Autoimmune Hemolytic Anemia, Evans Syndrome, Immune Thrombocytopenia
Conditions
Brief 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.
Interventions
OTHERdata 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.
Sponsors
University Hospital, Bordeaux
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of ITP, AIHA, Evans Syndrome * Onset before the age of 18
Exclusion criteria
* Opposition of legal representative or to data collection
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| AIC context | Baseline | Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment lines | every 6 months after baseline up to 19 years | Percentage of patients with each treatment by line of treatments |
| Adverse drug reactions | every 6 months after baseline up to 19 years | Percentage of patients with adverse drug reaction reported by investigators |
| Events | every 6 months after baseline up to 19 years | Percentage of patients with other events of interest like cancer, infection, thrombosis, death |
| AIC context | every 6 months after baseline up to 19 years | Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID). |
Countries
France
Contacts
Primary ContactNathalie ALADJIDI, MD
Backup ContactAurore Capelli
Outcome results
None listed