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

TL;DR

This study compared the effectiveness and safety of generic and original eltrombopag in treating immune thrombocytopenia and found both to be similarly effective with minor differences in side effects.

Contribution

The study provides a direct comparison of generic and original eltrombopag in a real-world, multi-center setting for immune thrombocytopenia.

Findings

Both formulations achieved high platelet response rates with no significant difference.

Bleeding rates decreased significantly in both groups.

Rompag® caused more arthralgia and vomiting compared to Revolade®.

Abstract

Background/Objectives: 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. Methods: 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. Results: PLT response was achieved in 94.2% of patients in the Revolade® group and 85.7% in the Rompag® group (p = 0.16). Bleeding rates decreased significantly in both groups (Revolade®: 56.5% to 2.9%, p < 0.001; Rompag®: 62.9% to 2.9%, p < 0.001). Although overall AE rates were similar (30.4% in the Revolade® group and 42.9% in the Rompag® group; p = 0.22), arthralgia (28.6% vs. 7.2%, p = 0.01) and vomiting (11.4% vs. 0%, p = 0.008) were more frequent with Rompag®. Conclusions: 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.

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