Table 1.
Demographic characteristics of the safety and efficacy cohorts
| Safety (N = 83 803) |
Efficacy (N = 55 284) |
Excluded (N = 28 519) |
|
|---|---|---|---|
| World region, n (%) | |||
| Europe | 52 135 (62.2) | 35 395 (64.0) | 16 740 (58.7) |
| USA | 16 737 (20.0) | 10 057 (18.2) | 6680 (23.4) |
| Asia | 11 023 (13.2) | 7668 (13.9) | 3355 (11.8) |
| Rest of the world | 3908 (4.7) | 2164 (3.9) | 1744 (6.1) |
| Race/ethnicity, n (%) | |||
| Black | 943 (1.1) | 540 (1.0) | 403 (1.4) |
| White | 59 022 (70.4) | 39 741 (71.9) | 19 281 (67.6) |
| Hispanic | 1989 (2.4) | 937 (1.7) | 1052 (3.7) |
| Asian | 12 082 (14.4) | 8470 (15.3) | 3612 (12.7) |
| Other | 2363 (2.8) | 1496 (2.7) | 867 (3.0) |
| Unknown | 7404 (8.8) | 4100 (7.4) | 3304 (11.6) |
| Sex, n (%) | |||
| Female | 35 183 (42.0) | 23 341 (42.2) | 11 842 (41.5) |
| Male | 48 620 (58.0) | 31 943 (57.8) | 16 677 (58.5) |
| Age at rhGH start, y | |||
| Mean ± SD | 10.2 ± 4.0 | 9.4 ± 3.8 | 10.7 ± 4.2 |
| Median (10th-90th percentile) | 10.7 (4.6 to 14.9) | 9.8 (4.2 to 14.1) | 11.2 (4.7 to 15.6) |
| Height SDS at rhGH start | n = 81 262 | n = 55 284 | n = 25 978 |
| Mean ± SD | −2.9 ± 1.3 | −3.0 ± 1.2 | −2.9 ± 1.4 |
| Median (10th-90th percentile) | −2.9 (−4.4 to −1.6) | −2.9 (−4.4 to −1.7) | −2.8 (−4.5 to −1.4) |
| Weight SDS at rhGH start | n = 81 262 | n = 55 284 | n = 25 978 |
| Mean ± SD | −1.9 ± 1.7 | −1.9 ± 1.7 | −1.8 ± 1.8 |
| Median (10th-90th percentile) | −1.9 (−3.7 to 0.2) | −1.9 (−3.7 to 0.1) | −1.8 (−3.7 to 0.3) |
| BMI SDS at rhGH start | n = 81 257 | n = 55 280 | n = 25 977 |
| Mean ± SD | −0.2 ± 2.2 | −0.2 ± 2.5 | −0.2 ± 1.5 |
| Median (10th-90th percentile) | −0.2 (−1.9 to 1.7) | −0.2 (−1.9 to 1.6) | −0.2 (−1.9 to 1.7) |
| Max GH peak, μg/L | n = 65 058 | n = 42 436 | n = 18 132 |
| Mean ± SD | 9. 5 ± 11.5 | 9.5 ± 11.6 | 9.2 ± 11.2 |
| Median (10th-90th percentile) | 7.4 (1.7 to 19.0) | 7.5 (1.8 to 19.0) | 7.2 (1.5 to 18.4) |
| Diagnosis of growth disorder, n (%) | |||
| Idiopathic GHD | 39 298 (46.9) | 25 810 (46.7) | 13 488 (47.3) |
| Isolated idiopathic GHD | 33 138 (39.5) | 21 263 (38.5) | 11 875 (41.6) |
| Combined idiopathic GHDa | 6160 (7.4) | 4547 (8.2) | 1613 (5.7) |
| Neurosecretory disfunction | 2187 (2.6) | 1537 (2.8) | 650 (2.3) |
| Congenital GHDb | 3323 (4.0) | 2189 (4.0) | 1134 (4.0) |
| Acquired GHD | |||
| Craniopharyngioma | 1381 (1.6) | 965 (1.7) | 416 (1.5) |
| Medulloblastoma | 998 (1.2) | 703 (1.3) | 295 (1.0) |
| Other cranial tumors | 1750 (2.1) | 1209 (2.2) | 541 (1.9) |
| Extracranial malignancy | 940 (1.1) | 680 (1.2) | 260 (0.9) |
| Non-GHD short stature disorders | |||
| Idiopathic short stature | 6867 (8.2) | 4336 (7.8) | 2531 (8.9) |
| Turner syndrome | 7714 (9.2) | 5580 (10.1) | 2134 (7.5) |
| Prader-Willi syndrome | 2338 (2.8) | 1501 (2.7) | 837 (2.9) |
| Other syndromesc | 2602 (3.1) | 1801 (3.3) | 801 (2.8) |
| Small for gestational age | 7936 (9.5) | 4892 (8.8) | 3044 (10.7) |
| Chronic renal failure | 2399 (2.9) | 1514 (2.7) | 885 (3.1) |
| Other causesd | 4070 (4.9) | 2567 (4.6) | 1503 (5.3) |
| Dose at rhGH start, mg/kg/wk | |||
| Mean ± SD | 0.2 ± 0.1 | 0.2 ± 0.1 | 0.2 ± 0.2 |
| Median (10th-90th percentile) | 0.2 (0.2 to 0.4) | 0.2 (0.2 to 0.4) | 0.2 (0.0 to 0.4) |
| Duration of rhGH treatment, y | |||
| Mean ± SD | 3.3 ± 2.8 | 4.2 ± 2.7 | 1.7 ± 2.2 |
| Median (10th-90th percentile) | 2.7 (0.3 to 7.2) | 3.5 (1.4 to 7.9) | 0.8 (0.0 to 4.7) |
| Duration of follow-up in KIGS, y | |||
| Mean ± SD | 3.9 ± 3.1 | 4.7 ± 3.0 | 2.2 ± 2.7 |
| Median (10th-90th percentile) | 3.1 (0.5 to 8.2) | 4.0 (1.6 to 9.0) | 1 (0.0 to 5.8) |
Abbreviations: BMI, body mass index; GH, growth hormone; GHD, growth hormone deficiency; KIGS, Pfizer International Growth Database; MPHD, multiple pituitary hormone deficiency; rhGH, recombinant human growth hormone; SDS, SD score; USA, United States of America.
Idiopathic GHD with additional pituitary hormone deficiencies.
Excluding GH receptor deficiency.
Other syndromes with 100 or more patients included Silver-Russell syndrome, Noonan syndrome, syndromes with/without chromosomal aberration, von Recklinghausen syndrome (neurofibromatosis), and gonadal dysgenesis.
Other causes with 100 or more patients included Kowarski-type bioinactive GH syndromes, other bioinactive GH syndromes, other functional GHD, head trauma, histiocytosis, other causes of acquired GHD, iatrogenic short stature due to medication, achondroplasia, hypochondroplasia, skeletal dysplasia, other chronic inflammatory disorders, precocious puberty, other endocrine disorders (not GHD).