Author:
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
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.
Publisher
Ferrata Storti Foundation (Haematologica)
Cited by
10 articles.
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