Health-related quality of life of young adults with Turner syndrome following a long-term randomized controlled trial of recombinant human growth hormone

Shayne P Taback, Guy Van Vliet, Shayne P Taback, Guy Van Vliet

Abstract

Background: There are limited long-term randomized controlled trials of growth hormone (GH) supplementation to adult height and few published reports of the health-related quality of life (HRQOL) following treatment. The present follow-up study of young adults from a long-term controlled trial of GH treatment in patients with Turner syndrome (TS) yielded data to examine whether GH supplementation resulted in a higher HRQOL (either due to taller stature or from the knowledge that active treatment and not placebo had been received) or alternatively a lower HRQOL (due to medicalization from years of injections).

Methods: The original trial randomized 154 Canadian girls with TS aged 7-13 years from 13 centres to receive either long-term GH injections at the pharmacologic dose of 0.3 mg/kg/week or to receive no injections; estrogen prescription for induction of puberty was standardized. Patients were eligible for the follow-up study if they were at least 16 years old at the time of follow-up. The instrument used to study HRQOL was the SF-36, summarized into physical and mental component scales (PCS and MCS); higher scores indicate better HRQOL.

Results: Thirty-four of the 48 eligible participants (71%) consented to participate; data were missing for one patient. Both groups (GH and no treatment) had normal HRQOL at this post-treatment assessment. The GH group had a (mean ± SD) PCS score of 56 ± 5; the untreated group 58 ± 4; mean score for 16-24 year old females in the general population 53.5 ± 6.9. The GH group had a mean MCS score of 52 ± 6; the untreated group 49 ± 13; mean score for 16-24 year old females in the general population 49.6 ± 9.8. Secondary analyses showed no relationship between HRQOL and height.

Conclusions: We found no benefit or adverse effect on HRQOL either from receiving or not receiving growth hormone injections in a long-term randomized controlled trial, confirming larger observational studies. We suggest that it remains ethically acceptable as well as necessary to maintain a long-term untreated control group to estimate the effects of pharmacological agents to manipulate adult height. Young adult women with TS have normal HRQOL suggesting that they adjust well to their challenges in life.

Trial registration: ClinicalTrials.gov Identifier NCT00191113.

References

    1. Taback SP, Van Vliet G, Guyda HJ. Pharmacologic manipulation of height: qualitative review of study populations and designs. Clin Invest Med. 1999;22:53–9.
    1. Stephure DK. Canadian Growth Hormone Advisory Committee. Impact of growth hormone supplementation on adult height in Turner Syndrome: results of the Canadian randomized controlled trial. J Clin Endocrinol Metab. 2005;90:3360–6.
    1. Ross JL, Quigley CA, Cao D, Feuillan P, Kowal K, Chipman JJ, Cutler JBJnr. Growth hormone plus childhood low-dose estrogen in Turner's syndrome. N Engl J Med. 2011;364:1230–42. doi: 10.1056/NEJMoa1005669.
    1. Sybert VP, McCauley E. Turner's syndrome. N Engl J Med. 2004;351(12):1227–38. doi: 10.1056/NEJMra030360.
    1. Baxter L, Bryant J, Cave CB, Milne R. Recombinant growth hormone for children and adolescents with Turner syndrome. Cochrane Database Syst Rev. 2007. p. CD003887.
    1. Bowling A. Measuring disease: a review of disease-specific quality of life measurement scales. Buckingham: Open University Press; 1995.
    1. Carel JC, Ecosse E, Bastie-Sigeac I, Cabrol S, Tauber M, Leger J, Nicolino M, Brauner R, Chaussain JL, Coste J. Quality of life determinants in young women with Turner's syndrome after growth hormone treatment: results of the StaTur population-based cohort study. J Clin Endocrinol Metab. 2005;90(4):1992–7. doi: 10.1210/jc.2004-1395.
    1. Bannink EM, Raat H, Mulder PG, de Muinck Keizer-Schrama SM. Quality of life after growth hormone therapy and induced puberty in women with Turner syndrome. J Pediatr. 2006;148(1):95–101. doi: 10.1016/j.jpeds.2005.08.043.
    1. Busschbach JJ, Rikken B, Grobbee DE, De Charro FT, Wit JM. Quality of life in short adults. Horm Res. 1998;49(1):32–8. doi: 10.1159/000023123.
    1. Boman UW, Bryman I, Halling K, Moller A. Women with Turner syndrome: psychological well-being, self-rated health and social life. J Psychosom Obstet Gynaecol. 2001;22(2):113–22. doi: 10.3109/01674820109049961.
    1. Naess EE, Bahr D, Gravholt CH. Health status in women with Turner syndrome - a questionnaire study on health status, education, work participation and aspects of sexual functioning. Clin Endocrinol (Oxf) 2009.
    1. Ware JE, Kosinski M. SF-36 Physical and Mental Health Summary Scales: a manual for users of version 1. 2. Lincoln, RI: QualityMetric Inc; 2001.
    1. Hopman WM, Berger C, Joseph L, Towheed T, Prior JC, Anastassiades T, Poliquin S, Zhou W, Adachi JD, Hanley DA, Papadimitropoulos E, Tenenhouse A. for the Camos Research Group. Health-related quality of life in Canadian adolescents and young adults: normative data using the SF-36. Can J Public Health. 2009;100:449–52.

Source: PubMed

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