Background: Recombinant human growth hormone (r-hGH) has been used for more than 30 years and indications for r-hGH have multiplied worldwide. There has been concern that it might raise mortality, but published data are limited.
Methods: The cohort comprised of 3902 UK patients over 18 years of age in 2009, treated with childhood r-hGH at all the major UK growth centres. The total European cohort was 24 232 from eight countries (including the UK), with > 400 000 patient years of follow-up. Patients were classified a priori based on pre-treatment perceived mortality risk from their underlying disease and followed for cause-specific mortality. Person-years at risk of mortality and expected rates from general population data were used to calculate standardized mortality ratios (SMRs).
Results: In the UK, low-risk patients with isolated GH deficiency or idiopathic short stature, all-cause mortality was not increased [SMR 1.0 (95% confidence interval 0.42.4)] nor was it increased in children born small for gestational age [SMR 1.1 (0.52.3)], but in contrast, increases in mortality in this sub-category were seen in the French sub-cohort. In patients at moderate, or high risk, mortality was clearly increased [SMR 3.6 (2.84.7) and 16.6 (14.019.7), respectively] in the UK cohort, similar to the findings from all countries. Mortality was not associated with mean daily or cumulative doses of r-hGH for any of the risk groups. Cause-specific mortality from diseases of the circulatory and haematological systems was increased in all risk groups.
Conclusions: In this, the largest cohort with the longest follow-up for r-hGH treated children, all-cause mortality was strongly related to underlying diagnosis. Results from patients with isolated GH deficiency or idiopathic short stature or SGA in the UK suggest that r-hGH treatment is not associated with increased all-cause mortality. However, mortality from certain causes was increased, emphasizing the need for further long-term surveillance.
27 - 29 Nov 2019
British Society for Paediatric Endocrinology and Diabetes