Clinical trial NCT05937828: OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia
Official title: "French National Cohort of Patients With Pediatric-onset of Autoimmune Cytopenia (OBS'CEREVANCE Cohort)"
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Immune Thrombocytopenia
Autoimmune Hemolytic Anemia
Evans Syndrome
Lead sponsor: University Hospital, Bordeaux
Sponsor class: OTHER
First posted: 2023-07-10
Enrollment: 3500
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Each of the 30 hemato-immunologic pediatric unit in France guaranties the prospective in real time inclusion in the cohort of all the children diagnosed in their unit, before 18 years old, with ITP, AIHA or Evans syndrome diagnosis, whatever the date of diagnosis at the date of inclusion.

This observational cohort is prospective from the day of initial diagnosis of the auto-immune cytopenia The pediatricians from this rare disease CEREVANCE national network follow national guidelines for evaluation and treatment (PNDS 2009, 2017)

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.

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

* Opposition of legal representative or to data collection

Primary outcomes:
measure: AIC context
timeFrame: Baseline
Secondary outcomes:
measure: Treatment lines
timeFrame: every 6 months after baseline up to 19 years
measure: Adverse drug reactions
timeFrame: every 6 months after baseline up to 19 years
measure: Events
timeFrame: every 6 months after baseline up to 19 years
measure: AIC context
timeFrame: every 6 months after baseline up to 19 years
Other outcomes:
Locations:
city: Amiens
country: France
facility: CHU Amiens Picardie Service d' Onco-Immuno-Hématologie Pédiatrique
city: Angers
country: France
facility: CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique
city: Besançon
country: France
facility: BESANCON CHU de Besançon Hôpital Jean MINJOZ Unité d'Hémato-Oncologie Pédiatrique, Pédiatrie 1
city: Bordeaux
country: France
facility: CHU de Bordeaux - Unité d'Hématologie et d'Oncologie pédiatrique
city: Brest
country: France
facility: CHU BREST Hôpital Morvan Unité d'Onco-Hématologie
city: Caen
country: France
facility: CHU de Caen Unité d'Onco-Hématologie
city: Clermont-Ferrand
country: France
facility: CHU de Clermont Ferrand
city: Paris
country: France
facility: APHP Hôpital Bicêtre Service de Pédiatrie générale
city: Quimper
country: France
facility: CH de Cornouaille Service de Pédiatrie
Study officials:
affiliation: University Hospital, Bordeaux
name: Nathalie ALADJIDI, MD
role: PRINCIPAL_INVESTIGATOR
References:
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