Table 3.

Response by dose level

	200 mg (n=6)	300 mg (n=10)	400 mg (n=8)	500 mg (n=7)	600 mg (n=10)	Safety expansion, 400 mg (n=8)	Total (n=49)
Overall response	6 (100%)	8 (80%)	6 (75%)	6 (86%)	9 (90%)	7 (88%)	42 (86%), 95% CI 73–94
Complete response or complete response with incomplete marrow recovery	2 (33%)	5 (50%)	6 (75%)	4 (57%)	5 (50%)	3 (38%)	25 (51%), 95% CI 36–66
Nodular partial response or partial response	4 (67%)	3 (30%)	0	2 (29%)	4 (40%)	4 (50%)	17 (35%), 95% CI 22–50
Stable disease	0	1 (10%)	1 (13%)	1 (14%)	1 (10%)	0	4 (8%)
Progressive disease	0	1 (10%)	0	0	0	1 (13%)	2 (4%)
Not assessed*	0	0	1 (13%)	0	0	0	1 (2%)
Negative marrow minimal residual disease response	3 (50%)	3 (30%)	6 (75%)	4 (57%)	7 (70%)	5 (62%)	28 (57%)

Data are n (%) or n (%), 95% CI.

^*Patient had fatal tumour lysis syndrome on day 1.