The management of gastrointestinal stromal tumors: a model for targeted and multidisciplinary therapy of malignancy

Heikki Joensuu1, Ronald P DeMatteo

  • 1Department of Oncology, Helsinki University Central Hospital, Helsinki, Finland. heikki.joensuu@hus.fi

Annual Review of Medicine
|October 25, 2011
PubMed

Insights

Gastrointestinal stromal tumor (GIST) is treatable with targeted therapies like imatinib, offering a chronic disease model. Surgery remains curative for localized GIST, while adjuvant therapy improves survival.

Area of Science:

  • Oncology
  • Molecular Biology
  • Gastroenterology

Background:

  • Gastrointestinal stromal tumor (GIST) serves as a key model for targeted cancer therapy.
  • Most GISTs harbor activating mutations in KIT or platelet-derived growth factor A (PDGFRA) genes.
  • GIST exhibits high responsiveness to selective tyrosine kinase inhibitors, transforming its management.

Purpose of the Study:

  • To review recent advancements in understanding GIST natural history and molecular biology.
  • To discuss progress in risk stratification and drug resistance mechanisms in GIST.
  • To highlight the role of surgery and adjuvant imatinib therapy in GIST management.

Main Methods:

  • Literature review of recent studies on GIST.
  • Analysis of molecular mechanisms driving GIST development and progression.
  • Evaluation of clinical trial data for targeted therapies and surgical outcomes.

Main Results:

  • Surgery is the only curative option for localized GIST, achieving >50% cure rates.
  • Adjuvant imatinib therapy extends recurrence-free survival and potentially overall survival.
  • Targeted therapy has converted GIST into a manageable chronic condition for some patients.

Conclusions:

  • While surgery is curative for localized disease, targeted therapies have revolutionized GIST treatment.
  • Future management strategies may involve molecular subtyping for combined or sequential tyrosine kinase inhibitor use.
  • Continued research into GIST molecular biology and drug resistance is crucial for further therapeutic advancements.

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