OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

NCT ID: NCT05937828

Last Updated: 2023-07-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

3500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2010-09-01

Study Completion Date

2029-12-31

Brief Summary

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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

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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.

Conditions

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Immune Thrombocytopenia Autoimmune Hemolytic Anemia Evans Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Immune thrombocytopenia (ITP)

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

data collection

Intervention Type OTHER

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.

data collection

Intervention Type OTHER

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).

data collection

Intervention Type OTHER

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.

Interventions

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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.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* Opposition of legal representative or to data collection
Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Bordeaux

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Nathalie ALADJIDI, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Bordeaux

Locations

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CHU Amiens Picardie Service d' Onco-Immuno-Hématologie Pédiatrique

Amiens, , France

Site Status RECRUITING

CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique

Angers, , France

Site Status RECRUITING

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

Besançon, , France

Site Status RECRUITING

CHU de Bordeaux - Unité d'Hématologie et d'Oncologie pédiatrique

Bordeaux, , France

Site Status RECRUITING

CHU BREST Hôpital Morvan Unité d'Onco-Hématologie

Brest, , France

Site Status RECRUITING

CHU de Caen Unité d'Onco-Hématologie

Caen, , France

Site Status RECRUITING

CHU de Clermont Ferrand

Clermont-Ferrand, , France

Site Status RECRUITING

APHP Hôpital Bicêtre Service de Pédiatrie générale

Paris, , France

Site Status RECRUITING

CH de Cornouaille Service de Pédiatrie

Quimper, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Nathalie ALADJIDI, MD

Role: CONTACT

+33 557820261

Aurore Capelli

Role: CONTACT

0557820877

Facility Contacts

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Valérie LI THIAO TE

Role: primary

Isabelle PELLIER

Role: primary

[email protected]

Role: backup

Nathalie CHEIKH

Role: primary

[email protected]

Role: backup

Nathalie ALADJIDI

Role: primary

LIANA CARAUSU

Role: primary

Marianna DEPARIS

Role: primary

Eric DORE

Role: primary

Corinne GUITTON

Role: primary

Philippe VIC

Role: primary

References

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Ducassou S, Leverger G, Fernandes H, Chambost H, Bertrand Y, Armari-Alla C, Nelken B, Monpoux F, Guitton C, Leblanc T, Fisher A, Lejars O, Jeziorski E, Fouissac F, Lutz P, Pasquet M, Pellier I, Piguet C, Vic P, Bayart S, Marie-Cardine A, Michel M, Perel Y, Aladjidi N. Benefits of rituximab as a second-line treatment for autoimmune haemolytic anaemia in children: a prospective French cohort study. Br J Haematol. 2017 Jun;177(5):751-758. doi: 10.1111/bjh.14627. Epub 2017 Apr 26.

Reference Type BACKGROUND
PMID: 28444729 (View on PubMed)

Aladjidi N, Jutand MA, Beaubois C, Fernandes H, Jeanpetit J, Coureau G, Gilleron V, Kostrzewa A, Lauroua P, Jeanne M, Thiebaut R, Leblanc T, Leverger G, Perel Y. Reliable assessment of the incidence of childhood autoimmune hemolytic anemia. Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26683. Epub 2017 Jul 27.

Reference Type BACKGROUND
PMID: 28748541 (View on PubMed)

Penel Page M, Bertrand Y, Fernandes H, Kherfellah D, Leverger G, Leblanc T, Libbrecht C, Michel G, Jeziorski E, Armari-Alla C, Nelken B, Pellier I, Pasquet M, Perel Y, Aladjidi N. Treatment with cyclosporin in auto-immune cytopenias in children: The experience from the French cohort OBS'CEREVANCE. Am J Hematol. 2018 May 14. doi: 10.1002/ajh.25137. Online ahead of print. No abstract available.

