Baseline subject demographic and clinical characteristics
| . | GMI-1070 (N = 43) . | Placebo (N = 33) . | P . |
|---|---|---|---|
| Characteristic | |||
| Male sex, N (%) | 18 (41.90) | 13 (39.40) | .829 |
| Age (y) | |||
| Mean (SD) | 25.4 (10.83) | 25.0 (10.20) | .855 |
| Median (range) | 23.0 (12, 50) | 22.0 (14, 51) | |
| Weight (kg) | |||
| Mean (SD) | 61.7 (17.01) | 69.9 (15.66) | .034 |
| Median (range) | 58.7 (32, 109) | 69.1 (46, 113) | |
| BMI | |||
| Mean (SD) | 22.7 (5.05) | 24.5 (5.50) | .152 |
| Median (range) | 21.5 (15, 41) | 23.2 (18, 42) | |
| Hemoglobin diagnosis | |||
| HbSS | 39 | 30 | .144 |
| Hb Sβ0 thalassemia | 1 | 3 | |
| HbSC | 3* | 0 | |
| Medical history | |||
| HU therapy, N (%) | 22 (51.2) | 23 (69.7) | .106 |
| Daily out-patient analgesics, N (%) | 18 (41.9) | 19 (57.6) | .177 |
| Admission for VOC, N (%) | |||
| In previous 12 mo | 33 (76.7) | 25 (75.8) | .921 |
| ≥3 episodes in previous 12 mo | 13 (30.2) | 14 (42.4) | .274 |
| ACS, N (%) | |||
| In previous 12 mo | 5 (11.6) | 6 (18.2) | .517 |
| History of avascular necrosis, N (%) | 15 (34.9) | 8 (24.2) | .320 |
| History of pulmonary hypertension, N (%) | 3 (7.0) | 9 (27.3) | .017 |
| Admission values | |||
| VAS at presentation | |||
| Mean (SD) | 8.28 (1.57) | 8.97 (1.52) | .079 |
| Median (range) | 8.0 (5, 10) | 10.0 (5, 10) | |
| Hemoglobin, g/dL | |||
| Mean (SD) | 8.32 (1.40) | 8.41 (1.57) | .745 |
| Median (range) | 8.60 (5.5, 11.7) | 8.30 (5.4, 13.9) | |
| WBC count, ×103/μL | |||
| Mean (SD) | 13.01 (5.09) | 13.78 (5.66) | .539 |
| Median (range) | 12.08 (3.90, 28.30) | 12.70 (4.31, 29.00) | |
| Absolute neutrophil count, ×103/μL | |||
| Mean (SD) | 7.41 (3.87) | 8.38 (5.16) | .389 |
| Median (range) | 6.60 (1.70, 15.97) | 7.32 (2.30, 26.77) | |
| Baseline hemoglobin A (N = 30) | |||
| <25%, N (%) | 12 (27.9) | 14 (42.4) | >.999 |
| 25 to <50%, N (%) | 1 (2.3) | 2 (6.1) | |
| >50 to <75%, N (%) | 0 (0) | 1 (3.0) |
| . | GMI-1070 (N = 43) . | Placebo (N = 33) . | P . |
|---|---|---|---|
| Characteristic | |||
| Male sex, N (%) | 18 (41.90) | 13 (39.40) | .829 |
| Age (y) | |||
| Mean (SD) | 25.4 (10.83) | 25.0 (10.20) | .855 |
| Median (range) | 23.0 (12, 50) | 22.0 (14, 51) | |
| Weight (kg) | |||
| Mean (SD) | 61.7 (17.01) | 69.9 (15.66) | .034 |
| Median (range) | 58.7 (32, 109) | 69.1 (46, 113) | |
| BMI | |||
| Mean (SD) | 22.7 (5.05) | 24.5 (5.50) | .152 |
| Median (range) | 21.5 (15, 41) | 23.2 (18, 42) | |
| Hemoglobin diagnosis | |||
| HbSS | 39 | 30 | .144 |
| Hb Sβ0 thalassemia | 1 | 3 | |
| HbSC | 3* | 0 | |
| Medical history | |||
| HU therapy, N (%) | 22 (51.2) | 23 (69.7) | .106 |
| Daily out-patient analgesics, N (%) | 18 (41.9) | 19 (57.6) | .177 |
| Admission for VOC, N (%) | |||
| In previous 12 mo | 33 (76.7) | 25 (75.8) | .921 |
| ≥3 episodes in previous 12 mo | 13 (30.2) | 14 (42.4) | .274 |
| ACS, N (%) | |||
| In previous 12 mo | 5 (11.6) | 6 (18.2) | .517 |
| History of avascular necrosis, N (%) | 15 (34.9) | 8 (24.2) | .320 |
| History of pulmonary hypertension, N (%) | 3 (7.0) | 9 (27.3) | .017 |
| Admission values | |||
| VAS at presentation | |||
| Mean (SD) | 8.28 (1.57) | 8.97 (1.52) | .079 |
| Median (range) | 8.0 (5, 10) | 10.0 (5, 10) | |
| Hemoglobin, g/dL | |||
| Mean (SD) | 8.32 (1.40) | 8.41 (1.57) | .745 |
| Median (range) | 8.60 (5.5, 11.7) | 8.30 (5.4, 13.9) | |
| WBC count, ×103/μL | |||
| Mean (SD) | 13.01 (5.09) | 13.78 (5.66) | .539 |
| Median (range) | 12.08 (3.90, 28.30) | 12.70 (4.31, 29.00) | |
| Absolute neutrophil count, ×103/μL | |||
| Mean (SD) | 7.41 (3.87) | 8.38 (5.16) | .389 |
| Median (range) | 6.60 (1.70, 15.97) | 7.32 (2.30, 26.77) | |
| Baseline hemoglobin A (N = 30) | |||
| <25%, N (%) | 12 (27.9) | 14 (42.4) | >.999 |
| 25 to <50%, N (%) | 1 (2.3) | 2 (6.1) | |
| >50 to <75%, N (%) | 0 (0) | 1 (3.0) |
BMI, body mass index; WBC, white blood cells.
Subjects enrolled with historical documentation of HbSS but found on Hb electrophoresis to have HbSC.