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Table 6 Efficacy

From: Phase 1 dose-ranging study of ezatiostat hydrochloride in combination with lenalidomide in patients with non-deletion (5q) low to intermediate-1 risk myelodysplastic syndrome (MDS)

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