Baseline characteristics of patients with SCD treated with mitapivat in the 8-week dose-finding period and the 1-year fixed-dose extension period
| . | Dose-finding period (n = 10) . | Fixed-dose extension period (n = 9) . |
|---|---|---|
| Age in y, median (range) | 26 (16-59) | 30 (16-59) |
| Female, n (%) | 6 (60) | 5 (56) |
| SCD genotype, n (%) | ||
| HbSS | 8 (80) | 7 (78) |
| HbS/β0-thalassemia | 1 (10) | 1 (11) |
| HbS/β+-thalassemia | 1 (10) | 1 (11) |
| Concomitant hydroxyurea therapy, n (%) | 6 (60) | 6 (67) |
| VOE∗ rate in previous 24 mo, median (range) | 2 (0-7) | 1 (0-7) |
| SCD-related hospital admission days in previous 24 mo, median (range) | 9 (0-42) | 8 (0-42) |
| RBC units in previous 12 mo, median (range) | 1 (0-3) | 1 (0-3) |
| . | Dose-finding period (n = 10) . | Fixed-dose extension period (n = 9) . |
|---|---|---|
| Age in y, median (range) | 26 (16-59) | 30 (16-59) |
| Female, n (%) | 6 (60) | 5 (56) |
| SCD genotype, n (%) | ||
| HbSS | 8 (80) | 7 (78) |
| HbS/β0-thalassemia | 1 (10) | 1 (11) |
| HbS/β+-thalassemia | 1 (10) | 1 (11) |
| Concomitant hydroxyurea therapy, n (%) | 6 (60) | 6 (67) |
| VOE∗ rate in previous 24 mo, median (range) | 2 (0-7) | 1 (0-7) |
| SCD-related hospital admission days in previous 24 mo, median (range) | 9 (0-42) | 8 (0-42) |
| RBC units in previous 12 mo, median (range) | 1 (0-3) | 1 (0-3) |
Includes VOEs as defined per study protocol based on chart review and patient recall for up to 24 months prior to study enrollment.