ESPE Abstracts

Objective: KIMS (Pfizer International Metabolic Survey) was a postmarketing surveillance study of growth hormone (GH) replacement in adults and adolescents with GH deficiency (GHD) that concluded in October 2012. This analysis aimed to evaluate the overall safety outcomes from the full cohort of GH-treated patients of KIMS.

Methods: Data were collected on adults and adolescents with confirmed GHD and closed epiphyses treated with GH (Genotropin^® [somatropin]; Pfizer, NY) and followed through routine clinical practice. Adverse events (AEs), serious adverse events (SAEs), and clinical characteristics (eg, lipid profile, glucose) are summarized.

Results: The analysis included 15,809 GH-treated patients with a mean follow up of 5.3 years (maximum 18.3 years and total 83,128 patient-years). Mean age at study entry was 43.9 years (standard deviation 15.3 years). Most patients had pituitary/hypothalamic tumors (62.6%) as the cause of their GHD, while 21.5% due to idiopathic/congenital GHD, and 15.2% due to other etiologies. GHD was adult-onset (AO-GHD) in 77.4 % of patients. AEs were reported in 51.2% of patients (treatment-related in 18.8%) during KIMS follow up; the most frequent being arthralgia (4.6%), while the most frequent SAE being pituitary tumor growth or recurrence (2.0%). A total of 606 deaths (3.8%) were reported, most commonly caused by neoplasms (n = 146). Other common death causes were cardiac or vascular disorders (n = 71) and infections/infestations (n = 60). Overall cancer incidence among patients without a prior history of cancer was comparable to that of the general population (standard incidence ratios [SIR] 0.92; 95% CI 0.83-1.01). Cancer SIRs were decreased in patients with idiopathic/congenital GHD (0.64; 95% CI 0.43-0.91), but were similar to the general population in those with pituitary/hypothalamic tumors (0.96; 95% CI 0.87-1.07) or other etiologies (0.80; 95% CI 0.56-1.10). Neither AO-GHD (0.90; 95% CI 0.81-1.00) nor childhood-onset GHD (1.21; 95% CI 0.81-1.74) was associated with elevated cancer risks. Mean (standard deviation) IGF-1 standard deviation score was -1.3 (2.2) at baseline, increased to +0.2 (1.73) at year 1, and reached +0.7 (1.47) at year 18. Neutral effects were observed for lipids and fasting blood glucose levels.

Conclusion: The present KIMS analysis of the full cohort of GH-treated adults, in one of the largest and longest real-world safety surveys, confirms and reinforces the favorable safety profile of GH replacement in adults and adolescents with GHD, with no new safety signals observed. The results support that GH replacement is safe and tolerable as currently prescribed in routine clinical practice.