Safety and Mortality Outcomes Following Pediatric Growth Hormone Therapy

Primary cancer rates and mortality risk were no higher for pediatric patients treated with growth hormone therapy.

Primary cancer rates and mortality risk were no higher for pediatric patients treated with growth hormone (GH) therapy and hemorrhagic strokes only occurred in patients with corresponding risk factors, according to a study published in The Journal of Clinical Endocrinology & Metabolism.

Researchers in this multinational, prospective, observational study from 1999 to 2015 sought to assess the validity of common concerns regarding the possible links between GH therapy and premature mortality, diabetes, primary cancer, neoplasm recurrence, and cerebrovascular disease. Using general population registries, children with growth disorders treated with GH therapy at 827 sites in 30 countries (N = 22,311) were evaluated for standardized mortality and incidence ratios for mortality, primary cancer, and diabetes with a mean follow-up period of 4.2 ± 3.2 years. Predominant diagnoses for participants were 8% Turner syndrome, 13% idiopathic short stature, and 63% GH deficiency.

In the cohort of patients eligible for inclusion in the mortality ratio calculation, 42 deaths occurred with an overall standardized mortality ratio of 0.61 (95% CI, 0.44-0.82). The standardized mortality ratio was elevated for patients with cancer-related organic GH deficiency (5.87; 95% CI, 3.21-9.85). Eighteen cases of type 2 diabetes were reported, showing an elevated risk (standardized incidence ratio [SIR], 3.77; 95% CI, 2.24-5.96), but 72% of these participants had diabetes risk factors. 

Fourteen primary cancers were observed in patients without cancer histories (SIR, 0.71; 95% CI, 0.39-1.20), intracranial tumor recurrences occurred in 8.1% (67 out of 823) of tumor survivors (16.9 cases/1000 person years; 95% CI, 13.3-21.5), and second neoplasms occurred in 5.0% (31 out of 622) of cancer survivors (10.7 cases/1000 person years; 95% CI, 7.5-15.2). Hemorrhagic strokes (n = 3) were seen only in participants with risk factors.

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Study investigators concluded that although the study’s results reflect favorably on GH therapy, the follow-up time was relatively short. The researchers added that “specific safety findings [emphasize] the need to monitor GH-treated patients for abnormalities in glucose metabolism and those with a history of previous neoplasm and irradiation for development of subsequent neoplasms.”

Disclosures: The GeNeSIS study was sponsored by Eli Lilly and Company. Please refer to original reference for a full list of authors’ disclosures.


Child CJ, Zimmermann AG, Chrousos GP, et al. Safety outcomes during pediatric GH therapy: final results from the prospective GeNeSIS observational program [published online September 13, 2018]. J Clin Endocrinol Metab. doi:10.1210/jc.2018-01189.