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. Author manuscript; available in PMC: 2014 Oct 1.
Published in final edited form as: Hematol Oncol Clin North Am. 2013 Oct;27(5):905–920. doi: 10.1016/j.hoc.2013.07.007

Table 2.

Clinical trials of imatinib in metastatic GIST

Study Study
Type
Dosages studied Number of
patients
Results

van Oosterom,
A.T., et al
Phase I 400mg q.d.,300mg b.i.d.,
400mg b.i.d., 500mg b.i.d
40 54% PR
37% SD

Demetri, G.D.,
et al.
Phase
II
400mg q.d vs. 600mg q.d 147 No statistically significant differences in toxicity or response
between the two doses.
Response rate in the 400mg q.d arm 49.3% PR, 31.5%SD,
and 16.4 % PD.
Response rate in the 600mg q.d arm 58.1%PR, 24.3%SD,
and 10.8% PD.

Verweij, J., et al. Phase II 400mg b.i.d 27 4% CR,67% PR, 19% SD,11% PD
73% free from disease at 1 year

Verweij, J., et al. Phase III 400mg q.d vs. 400mg b.i.d 946 Response rate in the 400mg q.d arm 4% CR, 45% PR, 32%
SD, 13% PD.
Response rate in the 400mg b.i.d arm 6% CR, 48% PR,
32% SD, 9% PD.
After a median follow-up of 760 days, 263 (56%) of 473
patients in the once a day arm had progressed compared with
235 (50%) of 473 in the twice a day arm hazard ratio 0·82
[95% CI 0·69–0·98]; p=0·026.
OS was 85% at 1 year and 69% at 2 years in patients treated
once a day, and 86% at 1 year and 74% at 2 years in those
treated twice a day.
Increased dose reductions (77 [16%] vs 282 [60%]) and
interruptions (189 [40%] vs 302 [64%]) in the twice a day
arm.

Blanke, C.D., et al Phase III 400mg q.d vs. 400mg b.i.d 694 Response rate in the 400mg q.d arm 5% CR, 40% PR, 25%
SD, 12% PD.
Response rate in the 400mg b.i.d arm 3% CR, 42% PR, 22%
SD, 10% PD
Median PFS was 18 and 20 months in the once daily and
twice daily arms respectively.
Median OS was 55 and 51 months, in the once daily and
twice daily arms respectively.
No statistically significant differences in objective response
rates, PFS or OS.

Data from Refs 712.