OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

Official title

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

Overview

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.

Interventions

• 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.

Primary outcome measures

• AIC context [Time frame: Baseline]

Secondary outcome measures (4)

• Treatment lines [Time frame: every 6 months after baseline up to 19 years]
• Adverse drug reactions [Time frame: every 6 months after baseline up to 19 years]
• Events [Time frame: every 6 months after baseline up to 19 years]
• AIC context [Time frame: every 6 months after baseline up to 19 years]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

France · 9 centers

• CHU Amiens Picardie Service d' Onco-Immuno-Hématologie Pédiatrique — Amiens
• CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique — Angers
• BESANCON CHU de Besançon Hôpital Jean MINJOZ Unité d'Hémato-Oncologie Pédiatrique, Pédiatr — Besançon
• CHU de Bordeaux - Unité d'Hématologie et d'Oncologie pédiatrique — Bordeaux
• CHU BREST Hôpital Morvan Unité d'Onco-Hématologie — Brest
• CHU de Caen Unité d'Onco-Hématologie — Caen
• CHU de Clermont Ferrand — Clermont-Ferrand
• APHP Hôpital Bicêtre Service de Pédiatrie générale — Paris
• … and 1 more center

Publications

• 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. PMID 28444729
• 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
• 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
• 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
• 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, Fische PMID 30940614
• 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, L PMID 31363182
• 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 th PMID 32130726
• 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;23 PMID 33340549

Identifiers

NCT: NCT05937828 · CHUBX 2023/08

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