<i>BRCA1/2</i> Functional Loss Defines a Targetable Subset in Leiomyosarcoma

Abstract

Soft-tissue sarcomas are a highly morbid, diverse set of tumors with limited treatment options. This study identifies an increased prevalence of functional <i>BRCA1/2</i> loss in patients with uterine leiomyosarcoma (uLMS). It also presents four patients with uLMS and <i>BRCA2</i> loss who achieved durable clinical benefit from poly (ADP-ribose) polymerase inhibition. These data suggest that patients with uLMS in particular should be screened for <i>BRCA1/2</i> alterations and may benefit from treatment targeted to these alterations.