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

Haematologica. 2022 Feb 1;107(2):457-466. doi: 10.3324/haematol.2020.271106.

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 (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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Hemolytic, Autoimmune* / diagnosis
  • Anemia, Hemolytic, Autoimmune* / therapy
  • Child
  • Child, Preschool
  • Follow-Up Studies
  • Humans
  • Infant
  • Prospective Studies
  • Retrospective Studies
  • Thrombocytopenia
  • Young Adult

Supplementary concepts

  • Evans Syndrome

Grants and funding

Finanzierung: This work was supported from 2004 by the French Ministry of Health (Programme Hospitalier de Recherche Clinique [PHRC] 2005, Rare Disease Plan 2007 and 2017), the Association Bordelaise pour l’Avancement des Sciences Pédiatriques (ABASP) research charity, the Association pour la Recherche et les Maladies Hématologiques de l’Enfant (RMHE) research charity, the Association Française du Syndrome d’Evans (AFSE), the O-CYTO patients’ association, and partially by GlaxoSmithKline, AMGEN and Novartis. TP is a recipient of a Charles Bruneau fellowship award.