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Complications in Hematology
Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden
Thomas Pincez,1,2 Helder Fernandes,1,3 Thierry Leblanc,4 Gérard Michel,5 Vincent Barlogis,5 Yves Bertrand,6 Bénédicte Neven,7,8,9 Wadih Abou Chahla,10 Marlène Pasquet,11 Corinne Guitton,12 Aude Marie-Cardine,13
Isabelle Pellier,14 Corinne Armari-Alla,15 Joy Benadiba,16 Pascale Blouin,17
Eric Jeziorski,18 Frédéric Millot,19 Catherine Paillard,20 Caroline Thomas,21 Nathalie Cheikh,22 Sophie Bayart,23 Fanny Fouyssac,24 Christophe Piguet,25 Marianna Deparis,26 Claire Briandet,27 Eric Doré,28 Capucine Picard,9,29 Frédéric Rieux-Laucat,8,9 Judith Landman-Parker,30 Guy Leverger30 and Nathalie Aladjidi1,3 on the behalf of members of the French Reference Center for Pediatric Autoimmune Cytopenia (CEREVANCE) and of collaborators from the French Reference Center for Adult Autoimmune Cytopenia (CERECAI).
1Centre de Référence National des Cytopénies Auto-immunes de l’Enfant (CEREVANCE), Bordeaux, France; 2Division of Pediatric Hematology-Oncology, Charles-Bruneau Cancer Center, Department of Pediatrics, Sainte-Justine University Hospital, Université de Montréal, Montréal, Québec, Canada; 3Pediatric Oncology Hematology Unit, University Hospital, Plurithématique CIC (CICP), Centre d’Investigation Clinique (CIC) 1401, INSERM, Bordeaux, France; 4Pediatric Hematology Unit, Robert Debré University Hospital, AP-HP, Paris, France; 5Department of Pediatric Hematology, La Timone Hospital, Marseille University Hospital, Marseille, France; 6Institute of Pediatric Hematology and Oncology, Lyon University Hospital, Lyon, France; 7Pediatric Immuno- Hematology and Rheumatology Department, Necker-Enfants Malades University Hospital, AP-HP, Paris, France; 8Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France; 9Imagine Institute, UMR 1163 INSERM, University of Paris, Paris, France; 10Department of Pediatric Hematology, Jeanne de Flandre Hospital, Lille University Hospital, Lille, France; 11Pediatric Oncology Immunology Hematology Unit, Children’s University Hospital, Toulouse, France; 12Department of Pediatrics, Bicêtre University Hospital, AP-HP, Le Kremlin-Bicêtre, France; 13Department of Pediatric Hematology and Oncology, Rouen University Hospital, Rouen, France; 14Pediatric Unit, Angers University Hospital, Angers, France; 15Pediatric Oncology Hematology Unit, Grenoble University Hospital, Grenoble, France; 16Department of Hemato-Oncology Pediatric, Nice University Hospital, Nice, France; 17Department of Pediatric Hematology- Oncology, Clocheville Hospital, Tours University Hospital, Tours, France; 18Pediatric Oncology Hematology Unit, Arnaud de Villeneuve University Hospital, Montpellier, France; 19Department of Pediatric Hematology, Poitiers University Hospital, Poitiers, France; 20Department of Pediatric Hematology and Oncology, Hautepierre University Hospital, Strasbourg, France; 21Pediatric Hematology Unit, Nantes University Hospital, Nantes, France; 22Department of Pediatric Hematology-Oncology, Besançon University Hospital, Besançon, France; 23Pediatric Hematology Unit, Rennes University Hospital, Rennes, France; 24Pediatric Hematology Unit, Nancy University Hospital, Nancy, France; 25Pediatric Oncology Hematology Unit, Limoges University Hospital, Limoges, France; 26Pediatric Oncology-Hematology Unit, Caen University Hospital, Caen, France; 27Department of Pediatrics, Dijon University Hospital, Dijon, France; 28Pediatric Unit, Clermont-Ferrand University Hospital, Clermont-Ferrand, France; 29Study Center for Primary Immunodeficiencies, Necker-Enfants Malades University Hospital, AP-HP, Paris, France and 30Pediatric Oncology Immunology Hematology Unit, Armand-Trousseau University Hospital, AP-HP, Paris, France
ABSTRACT
Pediatric-onset Evans syndrome (pES) is defined by both immune throm- bocytopenic purpura (ITP) and autoimmune hemolytic anemia (AIHA) before the age of 18 years. There have been no comprehensive long-term studies of this rare disease, which can be associated to various immunopatho- logical manifestations (IM). We report outcomes of the 151 patients with pES and more than 5 years of follow-up from the nationwide French prospective OBS’CEREVANCE cohort. Median age at final follow-up was 18.5 years (range, 6.8–50.0 years) and the median follow-up period was 11.3 years (range, 5.1–38.0 years). At 10 years, ITP and AIHA were in sustained complete
Ferrata Storti Foundation
Haematologica 2022 Volume 107(2):457-466
Correspondence:
NATHALIE ALADJIDI
nathalie.aladjidi@chu-bordeaux.fr
Received: August 31, 2020. Accepted: December 22, 2020. Pre-published: January 14, 2021.
https://doi.org/10.3324/haematol.2020.271106 ©2022 Ferrata Storti Foundation
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