PRIMARY-TREATMENT OF ACQUIRED APLASTIC-ANEMIA - OUTCOMES WITH BONE-MARROW TRANSPLANTATION AND IMMUNOSUPPRESSIVE THERAPY

Background: Both immunosuppressive therapy and bone marrow transplanta tion are accepted treatments for patients with aplastic anemia. Choosi ng one of these therapies for a given patient depends not only on dono r availability but also on such factors as patient age. Objective: io compare survival rates and long-term complications after bone marrow t ransplantation or immunosuppressive therapy in patients with acquired aplastic anemia and to identify prognostic factors associated with imp roved survival. Design: Center-based, retrospective analysis. Setting: Referral center for patients with aplastic anemia. Patients: 395 pati ents with acquired aplastic anemia. Intervention: Bone marrow transpla nt from an HLA-identical, related donor or immunosuppressive therapy. Measurements: Kaplan-Meier survival curves, results of log-rank tests, and cumulative incidence curves. Results: Of 168 bone marrow transpla nt recipients, 89% had sustained engraftment. Forty-six patients devel oped grade II to IV acute graft-versus-host disease, and 68 developed chronic graft-versus-host disease that required therapy. Of 227 patien ts who received immunosuppressive therapy, 44% achieved a complete, pa rtial, or minimal response. Fifty-four percent died or had no response to therapy. Actuarial survival at 15 years was 69% for bone marrow tr ansplant recipients and 38% for patients receiving immunosuppressive t herapy (P < 0.001). Improved survival was associated with having bone marrow transplantation as primary therapy, being younger, having no tr ansfusion before transplantation, and having a higher absolute neutrop hil count. Disease duration, year of therapy, sex, refractoriness to p latelet transfusions, and previous treatment with androgens or cortico steroids did not significantly affect survival. Conclusions: Data from this center suggest that bone marrow transplantation may be preferred for younger patients with acquired aplastic anemia who have matched, related donors. Long-term survival is excellent for patients who respo nd to either form of therapy.