Table 2.
Reported SAEs and TEAEs during GH treatment of patients with SHOX deficiency in GeNeSIS and the clinical trial
| GeNeSIS | Clinical trial | |
|---|---|---|
| Number of GH-treated patients | 521 | 49 |
| Patients with ≥1 SAE | 12 (2.3%) | 6 (12.2%) |
| Number of GH-treated patients with follow-up | 495 | 49 |
| Patients with ≥1 TEAE | 98 (19.8%) | 43 (87.8%) |
| TEAEs reported most frequentlya | ||
| Precocious puberty | 13 (2.6%) | 1 (2.0%) |
| Arthralgia | 12 (2.4%) | 6 (12.2%) |
| Headache | 8 (1.6%) | 9 (18.4%) |
| Hypothyroidism | 6 (1.2%) | 1 (2.0%) |
| Back pain | 5 (1.2%) | 4 (8.2%) |
| Madelung deformity | 2 (0.4%) | 8 (16.3%) |
| Nasopharyngitis | 1 (0.2%) | 7 (14.3%) |
| Ear infection | 1 (0.2%) | 6 (12.2%) |
| Cough | 1 (0.2%) | 6 (12.2%) |
| Congenital bowing of long bonesb | 0 | 9 (18.4%) |
| Vomiting | 0 | 6 (12.2%) |
GH, growth hormone; SAE, serious adverse event; SHOX, short stature homeobox-containing gene; TEAE, treatment-emergent adverse event.
a
MedDRA preferred terms; events reported in >1% of patients in GeNeSIS or >12% of clinical trial patients.
b
MedDRA preferred term states congenital, reflecting the initial clinical observation of Madelung deformity during study participation.