期刊
PEDIATRIC BLOOD & CANCER
卷 63, 期 8, 页码 1468-1470出版社
WILEY
DOI: 10.1002/pbc.26026
关键词
ETV6-NTRK3; gene fusion; infantile fibrosarcoma; LOXO-101; tropomyosin-related kinase (TRK)
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. (C) 2016 The Authors. Pediatric Blood & Cancer, published by Wiley Periodicals, Inc.
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