Adjuvant imatinib mesylate after resection of localised, primary gastrointestinal stromal tumour: a randomised, double-blind, placebo-controlled trial
- Study type
- Clinical trial
- Study name
- ACOSOG Z9001
- Phase
- Phase 3
- Design
- Randomised, double-blind, placebo-controlled, multicentre trial
- Sample size
- 713 patients (359 in the imatinib group and 354 in the placebo group)
- Source
- The Lancet · 2009
- Trial registration
- NCT00041197
In plain words
Gastrointestinal stromal tumour (GIST) is a type of sarcoma that grows in the digestive tract and may come back after surgery. In this study, taking an imatinib tablet every day for one year after surgery was compared with a placebo. In people who took imatinib, the tumour came back or death occurred less often, and the treatment was well tolerated. These results come from a fairly short follow-up, and decisions about treatment should be made with your own doctor.
Key results
Treatment arms and regimens
Imatinib mesylate 400 mg daily for 1 year after surgery (359 patients).
Placebo daily for 1 year after surgery (354 patients). On tumour recurrence, patients in this group could cross over to imatinib.
Source of the regimen details: Abstract of the paper
Doses are given for the information of specialists, as in this study’s protocol, and are not a prescription. Every drug must be prescribed by the treating physician for the individual patient.
Study population
Eligible patients had complete gross resection of a primary gastrointestinal stromal tumour at least 3 cm in size and positive for the KIT protein on immunohistochemistry.
Endpoints
Primary endpoint: recurrence-free survival. Analysis was by intention to treat. Accrual was stopped early because the results crossed the efficacy boundary for recurrence-free survival at the interim analysis.
Safety and side effects
Adjuvant imatinib was well tolerated. The most common serious adverse events in the imatinib group were: dermatitis (11 patients [3%] versus 0), abdominal pain (12 patients [3%] versus 6 patients [1%]) and diarrhoea (10 patients [2%] versus 5 patients [1%]). In the placebo group, hyperglycaemia (2 patients [less than 1%] versus 7 patients [2%]) was reported.
Clinical significance and place in guidelines
According to the abstract, one year of adjuvant imatinib after complete resection of a primary gastrointestinal stromal tumour (at least 3 cm and KIT positive) improved recurrence-free survival compared with placebo and its toxicity was tolerable; adjuvant imatinib is therefore an evidence-based option for these patients. The abstract gives no information on overall survival, optimal treatment duration, drug approvals or its place in guidelines, and these should be checked in up-to-date guidelines.
Limitations
Median follow-up was short (19.7 months) and accrual was stopped early because the efficacy boundary was crossed at the interim analysis. The primary endpoint was recurrence-free survival, and overall survival results are not reported in the abstract. Treatment duration was 1 year, and patients in the placebo group could cross over to imatinib on recurrence. The authors themselves concluded that imatinib "appears to" improve recurrence-free survival.
This summary was prepared by the editorial team from the published paper; the original paper is the final reference. Read the original abstract on PubMed.