordeaux · Academic / Other
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

From 2004, OBS'CEREVANCE is a national real-world prospective clinical cohort of patients with auto-immune cytopenia of pediatric-onset : Immune thrombocytopenia (ITP), Autoimmune Hemolytic anemia (AIHA), or Evans syndrome (all bi or tri cytopenias). Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.

Detailed description

Immune thrombocytopenic purpura (ITP) and autoimmune hemolytic anemia (AHAI) are rare childhood diseases that involve autoimmune destruction of platelets and erythrocytes respectively. They may be associated with an even rarer entity, Evans syndrome (ES). These three conditions are referred to as autoimmune cytopenias (AIC). In association with CAI, patients may present with various immunopathological (IM) manifestations such as lymphoproliferation, autoimmune autoimmune/autoinflammatory organ diseases that may be absent at the time of at the time of diagnosis of CAI and develop during follow-up. Since 2004, the CEREVANCE reference center for childhood autoimmune CEREVANCE has been coordinating a national prospective cohort of patients with pediatric-onset CAI including over 1900 patients (data 05/2023). Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.

Conditions

• Immune Thrombocytopenia
• Autoimmune Hemolytic Anemia
• Evans Syndrome

Interventions

Type	Name	Description
OTHER	data collection	description of the long-term health of adult patients, identification of the heterogenous genetic underlying pathophysiologic contexts, study of the benefit-risk balance of treatments, including the growing development of targeted therapies.

Timeline

• Start date: 2010-09-01
• Primary completion: 2029-12-31
• Completion: 2029-12-31
• First posted: 2023-07-10
• Last updated: 2023-07-10

Locations

9 sites across 1 country: France