Growth hormone treatment in children with short stature: impact of the diagnosis on parents

Stefanie Witt; Janika Bloemeke; Monika Bullinger; Helmuth-Günther Dörr; Neuza Silva; Julia Hannah Quitmann

doi:10.1515/jpem-2023-0420

Article

Growth hormone treatment in children with short stature: impact of the diagnosis on parents

Stefanie Witt , Janika Bloemeke , Monika Bullinger , Helmuth-Günther Dörr , Neuza Silva and Julia Hannah Quitmann

Published/Copyright: March 1, 2024

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From the journal Journal of Pediatric Endocrinology and Metabolism Volume 37 Issue 4

Abstract

Objectives

This prospective multicenter study aimed (1) to examine changes in parent-reported health-related quality of life (HRQOL) of children with short stature and the effects of the children’s condition on parents themselves within the first year of human growth hormone (hGH) treatment and (2) to predict effects on parents based on main and interaction effects of children’s HRQOL and increase in height.

Methods

A total of 110 parents of children aged 4–18 years, diagnosed with idiopathic growth hormone deficiency, small for gestational age, or idiopathic short stature, were recruited from 11 participating German pediatric endocrinologists and asked to fill out the short stature-specific Quality of Life in Short Stature Youth (QoLISSY) Questionnaire before hGH treatment was initiated and one year later.

Results

Negative effects of the children’s short stature on the parents decrease over time, independent of diagnosis and treatment status. Furthermore, treatment status and height increase moderated the links between children’s improved HRQOL as perceived by their parents and decreased caregiving burden.

Conclusions

Based on the children’s improved HRQOL and the parent’s decrease in caregiving burden, patient-reported outcomes that consider parental and child’s perspectives should be considered when deciding on hGH treatment for children.

Keywords: health-related quality of life; human growth hormone treatment; parental burden; short-statured children

Corresponding author: Dr. Stefanie Witt, Department of Medical Psychology, Center for Psychosocial Medicine, University Medical Center Hamburg-Eppendorf, Martinistraße 52, Hamburg 20251, Germany, E-mail: s.witt@uke.de

Neuza Silva and Julia Quitmann share last authorship.

Funding source: Pfizer

Acknowledgments

We thank all participating clinicians, namely Ilker Akkurt (Hamburg), Desiree Dunstheimer (Augsburg), Christian Vogel (Chemnitz), Volker Böttcher (Frankfurt/Main), Ursula Kuhnle Krahl (Gauting), Markus Bettendorf (Heidelberg), Eckhard Schönau (Cologne), Susanne Fricke-Otto (Krefeld), Alexandra Keller (Leipzig), Klaus Mohnike (Magdeburg), and Helmuth-Günther Dörr (Erlangen). In particular, we thank all participating families for sharing their experiences with us.

Research ethics: The study was approved by the appropriate Ethics Committees and was conducted following the ethical standard laid down in the 1964 Declaration of Helsinki and its later amendments.
Informed consent: Written informed consent was obtained from all participants and parents (where participants were under 16 years old) before the study. Patients signed informed consent regarding publishing their anonymized data.
Author contributions: Monika Bullinger, Helmuth-Günther Dörr, and Julia Quitmann contributed to the study conception and design. Stefanie Witt, Neuza Silva, Janika Bloemeke and Julia Quitmann performed material preparation, data collection, and analysis. Neuza Silva and Stefanie Witt wrote the manuscript’s first draft, and all authors commented on previous versions. All authors have read, approved, and accepted the responsibility for the entire content of this manuscript and its submission.
Competing interests: Monika Bullinger, Julia Quitmann, and the participating clinicians received research funding for conducting the study. The authors received no financial support for the preparation of the manuscript. 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 and comprehensive information of its development and validation process are published in the QoLISSY User’s Manual (The European QoLISSY Group 2011). The Manual is available upon request, including QoLISSY child and observer forms and scoring information (http://www.pfizerpatientreportedoutcomes.com/therapeutic-areas/cv-metabolic/endocrine).
Research funding: Pfizer, Inc. sponsored this study. The funding organization played no role in the study design, in the collection, analysis, and interpretation of data, in the writing of the report, or in the decision to submit the report for publication.
Data availability: De-identified data will be made readily available to qualified individuals within the scientific community upon request for research purposes.

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Received: 2023-09-22

Accepted: 2024-02-05

Published Online: 2024-03-01

Published in Print: 2024-04-25

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Articles in the same Issue

https://doi.org/10.1515/jpem-2023-0420

Keywords for this article

health-related quality of life; human growth hormone treatment; parental burden; short-statured children