Abstract
Intimal sarcoma is an ultra-rare, high-grade malignant neoplasm arising in the intima of large blood vessels, most frequently the pulmonary arteries and aorta. As murine double minute 2 (MDM2) amplification is found in over 70% of intimal sarcomas, inhibition of MDM2 could provide clinical benefit in this patient population. This study was conducted to evaluate the activity of milademetan, a novel specific small-molecule inhibitor of MDM2 in intimal sarcoma patients as a sub-study under the nationwide large registry for rare cancers in Japan (MASTERKEY Project).
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