Eltrombopag use in surgical procedures in inherited thrombocytopenia: expanding the role of thrombopoietin-receptor agonists

Background: Inherited thrombocytopenias (ITs) are a group of rare disorders characterized by low platelet counts and variable bleeding risk. Surgical procedures in these patients often require transient platelet elevation. Thrombopoietin-receptor agonists, such as eltrombopag, offer a potential alternative to platelet transfusions, which carry risks including alloimmunization and refractoriness. Objectives: To evaluate the efficacy and safety of short-term eltrombopag administration in patients with ITs undergoing elective surgical procedures. Methods: Nine patients with confirmed ITs (MYH9-related disease, ANKRD26-related thrombocytopenia [-RT], ITGA2B/ITGB3-RT, ACTN1-RT) underwent 15 surgical procedures. Eltrombopag was administered at 50 mg/d, initiated approximately 3 weeks prior to surgery. Platelet counts, bleeding complications, and adverse events were monitored. Results: /μL. No bleeding complications or transfusions were required. One patient with ITGA2B/ITGB3-RT failed to respond and required additional hemostatic support. No major adverse events were observed. Conclusion: Short-term eltrombopag administration was effective in increasing platelet counts and preventing bleeding complications in patients with IT undergoing surgery. The inclusion of the first reports of ACTN1-RT expands the therapeutic scope of thrombopoietin-receptor agonists. Although larger studies remain challenging due to the rarity of these conditions, our results contribute meaningful clinical evidence to guide perioperative management in IT.