OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

Recruiting

• Conditions: Immune Thrombocytopenia; Autoimmune Hemolytic Anemia; Evans Syndrome
• Interventions: Other: data collection

• Registration Number: NCT05937828
• Lead Sponsor: University Hospital, Bordeaux

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.

Study Timeline

• Study Start: 2010-09-01
• Study First Submitted: 2023-03-14
• Status Verified: 2023-07
• Study First Submitted QC: 2023-07-07
• Last Update Submitted: 2023-07-07
• Study First Posted: 2023-07-10
• Last Update Posted: 2023-07-10
• Primary Completion: 2029-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 3500

Inclusion Criteria

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

Exclusion Criteria

• Opposition of legal representative or to data collection

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Evans syndrome (all bi or tri cytopenias)	data collection	Evans syndrome (ES) : simultaneous (less than 1 month) or sequential association of at least two autoimmune cytopenia among ITP, AIHA and autoimmune neutropenia (AIN).
Immune thrombocytopenia (ITP)	data collection	Immune thrombocytopenia (ITP) : defined according to the international working group criteria (Rodeghiero et al., Blood 2009).
Autoimmune Hemolytic anemia (AIHA)	data collection	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.

Primary Outcome Measures

Name	Time	Method
AIC context	Baseline	Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).

Secondary Outcome Measures

Name	Time	Method
Events	every 6 months after baseline up to 19 years	Percentage of patients with other events of interest like cancer, infection, thrombosis, death
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
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).

Trial Locations

Locations (9)

CHU Amiens Picardie Service d' Onco-Immuno-Hématologie Pédiatrique; 🇫🇷 Amiens, France

BESANCON CHU de Besançon Hôpital Jean MINJOZ Unité d'Hémato-Oncologie Pédiatrique, Pédiatrie 1; 🇫🇷 Besançon, France

CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique; 🇫🇷 Angers, France

CHU de Clermont Ferrand; 🇫🇷 Clermont-Ferrand, France

CHU de Bordeaux - Unité d'Hématologie et d'Oncologie pédiatrique; 🇫🇷 Bordeaux, France

CHU BREST Hôpital Morvan Unité d'Onco-Hématologie; 🇫🇷 Brest, France

CHU de Caen Unité d'Onco-Hématologie; 🇫🇷 Caen, France

APHP Hôpital Bicêtre Service de Pédiatrie générale; 🇫🇷 Paris, France

CH de Cornouaille Service de Pédiatrie; 🇫🇷 Quimper, France