Abstract
Background
In addition to increasing linear growth, improvement in health-related quality of life (HRQOL) is an important endpoint in the treatment of short statured youth. Hence, condition-specific psychometric valid instruments that adequately assess HRQOL are needed. We aimed to confirmatorily examine the psychometric performance of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire used in a previously reported prospective randomized open-label trial.
Methods
This trial compared treatment of idiopathic short stature (ISS) in 76 adolescent males with either oral aromatase inhibitors (AIs), subcutaneous daily growth hormone (GH) or a combination treatment (AI/GH) for at least 2 years, demonstrating improvements in HRQOL with the GH and AI/GH interventions. HRQOL was assessed from the child’s and parent’s perspectives with the short stature-specific QoLISSY and the generic KIDSCREEN questionnaires before and 24 months into treatment. Scale scores and psychometric properties were examined regarding reliability and validity of the QoLISSY questionnaire using the dataset from the published trial.
Results
The QoLISSY questionnaire showed high internal consistency and satisfactory criterion, convergent and known-groups validity. Scale scores were evenly distributed with no major floor or ceiling effects. Responsiveness analyses suggest that the QoLISSY questionnaire detects significant changes in HRQOL after 2 years of treatment with growth-promoting therapies in children with short stature from both the child’s and parent’s perspectives.
Conclusions
The QoLISSY questionnaire is a psychometrically sound, reliable and valid instrument that can explore the experiences associated with short stature, track HRQOL changes over time and in response to treatment, and highlight HRQOL domains that can be improved through intervention.
Acknowledgments
We would like to thank all the families who participated in this study. Furthermore, we thank Ximena Gaete, Y. Miles Yu, Priscila Gagliardi and Ligeia Damaso who were involved in conducting the original study in the US and in Chile. In addition, we thank Rachel Sommer and Anja Rohenkohl for their support during the study. The authors are also grateful to AstraZeneca, Novartis, Pfizer and Genentech for providing study medications, to the Thrasher Research Fund for funding the study, and to Pfizer for permission to use the QoLISSY questionnaire, developed by the QoLISSY study group. QoLISSY is a joint initiative between Pfizer Limited and the University Medical Center Hamburg-Eppendorf. Copyright Pfizer Limited all rights reserved
Author contributions: All the authors have accepted responsibility for the entire content of this submitted manuscript and approved submission.
Research funding: None declared.
Employment or leadership: None declared.
Honorarium: None declared.
Competing interests: The principle investigator of the study, Dr. Nelly Mauras, received research support in drug supply agreements from Genentech, Pfizer, Novartis and AstraZeneca; and consulted for Opko. Dr. Judith L Ross receives research support from Versartis and Novo Nordisk and consults for Novo Nordisk. Prof. Monika Bullinger consulted and received research support from Pfizer, Otsuka Lundbeck and Janssen-Cielag. The principal study sponsors, the Thrasher Research Fund (N Mauras – principal investigator), nor the companies here listed who provided study drugs had any role in the study design, data collection, analysis, writing or submission of this manuscript. All other authors have nothing to declare.
References
1. Brütt AL, Sandberg DE, Chaplin J, Wollmann H, Noeker M, et al. 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 2009;72:65–73.10.1159/000232158Search in Google Scholar PubMed
2. Bullinger M. Assessing health related quality of life in medicine. An overview over concepts, methods and applications in international research. Horm Res Paediatr 2002;20:93–101.Search in Google Scholar
3. Rüther A, Wang-Rieger D, Elstein D, Guyatt G. Aspects of patient reported outcomes in rare diseases: a discussion paper. Int J Technol Assess Health Care 2016;32:126–30.10.1017/S0266462316000271Search in Google Scholar PubMed
4. Jafari-Adli S, Qorbani M, Heshmat R, Ranjbar SH, Taheri E, et al. Association of short stature with life satisfaction and self-rated health in children and adolescents: the CASPIAN-IV study. J Pediatr Endocrinol Metab 2016;29:1299–306.10.1515/jpem-2016-0215Search in Google Scholar PubMed
5. GHResearchSociety. Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: summary statement of the GH Research Society. GH Research Society. J Clin Endocrinol Metab 2000;85:3990–3.10.1210/jc.85.11.3990Search in Google Scholar PubMed
6. Drosatou C, Vlachopapadopoulou EA, Bullinger M, Quitmann J, Silva N, et al. Validation of the Greek version of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire. J Pediatr Endocrinol Metab 2019;32:215–24.10.1515/jpem-2018-0403Search in Google Scholar PubMed
7. Rohenkohl AC, Stalman S, Kamp G, Bullinger M, Quitmann J. Psychometric performance of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the Netherlands. Eur J Pediatr 2016;175:347–54.10.1007/s00431-015-2656-8Search in Google Scholar PubMed
