Demographics (safety population) and baseline iron burden (ITT population) of patients in the FIRST-EXT study
| . | Deferiprone (N = 134) . |
|---|---|
| Age, y | |
| Mean (SD) | 16.2 (8.6) |
| Median (P25, P75) [min-max] | 14.0 (10, 20) [4-47] |
| Sex, n (%) | |
| Female | 53 (39.6) |
| Male | 81 (60.4) |
| Race, n (%) | |
| White | 114 (85.1) |
| Black | 20 (14.9) |
| Primary diagnosis, n (%) | |
| Sickle cell disease | 115 (85.8) |
| Sickle cell anemia | 73 (54.5) |
| Hemoglobin S-beta-thalassemia | 40 (29.9) |
| Sickle cell with hemoglobin C disease | 2 (1.5) |
| Other anemias | 19 (14.2) |
| Hereditary spherocytosis | 8 (6.0) |
| Congenital dyserythropoietic anemia | 4 (3.0) |
| Spherocytic anemia∗ | 4 (3.0) |
| Hemoglobinopathy† | 1 (0.7) |
| Chronic nonspherocytic hemolytic anemia | 1 (0.7) |
| Autoimmune hemolytic anemia | 1 (0.7) |
| Baseline iron burden, mean (SD) [min-max]‡ | |
| LIC, mg/g dw | 14.93 (7.61) [2.33-36.84] |
| Cardiac T2∗ MRI, ms | 32.69 (17.66) [20.70-48.20] |
| SF, μg/L | 3894 (2591) [134-12397] |
| Concomitant hydroxyurea, n (%) | 22 (16.4) |
| . | Deferiprone (N = 134) . |
|---|---|
| Age, y | |
| Mean (SD) | 16.2 (8.6) |
| Median (P25, P75) [min-max] | 14.0 (10, 20) [4-47] |
| Sex, n (%) | |
| Female | 53 (39.6) |
| Male | 81 (60.4) |
| Race, n (%) | |
| White | 114 (85.1) |
| Black | 20 (14.9) |
| Primary diagnosis, n (%) | |
| Sickle cell disease | 115 (85.8) |
| Sickle cell anemia | 73 (54.5) |
| Hemoglobin S-beta-thalassemia | 40 (29.9) |
| Sickle cell with hemoglobin C disease | 2 (1.5) |
| Other anemias | 19 (14.2) |
| Hereditary spherocytosis | 8 (6.0) |
| Congenital dyserythropoietic anemia | 4 (3.0) |
| Spherocytic anemia∗ | 4 (3.0) |
| Hemoglobinopathy† | 1 (0.7) |
| Chronic nonspherocytic hemolytic anemia | 1 (0.7) |
| Autoimmune hemolytic anemia | 1 (0.7) |
| Baseline iron burden, mean (SD) [min-max]‡ | |
| LIC, mg/g dw | 14.93 (7.61) [2.33-36.84] |
| Cardiac T2∗ MRI, ms | 32.69 (17.66) [20.70-48.20] |
| SF, μg/L | 3894 (2591) [134-12397] |
| Concomitant hydroxyurea, n (%) | 22 (16.4) |
dw, dry weight; ITT, intent-to-treat; LIC, liver iron concentration; max, maximum; min, minimum; MRI, magetic resonance imaging; ms, milliseconds; P25, twenty-fifth percentile; P75, seventy-fifth percentile; SD, standard deviation; SF, serum ferritin.
Hereditary status was not captured.
Hemoglobinopathy other than beta-thalassemia.
Baseline was defined as the start of deferiprone treatment. Baseline LIC and cardiac T2∗ MRI values were missing for 1 patient and 3 patients, respectively.