Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial

摘要: Summary Background No effective therapeutic options for patients with unresectable imatinib-resistant gastrointestinal stromal tumour are available. We did a randomised, double-blind, placebo-controlled, multicentre, international trial to assess tolerability and anticancer efficacy of sunitinib, multitargeted tyrosine kinase inhibitor, in advanced who were resistant or intolerant previous treatment imatinib. Methods Blinded sunitinib placebo was given orally once daily at 50-mg starting dose 6-week cycles 4 weeks on 2 off treatment. The primary endpoint time progression. Intention-to-treat, modified intention-to-treat, per-protocol analyses done. This study is registered ClinicalTrials.gov, number NCT00075218. Findings 312 randomised 2:1 ratio receive (n=207) (n=105); the unblinded early when planned interim analysis showed significantly longer progression sunitinib. Median 27·3 (95% CI 16·0–32·1) receiving 6·4 (4·4–10·0) those (hazard 0·33; p Interpretation noted significant clinical benefit, including disease control superior survival, compared after failure discontinuation imatinab. Tolerability acceptable.