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Article Dans Une Revue Frontiers in Pediatrics Année : 2015

Evans Syndrome in Children: Long-Term Outcome in a Prospective French National Observational Cohort

1 Service d'Hémato-oncologie Pédiatrique
2 CIC Bordeaux
3 CEREVANCE - Centre de Référence National des Cytopénies Auto-immunes de l'Enfant
4 CHU Bordeaux
5 Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris]
6 IMAGINE - U1163 - Imagine - Institut des maladies génétiques
7 IHOPe - Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL]
8 Service d'hématologie pédiatrique
9 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
10 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
11 Service d'hématologie, immunologie biologiques et cytogénétique
12 CRCNA - Centre de Recherche en Cancérologie Nantes-Angers
13 CHU Nice [Cimiez]
14 Hôpital la Tronche
15 CHU Nantes - Centre Hospitalier Universitaire de Nantes
16 Hôpital Charles Nicolle [Rouen]
17 PANTHER - Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices
18 Service d'hématologie pédiatrique
19 Service d'Hématologie et d'Oncologie Pédiatrique [CHRU Nancy]
20 Hématogoie pédiatrique
21 Service de pédiatrie
22 Service de Pédiatrie médicale - Spécialités médicales [CHU Limoges]
23 CHRU Montpellier - Centre Hospitalier Régional Universitaire [Montpellier]
24 Service d'hématologie
25 Hôpital Morvan [Brest]
26 CHU Caen
27 CIC 1405 - Centre d'Investigation Clinique [CHU Clermont-Ferrand]
28 Service de pédiatrie (CHU de Dijon)
29 CHU de Poitiers - Centre hospitalier universitaire de Poitiers = Poitiers University Hospital
30 Hôpital Necker - Enfants Malades [AP-HP]
31 Hôpital Pellegrin
32 CHU Trousseau [APHP]
Damien Bodet
  • Fonction : Auteur
Yves Perel
  • Fonction : Auteur

Résumé

Evans syndrome (ES) is a rare autoimmune disorder whose long-term outcome is not well known. In France, a collaborative pediatric network set up via the National Rare Disease Plan now provides comprehensive clinical data in children with this disease. Patients aged less than 18 years at the initial presentation of autoimmune cytopenia have been prospectively included into a national observational cohort since 2004. The definition of ES was restricted to the simultaneous or sequential association of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP). Cases were deemed secondary if associated with a primitive immunodeficiency or systemic lupus erythematosus. In December 2014, we analyzed the data pertaining to 156 children from 26 centers with ES whose diagnosis was made between 1981 and 2014. Median age (range) at the onset of cytopenia was 5.4 years (0.2–17.2). In 85 sequential cases, the time lapse between the first episodes of AIHA and ITP was 2.4 years (0.1–16.3). The follow-up period as from ES diagnosis was 6.5 years (0.1–28.8). ES was secondary, revealing another underlying disease, in 10% of cases; various associated immune manifestations (mainly lymphoproliferation, other autoimmune diseases, and hypogammaglobulinemia) were observed in 60% of cases; and ES remained primary in 30% of cases. Five-year ITP and AIHA relapse-free survival were 25 and 61%, respectively. Overall, 69% of children required one or more second-line immune treatments, and 15 patients (10%) died at the age of 14.3 years (1.7–28.1). To our knowledge, this is the first consistent long-term clinical description of this rare syndrome. It underscores the high rate of associated immune manifestations and the burden of long-term complications and treatment toxicity. Future challenges include (1) the identification of the underlying genetic defects inducing immune dysregulation and (2) the need to better characterize patient subgroups and second-line treatment strategies.
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Dates et versions

hal-02413085 , version 1 (14-02-2024)

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Nathalie Aladjidi, Helder Fernandes, Thierry Leblanc, Amélie Vareliette, Frédéric Rieux-Laucat, et al.. Evans Syndrome in Children: Long-Term Outcome in a Prospective French National Observational Cohort. Frontiers in Pediatrics, 2015, 3, ⟨10.3389/fped.2015.00079⟩. ⟨hal-02413085⟩
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