Table 2. Results of the per-sample multivariate logistic regression analysis related to total and free drug exposure.

	Total exposure
	All CMLa		All GIST		Exon 11 mutation GIST		Exon 9 mutation or wt GIST
	53		227		86		36
n	OR	P-value	OR	P-value	OR	P-value	OR	P-value
Response vs
Dose	0.3 (±0.2)	0.073	0.7 (±0.2)	0.206	0.6 (±0.2)	0.306	→ ∞b	0.008
mut			0.8 (±0.2)	0.645	1.4 (±0.6)	0.398	0.9 (±0.6)	0.821
AUC	0.5 (±0.1)	0.012	0.5 (±0.1)	0.012
AUC			1.2 (±0.4)	0.541
+ mut			0.8 (±0.3)	0.661	0.5 (±0.2)	0.082	8.9 (±8.0)	0.015
CL	2.6 (±1.1)	0.023	1.5 (±0.4)	0.097
CL			1.0 (±0.3)	0.976
+ mut			0.8 (±0.3)	0.646
Toxicity vs
Dose	3.2 (±2.1)	0.062	2.8 (±0.7)	0.000	2.4 (±0.9)	0.027	3.3 (±2.5)	0.114
mut			1.9 (±0.8)	0.071	1.2 (±0.4)	0.544	0.5 (±0.3)	0.224
AUC	1.4 (±0.4)	0.265	2.2 (±0.4)	0.000
AUC			1.0 (±0.3)	0.906
+ mut			1.9 (±0.7)	0.071	0.5 (±0.3)	0.147	3.2 (±1.7)	0.032
CL	0.9 (±0.4)	0.816	0.9 (±0.2)	0.708
CL			2.0 (±0.6)	0.017
+ mut			1.7 (±0.6)	0.132
	Free exposure
	44		193		78		33
n	OR	P-value	OR	P-value	OR	P-value	OR	P-value
Response vs
Dose	0.4 (±0.3)	0.242	0.8 (±0.3)	0.630	0.7 (±0.3)	0.321	→ ∞b	0.037c
mut			0.6 (±0.3)	0.289	1.3 (±0.6)	0.621	→ ∞b	0.029
AUCu	1.6 (±1.0)	0.481	0.9 (±0.2)	0.548
AUCu			2.6 (±1.1)	0.026
+ mut			0.6 (±0.2)	0.299	0.1 (±0.1)	0.007	0.1 (±0.1)	0.104
CLu	0.8 (±0.9)	0.024	1.2 (±0.5)	0.750
CLu			0.1 (0.1)	0.002
+ mut			1.1 (0.6)	0.807
Toxicity vs
Dose	7.2 (±6.2)	0.022	2.4 (±0.7)	0.001	2.1 (±0.8)	0.064	4.9 (±4.0)	0.051
mut			1.7 (±0.6)	0.167	2.1 (±1.0)	0.118	3.7 (±2.3)	0.035
AUCu	6.1 (±4.5)	0.013	2.7 (±0.6)	0.000
AUCu			2.4 (±0.9)	0.014
+ mut			1.7 (±0.6)	0.148	2.4 (±1.8)	0.240	0.2 (±0.2)	0.154
CLu	0.4 (±0.4)	0.330	0.5 (±0.2)	0.063
CLu			1.3 (±0.8)	0.726
+ mut			1.6 (±0.6)	0.241

OR=odds ratios associated with doubling value of the predictor (AUC/AUC_u, CL/CL_u, Dose) or with mutation profile (mut).

PK parameters expressed as log₂ values; response on a 3-point scale for CML and 2-point scale for GIST (OR vs SD+PD), tolerability on a 4-point scale, and dichotomous mutation profile. The odds ratios (± s.e.) represent the effect on efficacy and toxicity score of a doubling of the PK parameter (AUC/AUC_u or CL/CL_u) or the Dose.

^aNo BCR-ABL mutation detected in the CML population.

^bGroups entirely distinct.

^cApproximated value only, due to a lack of sufficient different samples.