OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia (OBS'CEREVANCE)

July 7, 2023 updated by: University Hospital, Bordeaux

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

3500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Amiens, France
      • Angers, France
        • Recruiting
        • CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique
        • Contact:
          • Isabelle PELLIER
        • Contact:
          • ispellier@chu-angers.fr
      • Besançon, France
        • Recruiting
        • BESANCON CHU de Besançon Hôpital Jean MINJOZ Unité d'Hémato-Oncologie Pédiatrique, Pédiatrie 1
        • Contact:
          • Nathalie CHEIKH
        • Contact:
          • ncheikh@chu-besancon.fr
      • Bordeaux, France
      • Brest, France
      • Caen, France
        • Recruiting
        • CHU de Caen Unité d'Onco-Hématologie
        • Contact:
      • Clermont-Ferrand, France
      • Paris, France
        • Recruiting
        • APHP Hôpital Bicêtre Service de Pédiatrie générale
        • Contact:
      • Quimper, France

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

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)

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
AIC context
Time Frame: Baseline
Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).
Baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment lines
Time Frame: every 6 months after baseline up to 19 years
Percentage of patients with each treatment by line of treatments
every 6 months after baseline up to 19 years
Adverse drug reactions
Time Frame: every 6 months after baseline up to 19 years
Percentage of patients with adverse drug reaction reported by investigators
every 6 months after baseline up to 19 years
Events
Time Frame: every 6 months after baseline up to 19 years
Percentage of patients with other events of interest like cancer, infection, thrombosis, death
every 6 months after baseline up to 19 years
AIC context
Time Frame: every 6 months after baseline up to 19 years
Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).
every 6 months after baseline up to 19 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Bordeaux

Investigators

  • Principal Investigator: Nathalie ALADJIDI, MD, University Hospital, Bordeaux

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2010

Primary Completion (Estimated)

December 31, 2029

Study Completion (Estimated)

December 31, 2029

Study Registration Dates

First Submitted

March 14, 2023

First Submitted That Met QC Criteria

July 7, 2023

First Posted (Actual)

July 10, 2023

Study Record Updates

Last Update Posted (Actual)

July 10, 2023

Last Update Submitted That Met QC Criteria

July 7, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CHUBX 2023/08

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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