Serious and treatment-emergent adverse events reported in Canada
| Adverse event | Diagnostic category; no. (%) of patients who reported adverse event | |||||
|---|---|---|---|---|---|---|
| All patients | Organic growth hormone deficiency | Idiopathic growth hormone deficiency | Turner syndrome | Idiopathic short stature | Small for gestational age | |
| All patients | n = 850 | n = 379 | n = 147 | n = 156 | n = 38 | n = 19 |
| Death* | 11 (1.3) | 7 (1.8) | 0 (0.0) | 1 (0.6) | 0 (0.0) | 0 (0.0) |
| Considered related to growth hormone treatment† | 2 (0.2) | 2 (0.5) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Serious adverse event | 97 (11.4) | 61 (16.1) | 7 (4.8) | 9 (5.8) | 3 (7.9) | 1 (5.3) |
| Considered related to growth hormone treatment‡ | 19 (2.2) | 12 (3.2) | 1 (0.7) | 4 (2.6) | 2 (5.3) | 0 (0.0) |
| Patients with ≥ 1 follow-up visit | n = 833 | n = 377 | n = 137 | n = 153 | n = 38 | n = 19 |
| ≥ 1 treatment-emergent adverse event§ | 587 (70.5) | 299 (79.3) | 72 (52.6) | 108 (70.6) | 21 (55.3) | 13 (68.4) |
| Headache | 88 (10.6) | 55 (14.6) | 12 (8.8) | 9 (5.9) | 2 (5.3) | 3 (15.8) |
| Secondary hypothyroidism | 60 (7.2) | 45 (11.9) | 6 (4.4) | 1 (0.6) | 0 (0.0) | 0 (0.0) |
| Scoliosis | 54 (6.5) | 22 (5.8) | 10 (7.3) | 12 (7.8) | 0 (0.0) | 3 (15.8) |
| Ovarian failure | 50 (6.0) | 9 (2.4) | 0 (0.0) | 41 (26.8) | 0 (0.0) | 0 (0.0) |
| Arthralgia | 48 (5.8) | 16 (4.2) | 5 (3.6) | 13 (8.5) | 1 (2.6) | 1 (5.3) |
| Upper respiratory tract infection | 44 (5.3) | 31 (8.2) | 5 (3.6) | 6 (3.9) | 0 (0.0) | 1 (5.3) |
| Hypothyroidism | 42 (5.0) | 29 (7.7) | 4 (2.9) | 8 (5.2) | 0 (0.0) | 0 (0.0) |
| Adverse event considered related to growth hormone treatment¶ | 87 (10.4) | 48 (12.7) | 6 (4.4) | 18 (11.8) | 3 (7.9) | 2 (10.5) |
| Headache | 19 (2.3) | 16 (4.2) | 0 (0.0) | 2 (1.3) | 0 (0.0) | 1 (5.3) |
| Scoliosis | 10 (1.2) | 9 (2.4) | 1 (0.7) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Arthralgia | 9 (1.1) | 4 (1.1) | 0 (0.0) | 3 (2.0) | 0 (0.0) | 0 (0.0) |
| Increase in insulin-like growth factor level | 9 (1.1) | 0 (0.0) | 0 (0.0) | 5 (3.3) | 0 (0.0) | 1 (5.3) |
| Melanocytic nevus | 7 (0.8) | 4 (1.1) | 0 (0.0) | 3 (2.0) | 0 (0.0) | 0 (0.0) |
| Otitis media | 6 (0.7) | 4 (1.1) | 0 (0.0) | 2 (1.3) | 0 (0.0) | 0 (0.0) |
↵* See Supplementary Table A2, Appendix 1 for details.
↵† Both due to recurrence of medulloblastoma.
↵‡ All serious adverse events considered related to growth hormone treatment occurred in single patients except for medulloblastoma recurrence in 2 patients (both with organic growth hormone deficiency) and adenoidal hypertrophy in 2 patients (1 with organic growth hormone deficiency, 1 with Turner syndrome). Other serious adverse events reported as related to growth hormone treatment were meningioma, neoplasm progression, aortic valve incompetence, rectal hemorrhage, edema, death, parotitis, upper limb fracture, type 1 diabetes mellitus, optic glioma, anaplastic astrocytoma, adrenal neoplasm, increased intracranial pressure, ischemic stroke, tonsillar hypertrophy, sleep apnea syndrome, scoliosis surgery, meningioma surgery, adenoidectomy and angiodysplasia; some patients experienced more than 1 event.
↵§ Any treatment-emergent adverse event irrespective of relatedness with frequency of 5% or more among all patients.
↵¶ Any treatment-emergent adverse event considered related with frequency greater than 0.5% among all patients.