OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

NCT05937828 | Recruiting

• 1 other identifier: CHUBX 2023/08
• Study Type: observational
• Target: 3,500
• Locations: 1 country
• Sites: 9

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.

Conditions

Immune Thrombocytopenia; Autoimmune Hemolytic Anemia; Evans Syndrome

Outcome Measures

Primary Outcomes (1)

• AIC context

  Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).

  Baseline

Secondary Outcomes (4)

• Treatment lines

  every 6 months after baseline up to 19 years

• Adverse drug reactions

  every 6 months after baseline up to 19 years

• Events

  every 6 months after baseline up to 19 years

• AIC context

  every 6 months after baseline up to 19 years

Study Arms (3)

Immune thrombocytopenia (ITP)

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

Other: data collection

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

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

Interventions

data collection 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); Evans syndrome (all bi or tri cytopenias); Immune thrombocytopenia (ITP)

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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)

You may qualify if:

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

You may not qualify if:

• Opposition of legal representative or to data collection

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

Study Sites (9)

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

Amiens, France

RECRUITING

CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique

Angers, France

RECRUITING

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

Besançon, France

RECRUITING

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

Bordeaux, France

RECRUITING

CHU BREST Hôpital Morvan Unité d'Onco-Hématologie

Brest, France

RECRUITING

CHU de Caen Unité d'Onco-Hématologie

Caen, France

RECRUITING

CHU de Clermont Ferrand

Clermont-Ferrand, France

RECRUITING

APHP Hôpital Bicêtre Service de Pédiatrie générale

Paris, France

RECRUITING

CH de Cornouaille Service de Pédiatrie

Quimper, France

RECRUITING

Related Publications (12)

• 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.

  PMID: 28444729 BACKGROUND

• 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.

  PMID: 28748541 BACKGROUND

• 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.

  PMID: 29756344 BACKGROUND

• 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.

  PMID: 30671780 BACKGROUND

• 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.

  PMID: 30940614 BACKGROUND

• 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.

  PMID: 31363182 BACKGROUND

• 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.

  PMID: 32130726 BACKGROUND

• 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.

  PMID: 33340549 BACKGROUND

• 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.

  PMID: 33440924 BACKGROUND

• 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.

  PMID: 35443028 BACKGROUND

• 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.

  PMID: 36882195 BACKGROUND

• 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.

  PMID: 38227934 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Anemia, Hemolytic, Autoimmune; Evans Syndrome

Interventions

Data Collection

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Anemia, Hemolytic; Anemia

Intervention Hierarchy (Ancestors)

Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Nathalie ALADJIDI, MD

  University Hospital, Bordeaux

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nathalie ALADJIDI, MD

CONTACT

+33 557820261; nathalie.aladjidi@chu-bordeaux.fr

Aurore Capelli

CONTACT

0557820877; aurore.capelli@chu-bordeaux.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 19 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2023
• First Posted: July 10, 2023
• Study Start: September 1, 2010
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2029
• Last Updated: July 10, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (9)