Table 1.

Pivotal clinical studies with imatinib in the adjuvant setting.

Study First Author/Publication Information	Number of Patients, Patient Population	Clinical Phase	Intervention	Molecular Analysis	Primary Endpoint	Results
Dematteo et al., Lancet 2009 [70]	713 Patients resected for ≥3 cm GIST	3	1 y adjuvant imatinib 400 mg/d vs. placebo	No	RFS (primary endpoint changed from OS to RFS)	1 y RFS: 98% (imatinib) vs. 83% (placebo), HR 0.35. No OS benefits
Casali et al., JCO 2015 [71]	908 Patients after R0-1 surgery for localized high- or intermediate-risk GISTs according to NIH criteria [72]	3	2 y adjuvant imatinib 400 mg/d vs. no treatment	No	IFFS (primary endpoint changed from OS to IFFS)	5 y IFFS: 87.0% (imatinib) vs. 84.1% (control), p = 0.21, HR 0.79. No OS benefits
Joensuu et al., JAMA 2020 [25]	397 Patients resected for high-risk GISTs according to modified NIH criteria [62]	3	1 y vs. 3 y adjuvant imatinib 400 mg/d	Yes (366/397)	RFS	10 y RFS: 52.5% (3 y treatment) and 41.8% (1 y treatment), HR 0.66, p = 0.003 10 year OS: 79.0% (3 y treatment) and 65.3% (1 y treatment), p = 0.004, HR: 0.55.

R0—radical surgery, R1—surgery with positive microscopic margin, OS—overall survival, RFS—recurrence-free survival, NIH—National Institutes of Health, IFFS—imatinib failure-free survival, d—day, y—year, HR—hazard ratio.