Introduction Prescribing of recombinant human growth hormone (rhGH) for growth failure in UK children is based on guidance from the National Institute for Health and Care Excellence. In 2013, the British Society for Paediatric Endocrinology and Diabetes initiated a national audit of newly prescribed rhGH treatment for children and adolescents. In this review, we have examined prescribing practices between 2013 and 2016.
Methods All patients ≤16.0 years of age starting rhGH for licensed and unlicensed conditions in the UK were included. Anonymised data on indication and patient demographics were analysed.
Results During the 4 years, 3757 patients from 76 of 85 (89%) centres started rhGH. For each licensed indication, proportions remained stable over this period: 56% growth hormone deficiency (GHD), 17% small for gestational age (SGA), 10% Turner syndrome, 6% Prader-Willi syndrome (PWS), 3% chronic renal insufficiency (CRI) and 2% short stature homeobox deficiency (SHOXd). However, the unlicensed category decreased from 10% (n=94) in 2013 to 5% (n=50) in 2016. The median age of patients starting rhGH was 7.6 years (range 0.1–16.0). Patients with PWS were significantly younger (median 2.2 years, range 0.2–15.1) compared with other indications (p<0.0001) and were followed by the SGA group (median 6.2 years, range 1.3–15.6, p<0.0001). Boys predominated in all groups except for PWS and SHOXd.
Conclusion We demonstrate significant engagement of prescribing centres in this audit and a decline in unlicensed prescribing by half in this 4-year period. Patients in the PWS group were younger at initiation of rhGH compared with other indications and had no male predominance unlike GHD, SGA and CRI.
- growth hormone
- growth hormone treatment
- growth hormone deficiency
- short stature
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Patient consent for publication Not required.
Contributors SS has coordinated and collected the data for this audit, done the preliminary analysis and wrote the initial draft. VS has reviewed and advised SS for the data collected. NS, LP and IB have overseen the audit as successive BSPED audit leads. LP and IB analysed the data and completed the paper. NS provided comments, which have been incorporated in the paper.
Funding This work was supported as part of an ongoing national audit being carried out by the BSPED.
Competing interests SS, VS and NS have nothing to disclose. IB reports other from one-off (not continuous) professional fees paid by growth hormone pharmaceutical companies, outside the submitted work. LP reports other from expenses to attend paediatric endocrinology conferences, outside the submitted work.
Ethics approval Approval for the audit was obtained from the Caldicott Guardian for NHS Greater Glasgow and Clyde. As the data did not contain patient identifiable information, written informed consent was not required from patients and families.
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement Data from this audit are available to the BSPED Clinical Committee, members and benefactors.
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