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

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. XX.