Is it Time to Resist using RECIST as a Primary Endpoint for Rare Tumors Trials?

Published in: Clinical cancer research : an official journal of the American Association for Cancer Research (2024)

Epithelioid hemangioendothelioma (EHE) is an ultra-rare cancer driven by the 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.

Keyphrases

patient reported outcomes
chronic pain
papillary thyroid
pain management
high resolution
neuropathic pain
squamous cell carcinoma
pi k akt
cell proliferation
lymph node metastasis