Table 4.
Summary of secondary endpoint results (ITT analysis set)
| Endpoint | 3-day treatment group (n = 34) | 5-day treatment group (n = 98) |
Total (N = 132) | |||
|---|---|---|---|---|---|---|
| n | n | n | ||||
| CR + PR + HIa (95% CI) | 16 | 47.1 (29.8, 64.9) | 47 | 48.0 (37.8, 58.3) | 63 | 47.7 (39.0, 56.6) |
| HIb (95% CI) | 13 | 38.2 (22.2, 56.4) | 37 | 38.1 (28.5, 48.6) | 50 | 38.2 (29.8, 47.1) |
| Cytogenetic response rate | ||||||
| Overall population | 6 | 4 (66.7) | 24 | 16 (66.7) | 30 | 20 (66.7) |
| Responders evaluated by clinical efficacy assessment (CR + mCR + PR), n (%) | 3 | 3 (100.0) | 13 | 11 (84.6) | 16 | 14 (87.5) |
| Best response as CR | 2 | 2 (100.0) | 3 | 2 (66.7) | 5 | 4 (80.0) |
| Best response mCR | 1 | 1 (100.0) | 10 | 9 (90.0) | 11 | 10 (90.9) |
| Non-responders evaluated by clinical efficacy | 3 | 1 (33.3) | 11 | 5 (45.5) | 14 | 6 (42.9) |
| Time to AML transformationc or deathd (months) | 34 | 98 | 132 | |||
| Event (AML or death) occurs | 9 (26.5) | 44 (44.9) | 53 (40.2) | |||
| Censored | 25 (73.5) | 54 (55.1) | 79 (59.8) | |||
| Median time | – | 21.4 | 23.8 | |||
| Overall survivald | 34 | 98 | 132 | |||
| Death | 16 (47.1) | 58 (59.2) | 74 (56.1) | |||
| Censored | 18 (52.9) | 40 (40.8) | 58 (43.9) | |||
| Survival rate (%) | ||||||
| 6-month (95% CI) | 91.1 (74.8, 97.0) | 84.7 (75.9, 90.5) | 86.3 (79.1, 91.1) | |||
| 12-month (95% CI) | 75.9 (57.5, 87.2) | 65.9 (55.5, 74.4) | 68.4 (59.7, 75.7) | |||
| 18-month (95% CI) | 69.3 (50.3, 82.2) | 53.7 (43.1, 63.2) | 57.6 (48.5, 65.7) | |||
| 24-month (95% CI) | 62.0 (42.6, 76.5) | 44.6 (34.3, 54.4) | 48.9 (39.8, 57.4) | |||
| Transfusion independence, N (%) | ||||||
| Baseline (before first dose of decitabine) | 34 | 7 (20.6) | 98 | 37 (37.8) | 132 | 44 (33.3) |
| Treatment phase | 34 | 18 (52.9) | 97 | 47 (48.5) | 131 | 65 (49.6) |
ITT analysis set included all patients who received at least 1 dose of decitabine
AML acute myeloid leukemia, CI confidence interval, CR complete response, HI hematological improvement, ITT intent to treat, mCR marrow complete response, PR partial response
aImprovement rate was calculated with total ITT patients of each treatment group as denominator
bProportion of patients with simple hematological improvement (HI) as the best response calculated in total ITT patients of each treatment group as denominator (excluding patients with all cell lines assessed as ‘not applicable’ at second treatment cycle, e.g., patients with normal hematology at baseline); patients who showed improvement in any of the 3 lineages were counted in the numerator
cProgression of MDS to AML; defined as the occurrence of >30% blasts in bone marrow
dUsing Kaplan–Meier method