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  • Long term follow-up of pedi...
    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

    Haematologica, 02/2022, Volume: 107, Issue: 2
    Journal Article

    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.