Your browser doesn't support javascript.
loading
Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden.
Pincez, Thomas; Fernandes, Helder; Leblanc, Thierry; Michel, Gérard; Barlogis, Vincent; Bertrand, Yves; Neven, Bénédicte; Chahla, Wadih Abou; Pasquet, Marlène; Guitton, Corinne; Marie-Cardine, Aude; Pellier, Isabelle; Armari-Alla, Corinne; Benadiba, Joy; Blouin, Pascale; Jeziorski, Eric; Millot, Frédéric; Paillard, Catherine; Thomas, Caroline; Cheikh, Nathalie; Bayart, Sophie; Fouyssac, Fanny; Piguet, Christophe; Deparis, Marianna; Briandet, Claire; Dore, Eric; Picard, Capucine; Rieux-Laucat, Frédéric; Landman-Parker, Judith; Leverger, Guy; Aladjidi, Nathalie.
Afiliação
  • Pincez T; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France; Division of Pediatric Hematology-Oncology, Charles-Bruneau Cancer Center, Department of Pediatrics, Sainte-Justine University Hospital, Université de Montréal, Montréal, Québec.
  • Fernandes H; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France; Pediatric Oncology Hematology Unit, University Hospital, Plurithématique CIC (CICP), Centre d'Investigation Clinique (CIC) 1401, INSERM Bordeaux.
  • Leblanc T; Pediatric Hematology Unit, Robert Debré University Hospital, AP-HP, Paris.
  • Michel G; Department of Pediatric Hematology, La Timone Hospital, Marseille University Hospital, Marseille.
  • Barlogis V; Department of Pediatric Hematology, La Timone Hospital, Marseille University Hospital, Marseille.
  • Bertrand Y; Institute of Pediatric Hematology and Oncology, Lyon University Hospital, Lyon.
  • Neven B; Pediatric Immuno-Hematology and Rheumatology Department, Necker-Enfants Malades University Hospital, AP-HP, Paris, France; Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France; Imagine Institute, UMR 1163 INSERM and Paris University, Paris.
  • Chahla WA; Department of Pediatric Hematology, Jeanne de Flandre Hospital, Lille University Hospital, Lille.
  • Pasquet M; Pediatric Oncology Immunology Hematology Unit, Children's University Hospital, Toulouse.
  • Guitton C; Department of Pediatrics, Bicêtre University Hospital, AP-HP, Le Kremlin-Bicêtre.
  • Marie-Cardine A; Department of Pediatric Hematology and Oncology, Rouen University Hospital, Rouen.
  • Pellier I; Pediatric Unit, Angers University Hospital, Angers.
  • Armari-Alla C; Pediatric Oncology Hematology Unit, Grenoble University Hospital, Grenoble.
  • Benadiba J; Department of Hemato-Oncology Pediatric, Nice University Hospital, Nice.
  • Blouin P; Department of Pediatric Hematology-Oncology, Clocheville Hospital, Tours University Hospital, Tours.
  • Jeziorski E; Pediatric Oncology Hematology Unit, Arnaud de Villeneuve University Hospital, Montpellier.
  • Millot F; Department of Pediatric Hematology, Poitiers University Hospital, Poitiers.
  • Paillard C; Department of Pediatric Hematology and Oncology, Hautepierre University Hospital, Strasbourg.
  • Thomas C; Pediatric Hematology Unit, Nantes University Hospital, Nantes.
  • Cheikh N; Department of Pediatric Hematology-Oncology, Besanc_on University Hospital, Besanc_on.
  • Bayart S; Pediatric Hematology Unit, Rennes University Hospital, Rennes.
  • Fouyssac F; Pediatric Hematology Unit, Nancy University Hospital, Nancy.
  • Piguet C; Pediatric Oncology Hematology Unit, Limoges University Hospital, Limoges.
  • Deparis M; Pediatric Oncology-Hematology Unit Department, Caen University Hospital, Caen.
  • Briandet C; Department of Pediatrics, Dijon University Hospital, Dijon.
  • Dore E; Pediatric Unit, Clermont-Ferrand University Hospital, Clermont-Ferrand.
  • Picard C; Imagine Institute, UMR 1163 INSERM and Paris University, Paris, France; Study Center for Primary Immunodeficiencies, Necker-Enfants Malades University Hospital, AP-HP, Paris.
  • Rieux-Laucat F; Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France; Imagine Institute, UMR 1163 INSERM and Paris University, Paris.
  • Landman-Parker J; Pediatric Oncology Immunology Hematology Unit, Armand-Trousseau University Hospital, AP-HP, Paris.
  • Leverger G; Pediatric Oncology Immunology Hematology Unit, Armand-Trousseau University Hospital, AP-HP, Paris.
  • Aladjidi N; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France; Pediatric Oncology Hematology Unit, University Hospital, Plurithématique CIC (CICP), Centre d'Investigation Clinique (CIC) 1401, INSERM Bordeaux. nathalie.aladjidi@chu-bordeaux.fr.
Haematologica ; 2021 Jan 14.
Artigo em Inglês | MEDLINE | ID: mdl-33440924
ABSTRACT
Pediatric-onset Evans syndrome (pES) is defined by both immune thrombocytopenic 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 immunopathological manifestations (IMs). 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 (6.8-50.0) years and the median follow-up period was 11.3 (5.1-38.0) years. At 10 years, ITP and AIHA were in sustained complete remission in 54.5% and 78.4% of patients, respectively. The frequency and number of clinical and biological IMs increased with age at 20 years old, 74% had at least one clinical cIM. A wide range of cIMs occurred, mainly lymphoproliferation, dermatological, gastrointestinal/hepatic and pneumological IMs. The number of cIMs was associated with a subsequent increase in the number of second-line treatments received (other than steroids and immunoglobulins; hazard ratio, 1.4; 95% confidence interval, 1.15-1.60; p = 0.0002, Cox proportional hazards method). Survival at 15 years after diagnosis was 84%. Death occurred at a median age of 18 (1.7-31.5) years, and the most frequent cause was infection. The number of second-line treatments and severe/recurrent infections were independently associated with mortality. In conclusion, longterm outcomes of pES showed remission of cytopenias but frequent IMs linked to high secondline treatment burden. Mortality was associated to drugs and/or underlying immunodeficiencies, and adolescents-young adults are a high-risk subgroup.
Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Idioma: Inglês Ano de publicação: 2021 Tipo de documento: Artigo

Similares

MEDLINE

...
LILACS

LIS

Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Idioma: Inglês Ano de publicação: 2021 Tipo de documento: Artigo
...