Patient demographics and baseline characteristics
| Voxelotor/placebo, mg/d . | Multiple dose for 28 d . | Multiple dose for 90 d . | |||||
|---|---|---|---|---|---|---|---|
| Placebo . | 500 . | 700 . | 1000 . | Placebo . | 700 . | 900 . | |
| N | 10 | 10 | 12 | 6 | 4 | 6 | 6 |
| Median age (range), y | 38 (21-53) | 29 (20-48) | 29 (20-56) | 40 (25-47) | 28 (18-48) | 41 (29-53) | 37 (25-42) |
| Male sex, n (%) | 6 (60) | 8 (80) | 4 (33) | 2 (33) | 3 (75) | 4 (67) | 3 (50) |
| Median BMI (range), kg/m2 | 23.5 (19.6-30.5) | 21.4 (17.2-27.5) | 23.0 (17.8-34.4) | 22.4 (20.2-26.1) | 18.8 (17.2-28.4) | 26.9 (21.9-35.3) | 22.8 (20.0-27.3) |
| Median baseline Hb (range), g/dL | 8.1 (7.2-10.0) | 7.9 (7.0-9.7) | 9.1 (7.5-9.8) | 8.2 (7.5-8.4) | 7.9 (7.2-9.3) | 8.3 (7.1-9.7) | 9.0 (7.6-9.7) |
| Hospitalizations due to painful crisis in the previous 12 mo, median (range) | 1 (0-7) | 0 (0-1) | 1 (0-7) | 1 (0-4) | 1 (0-2) | 0 (0-2) | 0 (0-1) |
| Patients with 0 events in previous 12 mo, n | 5 | 7 | 5 | 3 | 2 | 4 | 5 |
| Blood transfusions in previous 12 mo, median (range) | 0 (0-1) | 0 (0) | 0 (0-4) | 0 (0-5) | 0 (0) | 0 (0) | 0 (0-1) |
| Current use of HU, n (%) | 3 (30) | 1 (10) | 3 (25) | 2 (33) | 1 (25) | 0 (0) | 2 (33) |
| Voxelotor/placebo, mg/d . | Multiple dose for 28 d . | Multiple dose for 90 d . | |||||
|---|---|---|---|---|---|---|---|
| Placebo . | 500 . | 700 . | 1000 . | Placebo . | 700 . | 900 . | |
| N | 10 | 10 | 12 | 6 | 4 | 6 | 6 |
| Median age (range), y | 38 (21-53) | 29 (20-48) | 29 (20-56) | 40 (25-47) | 28 (18-48) | 41 (29-53) | 37 (25-42) |
| Male sex, n (%) | 6 (60) | 8 (80) | 4 (33) | 2 (33) | 3 (75) | 4 (67) | 3 (50) |
| Median BMI (range), kg/m2 | 23.5 (19.6-30.5) | 21.4 (17.2-27.5) | 23.0 (17.8-34.4) | 22.4 (20.2-26.1) | 18.8 (17.2-28.4) | 26.9 (21.9-35.3) | 22.8 (20.0-27.3) |
| Median baseline Hb (range), g/dL | 8.1 (7.2-10.0) | 7.9 (7.0-9.7) | 9.1 (7.5-9.8) | 8.2 (7.5-8.4) | 7.9 (7.2-9.3) | 8.3 (7.1-9.7) | 9.0 (7.6-9.7) |
| Hospitalizations due to painful crisis in the previous 12 mo, median (range) | 1 (0-7) | 0 (0-1) | 1 (0-7) | 1 (0-4) | 1 (0-2) | 0 (0-2) | 0 (0-1) |
| Patients with 0 events in previous 12 mo, n | 5 | 7 | 5 | 3 | 2 | 4 | 5 |
| Blood transfusions in previous 12 mo, median (range) | 0 (0-1) | 0 (0) | 0 (0-4) | 0 (0-5) | 0 (0) | 0 (0) | 0 (0-1) |
| Current use of HU, n (%) | 3 (30) | 1 (10) | 3 (25) | 2 (33) | 1 (25) | 0 (0) | 2 (33) |
BMI, body mass index; HU, hydroxyurea.