SAT-694 Long-term Effectiveness And Safety Of GH Replacement Therapy In Adults <60 Years: Real-world Data From NordiNet® IOS And ANSWER

Abstract J.D. Carmichael: Consulting Fee; Self; Novo Nordisk, Camurus, Xeris. A.Y. Kargi: Consulting Fee; Self; Corcept Therapeutics, Ascendis Pharma, Novo Nordisk, Recordati, Xeris Biopharma. L. Dichtel: Consulting Fee; Self; Lumos Pharma. Employee; Self; Mass General Brigham. Research Investigator; Self; Pfizer, Inc., Novo Nordisk, Perspectum Ltd., Lumos Pharma, Recordati. Stock Owner; Self; Marea Therapeutics. N. Kelepouris: Employee; Self; Novo Nordisk. N. Nedjatian: Employee; Self; Novo Nordisk. M. Fridman: Other; Self; Novo Nordisk. M.M. Weber: Speaker; Self; Ipsen, Eli Lilly & Company, Novo Nordisk. Background: The large-scale, noninterventional NordiNet® International Outcome Study (NordiNet® IOS) and American Norditropin® Studies: Web-Enabled Research (ANSWER) Program collected real-world data on the long-term effectiveness and safety of growth hormone replacement therapy (GHRT). Objective: To compare safety and clinical outcomes of GHRT in adults with adult growth hormone deficiency (AGHD) across 3 age groups. Methods: Real-world data from NordiNet® IOS and ANSWER were used to analyze GHRT safety and clinical outcomes in adults with AGHD by age: 18 to 29 years, 30 to 39 years, and 40 to <60 years in groups 1, 2, and 3, respectively. Safety was assessed in the full analysis set (FAS), including all GH-naïve and nonnaïve patients with a GHD diagnosis who initiated GH replacement after age 18 years and continued beyond age 20 years. Incidence rates/1000 patient-years were used for serious adverse reactions (SARs), nonserious adverse reactions (NSARs), and serious adverse events (SAEs). SAEs assessed included depression, seizure, arrhythmia, and adrenocortical insufficiency rate. Clinical outcomes were assessed in the effectiveness analysis set (EAS), including patients who were GH-naïve at baseline with valid baseline body mass index, age, and dosing information. Clinical outcomes were assessed yearly for ≤10 years of follow-up; clinical outcome differences between groups were compared at 2 years. Results: Pooled analyses from NordiNet® IOS and ANSWER included 2469 patients from the FAS (558, 494, and 1417 in groups 1, 2, and 3, respectively) and 1006 patients from the EAS (148, 209, and 649 in groups 1, 2, and 3, respectively). The mean (SD) durations of follow-up/patient were 5.1 (4.5) and 5.0 (4.1) years for FAS and EAS patients, respectively. In the FAS, no statistically significant differences in the incidence rates of adverse events or reactions were observed between the 3 groups. The most common SAEs in all groups were “neoplasms benign, malignant, and unspecified” (0.54%, 0.61%, and 1.34% in groups 1, 2, and 3, respectively) and “nervous system disorders” (0.90%, 0.61%, and 0.64% in groups 1, 2, and 3, respectively). Of the 10 total deaths recorded, 2 cases (1 due to metastatic colon cancer and 1 due to anaplastic astrocytoma) were deemed possibly related to treatment. In the EAS, there was a general increase in mean GH dose for females of all groups, no change for males in group 2 and 3, and decrease for males in group 1 over the follow-up years. By year 2, the proportion of patients within the normal range of insulin-like growth factor-1 (IGF-I) standard deviation score (SDS) had increased in all groups to ≥80% and remained >80% over most of the follow-up years and up to year 10. Conclusion: No significant differences in the risk of GHRT-related adverse reactions were observed across age groups, and by year 2, ≥80% of patients across all age groups had a normal IGF-I SDS. Saturday, June 1, 2024