Diego G Lanza, Leonardo J Enciso, Luis G Moncaleano, María A Rodríguez
In this real-world retrospective study, no statistically significant differences were observed between ELT and ROM in time to platelet response or the probability of platelet response. Safety outcomes were infrequent in both groups. Patients treated with ROM had received more prior therapies and more frequently had undergone splenectomy, suggesting a population with more advanced disease at treatment initiation. These findings provide comparative real-world data from a Latin American cohort and should be interpreted as exploratory and hypothesis-generating.
INTRODUCTION: Evidence on the comparative effectiveness and safety of thrombopoietin receptor agonists (TPO-RAs) in adults with primary immune thrombocytopenia (ITP) is scarce. We conducted a retrospective cohort study at a Colombian university hospital to describe the effectiveness and safety of romiplostim (ROM) and eltrombopag (ELT) and to explore comparative outcomes between treatment groups.
METHODOLOGY: A retrospective cohort study was conducted using medical records of adults with primary ITP treated at the Hospital Universitario Nacional de Colombia (Colombia National University Hospital) between 2016 and 2023. Effectiveness and safety outcomes are described, and the probability of platelet response was determined using Kaplan-Meier analysis.
RESULTS: A total of 58 patients were included (63.8% women), with a mean age of 48 years. The median number of prior treatments was 3.00 (interquartile range (IQR), 3.00-4.00) for the ROM group and 2.00 (IQR, 2.00-3.00) for the ELT group (p < 0.001). The median time to achievement of the first platelet response was 37 days (95% confidence interval (CI), 19-61) in patients treated with ELT and 71 days (95% CI, 12-162) in those treated with ROM (p = 0.50). The proportion of patients achieving at least one platelet response during follow-up was 97.3% for ELT and 100% for ROM (p = 1.000). No statistically significant difference in the probability of achieving a platelet response was observed between treatment groups (log-rank p = 0.461). Thrombotic events and treatment-related adverse events were infrequent.
CONCLUSIONS: In this real-world retrospective study, no statistically significant differences were observed between ELT and ROM in time to platelet response or the probability of platelet response. Safety outcomes were infrequent in both groups. Patients treated with ROM had received more prior therapies and more frequently had undergone splenectomy, suggesting a population with more advanced disease at treatment initiation. These findings provide comparative real-world data from a Latin American cohort and should be interpreted as exploratory and hypothesis-generating.