Real-world insights into acquired aplastic anemia: the Latin American Registry of Aplastic Anemia

Aplastic anemia (AA) is a rare condition characterized by bone marrow hypocellularity and pancytopenia, with a global prevalence of 2 cases per million individuals per year. The Latin American Registry of Aplastic Anemia was established in 2017 to evaluate the prevalence, clinical characteristics, diagnostic tools, treatment patterns, and outcomes of patients in the region. This retrospective analysis includes 343 patients diagnosed between 2015 and 2024 from 5 countries in Latin America. The mean age at diagnosis was 41 years (range, 5-85 years). First-line treatment was mainly immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine in 44.6%, with the addition of eltrombopag in 11.4%. Hematopoietic stem cell transplantation (HSCT) was performed in 10.7% of patients. The overall response rate for first-line treatment was 91% among HSCT recipients and 63% among patients who received IST. The median follow-up of the entire group was 1.5 years, with an estimated 3-year overall survival of 80%. This study provides real-world data from participating centers in Latin America, predominantly from Colombia and Peru, highlighting heterogeneity in management of patients with AA in this context, and the need for standardized care protocols to improve patient access and outcomes.