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Effect of oxandrolone and timing of pubertal induction on final height in Turner syndrome: final analysis of the UK randomised placebo-controlled trial
  1. Emma Jane Gault1,
  2. Tim J Cole2,
  3. Sarah Casey3,
  4. Peter C Hindmarsh4,
  5. Peter Betts5,
  6. David B Dunger6,
  7. Malcolm D C Donaldson7
  1. 1University of Glasgow College of Medical, Veterinary and Life Sciences, Glasgow, UK
  2. 2Policy and Practice Programme, UCL Great Ormond Street, Institute of Child Health, London, UK
  3. 3Pharmacy (Clinical Trials) Department, NHS Greater Glasgow and Clyde, Glasgow, UK
  4. 4Clinical and Molecular Genetics Unit, UCL Great Ormond Street Institute of Child Health, London, UK
  5. 5Paediatrics, Southampton University Hospitals NHS Trust, Southampton, UK
  6. 6Paediatrics, University of Cambridge, Cambridge, UK
  7. 7Section of Child Health, Royal Hospital for Sick Children, University of Glasgow School of Medicine, Glasgow, UK
  1. Correspondence to Dr Malcolm D C Donaldson, Section of Child Health, Royal Hospital for Sick Children, University of Glasgow School of Medicine, Glasgow G51 4TF, UK; malcolm.donaldson{at}glasgow.ac.uk

Abstract

The UK Turner syndrome (TS) study examined the effect on final height of oxandrolone 0.05 mg/kg/day (maximum dose 2.5 mg) versus placebo from 9 years of age; and delaying ethinylestradiol induction of puberty by 2 years from 12 (E12) to 14 (E14) years in growth hormone-treated girls with TS. The study ran from 1999 to 2013. By 2011, eighty-two of 92 participants had reached final height and an interim analysis using the Super-Imposition by Translation And Rotation model showed significant increases in final height with both oxandrolone and E14. The analysis has been repeated now that all 92 patients have reached final height. Oxandrolone still significantly increased final height by 4.1 cm (95% CI 1.6 to 6.6, n=92) compared with 4.6 cm previously. However, the E14 effect was no longer significant at 2.7 cm (95% CI −0.8 to 6.1, n=56) compared with 3.8 cm previously.

  • Turner syndrome
  • growth
  • endocrinology
  • therapeutics
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Footnotes

  • Contributors PB, TJC, MDCD, DBD and PCH were responsible for designing the study. PB, SC, MDCD and EJG carried out the study. TJC, MDCD and EJG analysed the data. TJC, MDCD, DBD and EJG prepared the manuscript. MDCD is the guarantor of the study.

  • Funding Funding was provided by the Scottish Executive Chief Scientist Office (1999–2004) (K/MRS/50/C2713) and by the British Society for Paediatric Endocrinology and Diabetes (2004–2013), with a contribution to funding of pharmacy staff from the Child Growth Foundation. Additional funding was received from the NIHR; TJC was funded by Medical Research Council (grant MR/R010692/1).

  • Competing interests None declared.

  • Patient consent for publication Written informed consent from parents and assent from the girls at enrolment was obtained.

  • Ethics approval The study has been approved previously by the Scotland A Research Ethics Committee (formerly Multicentre Research Ethics Committee for Scotland) (Ref 98/0/92) as well as by local research ethics committees at participating centres.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data availability statement No data are available.

  • Author note We deeply regret that Dr Peter Betts, co-author, died during the submission of this work. This paper is dedicated to the memory of Wendy Paterson (1955–2012), auxologist, who was an integral part of the UK Turner study team and made a fundamental contribution to its successful completion.

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