1095 - Aplastic Anemia: A Single Institution Experience

Abstract

ABSTRACT Background Aplastic anemia (AA) is an extremely rare disease characterized by pancytopenia and hypocellular bone marrow (BM). Diagnosis is difficult and requires high degree of suspicion. Bone marrow transplantation (BMT) and Immunosuppressive Therapy (IST) are the only therapeutic options with a curative aim. Objectives Analysis of clinical and pathological features, therapeutic approach and overall survival of patients diagnosed with idiopathic AA in IPO-Porto. Methods Retrospective analysis of idiopathic AA patients from September/1995 to October/2011. The overall survival (OS) analysis was performed by Kaplan Meier method. Results During this period, 12 patients were identified with a median age of 31 years (16-49), 75% were younger than 40 years. 50% were female. The main symptom at diagnosis was asthenia (58.3%), followed by ecchymosis (16.7%) and fever (16.7%). At diagnosis, 33.3% had neutrophil counts less than 1.5x109/L and 41.7% had platelet counts less than 20.0x109/L. All patients had BM cellularity less than 25%. Only one patient had BMT as 1st line treatment; this patient is in complete remission with a follow-up of 15 years. The remaining patients underwent IST with anti-thymocyte immunoglobulin, cyclosporine and methylprednisolone; three patients had complete remission but relapsed, with a disease-free interval of 55 months. As a 2nd line treatment, three patients received unrelated BMT and two had complete remission. Of the seven patients who underwent 2nd line IST, three had complete remission, two underwent BMT on the 3rd line, and two patients were treated, respectively, with alemtuzumab and cyclosporine. Patients receiving IST had a median follow-up of 56 months. Three deaths occurred and OS at 15 years is 70%. Patients with neutrophil counts less than 1.5x109/L had a lower OS (P = 0.01). There was no secondary myelodysplastic syndromes or acute leukemias. Conclusion BMT is considered to be the most appropriate treatment in young patients with a compatible donor. However, IST is also a valid treatment for those without a donor, with a good survival. Disclosure All authors have declared no conflicts of interest.