Is It Time to Resist Using RECIST as a Primary Endpoint for Rare Tumor Trials?

Van Tine, Brian A; Haarberg, Sasha · Clin Cancer Res · 2024

editorial · Level V

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Abstract

Epithelioid hemangioendothelioma is an ultra-rare cancer driven by YAP-CAMTA1 fusion. Based on the link of the fusion to the MEK pathway, SARC33 was performed. It is a phase 2 trial examining trametinib that missed its primary objective by RECIST but demonstrated patient-reported outcome benefits in improved pain. See related article by Schuetze et al., p. 4584.

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