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Article Dans Une Revue Haematologica Année : 2022

Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden

1 CEREVANCE - Centre de Référence National des Cytopénies Auto-immunes de l'Enfant
2 Centre de recherche du CHU Sainte-Justine / Research Center of the Sainte-Justine University Hospital [Montreal, Canada]
3 CIC Bordeaux
4 CHU Bordeaux
5 Hôpital Robert Debré
6 AP-HP - Assistance publique - Hôpitaux de Paris (AP-HP)
7 TIMONE - Hôpital de la Timone [CHU - APHM]
8 CEReSS - Centre d'études et de recherche sur les services de santé et la qualité de vie
9 HCL - Hospices Civils de Lyon
10 Imagine - U1163 - Imagine - Institut des maladies génétiques (IHU)
11 Hôpital Necker - Enfants Malades [AP-HP]
12 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
13 Hôpital Bicêtre [AP-HP, Le Kremlin-Bicêtre]
14 CHU Rouen
15 CHU Angers - Centre Hospitalier Universitaire d'Angers
16 UA - Université d'Angers
17 CHUGA - Centre Hospitalier Universitaire [CHU Grenoble]
18 CHU Nice - Centre Hospitalier Universitaire de Nice
19 CHU Trousseau [Tours]
20 Hôpital Arnaud de Villeneuve [CHRU Montpellier]
21 CHU de Poitiers [La Milétrie] - Centre hospitalier universitaire de Poitiers = Poitiers University Hospital
22 HUS - Les Hôpitaux Universitaires de Strasbourg
23 Hôpital de Hautepierre [Strasbourg]
24 CHU Nantes - Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
25 Service d'Hématologie et Oncologie pédiatrique [CHRU Besançon]
26 Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Ponchaillou]
27 CHU Nancy - Centre Hospitalier Universitaire de Nancy
28 Service de Pédiatrie médicale [CHU Limoges]
29 CHU Caen
30 CHU Dijon
31 CHU Clermont-Ferrand
32 Equipe Inserm U1163 - Immunogenetics of pediatric autoimmune diseases
33 CHU Trousseau [APHP]
Yves Bertrand
  • Fonction : Auteur
  • PersonId : 899520
Aude Marie-Cardine
  • Fonction : Auteur
Fanny Fouyssac
  • Fonction : Auteur
  • PersonId : 908404
Marianna Deparis
  • Fonction : Auteur
Claire Briandet
  • Fonction : Auteur
Éric Doré
  • Fonction : Auteur
Guy Leverger
  • Fonction : Auteur
  • PersonId : 1093080

Résumé

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 (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 remission in 54.5% and 78.4% of patients, respectively. The frequency and number of clinical and biological IM increased with age: at the age of 20 years, 74% had at least one clinical IM (cIM). A wide range of cIM occurred, mainly lymphoproliferation, dermatological, gastrointestinal/hepatic and pneumological IM. The number of cIM 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 years (range, 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, long-term outcomes of pES showed remission of cytopenias but frequent IM linked to high second-line treatment burden. Mortality was associated to drugs and/or underlying immunodeficiencies, and adolescents-young adults are a high-risk subgroup.
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Dates et versions

hal-03784076 , version 1 (15-09-2023)

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Paternité - Pas d'utilisation commerciale

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Citer

Thomas Pincez, Helder Fernandes, Thierry Leblanc, Gérard Michel, Vincent Barlogis, et al.. Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden. Haematologica, 2022, 107 (2), pp.457-466. ⟨10.3324/haematol.2020.271106⟩. ⟨hal-03784076⟩
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