Evans syndrome in adults: an observational multicenter study

Authors: Fattizzo, B; Michel, M; Alessandro Giannotta, J; Lund Hansen, D; Arguello, M; Sutto, E; Bianchetti, N; Patriarca, A; Cantoni, S; Eva Mingot-Castellano, M; McDonald, V; Capecchi, M; Zaninoni, A; Consonni, D; Mathilde Vos, J; Vianelli, N; Chen, F; Glenthoj, A; Frederiksen, H; Jose Gonzalez-Lopez, T; Barcellini, W

Affiliations: Fdn Ist Ricovero & Cura Carattere Sci Ca Granda O, Hematol Unit, Milan, Italy; Univ Milan, Dept Oncol & Hematooncol, Milan, Italy; Creteil Univ Paris Est Creteil, Ctr Hosp Univ Henri Mondor, AP HP, Ctr Reference Maladies Rares Sur Cytopenies Autoimmunes, Paris, France; Odense Univ Hosp, Dept Hematol, Odense, Denmark; Hosp Univ Principe Asturias Alcala de Henares, Madrid, Spain; Univ Bologna, Inst Hematol LeA Seragnoli, Bologna, Italy; Spedali Civili Brescia, Ematol, Brescia, Italy; Univ Piemonte Orientale, Azienda Osped Univ Maggiore della Carita, Dept Translat Med, Novara, Italy; Azienda Socio Sanitaria Terr Osped Niguarda, Niguarda Canc Ctr, Dipartimento Ematol & Oncol, Milan, Italy; Hosp Univ Virgen del Rocio, Serv Hematol, Seville, Spain; Queen Mary Univ, Barts Hlth Natl Hlth Serv Trust, Clin Haematol, London, England; Fdn Ist Ricovero & Cura Carattere Sci Ca Granda O, Epidemiol Unit, Milan, Italy; Univ Amsterdam, Amsterdam Univ Med Ctr, Sanquin Landsteiner Lab, Amsterdam, Netherlands; Rigshosp, Dept Hematol, Copenhagen, Denmark; Hosp Univ Burgos, Serv Hematol, Burgos, Spain

Publication: Blood Adv; 2021; 5. 5468–5478 January 2022

Abstract: Evans syndrome (ES) is a rare condition, defined as the presence of 2 autoimmune cytopenias, most frequently autoimmune hemolytic anemia and immune thrombocytopenia (ITP) and rarely autoimmune neutropenia. ES can be classified as primary or secondary to various conditions, including lymphoproliferative disorders, other systemic autoimmune diseases, and primary immunodeficiencies, particularly in children. In adult ES, little is known about clinical features, disease associations, and outcomes. In this retrospective international study, we analyzed 116 adult patients followed at 13 European tertiary centers, focusing on treatment requirements, occurrence of complications, and death. ES was secondary to or associated with underlying conditions in 24 cases (21%), mainly other autoimmune diseases and hematologic neoplasms. Bleeding occurred in 42% of patients, mainly low grade and at ITP onset. Almost all patients received first-line treatment (steroids with or without intravenous immunoglobulin), and 23% needed early additional therapy for primary refractoriness. Additional therapy lines included rituximab, splenectomy, immunosuppressants, thrombopoietin receptor agonists, and others, with response rates >80%. However, a remarkable number of relapses occurred, requiring >3 therapy lines in 54% of cases. Infections and thrombotic complications occurred in 33% and 21% of patients, respectively, mainly grade >= 3 and correlated with the number of therapy lines. In addition to age, other factors negatively affecting survival were severe anemia at onset and occurrence of relapse, infection, and thrombosis. These data show that adult ES is often severe and marked by a relapsing clinical course and potentially fatal complications, pinpointing the need for high clinical awareness, prompt therapy, and anti-infectious/anti-thrombotic prophylaxis.