Use of Cabozantinib to Treat MET -amplified Pediatric Colorectal Cancer

• Published in: Journal of pediatric hematology/oncology Vol. 45; no. 3; p. e423
• Main Authors: Fukui, Sadahiro, Umeda, Katsutsugu, Kubota, Hirohito, Iwai, Atsushi, Akazawa, Ryo, Isobe, Kiyotaka, Tanaka, Kuniaki, Kouzuki, Kagehiro, Kawabata, Naoko, Saida, Satoshi, Kato, Itaru, Hiramatsu, Hidefumi, Itatani, Yoshiro, Funakoshi, Taro, Adachi, Souichi, Takita, Junko
• Format: Journal Article
• Language: English
• Published: United States 01.04.2023
• Subjects: Adolescent; Adult; Anilides - therapeutic use; Child; Colorectal Neoplasms - drug therapy; Colorectal Neoplasms - genetics; Humans; Male; Mutation; Pyridines - therapeutic use
• ISSN: 1536-3678
• DOI: 10.1097/MPH.0000000000002482

Pediatric colorectal cancer (CRC) is extremely rare, with little information about genetic profiles compared with adult CRC. Here, a 13-year-old male with advanced CRC underwent cancer gene panel testing, which detected 4 genetic abnormalities ( MET amplification in addition to TP53 , SMAD4 , and CTNNA1 mutations) that might be associated with a poor prognosis. Based on high-level MET amplification, he received a multikinase inhibitor, cabozantinib, after failure of first-line and second-line chemotherapy, resulting in transient disease stabilization. Tailored targeted therapy based on molecular profiling can be an effective treatment strategy for rare cancers such as pediatric CRC.