Pediatric Evans Syndrome: A 20-year experience from a tertiary center in Brazil

Introduction: The Evans syndrome (ES) is a rare, often chronic, relapsing and treatment -refractory hematological disorder. We described the clinical features, diagnostic workup, treatment and outcome in patients with ES.Method: We performed a retrospective chart review of patients aged < 18 years with ES admitted to a tertiary center in Brazil from 2001 to 2021. The analysis of the data was pri-marily descriptive, using median, interquartile range and categorical variables presented in absolute frequencies.Main results: Twenty patients (12 female, 8 male) were evaluated in this study. The median age at the initial cytopenia was 4.98 years (1.30-12.57). The ES was secondary in nine cases (45%), of which six patients (30%) showed autoimmune disease (AID) or primary immuno-deficiencies (PID) and one presented a spontaneous recovery. Steroids and intravenous immunoglobulin were first-line therapy in 19 cases. Twelve patients (63%) required sec-ond-line treatments (rituximab, cyclosporine, splenectomy, sirolimus, cyclophosphamide, mycophenolate mofetil, azathioprine and eltrombopag). The median follow-up period was 2.41 years (1.4 -7.52). One patient (5%) died of underlying neuroblastoma, one case (5%) was lost to follow-up and four patients (20%) received a medical discharge. The median age for the 14 remaining cases was 12.6 years. Twelve patients (85.7%) were in complete response (CR) with no therapies. Two patients (14.3%) were in CR with chronic therapy.Conclusion: As ES may be a symptom of AID and PID, a thorough rheumatological, immuno-logic and genetic workup and a careful follow-up are essential. The second-line treatment remains a dilemma. Further prospective studies are needed to address the optimal thera-peutic combinations, morbidity and mortality in this disorder.& COPY; 2022 Associacao Brasileira de Hematologia, Hemoterapia e Terapia Celular. Published by Elsevier Espana, S.L.U. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).