Eltrombopag: Efficacy and Safety in Steroid Refractory Lupus-Associated Immune Thrombocytopenia.

Abstract

Background: Eltrombopag, a thrombopoietin receptor agonist, is effective in chronic immune thrombocytopenia (ITP). The data in lupus ITP are sparse. This study aimed to assess the efficacy and safety of eltrombopag in lupus ITP.

Methods: This is a single-center study conducted between 2012 and 2017 of 12 subjects with systemic lupus erythematosus-associated ITP. Patients with inadequate or suboptimal response to steroids and other immunosuppressants treated with eltrombopag were included in the study. Time taken for response to therapy (defined as platelets >1 lakh), dose, duration of treatment, and adverse effects of the drug were noted.

Results: A total of 12 subjects were included in the study. The median platelet count at initiation of eltrombopag was 19,000 per μL. The time taken for response to therapy was 8 days. The median platelet count at 1-month follow-up was 241,000 per μL. All patients were concurrently treated with steroids and other immunosuppressants. Sustained benefit after stopping eltrombopag was noted in all patients. No adverse events including thrombotic complication were noted.

Conclusions: Eltrombopag is a new drug in our arsenal for treatment of ITP in lupus. It is a rapidly effective, safe, and orally administered medication. It indirectly contributes to reduction in the dose of steroids and immunosuppressants, thereby minimizing their cumulative adverse effects. It is a promising and safe option for the treatment of lupus-associated thrombocytopenia, but this needs further confirmation from multicenter, multiethnic, randomized controlled trials.