OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

University Hospital of Bordeaux

Status: Enrolling

Conditions: Evans Syndrome; Immune Thrombocytopenia; Autoimmune Hemolytic Anemia

Treatments: Other: data collection

Study type: Observational

Funder types: Other

Identifiers

NCT05937828

CHUBX 2023/08

Details and patient eligibility

About

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.

Full 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.

Enrollment: 3,500 estimated patients

Sex: All

Ages: Under 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of ITP, AIHA, Evans Syndrome
• Onset before the age of 18

Exclusion criteria

• Opposition of legal representative or to data collection

Trial design

3,500 participants in 3 patient groups

Immune thrombocytopenia (ITP)

Description:

Immune thrombocytopenia (ITP) : defined according to the international working group criteria (Rodeghiero et al., Blood 2009).

Treatment:

Other: data collection

Autoimmune Hemolytic anemia (AIHA)

Description:

Autoimmune haemolytic anaemia (AIHA) : Hb \< 110 g/L with a positive direct antiglobulin test (DAT) and at least one of the following haemolysis criteria: reticulocyte count \> 120 G/L, free bilirubin \> 17 mmol/L, or haptoglobin \< 10 mg/dL.

Treatment:

Other: data collection

Evans syndrome (all bi or tri cytopenias)

Description:

Evans syndrome (ES) : simultaneous (less than 1 month) or sequential association of at least two autoimmune cytopenia among ITP, AIHA and autoimmune neutropenia (AIN).

Treatment:

Other: data collection