Reference Type BACKGROUND
PMID: 29756344 (View on PubMed)

Pincez T, Neven B, Le Pointe HD, Varlet P, Fernandes H, Gareton A, Leverger G, Leblanc T, Chambost H, Michel G, Pasquet M, Millot F, Hermine O, Mathian A, Hully M, Zephir H, Hamidou M, Durand JM, Perel Y, Landman-Parker J, Rieux-Laucat F, Aladjidi N. Neurological Involvement in Childhood Evans Syndrome. J Clin Immunol. 2019 Feb;39(2):171-181. doi: 10.1007/s10875-019-0594-3. Epub 2019 Jan 22.

Reference Type BACKGROUND
PMID: 30671780 (View on PubMed)

Hadjadj J, Aladjidi N, Fernandes H, Leverger G, Magerus-Chatinet A, Mazerolles F, Stolzenberg MC, Jacques S, Picard C, Rosain J, Fourrage C, Hanein S, Zarhrate M, Pasquet M, Abou Chahla W, Barlogis V, Bertrand Y, Pellier I, Colomb Bottollier E, Fouyssac F, Blouin P, Thomas C, Cheikh N, Dore E, Pondarre C, Plantaz D, Jeziorski E, Millot F, Garcelon N, Ducassou S, Perel Y, Leblanc T, Neven B, Fischer A, Rieux-Laucat F; members of the French Reference Center for Pediatric Autoimmune Cytopenia (CEREVANCE). Pediatric Evans syndrome is associated with a high frequency of potentially damaging variants in immune genes. Blood. 2019 Jul 4;134(1):9-21. doi: 10.1182/blood-2018-11-887141. Epub 2019 Apr 2.

Reference Type BACKGROUND
PMID: 30940614 (View on PubMed)

Margot H, Boursier G, Duflos C, Sanchez E, Amiel J, Andrau JC, Arpin S, Brischoux-Boucher E, Boute O, Burglen L, Caille C, Capri Y, Collignon P, Conrad S, Cormier-Daire V, Delplancq G, Dieterich K, Dollfus H, Fradin M, Faivre L, Fernandes H, Francannet C, Gatinois V, Gerard M, Goldenberg A, Ghoumid J, Grotto S, Guerrot AM, Guichet A, Isidor B, Jacquemont ML, Julia S, Khau Van Kien P, Legendre M, Le Quan Sang KH, Leheup B, Lyonnet S, Magry V, Manouvrier S, Martin D, Morel G, Munnich A, Naudion S, Odent S, Perrin L, Petit F, Philip N, Rio M, Robbe J, Rossi M, Sarrazin E, Toutain A, Van Gils J, Vera G, Verloes A, Weber S, Whalen S, Sanlaville D, Lacombe D, Aladjidi N, Genevieve D. Immunopathological manifestations in Kabuki syndrome: a registry study of 177 individuals. Genet Med. 2020 Jan;22(1):181-188. doi: 10.1038/s41436-019-0623-x. Epub 2019 Jul 31.

Reference Type BACKGROUND
PMID: 31363182 (View on PubMed)

Ducassou S, Gourdonneau A, Fernandes H, Leverger G, Pasquet M, Fouyssac F, Bayart S, Bertrand Y, Michel G, Jeziorski E, Thomas C, Abouchallah W, Viard F, Guitton C, Cheikh N, Pellier I, Carausu L, Droz C, Leblanc T, Aladjidi N; Centre de Reference National des Cytopenies Auto-immunes de l'Enfant (CEREVANCE). Second-line treatment trends and long-term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years. Br J Haematol. 2020 Jun;189(5):931-942. doi: 10.1111/bjh.16448. Epub 2020 Mar 4.

Reference Type BACKGROUND
PMID: 32130726 (View on PubMed)

Ducassou S, Fernandes H, Savel H, Bertrand Y, Leblanc T, Abou Chahla W, Pasquet M, Leverger G, Barlogis V, Thomas C, Bayart S, Pellier I, Armari-Alla C, Guitton C, Cheikh N, Kherfellah D, Vassal G, Thiebaut R, Laghouati S, Aladjidi N. Prospective Evaluation of the First Option, Second-Line Therapy in Childhood Chronic Immune Thrombocytopenia: Splenectomy or Immunomodulation. J Pediatr. 2021 Apr;231:223-230. doi: 10.1016/j.jpeds.2020.12.018. Epub 2020 Dec 17.