8. Rohenkohl AC, De Schepper J, Vanderfaeillie J, Fricke K, Hendrickx S, et al. Validation of the Flemish version of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire. Acta Clin Belg 2014;69:177–82.10.1179/2295333714Y.0000000024Search in Google Scholar PubMed
9. Quitmann J, Giammarco A, Maghnie M, Napoli F, Di Giovanni I, et al. Validation of the Italian Quality of Life in Short Stature Youth (QoLISSY) questionnaire. J Endocrinol Invest 2017;40:1077–84.10.1007/s40618-017-0667-1Search in Google Scholar PubMed
10. Bullinger M, Sommer R, Pleil A, Mauras N, Ross J, et al. Evaluation of the American-English Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the United States. Health Qual Life Outcomes 2015;13:43.10.1186/s12955-015-0236-2Search in Google Scholar PubMed PubMed Central
11. Sommer R, Blömeke J, Bullinger M, Quitmann J. The psychometric evaluation of the quality of life in short stature youth (QoLISSY) instrument for German children born small for gestational age. J Endcrinol Invest 2018;41:1185–91.10.1007/s40618-018-0850-zSearch in Google Scholar PubMed
12. Bullinger M, Quitmann J, Power M, Herdman M, Mimoun E, et al. 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 2013;11:76.10.1186/1477-7525-11-76Search in Google Scholar PubMed PubMed Central
13. Rohenkohl AC, Bullinger M, Quitmann J. Quality of life in children, adolescents, and young adults with achondroplasia. Orthopade 2014;44:212–8.10.1007/s00132-014-3020-9Search in Google Scholar PubMed
14. European QoLISSY Group. Quality of Life in Short Stature Youth. The QoLISSY Questionnaire – User’s Manual. Lengerich: Pabst Science Publishers, 2013.Search in Google Scholar
15. Mauras N, Ross JL, Gagliardi P, Yu YM, Hossain J, et al. Randomized trial of aromatase inhibitors, growth hormone, or combination in pubertal boys with idiopathic, short stature. J Clin Endocinol Metab 2016;101:4984–93.10.1210/jc.2016-2891Search in Google Scholar PubMed PubMed Central
16. Bullinger M, Bloemeke J, Meriq V, Sommer R, Gaute X, et al. Quality of life in adolescent boys with idiopathic short stature: positive impact of Growth hormone and aromatase inhibitor. Horm Res Peadiatr 2018;90:381–92.10.1159/000496353Search in Google Scholar PubMed
17. Rohenkohl A, Bullinger M, Pleil A, Kriston L, Quitmann J, et al. A brief version of the Quality of Life in Short Stature Youth Questionnaire – The QoLISSY-Brief. Child Indic Res 2015;9:971.10.1007/s12187-015-9350-2Search in Google Scholar
18. Igl W, Zwingmann C, Faller H, Beutel M, Beyer W, et al. Änderungssensitivität von generischen Patientenfragebogen – Ergebnisse einer verbundübergreifenden Reanalyse. [Sensitivity to Change of Generic Patient Questionnaires – Results of a Reanalysis of Pooled Data]. Phys Med Rehab Kuror 2006;16:69–81.10.1055/s-2005-915386Search in Google Scholar
19. Irwing P, Booth T, Hughes DJ. The Wiley Handbook of Psychometric testing. A Multidisciplinary Reference on Survey, Scale and Test Development. Edition F, editor. Hoboken, New Jersey: Wiley Blackwell, 2018.10.1002/9781118489772Search in Google Scholar
20. The KIDSCREEN Group Europe. The KIDSCREEN questionnaires: quality of life for children and adolescents – Handbook. Lengerich, Germany: Pabst Science Publishers, 2006.Search in Google Scholar
21. Lim CR, Harris K, Dawson J, Beard DJ, Fitzpatrick R, et al. Floor and ceiling effects in the OHS: an analysis of the NHS PROMs data set. BMJ Open 2015;5:e007765.10.1136/bmjopen-2015-007765Search in Google Scholar PubMed PubMed Central
22. McHorney C. Health status assessment methods for adults: past accomplishments and future challenges. Annu Rev Public Health 1999;20:309–35.10.1146/annurev.publhealth.20.1.309Search in Google Scholar PubMed
23. Terwee CB, Bot SD, de Boer MR, van der Windt DA, Knol DL, et al. Quality criteria were proposed for measurement properties of health status questionnaires. J Clin Epidemiol 2007;60:34–42.10.1016/j.jclinepi.2006.03.012Search in Google Scholar PubMed
24. Field AP. Discovering Statistics Using SPSS. London: SAGE, 2009.Search in Google Scholar
25. Nunnally JC. Psychometric theory. 3 ed. New York, NY: McGraw-Hill, 1994.Search in Google Scholar
26. Mukaka M. A guide to appropriate use of Correlation coefficient in medical research. Malawi Med J 2012;24:69–71.Search in Google Scholar PubMed
27. Cohen J. Statistical power analysis for the behavioral sciences. Erlbaum L, editor. Hillsdale, MI, USA: Lawrence Erlbaum Associates, Inc, 1988.Search in Google Scholar
28. Bullinger-Naber M, Quitmann J, Silva N, Rohenkohl A, Chaplin J, et al. Cross-cultural equivalence of the patient- and parent-reported Quality of Life in Short Stature Youth Questionnaire (QoLISSY) questionnaire. Horm Res Paediatr 2014;82:18–30.10.1159/000358832Search in Google Scholar PubMed
29. Folkman S, Moskowitz JT. Coping: pitfalls and promise. Annu Rev Psychol 2004;55:745–74.10.1146/annurev.psych.55.090902.141456Search in Google Scholar PubMed
30. Hartmann DP, Roper BL, Bradford DC. Some relationships between behavioral and traditional assessment. J Behav Assess 1979;1:3–21.10.1007/BF01322415Search in Google Scholar
31. Lem A, Jobse I, van Der Kaay D, de Ridder M, Raat H, et al. Health-related quality of life in short children born small for gestational age: effects of growth hormone treatment and postponement of puberty. Horm Res Paediatr 2012;77: 170–9.10.1159/000337218Search in Google Scholar PubMed
©2019 Walter de Gruyter GmbH, Berlin/Boston
