Is switching to romiplostim still worthwhile after failure of multiple oral TPO-RAs in pediatric refractory ITP?

Abstract Background: Thrombopoietin receptor agonists (TPO-RAs) are recommended second-line therapies for pediatric immune thrombocytopenia (ITP). If one agent proves ineffective, switching between TPO-RAs, particularly from oral agents such as eltrombopag (ELT) or avatrombopag (AVA) to the injectable romiplostim (ROM), has been suggested. However, it is unclear whether switching to ROM still provides meaningful benefit if multiple oral TPO-RAs have already failed. In China, the late marketing of ROM led to late use after failing oral TPO-RAs, providing a unique real-world scenario. Methods: This single-center, retrospective, observational study included patients aged 1–18 years with persistent or chronic ITP who received ROM for at least four weeks. Clinical characteristics, prior therapies, platelet response, bleeding outcomes, rescue therapy, and adverse drug reactions (ADRs) were assessed. Results: Sixteen patients with persistent or chronic ITP were analyzed. Thirteen of them had refractory ITP and had all previously used two or more TPO-RAs. 11/13 showed no response (NR), and 2/13 exhibited transient partial response (PR) to ROM. 12/13 patients had bleedings, 8/13 required rescue therapy and 13/13 switched to other drugs. Three patients chose ROM due to drug interactions or liver dysfunction, rather than treatment failure of other TPO-RAs, only 1/3 used ELT. 2/3 achieved a complete response (CR), while 1/3 had an NR. No adverse drug reactions (ADRs) occurred. Conclusion: ROM is safe and effective in selected pediatric ITP patients. However, children who have failed multiple oral TPO-RAs derive minimal benefit from switching to ROM, and alternative therapies targeting immune dysregulation should be prioritized instead of further within-class switching.