Refractoriness to eltrombopag in adult primary immune thrombocytopenia: utility of next-generation sequencing techniques.

ABSTRACT: Thrombopoietin receptor agonists, for example eltrombopag, are standard second-line treatment for immune thrombocytopenia (ITP). Eltrombopag has demonstrated high response rates, both in clinical trials and in routine practice studies. However, some patients with ITP are refractory to this drug. Next-generation sequencing (NGS) may help us identify underlying molecular biology variants that may be involved in eltrombopag refractoriness. Our multicenter national NGS study investigated 110 genes of the most important cell-signaling pathways involved in the mechanism of action of eltrombopag in 35 refractory cases and 35 eltrombopag-responsive controls. Our refractory population comprised 51.4% men with a median age at diagnosis of 48 (range, 38-69) years and a median platelet count of 7 × 10

SUBMITTER: Gonzalez-Lopez TJ 

PROVIDER: S-EPMC12320395 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature