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Table 5 Efficacy

From: Phase 1-2a multicenter dose-escalation study of ezatiostat hydrochloride liposomes for injection (Telintra®, TLK199), a novel glutathione analog prodrug in patients with myelodysplastic syndrome

Hematologic Improvement Response Rate (IWG 2000)
Baseline Cell-Line Cytopenia n (%)
Trilineage 4/16 (25)
Bilineage 1/13 (8)
Unilineage 7/14 (50)
Hematologic Responses by Cell-Line Cytopenia (IWG 2000)
  Number of Patients Major Response
n (%)
Minor Response
n (%)
Total Response
n (%)
HI-E 38 5 (13) 4 (11) 9 (24)
HI-N 26 9 (35) 2 (8) 11 (42)
HI-P 24 4 (17) 8 (33) 12 (50)
Hematologic Improvement (HI) Response by Patient Demographics & MDS Disease Characteristics
   HI-E Response
n (%)
HI-N Response
n (%)
HI-P Response
n (%)
Number of Patients (%) 9/38 (24) 11/26 (42) 12/24 (50)
Gender    
   Female 2/13 (15) 3/8 (38) 3/9 (33)
   Male 7/25 (28) 8/18 (44) 9/15 (60)
Baseline FAB    
   RA 6/20 (30) 5/12 (42) 7/11 (64)
   RARS 1/6 (17) 2/4 (50) 1/1 (100)
   RAEB 2/8 (25) 4/7 (57) 3/7 (43)
   RAEB-t 0/3 (0) 0/3 (0) 0/3 (0)
   CMML 0/1 (0) 0 0/1 (0)
   Unknown 0 0 1/1 (100)
Baseline Karyotype    
   Normal 3/20 (15) 6/16 (38) 5/14 (36)
   Abnormal 6/18 (33) 5/10 (50) 7/10 (70)
Transfusion Requirements and Clinical Symptoms Improvement (IWG 2000)
Phase 1: Comparison of Decreased Transfusion Requirements and Clinical Symptoms Improvement
    All Dose Levels Combined
n (%)
50% Decreased Transfusion Requirements 3/15 (20)
Clinical Symptoms Improvement 7/21 (33)
Phase 2a: Comparison of Decreased Transfusion Requirements and Clinical Symptoms Improvement by Dose Schedule
   Dose Schedule 1 Days 1–5 of 21-Day Treatment Cycle
n (%)
Dose Schedule 2 Days 1–3 of 21-Day Treatment Cycle
n (%)
Total
n (%)
50% Decreased Transfusion Requirements 1/5 (20) 1/11 (9) 2/16 (13)
Clinical Symptoms Improvement 9/10 (90) 3/12 (25) 12/22 (55)
  1. Abbreviations: RA, refractory anemia; RARS, refractory anemia with ringed sideroblasts; RAEB, refractory anemia with excess blasts; RAEB-t, refractory anemia with excess blasts in transformation; CMML, chronic myelomonocytic leukemia; IWG, International Working Group.