Evans syndrome: results of a national survey

Our goal was to improve the management of Evans Syndrome, an uncommon and frequently refractory condition. We conducted a retrospective survey to assess the demography, presentation, clinical course, and treatment response of affected children.

Information was analyzed from a detailed questionnaire completed by pediatric hematologists mainly in the U.S. and Canada. We sought information regarding demographics, findings at presentation, approach to diagnosis, treatments used (with specific reference to splenectomy, corticosteroids, and intravenous immunoglobulin (IVIG)), course of the disease with emphasis on recurrences, and status at last follow-up.

Forty-two patients (22 male, 20 female) were included in the study. The median age was 7.7 years (range 0.2-26.6 years). At presentation, thrombocytopenia (32 patients) and anemia (28) were common; neutropenia occurred in 10 and pancytopenia in 6. Patients received a median of 5 (range 0-12) modalities of treatment. Courses of IVIG and corticosteroids were given to almost all patients; responses were varied but the effects lasted as long as 2 years. Splenectomy was performed for 15 patients but the median duration of response was only 1 month. Other treatments included cyclosporine, vincristine, danazol, azathioprine, cyclophosphamide, and plasmapheresis. The course of the disease was characterized by recurrent thrombocytopenia, hemolytic anemia, and neutropenia. After a median follow-up of 3 years, 3 patients had died, 20 had active disease on treatment, 5 had persistent disease (not on treatment), and 14 had no evidence of disease.

Evans syndrome is a chronic and recurrent condition which is often refractory to IVIG, corticosteroids, and splenectomy. Responses to other agents have been anecdotal and inconclusive. A prospective study involving these agents is needed to determine optimal therapeutic combinations.