Table 3. Baseline Demographics and Disease Characteristics  VIDAZA
(N=99) Observation
(N=92)
Gender (n%)  
Male 72 (72.7) 60 (65.2)
Female 27 (27.3) 32 (34.8)
Race (n%)  
White 93 (93.9) 85 (92.4)
Black  1 (1.0) 1 (1.1)
Hispanic 3 (3.0) 5 (5.4)
Asian/Oriental 2 (2.0) 1 (1.1)
Age (years)  
N 99 91
Mean ± SD 67.3 ± 10.39 68.0 ± 10.23
Range 31 - 92 35 - 88
Adjudicated MDS diagnosis at study entry (n%)  
RA 21 (21.2) 18 (19.6)
RARS 6 (6.1) 5 (5.4)
RAEB 38 (38.4) 39 (42.4)
RAEB-T 16 (16.2) 14 (15.2)
CMMoL 8 (8.1) 7 (7.6)
AML 10 (10.1) 9 (9.8)
Transfusion product used in 3 months before study entry (n%)  
Any transfusion product 70 (70.7) 59 (64.1)
Blood cells, packed human 66 (66.7) 55 (59.8)
Platelets, human blood 15 (15.2) 12 (13.0)
Hetastarch 0(0.0) 1(1.1)
Plasma protein fraction 1(1.0) 0(0.0)
Other 2(2.0) 2(2.2)
Table 4. Response Criteria  RA RARS RAEB RAEB-T CMMoL
Complete
Response
(CR),
duration ≥4weeks Marrow <5% blasts
Peripheral
Blood Normal CBC if abnormal at baseline
Absence of blasts in the peripheral circulation
Partial
Response
(PR),
duration ≥4weeks Marrow No marrow requirements
 ≥50% decrease in blasts
Improvement of marrow dyspoiesis

Peripheral
Blood ≥50% restoration in the deficit from normal levels of baseline white cells, hemoglobin and platelets if abnormal at baseline

No blasts in the peripheral circulation

For CMMoL, if WBC is elevated at baseline, a ≥75% reduction in the excess count over the upper limit of normal
The overall response rate (CR + PR) of 15.7% in VIDAZA-treated patients without AML (16.2%for all VIDAZA randomized patients including AML) was statistically significantly higher than the response rate of 0% in the observation group (p<0.0001) (Table 5). The majority of patients who achieved either CR or PR had either 2 or 3 cell line abnormalities at baseline (79%; 11/14) and had elevated bone marrow blasts or were transfusion dependent at baseline. Patients responding to VIDAZA had a decrease in bone marrow blasts percentage, or an increase in platelets, hemoglobin or WBC. Greater than 90% of the responders initially demonstrated these changes by the 5th treatment cycle. All patients who had been transfusion dependent became transfusion independent during PR or CR. The mean and median duration of clinical response of PR or better was estimated as 512 and 330 days, respectively; 75% of the responding patients were still in PR or better at completion of treatment. Response occurred in all MDS subtypes as well as in patients with adjudicated baseline diagnosis of AML.
Table 5. Response Rates  VIDAZA
(N=89) Observation Before Crossover
(N=83) 
Response n(%) n(%) P value
Overall (CR+PR) 14 (15.7) 0 ( 0.0) (<0.0001)
Complete (CR) 5 ( 5.6) 0 ( 0.0) (0.06)
Partial (PR) 9 (10.1) 0 ( 0.0) --
Patients in the observation group who crossed over to receive VIDAZA treatment (47 patients) had a response rate of 12.8%.
Study 2, a multi-center, open-label, single-arm study of 72 patients with RAEB, RAEB-T, CMMoL, or AML was also carried out. Treatment with subcutaneous VIDAZA resulted in a response rate (CR + PR) of 13.9%, using criteria similar t