Reference Type BACKGROUND
PMID: 33340549 (View on PubMed)

Pincez T, Fernandes H, Leblanc T, Michel G, Barlogis V, Bertrand Y, Neven B, Chahla WA, Pasquet M, Guitton C, Marie-Cardine A, Pellier I, Armari-Alla C, Benadiba J, Blouin P, Jeziorski E, Millot F, Paillard C, Thomas C, Cheikh N, Bayart S, Fouyssac F, Piguet C, Deparis M, Briandet C, Dore E, Picard C, Rieux-Laucat F, Landman-Parker J, Leverger G, Aladjidi N. Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden. Haematologica. 2022 Feb 1;107(2):457-466. doi: 10.3324/haematol.2020.271106.

Reference Type BACKGROUND
PMID: 33440924 (View on PubMed)

Pincez T, Aladjidi N, Heritier S, Garnier N, Fahd M, Abou Chahla W, Fernandes H, Dichamp C, Ducassou S, Pasquet M, Bayart S, Moshous D, Cheikh N, Paillard C, Plantaz D, Jeziorski E, Thomas C, Guitton C, Deparis M, Marie Cardine A, Stephan JL, Pellier I, Dore E, Benadiba J, Pluchart C, Briandet C, Barlogis V, Leverger G, Leblanc T. Determinants of long-term outcomes of splenectomy in pediatric autoimmune cytopenias. Blood. 2022 Jul 21;140(3):253-261. doi: 10.1182/blood.2022015508.

Reference Type BACKGROUND
PMID: 35443028 (View on PubMed)

Pincez T, Fernandes H, Pasquet M, Abou Chahla W, Granel J, Heritier S, Fahd M, Ducassou S, Thomas C, Garnier N, Barlogis V, Jeziorski E, Bayart S, Chastagner P, Cheikh N, Guitton C, Paillard C, Lejeune J, Millot F, Li-Thiao Te V, Mallebranche C, Pellier I, Neven B, Armari-Alla C, Carausu L, Piguet C, Benadiba J, Pluchart C, Stephan JL, Deparis M, Briandet C, Dore E, Marie-Cardine A, Leblanc T, Leverger G, Aladjidi N. Impact of age at diagnosis, sex, and immunopathological manifestations in 886 patients with pediatric chronic immune thrombocytopenia. Am J Hematol. 2023 Jun;98(6):857-868. doi: 10.1002/ajh.26900. Epub 2023 Mar 21.

Reference Type BACKGROUND
PMID: 36882195 (View on PubMed)

Granel J, Fernandes H, Bader-Meunier B, Guth A, Richer O, Pillet P, Leverger G, Ducassou S, Fahd M, Pasquet M, Garnier N, Barlogis V, Guitton C, Jeziorski E, Thomas C, Bayart S, Cheikh N, Paillard C, Abou Chahla W, Chastagner P, Neven B, Millot F, Lejeune J, Li-Thiao Te V, Armari-Alla C, Briandet C, Carausu L, Deparis M, Piguet C, Benadiba J, Marie-Cardine A, Stephan JL, Pellier I, Pluchart C, Dore E, Michaux K, Heritier S, Leblanc T, Aladjidi N. Antinuclear antibody-associated autoimmune cytopenia in childhood is a risk factor for systemic lupus erythematosus. Blood. 2024 Apr 18;143(16):1576-1585. doi: 10.1182/blood.2023021884.

Reference Type DERIVED
PMID: 38227934 (View on PubMed)

Other Identifiers

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CHUBX 2023/08

Identifier Type: -

Identifier Source: org_study_id

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