Summary:Evans syndrome is a rare disorder characterized by combined autoimmune thrombocytopenia (ITP) and autoimmune hemolytic anemia (AIHA). Standard treatments consist of transfusions, corticosteroids, splenectomy, IVIG, anabolic steroids, vincristine, alkylating agents, or cyclosporine. In a patient with refractory disease, an allogeneic hematopoietic stem cell transplant (HSCT) resulted in complete clinical and serologic remission for more than 30 months. Allogeneic HSCT may be the only current curative therapy for Evans syndrome but may also be complicated by significant toxicities. Bone Marrow Transplantation (2001) 28, 903-905. Keywords: Evans syndrome; allogeneic stem cell transplantationIn December 1998, a 28-year-old male patient with refractory Evans syndrome (Hb of 9.1g/dl and platelets of 27 × 10 9 /l) was referred to Northwestern University for an allogeneic matched sibling HSCT. Symptoms first occurred in February 1996 when epistaxis and ecchymosis developed following flu-like symptoms of headache and diarrhea. Thrombocytopenia and anemia were present with platelets of 2 × 10 9 /l, hemoglobin of 8.8 g/dl, and WBC of 6.3 × 10 9 /l. Evidence of hemolysis included hyperbilirubinemia (3.8 mg/dl), an increased LDH (424 U/l), decreased haptoglobin (7 mg/dl), reticulocytosis (corrected reticulocyte count 5.5%), bone marrow erythroid hyperplasia, and a direct Coombs test (DAT) positive for warm reactive antiIgG and anti-C3D. Evidence for ITP included anti-platelet antibodies and increased megakaryocytes within the marrow. Cytomegalovirus (CMV) antibody was positive for IgM but negative for IgG indicating recent infection. There was no other evidence of coincidental or precipitating infections. Serology was negative for HIV, hepatitis A, B, and C, and indicated past Epstein-Barr virus infection. Anti-nuclear and anti-double stranded DNA antibodies were negative. An initial response was obtained with intravenous immunoglobulin (IVIG) and corticosteroids. Due to inability to wean high-dose corticosteroid therapy, or transfusion-dependent cytopenias, the patient was treated and subsequently failed multiple interventions including splenectomy (August 1996), IVIG, danazol, and vincristine (April 1997), azathioprine 100 mg/day (August 1997), IVIG (March 1998), and cyclosporine, IVIG, and vincristine (April 1998). Upon referral for transplant, the patient was platelet and red blood cell (RBC) transfusion-dependent despite prednisone, cyclosporine, and IVIG.Pre-transplant, the disease and treatment-related complications included hemoglobinuria, malaise, steroid-induced diabetes, vincristine-induced peripheral neuropathy, cyclosporine-induced tremor and nephropathy necessitating daily intravenous hydration, anemia-related syncope, retinal hemorrhage, and respiratory and renal failure that necessitated ICU transfer and respiratory support. Pre-transplant opportunistic infectious included cryptococcal fungemia, persistent CMV viremia, and mycobacterium avium positive blood cultures. Maintenance antimicrobial therapy ...