Table 1.
Patient demographic and baseline characteristics for participants ≥ 5 years at screening and for all patients (aged 1–12 years)
| Characteristic | Patients aged ≥ 5 years at screening | All patients | ||||
|---|---|---|---|---|---|---|
| Burosumab (n = 15) | Conventional therapy (n = 20) | Total (n = 35) | Burosumab (n = 29) | Conventional therapy (n = 32) | Total (n = 61) | |
| Age, years, | ||||||
| Mean (SD) | 8.6 (2.4) | 8.4 (2.2) | 8.5 (2.2) | 5.8 (3.4) | 6.3 (3.2) | 6.3 (3.3) |
| Sex, n (%) | ||||||
| Boys | 10 (66.7) | 9 (45.0) | 54.3) | 13 (44.8) | 14 (43.8) | 27 (44.3) |
| Girls | 5 (33.3) | 11 (55.0) | 16 (45.7) | 16 (55.2) | 18 (56.3) | 34 (55.7) |
| Ethnic origin, n (%) | ||||||
| White | 12 (80.0) | 14 (70.0) | 26 (74.3) | 25 (86.2) | 25 (78.1) | 50 (82.0) |
| Asian | 1 (6.7) | 5 (25.0) | 6 (17.1) | 2 (6.9) | 6 (18.8) | 8 (13.1) |
| Other | 1 (13.3) | 1 (5.0) | 3 (8.6) | 2 (6.9) | 1 (3.1) | 3 (4.9) |
| Geographic region, n (%) | ||||||
| Japan | 1 (6.7) | 3 (15.0) | 4 (11.4) | 2 (6.9) | 3 (9.4) | 5 (8.2) |
| ROW | 14(93.3) | 17(85.0) | 31 (88.6) | 27 (93.1) | 28 (90.6) | 56 (91.8) |
| TmP/GFR, mmol/L | ||||||
| Mean (SD) | 0.67 (0.12) | 0.66 (0.11) | 0.67 (0.11) | 0.65 (0.11) | 0.71 (0.12) | 0.68 (0.12) |
| Height Z score | ||||||
| Mean (SD) | − 2.4 (1.2) | − 1.9 (0.8) | − 2.1 (1.0) | − 2.3 (1.2) | − 2.1 (0.9) | − 2.2 (1.0) |
| Median (min, max) | − 2.0 (− 5.0, − 0.5) | − 2.1 (− 3.1, − 0.1) | − 2.0 (− 5.0, − 0.1) | − 2.3 (− 3.1, − 1.5) | − 2.1 (− 2.5, − 1.4) | − 2.2 (− 5.0, − 0.1) |
| Weight, Z score | ||||||
| Mean (SD) | − 1.0 (1.4) | − 0.4 (0.8) | − 0.9 (1.2) | − 0.9 (1.2) | − 0.6 (0.9) | − 0.8 (1.0) |
| Median (min, max) | − 0.8 (− 3.2, 1.3) | − 0.6 (− 1.6, 1.5) | − 0.8 (− 3.2, 1.4) | − 0.8 (− 3.2, 1.4) | − 0.7 (− 2.3, 1.5) | − 0 8 (− 3.2, 1.5) |
| Serum phosphorus concentration, mmol/L | ||||||
| Mean (SD) | 0.75 (0.07) | 0.73 (0.09) | 0.74 (0.08) | 0.78 (0.08) | 0.74 (0.08) | 0.76 (0.08) |
| Serum 1,25(OH)2D concentration, pmol/L | ||||||
| Mean (SD) | 96 (38) | 89 (36) | 91 (36) | 110 (48) | 96 (36) | 103 (43) |
| Alkaline phosphatase concentration, U/L | ||||||
| Mean (SD) | 493.3 (146.4) | 516.8 (168.1) | 506.7 (157.3) | 510.8 (124.9) | 523.4 (154.4) | 517.4 (140.2) |
| Total Thacher RSS | ||||||
| Mean (SD) | 3.1 (0.8) | 3.0 (0.9) | 3.0 (0.8) | 3.2 (1.0) | 3.2 (1.1) | 3.2 (1.1) |
| Median (min, max) | 3.0 (2.0, 5.0) | 3.0 (2.0, 4.5) | 3.0 (2.0, 5.0) | 3.0 (2.5, 4.0) | 3.0 (2.5, 3.5) | 3.0 (2.0, 6.5) |
Max maximum, min minimum, ROW rest of world, RSS Rickets Severity Score, SD standard deviation, TmP/GFR tubular maximum for phosphate reabsorption per glomerular filtration rate