Rituximab for Immune Cytopenia in Adults: Idiopathic Thrombocytopenic Purpura, Autoimmune Hemolytic Anemia, and Evans Syndrome

Objective: To evaluate the efficacy of rituximab for the treatment of adult patients with immune cytopenia, including idiopathic thrombocytopenic purpura (ITP), autoimmune hemolytic anemia, and Evans syndrome. Patients and Methods: We retrospectively reviewed the medical charts of all patients treated with rituximab for immune cytopenia at the Mayo Clinic in Rochester, Minn, through January 1, 2003. Fourteen patients (median age at first diagnosis, 51 years; range, 21-79 years) were identified who received 1 or more treatment courses of rituximab for treatment of refractory ITP (12 patients), autoimmune hemolytic anemia (AIHA) (5 patients), or both ITP and AIHA (classified as Evans syndrome) (4 patients). Data regarding age, diagnosis, date of diagnosis, previous treatments, comorbid conditions, blood cell counts before taking rituximab, number of rituximab treatments, and response to treatment were extracted and analyzed. Results: Of 12 patients treated for ITP, 6 were receiving corticosteroid-based treatment either alone or combined with other immunosuppressive therapy at the time they received rituximab. Complete remission occurred in 5 (42%) of 12 patients with ITP and in 2 (40%) of 5 patients with AIHA. Response to rituximab in patients with Evans syndrome was seen in either ITP or AIHA, but not both. Complete response was often durable in ITP. Responses were seen in both splenectomized and nonsplenectomized patients. Conclusions: Our findings, considered with the results of other studies, suggest that rituximab deserves early consideration as salvage therapy for immune cytopenias that are refractory to both corticosteroid treatment and splenectomy. This series represents the largest series of adult patients with AIHA and Evans syndrome.