{"entity":{"id":"paper-demetri-imatinib-gist-nejm-2002","kind":"paper","name":"Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumours","aka":[],"tldr":"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.\n\nPartial 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.","asOf":"2026-09-17","links":[{"label":"N Engl J Med 2002","url":"https://doi.org/10.1056/NEJMoa020461"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/12181401/"}],"tags":[],"related":[],"cancers":["gist-kit-exon-11"],"sections":[],"technologies":[],"targets":[],"drugs":["imatinib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nejm"],"dependsOn":[],"notes":[],"journal":"New England Journal of Medicine","year":2002,"doi":"10.1056/NEJMoa020461","pmid":"12181401","authors":"Demetri GD, von Mehren M, Blanke CD, et al.","paperType":"rct","findings":["Partial response 53.7 percent; stable disease 27.9 percent.","No significant difference between 400 and 600 mg doses."],"whatItMeans":"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)."],"changedPractice":true,"participants":147},"route":"/key-papers/paper-demetri-imatinib-gist-nejm-2002/","neighbours":{"cancer":[{"id":"gist-kit-exon-11","kind":"cancer","name":"KIT exon 11-mutant GIST","route":"/cancers/gist-kit-exon-11/"}],"drug":[{"id":"imatinib","kind":"drug","name":"Imatinib","route":"/drugs/imatinib/"}],"journal":[{"id":"nejm","kind":"journal","name":"New England Journal of Medicine","route":"/journals/nejm/"}]}}