期刊
CANCER DISCOVERY
卷 4, 期 8, 页码 889-895出版社
AMER ASSOC CANCER RESEARCH
DOI: 10.1158/2159-8290.CD-14-0377
关键词
-
类别
资金
- Sarcoma Foundation of America
- Joyce Family Foundation
- NIH K12 training grant [K12 CA9060625]
- Damon Runyon Clinical Investigator Award
Inflammatory myofibroblastic tumor (IMT) is a neoplasm that typically occurs in children. The genetic landscape of this tumor is incompletely understood and therapeutic options are limited. Although 50% of IMTs harbor anaplastic lymphoma kinase (ALK) rearrangements, no therapeutic targets have been identified in ALK-negative tumors. We report for the first time that IMTs harbor other actionable targets, including ROS1 and PDGFR beta fusions. We detail the case of an 8-year-old boy with treatment-refractory ALK-negative IMT. Molecular tumor profiling revealed a ROS1 fusion, and he had a dramatic response to the ROS1 inhibitor crizotinib. This case prompted assessment of a larger series of IMTs. Next-generation sequencing revealed that 85% of cases evaluated harbored kinase fusions involving ALK, ROS1, or PDGFR beta. Our study represents the most comprehensive genetic analysis of IMTs to date and also provides a rationale for routine molecular profiling of these tumors to detect therapeutically actionable kinase fusions. SIGNIFICANCE: Our study describes the most comprehensive genomics-based evaluation of IMT to date. Because there is no standard-of-care therapy for IMT, the identification of actionable genomic alterations, in addition to ALK, is expected to redefine management strategies for patients with this disease. (C) 2014 AACR.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据