Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review

Thrombopoietin receptor agonists (TPO-RAs) have become an important component of pediatric immune thrombocytopenia (ITP) management, with growing evidence supporting their use in other hematologic disorders. We conducted a retrospective single-center study evaluating TPO-RA use at a pediatric oncohematology center in Poland between 1 January 2016, and 31 March 2026. Clinical indications, treatment patterns, efficacy, and safety outcomes were analyzed. Treatment response was defined as initial response (IR, platelet count [PLT] > 50 × 109/L), complete response (CR, PLT > 100 × 109/L), and sustained response (SR, PLT > 50 × 109/L maintained in ≥75% of visits over 6 months). Thirty-five patients were included in the analysis. The most common indication was ITP (27/35, 77%), followed by severe aplastic anemia (5/35, 14%), poor graft function (2/35, 6%), and inherited platelet disorders (1/35, 3%). TPO-RA use increased over time, with more than half of patients (20/35, 57%) initiating therapy between 2024 and 2026. Overall IR, CR, and SR rates were 77%, 63%, and 61%, respectively, with a median time to response of 7 days (interquartile range [IQR], 7–12.5). TPO-RAs were increasingly used earlier in the disease course, including in rescue and off-label settings. Treatment was well tolerated, with only grade 1–2 adverse events reported. Our real-world data confirm the high efficacy and favorable safety profile of TPO-RAs in pediatric patients and support their expanding role in pediatric hematology beyond disease-specific indications.