Infantile Fibrosarcoma With NTRK3–ETV6 Fusion Successfully Treated With the Tropomyosin‐Related Kinase Inhibitor LOXO‐101. Issue 8 (19th April 2016)

• Record Type: Journal Article
• Authors: Nagasubramanian, Ramamoorthy; Wei, Julie; Gordon, Paul; Rastatter, Jeff C.; Cox, Michael C.; Pappo, Alberto
• Is Part Of: Pediatric blood & cancer. Volume 63:Issue 8(2016)
• Page Start: 1468
• Page End: 1470
• Publication Date: 2016-04-19
• Subjects: ETV6–NTRK3 -- gene fusion -- infantile fibrosarcoma -- LOXO‐101 -- tropomyosin‐related kinase (TRK); Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.26026
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract : Infantile fibrosarcoma (IFS) is a rare pediatric cancer typically presenting in the first 2 years of life. Surgical resection is usually curative and chemotherapy is active against gross residual disease. However, when recurrences occur, therapeutic options are limited. We report a case of refractory IFS with constitutive activation of the tropomyosin‐related kinase (TRK) signaling pathway from an ETS variant gene 6–neurotrophin 3 receptor gene ( ETV6–NTRK3 ) gene fusion. The patient enrolled in a pediatric Phase 1 trial of LOXO‐101, an experimental, highly selective inhibitor of TRK. The patient experienced a rapid, radiographic response, demonstrating the potential for LOXO‐101 to provide benefit for IFS harboring NTRK gene fusions.