Improved survival in severe acquired aplastic anemia of childhood

Multi-agent immunosuppressive therapy has produced improved survival for se vere acquired aplastic anemia in children. Recently, some investigators hav e suggested that immunosuppressive therapy may replace bone marrow transpla ntation as first-line therapy for this disorder. To assess its validity, we compared the outcomes of bone marrow transplantation vs immunosuppressive therapy in one institution from 1987 to 1997. We studied 46 consecutive pat ients less than 18 years of age who presented between January 1987 and Apri l 1997. Inherited marrow failure syndromes and myelodysplastic syndromes we re excluded. Patients received immunosuppressive therapy vs bone marrow tra nsplantation based on availability of HLA-matched donors. The main outcome measures were survival, complete marrow and hematological remission, or par tial remission but achieving independence from transfusional support. Twent y patients received multi-agent immunosuppressive therapy (cyclosporine, an tithymocyte globulin and methylprednisolone); 11 attained complete remissio n and three partial remission for a transfusion-independent survival of 70% . Six patients died of infectious and hemorrhagic complications. Twenty-six patients were transplanted and 24 (93%) achieved complete remission; one a chieved a PR, 25 remain transfusion independent with a median follow-up of 5.9 years or 70 months. One patient developed AML 34 months after successfu l transplant and one patient died due to graft failure and complications of transplant. There has been a striking improvement in survival for pediatri c patients treated with multi-agent immunosuppression in the last decade. H owever, transplantation results have also improved and this remains the def initive first-line therapy for severe acquired aplastic anemia in this age group.