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Is It Time to Resist Using RECIST as a Primary Endpoint for Rare Tumor Trials?
Brian A Van Tine1,2,3, Sasha Haarberg1,2
1Division of Medical Oncology, Department of Medicine, School of Medicine, Washington University in St. Louis, St. Louis, Missouri.
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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