First-year predictors of health-related quality of life changes in short-statured children treated with human growth hormone
- 110 Downloads
Little attention has been directed towards examining the impact of predictors on change in health-related quality of life (HRQOL) within the course of growth hormone (GH) treatment in pediatric short stature. We aimed to assess changes in HRQOL and its sociodemographic, clinical and psychosocial predictors in children and adolescents diagnosed with growth hormone deficiency (GHD), and born short for gestational age (SGA) before and 12-month after start of GH treatment from the parents’ perspective. Results were compared with an untreated group with idiopathic short stature (ISS). In this prospective multicenter study, 152 parents of children/adolescents (aged 4–18 years) provided data on their children’s HRQOL at baseline and at 12-month follow-up.
Repeated-measures multivariate analyses of covariance were performed to examine parent-reported HRQOL changes from baseline to 1-year after treatment and hierarchical linear regressions to identify the predictors of HRQOL changes.
Results showed that parents of children that were treated with GH report an increase in their children’s HRQOL after 1 year. Changes in HRQOL were mostly explained by psychosocial predictors followed by sociodemographic and clinical variables. Specifically, the diagnosis SGA significantly predicted a greater increase in parent-reported HRQOL. Furthermore, a lower caregiving burden significantly predicted a decrease in parent-reported HRQOL.
In conclusion, a substantial percentage of explained variance in HRQOL relates to psychosocial and sociodemographic predictors. However, there appears to be other important factors that are predictors of HRQOL, which need to be determined in large, population-based samples.
KeywordsShort stature Growth hormone treatment Children and adolescents HRQOL Predictors of change
The QoLISSY prospective project would like to thank the children, parents, and staff from the participating clinical centers for their participation and contributions. Besides, we thank Dr. Ilker Akkurt, Dr. Desiree Dunstheimer, Dr. Christian Vogel, Dr. Volker Böttcher, Dr. Ursula Kuhnle Krahl, PD. Dr. Markus Bettendorf, Prof. Dr. Eckhard Schönau, Dr. Susanne Fricke-Otto, Dr. Alexandra Keller and Prof. Dr. Klaus Mohnike who helped to conduct the study, as well as Prof. Dr. H. Wollmann, Dr. Anja Rohenkohl and Dr. Rachel Sommer for supporting and helping to realize the study.
This study was sponsored by Pfizer, Inc. and while the authors received research funding for the conduct of the study, no financial support was given for the writing of this manuscript.
Compliance with ethical standards
Conflict of interest
The authors declare that they have no conflict of interest.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
Informed consent was obtained from all individual participants included in the study.
Access to the QoLISSY instrument
QoLISSY is a joint initiative between Pfizer Limited and the University Medical Center Hamburg–Eppendorf. Copyright Pfizer Limited all rights reserved. The European QoLISSY instrument, together with comprehensive information of its development and validation process is published in the QoLISSY’s User’s Manual (Pabst Science Publishers, Lengerich, 2013). The Manual, which is available upon request, includes QoLISSY child and parent forms, as well as scoring information (http://www.pfizerpatientreportedoutcomes.com/therapeutic-areas/cv-metabolic/endocrine).
- 3.QoLISSY Group (2017) Quality of life of short statured children and adolescents during growth hormone treatment: final study report. Medical University Center Hamburg-Eppendorf, HamburgGoogle Scholar
- 10.Finkelstein BS, Singh J, Silvers JB, Marreroa U, Neuhauser D, Cuttler L (1999) Patient attitudes and preferences regarding treatment: GH therapy for childhood short stature. Horm Res 51:67–72Google Scholar
- 11.Hardin D, Woo J, Butsch R, Huett B (2007) Current Prescribing practices and opinions about growth hormone therapy: results of a nationwide survey of paediatric endocrinologists. Clin Endocrinol (Oxf) 66:85–94Google Scholar
- 15.Migliaretti G, Ditaranto S, Guiot C, Vannelli S, Matarazzo P, Cappello N, Stura I, Cavallo F (2018) Long-term response to recombinant human growth hormone treatment: a new predictive mathematical method. J Endcrinol Investig 41:839–848. https://doi.org/10.1007/s40618-017-0816-6 CrossRefGoogle Scholar
- 17.Ranke M, Lindberg A, Tanaka T, Camacho-Hübner C, Dunger D, Geffner M (2017) Baseline characteristics and gender differences in prepubertal children treated with growth hormone in Europe, USA, and Japan: 25 Years’ KIGS ® experience (1987–2012) and review. Horm Res Paediatr 87:30–41CrossRefGoogle Scholar
