Table 6.

Efficacy

	Dose Group 1	Dose Group 2
Intent-to-Treat (ITT) Population	2000/ 10 mg	2500/ 10 mg
HI–E (%) (95% CI)	(N = 13)	(N = 6)
	4 (30.8)	1 (16.7)
	(9.1−61.4)	(0.4−64.1)
HI–N (%) (95% CI)	(N = 3)	(N = 2)
	1 (33.3)	0
	(0.8−90.6)	(0−84.2)
HI–P (%) (95% CI)	(N = 5)	(N = 4)
	3 (60)	0
	(14.7−94.7)	(0−60.2)
Platelet Transfusion Dependent (%)	(N = 1)	(N = 1)
Platelet Transfusion Independent (%)	1 (100)	0 (0)
Bilineage (HI-E and HI-N) (%) (95% CI)	(N = 3)	(N = 2)
	1 (33.3)	0
	(0.8−90.6)	(0−84.2)
Bilineage (HI-E and HI-P) (%) (95% CI)	(N = 5)	(N = 4)
	3 (60)	0
	(14.7−94.7)	(0−60.2)
Bilineage (HI-N and HI-P) (%) (95% CI)	(N = 3)	(N = 1)
	1 (33.3)	0 (0)
	(0.8−90.6)	(0−97.5)
Trilineage (HI-E, HI-N, and HI-P) (%) (95% CI)	(N = 3)	(N = 1)
	1 (33.3)	0
	(0.8−90.6)	(0−97.5)
No. of RBC Transfusion Dependent Patients (%)	10 (76.9)	3 (50)
RBC Transfusion Independence (%) (95% CI)	3 (30)	0
	(6.7−65.2)	(0−70.8)
Efficacy Evaluable (EE) Population
HI–E (%) (95% CI)	(N = 10)	(N = 4)
	4 (40)	1 (25)
	(12.2−73.8)	(0.6−80.6)
HI–N (%) (95% CI)	(N = 3)	(N = 2)
	1 (33.3)	0 (0)
	(0.8−90.6)	(0−84.2)
HI–P (%) (95% CI)	(N = 5)	(N = 2)
	3 (60)	0 (0)
	(14.7−94.7)	(0−84.2)
Platelet Transfusion Dependent	(N = 1)	(N = 0)
Platelet Transfusion Independent (%)	1 (100)	0 (0)
Bilineage (HI-E and HI-N) (%) (95% CI)	(N = 3)	(N = 2)
	1 (33.3)	0 (0)
	(0.8−90.6)	(0−84.2)
Bilineage (HI-E and HI-P) (%) (95% CI)	(N = 5)	(N = 2)
	3 (60)	0 (0)
	(14.7−94.7)	(0−84.2)
Bilineage (HI-N and HI-P) (%) (95% CI)	(N = 3)	(N = 1)
	1 (33.3)	0 (0)
	(0.8−90.6)	(0−97.5)
Trilineage (HI-E, HI-N, and HI-P) (%) (95% CI)	(N = 3)	(N = 1)
	1 (33.3)	0 (0)
	(0.8−90.6)	(0−97.5)
No. of RBC Transfusion Dependent Patients (%)	7 (70)	2 (50)
RBC Transfusion Independence (%) (95% CI)	3 (42.9)	0
	(9.9−81.6)	(0−84.2)

Abbreviations: HI-E hematologic improvement-erythroid; HI-N hematologic improvement-neutrophil; HI-P hematologic improvement-platelet; CI confidence interval; RBC red blood cell.