Original Versus Generic Eltrombopag in Patients with Immune Thrombocytopenia: A Prospective Multi-Center Experience on Efficacy and Safety

<b>Background/Objectives</b>: Eltrombopag, a thrombopoietin receptor agonist, is widely used in the treatment of relapsed or refractory (R/R) immune thrombocytopenia (ITP). This study aimed to compare the efficacy, safety, and tolerability of generic eltrombopag (Rompag^®) with original eltrombopag (Revolade^®) in adult patients with R/R ITP. <b>Methods</b>: In this prospective, multicenter study conducted at 10 centers, 104 adult ITP patients were followed for at least 3 months. A total of 35 (33.7%) patients received Rompag^® and 69 (66.3%) received Revolade^®. The primary endpoint was platelet (PLT) response, defined as achieving a PLT count ≥50 × 10⁹/L and at least a twofold increase from baseline, without the need for rescue therapy or transfusion. Secondary endpoints included bleeding rates, fatigue-related quality of life, adverse events (AEs), and rescue therapy requirements. <b>Results</b>: PLT response was achieved in 94.2% of patients in the Revolade^® group and 85.7% in the Rompag^® group (<i>p</i> = 0.16). Bleeding rates decreased significantly in both groups (Revolade^®: 56.5% to 2.9%, <i>p</i> < 0.001; Rompag^®: 62.9% to 2.9%, <i>p</i> < 0.001). Although overall AE rates were similar (30.4% in the Revolade^® group and 42.9% in the Rompag^® group; <i>p</i> = 0.22), arthralgia (28.6% vs. 7.2%, <i>p</i> = 0.01) and vomiting (11.4% vs. 0%, <i>p</i> = 0.008) were more frequent with Rompag^®. <b>Conclusions</b>: Both generic and original eltrombopag demonstrated no statistically significant difference in efficacy in achieving PLT response, reducing bleeding, and improving fatigue-related quality of life in adult patients with R/R ITP. Although minor differences in AE profiles were observed, particularly arthralgia and vomiting, both formulations showed acceptable safety and tolerability.