- 22.Brütt AL, Sandberg DE, Chaplin J, Wollmann H, Noeker M, Koltowska-Haggstrom M, Bullinger M (2009) Assessment of health-related quality of life and patient satisfaction in children and adolescents with growth hormone deficiency or idiopathic short stature—part 1: a critical evaluation of available tools. Horm Res 72:65–73Google Scholar
- 25.Bullinger M, Dellenmark Blom M, Feigerlova E, Herdman M, Lunde C, Mimoun E, Pleil A, Power M, Quitmann J, Rohenkohl A, Sanz D, Skoropadskaya A, Wollmann H, Chaplin J (2010) Assessing quality of life in short stature youth-the QoLiSSY project focus group and cognitive debriefing experience. Value Health 13:63CrossRefGoogle Scholar
- 29.Sandberg DE, Colsman M (2005) Growth hormone treatment of short stature: status of the quality of life rationale. Horm Res 63:275–283Google Scholar
- 30.Bullinger M (2002) Assessing health related quality of life in medicine. An overview over concepts, methods and applications in international research. Restor Neurol Neurosci 20:93–101Google Scholar
- 32.Sheppard L, Eiser C, Davies HA, Carney S, Clarke SA, Urquhart T, Ryder MJ, Stoner A, Wright NP, Butler G (2006) The effects of growth hormone treatment on health-related quality of life in children. Horm Res 65:243–249Google Scholar
- 33.Stephen MD, Varni JW, Limbers CA, Yafi M, Heptulla RA, Renukuntla VS, Bell CS, Brosnan PG (2011) Health-related quality of life and cognitive functioning in pediatric short stature: comparison of growth-hormone-naive, growth-hormone-treated, and healthy samples. Eur J Pediatr 170:351–358CrossRefGoogle Scholar
- 35.Al-Uzri A, Matheson M, Gipson DS, Mendley SR, Hooper SR, Yadin O, Rozansky DJ, Moxey-Mims M, Furth SL, Warady BA, Gerson AC, Chronic Kidney Disease in Children Study G (2013) The impact of short stature on health-related quality of life in children with chronic kidney disease. J Pediatr 163(736–741):e731Google Scholar
- 42.The KIDSCREEN Group Europe (2006) The KIDSCREEN questionnaires: Quality of life for children and adolescents—Handbook. Pabst Science Publishers, LengrichGoogle Scholar
- 43.Cole TJ (1990) The LMS method for constructing normalized growth standards. Eur J Clin Nutr 44:45–60Google Scholar
- 45.Bullinger M, Quitmann J, Power M, Herdman M, Mimoun E, DeBusk K, Feigerlova E, Lunde C, Dellenmark-Blom M, Sanz D, Rohenkohl A, Pleil A, Wollmann H, Chaplin JE (2013) Assessing the quality of life of health-referred children and adolescents with short stature: development and psychometric testing of the QoLISSY instrument. Health Qual Life Outcomes 11:76CrossRefGoogle Scholar
- 47.Bullinger M, Sommer R, Pleil A, Mauras N, Ross J, Newfield R, Silverman L, Rohenkohl A, Fox J, Quitmann J (2015) Evaluation of the American-english quality of life in short stature youth (QoLISSY) questionnaire in the United States. Health Qual Life Outcomes. https://doi.org/10.1186/s12955-015-0236 Google Scholar
- 51.The European QoLISSY Group (2013) Quality of life in short stature youth. In: The QoLISSY questionnaire—User’s manual. Pabst Science Publishers, LengerichGoogle Scholar
- 52.IBM Corp (2012) IBM SPSS statistics for windows. 21.0 edn. IBM Corp., Armonk, NYGoogle Scholar
- 53.Mukaka M (2012) A guide to appropriate use of correlation coefficient in medical research. Malawi Med J 24:69–71Google Scholar
- 55.Thomas M, Beckers D, Brachet C, Dotremont H, Lebrethon MC, Lysy P, Massa G, Reynaert N, Rooman R, van der Straaten S, Roelants M, De Schepper J (2018) Adult height after growth hormone treatment at pubertal onset in short adolescents born small for gestational age: results from a belgian registry-based study. Int J Endocrinol. https://doi.org/10.1155/2018/6421243 Google Scholar
- 59.Cutfield WS, Lindberg A, Rapaport R, Wajnrajch MP, Saenger P (2006) Safety of growth hormone treatment in children born small for gestational age: the US trial and KIGS analysis. Horm Res 65(Suppl 3):153–159Google Scholar
- 62.Ranke MB, Lindberg A, Cowell CT, Wikland KA, Reiter EO, Wilton P, Price DA (2003) Prediction of response to growth hormone treatment in short children born small for gestational age: analysis of data from KIGS (Pharmacia International Growth Database). J Clin Endocrinol Metab 88:125–131CrossRefGoogle Scholar
- 64.Van Pareren Y, Mulder P, Houdijk M, Jansen M, Reeser M, Hokken-Koelega A (2003) Adult height after long-term, continuous growth hormone (GH) treatment in short children born small for gestational age: results of a randomized, double-blind, dose-response GH trial. J Clin Endocrinol Metab 88:3584–3590. https://doi.org/10.1210/jc.2002-021172 CrossRefGoogle Scholar