Demographics and baseline characteristics of pts treated
| . | Tamibarotene + azacitidine (n = 126) . | Placebo + azacitidine (n = 64) . | Total (N = 190) . |
|---|---|---|---|
| Age, y∗ | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 73.873 (8.4984) | 75.346 (8.4145) | 74.369 (8.4767) |
| Median | 74.955 | 75.530 | 74.975 |
| Min, max | 37.65, 91.50 | 47.95, 92.86 | 37.65, 92.86 |
| Age group, n (%), y | |||
| ≥18 to <65 | 13 (10.32) | 6 (9.38) | 19 (10.00) |
| ≥65 to <75 | 52 (41.27) | 25 (39.06) | 77 (40.53) |
| ≥75 | 61 (48.41) | 33 (51.56) | 94 (49.47) |
| ≥65 | 113 (89.68) | 58 (90.63) | 171 (90.00) |
| Sex, n (%) | |||
| Male | 86 (68.25) | 46 (71.88) | 132 (69.47) |
| Female | 40 (31.75) | 18 (28.13) | 58 (30.53) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 2 (1.59) | 0 (0.00) | 2 (1.05) |
| Not Hispanic or Latino | 44 (34.92) | 21 (32.81) | 65 (34.21) |
| Not Reported | 3 (2.38) | 2 (3.13) | 5 (2.63) |
| Missing† | 77 (61.11) | 41 (64.06) | 118 (62.11) |
| Race, n (%) | |||
| American Indian or Alaska Native | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Asian | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Black or African American | 4 (3.17) | 1 (1.56) | 5 (2.63) |
| Native Hawaiian or other Pacific Islander | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| White | 42 (33.33) | 20 (31.25) | 62 (32.63) |
| Other | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Multiple | 1 (0.79) | 0 (0.00) | 1 (0.53) |
| Not reported | 2 (1.59) | 2 (3.13) | 4 (2.11) |
| Missing† | 77 (61.11) | 41 (64.06) | 118 (62.11) |
| Body mass index, kg/m2‡ | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 26.659 (4.7545) | 26.609 (4.2877) | 26.642 (4.5912) |
| Median | 26.025 | 25.930 | 25.970 |
| Min, max | 16.06, 50.55 | 17.57, 38.01 | 16.06, 50.55 |
| Geographical region, n (%) | |||
| North America, Western Europe plus Israel | 122 (96.83) | 62 (96.88) | 184 (96.84) |
| Eastern Europe | 4 (3.17) | 2 (3.13) | 6 (3.16) |
| ECOG Performance Status, n (%) | |||
| 0 | 57 (45.24) | 31 (48.44) | 88 (46.32) |
| 1 | 61 (48.41) | 26 (40.63) | 87 (45.79) |
| 2 | 7 (5.56) | 7 (10.94) | 14 (7.37) |
| 3 | 1 (0.79) | 0 (0.00) | 1 (0.53) |
| Time since high-risk diagnosis of MDS, mo | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 2.898 (6.4125) | 4.452 (16.6073) | 3.421 (10.9395) |
| Median | 1.150 | 1.410 | 1.220 |
| Min, max | 0.07, 43.86 | 0.03, 131.19 | 0.03, 131.19 |
| Type of MDS, n (%) | |||
| Primary or not-treatment-related MDS | 111 (88.10) | 56 (87.50) | 167 (87.89) |
| Secondary or treatment-related MDS | 15 (11.90) | 8 (12.50) | 23 (12.11) |
| IPSS-R risk group, n (%) | |||
| Intermediate | 34 (26.98) | 16 (25.00) | 50 (26.32) |
| High | 40 (31.75) | 21 (32.81) | 61 (32.11) |
| Very high | 52 (41.27) | 27 (42.19) | 79 (41.58) |
| Bone marrow blasts (%) | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 9.87 (3.670) | 9.29 (3.558) | 9.67 (3.634) |
| Median | 10.00 | 8.00 | 9.00 |
| Min, max | 3.0, 19.0 | 5.2, 18.0 | 3.0, 19.0 |
| Bone marrow blast category, n (%) | |||
| <5% | 2 (1.59) | 0 (0.00) | 2 (1.05) |
| ≥5% to ≤10% | 73 (57.94) | 44 (68.75) | 117 (61.58) |
| >10% to ≤15% | 42 (33.33) | 14 (21.88) | 56 (29.47) |
| >15% | 9 (7.14) | 6 (9.38) | 15 (7.89) |
| >5% | 121 (96.03) | 64 (100.00) | 185 (97.37) |
| ≤10% | 75 (59.52) | 44 (68.75) | 119 (62.63) |
| >10% | 51 (40.48) | 20 (31.25) | 71 (37.37) |
| . | Tamibarotene + azacitidine (n = 126) . | Placebo + azacitidine (n = 64) . | Total (N = 190) . |
|---|---|---|---|
