Table 2.
Participants treatment history, baseline height, and baseline nephrocalcinosis in burosumab trials [7, 13, 50, 72]
| Open-label, phase 2 burosumab Q2W (N = 13) | Open-label, dose-finding phase 2 burosumab Q2W or Q4W (N = 52) | Randomized, double-blind, placebo-controlled phase 3 | Randomized, active-controlled, open-label, phase 3 burosumab Q4W (N = 134) | ||
|---|---|---|---|---|---|
| Conventional therapy (n = 32) | Burosumab Q2W (n = 29) | ||||
| Age, y | 1-4 | 5-12 | 1-12 | ≤ 18 | |
| Treatment history | |||||
| Conventional therapy, n (%) | 13 (100) | 50 (96) | 32 (100) | 29 (100) | 181 (98) |
| Mean (SD) duration of conventional therapy, y | 1.3 (1.2) | 6.9 (2.4) | 4.3 (3.0) | 3.3 (3.1) | 16.5 (10.4)a |
| 18.2 (11.0)b | |||||
| Mean (SD) age when conventional therapy was initiated, y | 1.7 (1.5) | 2.1 (91.3) | NR | NR | 10.9 (81)c |
| Baseline height | |||||
| Mean (SD) z score | –1.4 (1.2) | –1.9 (1.0) | –2.1 (0.9) | –2.3 (1.2) | –2.3 (1.3) |
| Mean (SD) percentile for age and sex | 18 (25) | 8.7 (11.5) | NR | NR | 6.8 (12.5) |
| Baseline nephrocalcinosis | |||||
| Grade > 0,d n (%) | 0 | 18 (34) | 9 (28) | 5 (17) | 73 (55) |
Abbreviations: NR, not reported; Q2W, every 2 weeks; Q4W, every 4 weeks.
aAmong individuals with any prior use of phosphate.
bAmong individuals with any prior use of vitamin D metabolites or analogs.
cn (percentage, %) of individuals who received conventional therapy before age 18 years.
dValues range from 0 (normal) to 4 (stone formation).