Diagnosis and management of Evans syndrome in adults: first consensus recommendations.

Evans syndrome is a rare disease marked by a severe clinical course, high relapse rate, infectious and thrombotic complications, and sometimes fatal outcome. Management is highly heterogeneous. There are several case reports but few large retrospective studies and no prospective or randomised trials. Here, we report the results of the first consensus-based expert recommendations aimed at harmonising the diagnosis and management of Evans syndrome in adults. After reviewing the literature, we used a fuzzy Delphi consensus method, with two rounds of a 42-item questionnaire that were scored by a panel of 13 international experts from five countries using a 7-point Likert scale. Panellists were selected by the core panel on the basis of their personal experience and previous publications on Evans syndrome and immune cytopenias; they met virtually throughout 2023. The panellists recommended extensive clinical and laboratory diagnostic tests, including bone marrow evaluation and CT scan, and an aggressive front-line therapy with prednisone (with or without intravenous immunoglobulins), with different treatment durations and tapering for immune thrombocytopenia and autoimmune haemolytic anaemias (AIHAs). Rituximab was strongly recommended as first-line treatment in cold-type AIHA and as second-line treatment in warm-type AIHA and patients with immune thrombocytopenia and antiphospholipid antibodies, previous thrombotic events, or associated lymphoproliferative diseases. However, rituximab was discouraged for patients with immunodeficiency or severe infections, with the same applying to splenectomy. Thrombopoietin receptor agonists were recommended for chronic immune thrombocytopenia and in the case of previous grade 4 infection. Fostamatinib was recommended as third-line or further-line treatment and suggested as second-line therapy for patients with previous thrombotic events. Immunosuppressive agents have been moved to third-line or further-line treatment. The panellists recommended the use of recombinant erythropoietin in AIHA in the case of inadequate reticulocyte counts, use of the complement inhibitor sutimlimab for relapsed cold AIHA, and the combination of rituximab plus bendamustine in Evans syndrome secondary to lymphoproliferative disorders. Finally, recommendations were given for supportive therapy, platelet or red blood cell transfusions, and thrombotic and antibiotic prophylaxis. These consensus-based recommendations should facilitate best practice for diagnosis and management of Evans syndrome in clinical practice.
Competing Interests: Declaration of interests WB has received consultancy or advisory board honoraria and speaker's bureau from Alexion, Novartis, Agios, Pfizer, Sanofi, and Sobi. BF has received consultancy or advisory board honoraria and speaker's bureau from Agios, Alexion, Apellis, Janssen, Novartis, Roche, Samsung, Sanofi, and Sobi. HF has received research support from Sanofi and Novartis. AG has received consultancy and research funds from Agios, Bristol Myers Squibb, Novo Nordisk, Saniona, Sanofi, Pharmacosmos, Novartis, AbbVie, and Vertex Pharmaceuticals. TJG-L has received research support from Sobi, Novartis, Amgen, Grifols; speakers bureau from Sobi, Novartis, Amgen, Grifols, argenx, Sanofi, Momenta, and Alpine; and fees for participation in data safety monitoring for Novartis and Alpine. IM has received consultancy honoraria from Alexion, Janssen, Novartis, Apellis, and Sanofi. MaM has received consultancy or speaker's bureau from Alexion, argenx, Sanofi, Novartis, and Sobi. MoM has received consultancy from Gilead, Novartis, AbbVie, and Pfizer. M-EM has received research support, consultancy and speaker's bureau from Amgen, Novartis, Novo Nordisk, Sanofi, Grifols, Sobi, Takeda, and CSL Bering. MN has received speaker fees from Sobi, Novo Nordisk, Amegn, Novartis, Kedrion, Bayer, Takeda, CSL Behring, and Sanofi, and consultancy honoraria from Bayer and CSL Behring. AP has received consultancy or speaker's bureau from Sanofi, Alexion, Novartis, Sobi, Pfizer, Gilead, and Morphosys. SR has received consultancy honoraria from Sanofi, Novartis, and Alexion. NV has received speaker's bureau from Sanofi, Amgen, and Grifols. All other authors declare no competing interests.
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