Response to Eltrombopag in Steroid Refractory and Steroid Dependent Immune Thrombocytopenia Real-world experience

Abstract

Objective: To evaluate Response to Eltrombopag in steroids refractory and steroids dependent immune thrombocytopenia.

Methodology: This prospective study was conducted at tertiary care bone marrow transplant center Rawalpindi from August 2024 to August 2025, Patients with ITP who had steroids-refractory or steroids-dependent immune thrombocytopenia, were treated with Eltrombopag, and responses were assessed .

Results: Immune thrombocytopenia (ITP) is characterized by decreased platelet count in the peripheral circulation. The first-line therapy is corticosteroids. Eltrombopag has been used as second-line therapy in ITP. 50 ITP patients who had failed or become dependent on first-line corticosteroid therapy were included in this study. The cohort had a mean age of 35.28 ± 19.31 years (range: 3-74 years) with a slight male predominance (54%). At the initiation of therapy, the median platelet count was 9 × 10⁹/L (IQR: 7-15). The treatment yielded an overall response rate of 90%. Specifically, 72% of patients (36/50) achieved a complete response (CR), with a mean platelet count of 300.33 × 10/L. A further 18% of patients (9/50) attained a partial response (PR), with a mean platelet count of 76.33 × 10/L. Only 10% of patients (5/50) showed no response. The median time to achieve a response was 3 weeks (1-4 weeks) with no significant side effects observed, and responses were sustained for a mean duration of 7 months during follow-up. Statistical analysis confirmed a highly significant association between post-treatment platelet counts and the defined response categories (p=0.001).

Conclusion: Eltrombopag proves to be a second-line treatment with high efficacy and a favorable tolerability profile for a diverse demographic of steroid-refractory and steroid-dependent ITP patients. It facilitates a rapid and durable platelet response, achieving high rates of complete and partial response in a real-world clinical setting, thereby reducing the reliance on chronic steroid use.