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

Autor:	Blanco BP; Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HC FMUSP), São Paulo, SP, Brazil. Electronic address: bruna.blanco@hc.fm.usp.br., Garanito MP; Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HC FMUSP), São Paulo, SP, Brazil.
Jazyk:	angličtina
Zdroj:	Hematology, transfusion and cell therapy [Hematol Transfus Cell Ther] 2023 Apr-Jun; Vol. 45 (2), pp. 196-203. Date of Electronic Publication: 2022 Feb 26.
DOI:	10.1016/j.htct.2022.01.011
Abstrakt:	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 primarily 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 immunodeficiencies (PID) and one presented a spontaneous recovery. Steroids and intravenous immunoglobulin were first-line therapy in 19 cases. Twelve patients (63%) required second-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, immunologic 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 therapeutic combinations, morbidity and mortality in this disorder. Competing Interests: Conflicts of interest No authors have any conflict of interest that is directly relevant to the content of this manuscript. (Copyright © 2022 Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular. Published by Elsevier España, S.L.U. All rights reserved.)
Databáze:	MEDLINE
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