Eltrombopag for Chemotherapy-Induced Thrombocytopenia: A Multicenter Retrospective Real-World Study

Introduction: Chemotherapy-induced thrombocytopenia (CIT) is a common dose-limiting toxicity that disrupts on-time, full-dose chemotherapy, yet no pharmacologic therapy is formally approved. Growing evidence from randomized and late-phase studies with thrombopoietin receptor agonists (TPO-RAs) has renewed interest in targeted supportive care. We evaluated the effectiveness and safety of eltrombopag for CIT in routine clinical practice. Methods: We conducted a small, retrospective, single-arm multicenter cohort study of 31 adults with solid tumors (74.2% stage IV). Given the descriptive, hypothesis-generating nature of this study, no causal inference regarding efficacy can be drawn. Platelet counts and chemotherapy continuity were tracked from baseline through week 12 after eltrombopag initiation. Bleeding, thrombosis, and laboratory safety signals were recorded. Results: The median platelet count increased from 33 × 10⁹/L at baseline to 71 × 10⁹/L at week 1 and 99.5 × 10⁹/L by week 12. Overall, 18/31 patients (58.1%) resumed chemotherapy within 3 weeks, and 15/31 (48.4%) completed planned regimens by week 6. Adverse events were limited to mild, transient elevations in transaminases (n = 3); no major bleeding or thrombotic events occurred. Conclusions: In this real-world multicenter cohort, eltrombopag was associated with rapid platelet recovery and improved chemotherapy deliverability with an acceptable safety profile. The retrospective, single-arm design and the hypothesis-generating nature of these findings preclude definitive conclusions regarding causal efficacy. These observational data highlight the need for prospective controlled trials to characterize the clinical role, optimal dosing, and long-term safety of oral TPO-RAs in CIT.