Discontinuation of imatinib in patients with advanced gastrointestinal stromal tumours after 3 years of treatment: an open-label multicentre randomised phase 3 trial

摘要: Summary Background The effect of imatinib discontinuation on progression-free survival and overall in long-lasting responders with advanced gastrointestinal stromal tumours (GIST) is unknown. We assessed treatment interruption patients non-progressive disease according to the Response Evaluation Criteria In Solid Tumors criteria after 3 years a randomised trial. Methods this open-label national multicentre phase study France, GIST free progression 400 mg/day were randomly assigned continue or interrupt imatinib. Randomisation was done centrally independently from other procedures computer-generated permuted blocks two four stratified by participating centre presence absence residual CT scan. primary endpoint survival. An interim analysis planned first 50 patients. Analysis intention-to-treat principle—ie, all group included. This registered ClinicalTrial.gov, number NCT00367861. Findings 434 enrolled trial between May 27, 2002, 5, 2009. Between June 13, 2005, 30, 2007, who had received their treatment, 25 each group. By Dec 7, 2009, median follow-up 35 months (95% CI 33–38) random assignment, 2-year 80% 58–91) continuation 16% (5–33) (p Interpretation Imatinib results high risk rapid GIST. Discontinuation not recommended outside clinical trials unless experience significant toxic effects. Funding Conticanet, Ligue Contre Le Cancer du Rhone, Novartis.