The following is a summary of “Safety and Efficacy of Pediatric Growth Hormone Therapy: Results From the Full KIGS Cohort,” published in the December 2022 issue of Endocrinology & Metabolism by Maghnie, et al.

KIGS, or the Kabi/Pfizer International Growth Database, was a large, global database (1987–2012) of children who received recombinant human growth hormone (rhGH) in real-world settings. From the whole KIGS cohort, researchers, for a study attempted to assess the effectiveness and safety of rhGH.

They gathered data from children receiving rhGH treatment for development problems (Genotropin [somatropin]; Pfizer). All patients who received treatment were assessed for safety, and those who received treatment for a year or longer were assessed for effectiveness. Patients who had received treatment for five years or more ( ≥ 2 years of puberty) and had grown to a near-adult height (NAH) comprised a subgroup. Adverse events, major adverse events, and height increase.

Treatment was given for idiopathic GH deficiency (IGHD; 46.9%), organic GHD (10.0%), small for gestational age (SGA; 9.5%), Turner syndrome (TS; 9.2%), idiopathic short stature (ISS; 8.2%), and others (16.2%) in the whole KIGS cohort (N = 83,803 [58% male]). The median time spent receiving rhGH therapy was 2.7 years, while the median time spent being observed was 3.1 years. 3.7% of patients experienced SAEs, and 0.4% died. Recurrence of craniopharyngioma (n = 151), neoplasm (n = 99), cancer (n = 91), and scoliosis (n = 91) was the most prevalent SAEs. Prepubertal patients’ median first-year delta height-SD score (SDS) (Prader) was 0.66 (IGHD), 0.55 (ISS), 0.58 (TS), and 0.71 (SGA). For males, the NAH-SDS median increases were 1.79 (IGHD), 1.37 (ISS), and 1.34 (SGA), whereas for girls they were 2.07 (IGHD), 1.62 (ISS), 1.07 (TS), and 1.57 (SGA).

The biggest and longest-running worldwide database of children who had received rhGH treatment, KIGS, had data demonstrating that rhGH was safe and increased adult height in both GHD and non-GHD situations.