# Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumours

Source: https://onco.cc/key-papers/paper-demetri-imatinib-gist-nejm-2002/  
OnCo record `paper-demetri-imatinib-gist-nejm-2002` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Imatinib produced responses in more than half of patients with advanced gastrointestinal stromal tumours, a cancer that had been completely resistant to chemotherapy, and turned a lethal disease into a chronic one.

## Summary

Phase 2 trial of 147 patients with unresectable or metastatic KIT-positive gastrointestinal stromal tumour randomised to imatinib 400 or 600 mg daily.

Partial response occurred in 53.7 percent and stable disease in 27.9 percent, with only 13.6 percent early progression; oedema, nausea, diarrhoea and myalgia were common but serious toxicity was uncommon. Long-term follow-up showed median survival of about five years.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: New England Journal of Medicine
- Year: 2002
- DOI: 10.1056/NEJMoa020461
- Authors: Demetri GD, von Mehren M, Blanke CD, et al.
- Findings: Partial response 53.7 percent; stable disease 27.9 percent.; No significant difference between 400 and 600 mg doses.
- What it means: Imatinib is the standard first-line treatment for advanced GIST and the model for kinase-targeted therapy in solid tumours.
- Caveats: Randomised between doses only, without a control arm.; Response assessment by size criteria underestimates benefit (Choi criteria came later).

## Sources

- N Engl J Med 2002: https://doi.org/10.1056/NEJMoa020461
- PubMed: https://pubmed.ncbi.nlm.nih.gov/12181401/

## Connected records

- cancers: [KIT exon 11-mutant GIST](https://onco.cc/cancers/gist-kit-exon-11/)
- drugs: [Imatinib](https://onco.cc/drugs/imatinib/)
- journals: [New England Journal of Medicine](https://onco.cc/journals/nejm/)

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