Abstract
INTRODUCTION: aplastic anemia (AA) is a rare blood disorder with a considerable mortality rate, particularly if left untreated. Limited South African data on AA prompted this study to investigate the clinico-hematological features, management, and outcome of patients managed at our center. METHODS: a ten-year retrospective observational study of all patients at the Universitas Academic Hospital with confirmed AA was conducted. Data on patient demographics, clinical and laboratory data, management, and outcomes were collected and analyzed. RESULTS: twenty-seven patient files were included. Idiopathic AA was the most common (55.6%), while 12 patients (44.4%) had secondary causes. Most (70.4%) patients presented with severe AA. The majority (77.8%) received immunosuppressive therapy (IST) as first-line treatment, with most (80.9%) responding to treatment (complete, hematological and partial responses). Overall treatment response across available modalities was good (77.8%), with a few (22.2%) cases of refractory disease. Many patients were lost to follow-up (51.9%). Although access to hematopoietic stem cell transplantation (HSCT) in South Africa is limited, our center's treatment response rate to IST (80.9%) is comparable to international studies. The study found no association between HIV and AA, and no transfusion-related complications were observed. CONCLUSION: the study suggests that AA remains a disease of the young in South Africa. Most patients in this study presented with severe disease. The presence of paroxysmal nocturnal hemoglobinuria is in line with other studies. Addressing the high loss-to-follow-up rate is crucial for future studies.