Background: Young adult patients with childhood-onset GH deficiency (GHD) whose GH replacement therapy (GHRT) is discontinued exhibit negative metabolic and physiological effects, reversible through GHRT.
Objective and hypotheses: To report the characteristics and 5-year GHRT in adults with childhood-onset GHD.
Method: Analysis of the subgroup of adults with childhood-onset GHD included between March 2003 and October 2006 in KIMS. In France, KIMS was conducted in all centers with at least one adult treated with Genotonorm® (Genotropin®).
Results: Overall 120 patients (56 (47%) females) were included; GHD was acquired, congenital and idiopathic in 64 (53%), 41 (34%) and 15 (13%) patients respectively. All patients had severe GHD, associated with one or more other pituitary hormone deficiency in 104 (87%) patients. Median age at inclusion was 22 years (interquartile range 19; 30). Median GHRT dose prescribed at inclusion, was 0.20 mg/day (0.20; 0.40); in 95 (79%) patients inclusion corresponded to adult GHRT initiation. Overall, 23 (19%) patients were lost to follow-up. The percentage of patients treated with GHRT decreased over time especially after 2 years and was 77% at 5 years. In 29 (24%) patients GHRT was permanently discontinued (patients request in 17 (59%) cases). Prescribed GHRT dose increased up to 2 years (0.60 mg/day (0.40;0.80)). IGF1 level was available, at each visit, in more than 80% of the patients, except at 5 years where IGF1 data was available in 70% of the patients. Median IGF1 level was 80 μg/l (40; 164) at inclusion, 199 μg/l (116; 306) after 1 year and 172 μg/l (78; 220) after 5 years. Body mass index increased from 25.4 kg/m2 (21.9; 30.7) at inclusion up to 27.2 kg/m2 (24.0; 32.4) after 5 years. Increase in pituitary tumor size was reported in one female. No new safety concern was reported.
Conclusion: The relatively weak prescribed doses may preclude highlighting long-term benefit of GHRT.
Funding: This work was supported by Pfizer.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology