ESPE Abstracts

In the history of biosynthetic GH, first prediction models on long term outcome of therapy were based on large multinational cohorts of various growth disorders and have concentrated on GH dose. In this study we analyzed the 1st year and final height (FH) data in a large single center cohort (center of expertise for rare growth disorders) and compared patient outcome and predictions in GHD and SGA. Our aim was to predict treatment outcome using simple previously suggested parameters using linear regression models for group comparisons adjusted for age and height SDS at treatment start. Patients with ongoing treatment were extrapolated using multiple imputation. GHD and SGA were defined according to international consensus guidelines (cut-off 7ng/ml in GHD, birthweight or length < -2,0 SDS in SGA). The sample consisted of 103 patients with GHD and 53 patients with SGA, median age 6,5 and 7,7 years, respectively (p 0,0968 ns). GH dose was 25 mcg/kg/d in GHD and 35mcg/kg/d in SGA. Height at start of GH therapy was -3,08 (-3,73/2,44 upper/lower quartile) SDS in GHD and -2,88 (-3,63/-2,57) SDS in SGA (P=0,771). DeltaHSDS 1st year was 0,92 (0,83/1,01) SDS in GHD and 0,69 (0,57/0,82) SDS in SGA (p 0,004). DeltaHSDS from start to FH was +2,15 (1,93/2,63) SDS in GHD and 1,16 (0,86/1,45) SDS in SGA (<0,001), corresponding to target height adjusted FH of -0,44 (-0,67/-0,2) SDS in GHD and -1,19 (-1,53/-0,86) SDS in SGA. A prognostic model including age and height SDS at start as well as deltaHSDS 1st year predicted deltaSDS at FH in GHD with an R-square of 0.47 but of only <0.1 in SGA. In GHD, the addition of deltaHSDS at FH increased the R-square from 0.38 (age and height SDS at start only) to 0.47. Summary and conclusions: As expected, both short term and long term effects of GH therapy were significantly higher in GHD compared to SGA. In contrast to GHD, long term effect of GH could not be predicted by the 1st year height SDS increase in SGA. Overall, long-term results in our center with GH dosing in the low-dose segment yieled favourable results when compared to recently published multinational trials*. • JCEM 107: 3287-3301 (2022)