ESPE Abstracts

Background: GH deficiency remains the main indication for GH therapy in children. GH therapy has subsequently been approved in the USA and Europe for other conditions resulting in short stature, including Turner Syndrome, being born small for gestational age with failure to attain normal growth, Prader-Willi Syndrome, chronic renal insufficiency, short stature homeobox-containing gene deficiency, and in the USA exclusively Noonan Syndrome and idiopathic short stature.

Objectives: To describe the main indications and treatment outcomes of rGH therapy among a cohort of children with short stature followed at the endocrinology clinic at SKMC-Abu Dhabi over the past 12 years. Assessing treatment outcomes using a change in height SDS at 1 year and 3 years post rGH therapy for various indications, in addition to exploring potential predictors for good outcomes.

Methods: A cross-sectional retrospective review of children (age < 18 years) treated with GH and followed between Jan 2011 and Dec 2022.

Results: A total of 416 children received rGH therapy during the study period off of which 369 were rGH naïve at baseline and enrolled in subsequent analysis at 1-year and 3-year follow-ups. Clinical indications were GHD (n=83, 19.9%), ISS (n=142, 34.1%), SGA (n=116, 27.9%), CKD (n=20, 4.8%), TS (n=16, 3.8%). Mean age at GH initiation based on indications were 9.8 years (±3.6) for GHD, 10.9 years (±2.9) for ISS, 6.9 years (±0.87) for SGA, 6.5 years for CKD (±3.5) and 9.2 years (±2.2) for TS. Mean height SDS at baseline was -2.89 ± 0.72, comparable among all groups but worse for TS (-3.38±0.75). Mean height SDS gain at 1year was excellent for all indications mean 0.69±0.45, more significant in SGA (=0.82 SD) but lower than the mean for TS (0.54 SD). The responses at 3 years persisted (Mean height SD gain=1.2±0.7) with an overall 83.3% gain in height. Multivariate analysis revealed that both age and puberty status at GH therapy initiation were inversely correlated with responses at 1 year and 3 years, with P=0.04 and 0.01 respectively however gender had no effect on the outcome.

Conclusion: Established our clinic’s rGH therapy registry. GHD is the most common indication in our cohort. Results were consistent with those from international surveillance databases. Age and pre-pubertal status at GH initiation were independently associated with a significant response to therapy supporting the importance of early identification and prompt initiation of GH therapy.