Cited by 33Growth Hormone and Insulin-like Growth FactorsPituitary Gland Disorders and TreatmentsHypothalamic control of reproductive hormones

Mortality in children receiving growth hormone treatment of growth disorders: data from the genetics and neuroendocrinology of short stature international study

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Quigley, Charmian A, Child, Christopher J, Zimmermann, Alan G, Rosenfeld, Ron G, Robison, Leslie L, Blum, Werner F·2017·The Journal of Clinical Endocrinology & Metabolism

Abstract

Context: Although pediatric growth hormone (GH) treatment is generally considered safe for approved indications, concerns have been raised regarding potential for increased risk of mortality in adults treated with GH during childhood. Objective: To assess mortality in children receiving GH. Design: Prospective, multinational, observational study. Setting: Eight hundred twenty-seven study sites in 30 countries. Patients: Children with growth disorders. Interventions: GH treatment during childhood. Main Outcome Measure: Standardized mortality ratios (SMRs) and 95% confidence intervals (CIs) using age- and sex-specific rates from the general population. Results: Among 9504 GH-treated patients followed for ≥4 years (67,163 person-years of follow-up), 42 deaths were reported (SMR, 0.77; 95% CI, 0.56 to 1.05). SMR was significantly elevated in patients with history of malignant neoplasia (6.97; 95% CI, 3.81 to 11.69) and borderline elevated for those with other serious non-GH-deficient conditions (2.47; 95% CI, 0.99-5.09). SMRs were not elevated for children with history of benign neoplasia (1.44; 95% CI, 0.17 to 5.20), idiopathic GHD (0.11; 95% CI, 0.02 to 0.33), idiopathic short stature (0.20; 95% CI, 0.01 to 1.10), short stature associated with small for gestational age (SGA) birth (0.66; 95% CI, 0.08 to 2.37), Turner syndrome (0.51; 95% CI, 0.06 to 1.83), or short stature homeobox-containing (SHOX) gene deficiency (0.83; 95% CI, 0.02 to 4.65). Conclusions: No significant increases in mortality were observed for GH-treated children with idiopathic GHD, idiopathic short stature, born SGA, Turner syndrome, SHOX deficiency, or history of benign neoplasia. Mortality was elevated for children with prior malignancy and those with underlying serious non-GH-deficient medical conditions.

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  • No overall increase in mortality due to GH relative to other GH-treated pediatric populations has been reported in TS individuals in GH registries.

    “No overall increase in mortality due to GH relative to other GH-treated pediatric populations has been reported in individuals with TS followed in the GH registries.173,197,208,225”

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  1. Crowley RK, Argese N, Tomlinson JW, Stewart PM. Central hypo- adrenalism. J Clin Endocrinol Metab. 2014;99(11):4027-4036.

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Authors
Quigley, C. A., Child, C. J., Zimmermann, A. G., Rosenfeld, R. G., Robison, L. L., & Blum, W. F.
Affiliations
  • Indiana University School of Medicine (US)
  • Endocrinology, Lilly Research Laboratories, Windlesham, Surrey GU20 6PH, United Kingdom
  • Eli Lilly (United States) (US)
  • Oregon Health & Science University (US)
  • St. Jude Children's Research Hospital (US)
  • Endocrinology, University Children’s Hospital, 35392 Giessen, Germany
Corresponding author
Quigley, Charmian A
Venue
The Journal of Clinical Endocrinology & Metabolism
Published
2017-05-26
Volume
102
Issue
9
Pages
3195-3205
ISSN
  • 0021-972X (ISSN-L)
  • 1945-7197
OpenAlex
W2618242417
Cited by
33
References
48
Citations by year
2026: 1 · 2025: 3 · 2024: 2 · 2023: 1 · 2022: 3 · 2021: 4 · 2020: 3 · 2019: 9 · 2018: 7
FWCI
4.11
Type
journal article
Language
en
Open access
Locations
  • The Journal of Clinical Endocrinology & Metabolism (publishedVersion)
  • PubMed (publishedVersion)
Funders
Eli Lilly and Company
Topics
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Growth Hormone and Insulin-like Growth Factors
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Pituitary Gland Disorders and Treatments
  • Health Sciences › Medicine › Reproductive Medicine › Hypothalamic control of reproductive hormones
Keywords
growth hormone therapy, child mortality, growth disorders, idiopathic growth hormone deficiency, idiopathic short stature, small for gestational age, Turner syndrome, SHOX deficiency, prior malignancy, standardized mortality ratio, long-term safety, childhood cancer survivors
MeSH terms
Adolescent, Body Height, Child, Child, Preschool, Dose-Response Relationship, Drug, Drug Administration Schedule, Female, Growth Disorders, Humans, Male, Prospective Studies, Reference Values, Cohort Studies, Confidence Intervals, Survival Analysis, Treatment Outcome, Risk Assessment, Human Growth Hormone, Internationality, Kaplan-Meier Estimate

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