Table 3.
Safety Summary through Week 64
| Assessment | Conventional therapy N = 32 | Burosumab N = 29 |
|---|---|---|
| TEAEs | 27 (84·4) | 29 (100·0) |
| Related TEAE | 7 (21·9) | 17 (58·6) |
| Serious TEAE | 3 (9·4) | 3 (10·3) |
| Serious Related TEAE | 0 (0·0) | 0 (0·0) |
| Grade 3 or 4 TEAE | 3 (9·4) | 4 (13·8) |
| TEAE Leading to Study Discontinuation | 0 (0·0) | 0 (0·0) |
| TEAE Leading to Treatment Discontinuation | 0 (0·0) | 0 (0·0) |
| TEAE Leading to Death | 0 (0·0) | 0 (0·0) |
| Pre-defined TEAEs of Interest | ||
| Injection site reaction | 0 (0·0) | 15 (51·7) |
| Hypersensitivity | 6 (18·8) | 11 (37·9) |
| Hyperphosphatemia | 0 (0·0) | 0 (0·0) |
| Ectopic mineralization | 0 (0·0) | 0 (0·0) |
| Restless leg syndrome | 0 (0·0) | 0 (0·0) |
| TEAEs with ≥ 10% Incidence in Either Group | ||
| Pyrexia | 6 (18·8) | 16 (55·2) |
| Cough | 6 (18·8) | 15 (51·7) |
| Arthralgia | 10 (31·3) | 13 (44·8) |
| Vomiting | 8 (25·0) | 12 (41·4) |
| Nasopharyngitis | 14 (43·8) | 11 (37·9) |
| Pain in extremity | 10 (31·3) | 11 (37·9) |
| Headache | 6 (18·8) | 10 (34·5) |
| Injection site erythema | 0 (0·0) | 9 (31·0) |
| Dental caries | 2 (6·3) | 9 (31·0) |
| Tooth abscess | 3 (9·4) | 8 (27·6) |
| Injection site reaction | 0 (0·0) | 7 (24·1) |
| Rhinorrhoea | 2 (6·3) | 7 (24·1) |
| Diarrhoea | 2 (6·3) | 7 (24·1) |
| Vitamin D decrease | 1 (3·1) | 6 (20·7) |
| Constipation | 0 (0·0) | 5 (17·2) |
| Nasal congestion | 1 (3·1) | 5 (17·2) |
| Oropharyngeal pain | 1 (3·1) | 5 (17·2) |
| Vitamin D deficiency | 1 (3·1) | 5 (17·2) |
| Contusion | 0 (0·0) | 4 (13·8) |
| Ear pain | 1 (3·1) | 4 (13·8) |
| Nausea | 1 (3·1) | 4 (13·8) |
| Asthma | 1 (3·1) | 4 (13·8) |
| Seasonal allergy | 2 (6·3) | 4 (13·8) |
| Influenza | 6 (18·8) | 4 (13·8) |
| Injection site pruritus | 0 (0·0) | 3 (10·3) |
| Injection site swelling | 0 (0·0) | 3 (10·3) |
| Fall | 0 (0·0) | 3 (10·3) |
| Injection site rash | 0 (0·0) | 3 (10·3) |
| Rash | 2 (6·3) | 3 (10·3) |
| Upper respiratory tract infection | 3 (9·4) | 3 (10·3) |
| Abdominal pain upper | 3 (9·4) | 3 (10·3) |
Data are n (%). TEAE, treatment-emergent adverse event. The serious TEAE in the conventional therapy group were hospitalization or surgery for craniosynostosis, bilateral genu varum deformities, and hematuria. The serious TEAE in the burosumab group were craniosynostosis, a viral infection, and a migraine. Injection site reaction is a grouped term that includes injection site reaction, erythema, pruritus, rash, erosion, swelling, urticaria, discomfort, hypersensitivity, inflammation, and papule. Hypersensitivity is a grouped term that includes rash (generalized, erythematous, injection site), dermatitis allergic, drug eruption, and swelling face.