| Age, y∗ | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 73.873 (8.4984) | 75.346 (8.4145) | 74.369 (8.4767) |
| Median | 74.955 | 75.530 | 74.975 |
| Min, max | 37.65, 91.50 | 47.95, 92.86 | 37.65, 92.86 |
| Age group, n (%), y | |||
| ≥18 to <65 | 13 (10.32) | 6 (9.38) | 19 (10.00) |
| ≥65 to <75 | 52 (41.27) | 25 (39.06) | 77 (40.53) |
| ≥75 | 61 (48.41) | 33 (51.56) | 94 (49.47) |
| ≥65 | 113 (89.68) | 58 (90.63) | 171 (90.00) |
| Sex, n (%) | |||
| Male | 86 (68.25) | 46 (71.88) | 132 (69.47) |
| Female | 40 (31.75) | 18 (28.13) | 58 (30.53) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 2 (1.59) | 0 (0.00) | 2 (1.05) |
| Not Hispanic or Latino | 44 (34.92) | 21 (32.81) | 65 (34.21) |
| Not Reported | 3 (2.38) | 2 (3.13) | 5 (2.63) |
| Missing† | 77 (61.11) | 41 (64.06) | 118 (62.11) |
| Race, n (%) | |||
| American Indian or Alaska Native | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Asian | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Black or African American | 4 (3.17) | 1 (1.56) | 5 (2.63) |
| Native Hawaiian or other Pacific Islander | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| White | 42 (33.33) | 20 (31.25) | 62 (32.63) |
| Other | 0 (0.00) | 0 (0.00) | 0 (0.00) |
| Multiple | 1 (0.79) | 0 (0.00) | 1 (0.53) |
| Not reported | 2 (1.59) | 2 (3.13) | 4 (2.11) |
| Missing† | 77 (61.11) | 41 (64.06) | 118 (62.11) |
| Body mass index, kg/m2‡ | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 26.659 (4.7545) | 26.609 (4.2877) | 26.642 (4.5912) |
| Median | 26.025 | 25.930 | 25.970 |
| Min, max | 16.06, 50.55 | 17.57, 38.01 | 16.06, 50.55 |
| Geographical region, n (%) | |||
| North America, Western Europe plus Israel | 122 (96.83) | 62 (96.88) | 184 (96.84) |
| Eastern Europe | 4 (3.17) | 2 (3.13) | 6 (3.16) |
| ECOG Performance Status, n (%) | |||
| 0 | 57 (45.24) | 31 (48.44) | 88 (46.32) |
| 1 | 61 (48.41) | 26 (40.63) | 87 (45.79) |
| 2 | 7 (5.56) | 7 (10.94) | 14 (7.37) |
| 3 | 1 (0.79) | 0 (0.00) | 1 (0.53) |
| Time since high-risk diagnosis of MDS, mo | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 2.898 (6.4125) | 4.452 (16.6073) | 3.421 (10.9395) |
| Median | 1.150 | 1.410 | 1.220 |
| Min, max | 0.07, 43.86 | 0.03, 131.19 | 0.03, 131.19 |
| Type of MDS, n (%) | |||
| Primary or not-treatment-related MDS | 111 (88.10) | 56 (87.50) | 167 (87.89) |
| Secondary or treatment-related MDS | 15 (11.90) | 8 (12.50) | 23 (12.11) |
| IPSS-R risk group, n (%) | |||
| Intermediate | 34 (26.98) | 16 (25.00) | 50 (26.32) |
| High | 40 (31.75) | 21 (32.81) | 61 (32.11) |
| Very high | 52 (41.27) | 27 (42.19) | 79 (41.58) |
| Bone marrow blasts (%) | |||
| N | 126 | 64 | 190 |
| Mean (SD) | 9.87 (3.670) | 9.29 (3.558) | 9.67 (3.634) |
| Median | 10.00 | 8.00 | 9.00 |
| Min, max | 3.0, 19.0 | 5.2, 18.0 | 3.0, 19.0 |
| Bone marrow blast category, n (%) | |||
| <5% | 2 (1.59) | 0 (0.00) | 2 (1.05) |
| ≥5% to ≤10% | 73 (57.94) | 44 (68.75) | 117 (61.58) |
| >10% to ≤15% | 42 (33.33) | 14 (21.88) | 56 (29.47) |
| >15% | 9 (7.14) | 6 (9.38) | 15 (7.89) |
| >5% | 121 (96.03) | 64 (100.00) | 185 (97.37) |
| ≤10% | 75 (59.52) | 44 (68.75) | 119 (62.63) |
| >10% | 51 (40.48) | 20 (31.25) | 71 (37.37) |
ECOG, Eastern Cooperative Oncology Group; max, maximum; min, minimum; SD, standard deviation.
Age is calculated as the time from the date of birth to the date of main informed consent.
Missing includes data not collected and data not solicited because collection was not allowed per local regulations.
Body mass index (kg/m2) = weight (kg)/[height (cm)/100]2.