The management of metastatic GIST: Current standard and investigational therapeutics Review


Authors: Kelly, C. M.; Gutierrez Sainz, L.; Chi, P.
Review Title: The management of metastatic GIST: Current standard and investigational therapeutics
Abstract: Gastrointestinal stromal tumor (GIST) is the most common mesenchymal tumor of the gastrointestinal tract. The majority of GISTs harbor gain of function mutations in either KIT or PDGFRα. Determination of the GIST molecular subtype upon diagnosis is important because this information informs therapeutic decisions in both the adjuvant and metastatic setting. The management of GIST was revolutionized by the introduction of imatinib, a KIT inhibitor, which has become the standard first line treatment for metastatic GIST. However, despite a clinical benefit rate of 80%, the majority of patients with GIST experience disease progression after 2–3 years of imatinib therapy. Second and third line options include sunitinib and regorafenib, respectively, and yield low response rates and limited clinical benefit. There have been recent FDA approvals for GIST including ripretinib in the fourth-line setting and avapritinib for PDGFRA exon 18-mutant GIST. This article aims to review the optimal treatment approach for the management of patients with advanced GIST. It examines the standard treatment options available but also explores the novel treatment approaches in the setting of imatinib refractory GIST. © 2021, The Author(s).
Keywords: gastrointestinal stromal tumor; imatinib; tyrosine kinase inhibitors; metastatic gist
Journal Title: Journal of Hematology & Oncology
Volume: 14
ISSN: 1756-8722
Publisher: Biomed Central Ltd  
Date Published: 2021-01-05
Start Page: 2
Language: English
DOI: 10.1186/s13045-020-01026-6
PUBMED: 33402214
PROVIDER: scopus
PMCID: PMC7786896
DOI/URL:
Notes: Review -- Export Date: 1 February 2021 -- Source: Scopus
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  1. Ping Chi
    172 Chi
  2. Ciara Marie Kelly
    89 Kelly