Endocrine Abstracts

Background: The efficacy of recombinant human growth hormone replacement therapy (GHRT) has been demonstrated, in part, through clinical trials conducted worldwide. KIMS (Pfizer International Metabolic Database), established in 1994 and active until 2012, includes treatment outcome data and real world safety from 15 809 patients from 31 countries.

Objective: The main objective of this analysis was to evaluate the long-term safety of patients with hypopituitarism or with Idiopathic GH deficiency receiving GHRT in adult life.

Study design: This open label, international, multi-center study enrolled adult and adolescents with hypopituitarism and GH deficiency. Patients were treated according to the standard of care of their physicians. A total of 15 809 patients (94.4% Caucasian, 50.5% males, 22.2% childhood-onset) were enrolled, with a mean age (S.D.) of 43.9 yr (15.3) at time of enrollment; the mean duration of follow-up was 5.3 yrs and 83 128 patient-years. Safety outcomes included all reported causality adverse events (AEs), treatment related (TR) and serious AEs (SAEs), including neoplasm incidence.

Results: 8093 (51.2%) patients were reported to have 27118 AEs, of which 16.6% TR, and 3998 (25.3%) patients were reported to have 7154 SAEs. 12.2% were reported to have an AE that led to study medication discontinuation and 5.5% to a dose reduction. The most frequently reported all causality AEs were arthralgia (4.6%), headache (3.6%), influenza (2.8%) and depression (2.8%), whereas for TRAEs these were arthralgia (2.6%) and peripheral edema (1.8%). The most frequently reported SAE was pituitary tumor recurrence, all causality (n=320/15 809, 2.0%) and TR (n=154/15 809, 1%). The reported occurrence of death was highest for general disorders and neoplasms (benign, malignant and unspecified) in 148 and 146 patients, respectively.

Conclusion: The results of this large study of GHRT in adult/adolescent patients with GHD complement data from clinical trials and confirm the favorable safety profile of GHRT.