Table 5.
Growth outcomes at year 1 in the efficacy cohort
| Diagnosis | Prepubertala | Pubertala | ||||||
|---|---|---|---|---|---|---|---|---|
| n | Initial Ht-SDS | Y 1 Ht-SDS | Delta Ht-SDS | n | Initial Ht-SDS | Y 1 Ht-SDS | Delta Ht-SDS | |
| IGHD | 13 882 | −3.15 (−4.65 to −2.11) |
−2.45 (−3.83 to −1.37) |
0.66 (0.22 to 1.40) |
8365 | −2.49 (−3.55 to −1.55) |
−2.00 (−3.12 to −0.99) |
0.48 (0.02 to 0.96) |
| NSD | 800 | −2.96 (−4.08 to −2.13) |
−2.30 (−3.38 to −1.47) |
0.63 (0.23 to 1.12) |
592 | −2.59 (−3.58 to −1.72) |
−2.14 (−3.23 to −1.09) |
0.41 (−0.04 to 0.89) |
| congGHD | 1740 | −3.40 (−5.53 to −1.56) |
−2.32 (−4.41 to −0.37) |
1.01 (0.15 to 2.22) |
340 | −2.65 (−4.34 to −1.01) |
−2.14 (−3.70v−0.43) |
0.49 (−0.06 to 1.14) |
| CP | 652 | −2.25 (−4.13 to −0.40) |
−1.51 (−3.44 to 0.34) |
0.75 (0.07 to 1.50) |
255 | −2.10 (−3.64 to −0.17) |
−1.43 (−2.99 to 0.34) |
0.52 (−0.12 to 1.18) |
| MB | 306 | −2.10 (−3.62 to −0.78) |
−1.58 (−3.24 to −0.29) |
0.51 (0.07 to 0.90) |
334 | −1.57 (−3.10 to −0.14) |
−1.27 (−2.80 to 0.18) |
0.32 (−0.14 to 0.84) |
| OCT | 512 | −2.23 (−4.00 to −0.51) |
−1.67 (−3.38 to 0.13) |
0.59 (0.01 to 1.25) |
585 | −1.45 (−3.26 to 0.35) |
−1.05 (−2.73 to 0.75) |
0.45 (−0.16 to 1.08) |
| ECM | 265 | −2.26 (−3.66 to −0.85) |
−1.96 (−3.39 to −0.37) |
0.38 (−0.06 to 0.86) |
358 | −1.52 (−3.01 to −0.26) |
−1.25 (−2.78 to 0.08) |
0.28 (−0.18 to 0.83) |
| ISS | 2125 | −3.20 (−4.35 to −2.30) |
−2.62 (−3.83 to −1.63) |
0.55 (0.18 to 1.00) |
1443 | −2.61 (−3.67 to −1.43) |
−2.19 (−3.36 to −0.99) |
0.39 (−0.10 to 0.85) |
| TS | 4067 | −3.27 (−4.52 to −2.10) |
−2.68 (−4.05 to −1.44) |
0.58 (0.05 to 1.08) |
1269 | −3.14 (−4.65 to −1.97) |
−2.72 (−4.23 to −1.48) |
0.44 (−0.08 to 0.93) |
| PWS | 1198 | −2.02 (−3.84 to −0.21) |
−1.02 (−2.84 to 0.73) |
0.94 (0.12 to 1.78) |
224 | −1.81 (−3.57 to 0.50) |
−1.40 (−3.35 to 0.84) |
0.30 (−0.29 to 1.14) |
| OS | 1290 | −3.72 (−5.64 to −2.45) |
−3.05 (−4.81 to −1.75) |
0.63 (0.16 to 1.30) |
388 | −2.93 (−4.34 to −1.19) |
−2.54 (−4.06 to −0.74) |
0.40 (−0.08 to 0.87) |
| SGA | 3487 | −3.25 (−4.48 to −2.30) |
−2.51 (−3.76 to −1.57) |
0.71 (0.30 to 1.23) |
997 | −2.68 (−3.73 to −1.76) |
−2.18 (−3.37 to −1.19) |
0.48 (−0.03 to 0.99) |
| CRF | 935 | −3.07 (−4.84 to −1.83) |
−2.40 (−4.20 to −1.07) |
0.63 (−0.06 to 1.41) |
408 | −2.56 (−4.62 to −1.34) |
−2.20 (−4.24 to −0.76) |
0.41 (−0.19 to 1.04) |
| Other | 1416 | −3.37 (−5.17 to −1.93) |
−2.79 (−4.59 to −1.28) |
0.56 (−0.01 to 1.28) |
874 | −2.51 (−3.98 to −0.63) |
−2.12 (−3.55 to −0.32) |
0.36 (−0.14 to 0.94) |
Data are presented as median (10th-90th percentile) unless noted otherwise.
Abbreviations: congGHD, congenital GHD; CP, craniopharyngioma; CRF, chronic renal failure; ECM, extracranial malignancy; GHD, growth hormone deficiency; Ht-SDS, height SD score; IGHD, idiopathic GHD; ISS, idiopathic short stature; MB, medulloblastoma; NSD, neurosecretory dysfunction; OCT, other cranial tumors; OS, other syndromes; PWS, Prader-Willi syndrome; SGA, small for gestational age; TS, Turner syndrome.
a
Patients who remained prepubertal during year 1.
b
Patients who already reached puberty at treatment start or went into puberty during year 1.