Prevalence, disease severity, treatment options and median survival of patients with Aplastic Anemia who attended Muhimbili National Hospital from 2018 to 2023

Abstract

ABSTRACT Background: Aplastic anemia (AA) is a rare bone marrow failure disorder characterized by pancytopenia and hypocellular marrow, primarily due to immune-mediated hematopoietic stem cell destruction. Its prevalence varies globally, and survival outcomes have improved with immunosuppressive therapy and bone marrow transplantation. However, recent data on AA prevalence and survival in Tanzania are scarce. Aim: This study aimed to determine the prevalence, severity, treatment options, and factors influencing the median survival of AA patients treated at MNH from 2018 to 2023. Method: In this retrospective cohort study, AA patients diagnosed and treated at MNH between January 2018 and December 2023 was reviewed. Clinical, laboratory, and demographic data were extracted from medical records. Descriptive statistics analyzed AA prevalence, demographics, disease severity, and treatment options. Kaplan-Meier curves and log-rank tests estimated survival. Cox regression assessed mortality risk factors, with statistical significance set at p < 0.05. Results: Among 577 pancytopenia patients, 156 (27%) had idiopathic AA, with a median age of 23 and a male-to-female ratio of 1.5:1. Severe AA was present in 85.9%. Treatments included cyclosporine (59.8%), supportive care (21.8%), and intensive IST (14.1%). The median survival was 158 days, with intensive IST showing the best outcomes, while supportive care increased mortality. Conclusion: Intensive IST significantly improves survival in AA patients. However, many patients still rely on supportive care due to socioeconomic barriers, impacting outcomes. This study provides critical data on AA demographics, treatment, and survival in Tanzania, contributing valuable insights for healthcare practitioners and policymakers.