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First-year predictors of health-related quality of life changes in short-statured children treated with human growth hormone

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Abstract

Purpose

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.

Method

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

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.

Conclusion

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.

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Acknowledgements

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.

Funding

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.

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Authors and Affiliations

  1. Center for Psychosocial Medicine, Institute for Medical Psychology, University Medical Center Hamburg-Eppendorf, Martinistraße 52, 20246, Hamburg, Germany

    J. Quitmann, J. Bloemeke, M. Bullinger, S. Witt & N. Silva

  2. Division Paediatric Endocrinology, Hospital for Children and Adolescents, University Erlangen-Nürnberg, Erlangen, Germany

    H.-G. Dörr

  3. Faculty of Psychology and Education Sciences, Center for Research in Neuropsychology and Cognitive Behavioral Intervention, University of Coimbra, Coimbra, Portugal

    N. Silva

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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.

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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).

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Quitmann, J., Bloemeke, J., Dörr, HG. et al. First-year predictors of health-related quality of life changes in short-statured children treated with human growth hormone. J Endocrinol Invest 42, 1067–1076 (2019). https://doi.org/10.1007/s40618-019-01027-4

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