Mortality in Children Receiving Growth Hormone Treatment for Growth Disorders: Data from the GeNeSIS Observational Program.

Mortality in Children Receiving Growth Hormone Treatment for Growth Disorders: Data from the GeNeSIS Observational Program.

J Clin Endocrinol Metab. 2017 May 26;:

Authors: Quigley CA, Child CJ, Zimmermann AG, Rosenfeld RG, Robison LL, Blum WF

Abstract
Context: Although pediatric GH treatment is generally considered safe for approved indications, there have been long-held concerns regarding potential for increased risk of neoplasia and, more recently, of stroke and mortality in adults treated with GH during childhood.
Objective: To assess mortality in children receiving GH.
Design: Prospective, multi-national, observational study.
Setting: Genetics and Neuroendocrinology of Short-stature International Study (GeNeSIS).
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 (67163 person-years of follow-up), 42 deaths were reported (SMR for all diagnoses combined: 0.77 [CI 0.56-1.05]). The SMR was significantly elevated in patients with organic GH deficiency (GHD), mainly influenced by patients with history of malignant neoplasia (n=294, SMR 6.97 [3.81-11.69]). SMRs for patients with history of benign neoplasia (n=158) and idiopathic GHD (n=4324) were 1.44 (0.17-5.20) and 0.11 (0.02-0.33), respectively. SMRs also were not significantly elevated for children with idiopathic short stature (0.20 [0.01-1.10]), short stature associated with small-for-gestational age (SGA) birth (0.66 [0.08-2.37]), Turner syndrome (0.51 [0.06-1.83]), or SHOX deficiency (0.83 [0.02-4.65]).
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.

PMID: 28575299 [PubMed - as supplied by publisher]

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