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

A favorable platelet response occurred in 14 of 15 procedures. Major response criteria-platelet count ≥100 × 109/L or ≥100% increase from baseline-were met in 10 cases. Median baseline platelet count was 45 × 103/μL, with a median increase of 143%, reaching a median peak of 103 × 103/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.