Table 6 Efficacy

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)