Table 1.
Baseline Characteristics
| Characteristic | Conventional Therapy (N=32) | Burosumab (N=29) |
|---|---|---|
| Age, years | 6·3 (3·2) | 5·8 (3·4) |
| Patients <5-years-old (stratification criteria) | 12 (38) | 14 (48) |
| Male | 14 (43·8) | 13 (44·8) |
| White | 25 (78·1) | 25 (86·2) |
| Region (stratification criteria Japan vs rest of the world) | ||
| Japan | 3 (9) | 2 (7) |
| United States | 15 (47) | 16 (55) |
| Canada | 7 (22) | 2 (7) |
| Europe | 3 (9) | 2 (7) |
| Korea | 2 (6) | 0 (0) |
| Australia | 2 (6) | 7 (24) |
| Height Z-score, mean (SD) | −2·1 (0·9) | −2·3 (1·2) |
| median [Range] | −21 [−4·7, −0·1] | −2·3 [−5·0, −0·3] |
| Weight Z-score, mean (SD) | −0·6 (0·9) | −0·9 (1·2) |
| median [Range] | −0·7 [−2·3, 1·5] | −0·8 [−3·2, 1·4] |
| Tanner Stage | ||
| 1 | 31 (97) | 27 (93) |
| 2 | 1 (3) | 2 (7) |
| Serum phosphorus, mmol/L | 0·74 (0·08) | 0·78 (0·08) |
| Serum TmP/GFR, mmol/L | 0·65 (0·11) | 0·71 (0·12) |
| Serum 1,25(OH)2D, pmol/L | 96 (36) | 110 (48) |
| Serum 25(OH)D, nmol/L | 79·38 (25·14) | 80·63 (26·15) |
| Alkaline Phosphatase, U/L | 523·4 (154·4) | 510·8 (124·9) |
| Duration of previous conventional therapy, years mean (SD) | 4·3 (3·0) | 3·3 (3·1) |
| median [Range] | 3·5 [0·8–12·0] | 2·2 [0·5–12·2] |
| Total Rickets Severity Score, mean (SD) | 3·2 (1·1) | 3·2 (1·0) |
| median [Range] | 3·0 [2·0, 6·5] | 3·0 [2·0, 6·5] |
| Patients with a Total Thacher Rickets Severity Score >2·5 (stratification criteria) | 20 (63) | 19 (66) |
Data are n (%), mean (SD), and/or median [min, max]. Baseline values were assessed after a 7-day washout period, in which patients stopped treatment with conventional therapy.