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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.