Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries
- 819 Downloads
Duchenne muscular dystrophy (DMD) is a progressive, genetically determined neuromuscular disease that affects males and leads to severe physical disability in early teenage years. Over the last decades, patient-reported outcomes such as Health-Related Quality of Life (HRQoL) gained great interest in clinical research. However, little is known about factors affecting HRQoL in boys with DMD. Data from the multi-center CARE-NMD project of boys with DMD from six European countries collected between 2011 and 2012 were analyzed (8–17 years old; n = 321). HRQoL was measured using the KIDSCREEN-10 index, the Pediatric Quality of Life Inventory (PedsQL) and the Neuromuscular Module of the PedsQL (NMM). Linear regression models served to examine influences of socio-demographic, disease- and treatment-specific as well as participation- and environment-related factors on overall and disease-specific HRQoL. Proportions of explained variance varied across models using different outcomes (18–34%). Overall HRQoL according to the KIDSCREEN-10 index was associated with household income, the frequency of attending a clinic with specialized staff, the number of days spent outside home, and the attitude of the local community, but no significant association with age occurred. Overall HRQoL according to the generic PedsQL and disease-specific HRQoL were both positively associated with age and influenced by the country of residence, the disease stage, number of days spent outside home, and the attitude of the local community. Our results may be relevant for clinical practice and planning interventions for this population, but should be confirmed by future research. Further questions for future studies on boys with DMD are proposed.
KeywordsHealth-Related Quality of Life Psychosocial health Duchenne muscular dystrophy Children and adolescents Europe Linear regression
The CARE-NMD project was funded by the EU Executive Agency for Health and Consumers (20091205). This publication was funded by The Danish National Rehabilitation Centre for Neuromuscular Diseases. We gratefully acknowledge the work of further members of the CARE-NMD study group who contributed significantly to the project: Velina Guergueltcheva, Teodora Chamova, Ivailo Tournev, Anna Kostera-Pruszczyk, Anna Kaminska, Anna Lusakowska, Lenka Mrazova, Lenka Pavlovska, Petr Vondraček, Marta Garami, Veronika Karcagi, Agnes Herczegfalvi, and Adrian Tassoni. Further, the authors would like to thank Dr. Levente Kriston, Institute of Medical Psychology, University Medical Center Hamburg-Eppendorf, Germany, for statistical advice concerning this manuscript. Most of all, we are very grateful to all the participants, both boys with DMD and their families who took their time to participate in the CARE-NMD project.
Compliance with ethical standards
Conflicts of interest
The authors declare that they have no conflict of interest.
The CARE-NMD project was approved by the ethics committee of the University Medical Center Freiburg, Germany, and the TREAT-NMD Global Database Oversight Committee (www.treat-nmd.eu/about/governance/tgdoc/). All persons gave their informed consent prior to their inclusion in the study.
- 5.World Health Organization (2001) International Classification of Functioning, Disability and Health: ICF. World Health Organization, GenevaGoogle Scholar
- 6.U.S. Department of Health and Human Services Food and Drug Administration (2009) Guidance for Industry: Patient-reported outcome measures: Use in medical product development to support labelling claims. FDA, Clinical/Medical http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed 12 Jan 2017
- 8.Centers for Disease Control and Prevention (2000) Measuring Healthy Days. CDC, Atlanta, Georgia. http://www.cdc.gov/hrqol/pdfs/mhd.pdf. Accessed 12 Jan 2017
- 9.International Society for Quality of Life Research (2016) What is Health-Related Quality of Life Research? ISOQOL. http://www.isoqol.org/about-isoqol/what-is-health-related-quality-of-life-research. Accessed 10 Jan 2017
- 13.Davis SE, Hynan LS, Limbers CA, Andersen CM et al (2010) The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the pediatric quality of life inventory neuromuscular module and generic core scales. J Clin Neuromuscul Dis 11:97–109. doi: 10.1097/CND.0b013e3181c5053b CrossRefPubMedGoogle Scholar
- 14.Houwen van Opstal SLS, Jansen M, van Alfen N, de Groot IJM (2014) Health related quality of life and its relation to disease severity in boys with Duchenne muscular dystrophy: satisfied boys, worrying parents—a case control study. J Child Neurol 29:1486–1495. doi: 10.1177/0883073813506490 CrossRefPubMedGoogle Scholar
- 16.Bendixen RM, Senesac C, Lott DJ, Vandenborne K (2012) Participation and quality of life in children with Duchenne muscular dystrophy using the International Classification of functioning, Disability, and Health. Health Qual Life Outcomes 10:43. doi: 10.1186/1477-7525-10-43 CrossRefPubMedPubMedCentralGoogle Scholar
- 18.Henricson E, Abresch R, Han JJ, Nicorici A et al (2013) The 6-minute walk test and person-reported outcomes in boys with duchenne muscular dystrophy and typically developing controls: longitudinal comparisons and clinically-meaningful changes over one year. PLOS Curr Muscular Dystrophy. doi: 10.1371/currents.md.9e17658b007eb79fcd6f723089f79e06 Google Scholar
- 24.Ravens-Sieberer U, The KIDSCREEN Group Europe (2006) The KIDSCREEN Questionnaires. Quality of Life questionnaires for children and adolescents—Handbook. Papst Science Publisher, LengerichGoogle Scholar
- 27.eurostat (2011) Average and median household income in European countries. http://appsso.eurostat.ec.europa.eu/nui/show.do. Accessed 16 Feb 2011 and 20 March 2016
- 29.Ravens-Sieberer U, Erhart M, Rajmil L, Herdman M et al (2010) Reliability, construct and criterion validity of the KIDSCREEN-10 score: a short measure for children and adolescents’ well-being and Health-Related Quality of Life. Qual Life Res 19:1487–1500. doi: 10.1007/s11136-010-9706-5 CrossRefPubMedPubMedCentralGoogle Scholar
- 31.Varni JW (2016) The PedsQL™—Measurement Model for the Pediatric Quality of Life Inventory™—PedsQL™ translations. http://www.pedsql.org/translations.html. Accessed 10 Jan 2017
- 35.Berkes A, Pataki I, Kiss M, Kemény C et al (2010) Measuring Health-Related Quality of Life in Hungarian children with heart disease: psychometric properties of the Hungarian version of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales and the Cardiac Module. Health Qual Life Outcomes 28(8):14. doi: 10.1186/1477-7525-8-14 CrossRefGoogle Scholar
- 37.Varni JW (2014) Scaling and scoring of the Pediatric Quality of Life Inventory PedsQL™ (version 14, updated March 2014) Mapi Research Trust, Lyon, France, and TAMU College station, Texas. http://www.pedsql.org/PedsQL-Scoring.pdf. Accessed 10 Jan 2017
- 38.Kline R (2000) Reliability of tests: practical issues. The handbook of psychological testing, 2nd edn. Routledge, London, pp 7–16Google Scholar
- 39.de Vaus D (2002) How to assess reliability. Analysing social science data. Sage Ltd, London, pp 17–21Google Scholar
- 40.Heck RH, Thomas SL, Tabata LN (2014) Defining a basic two-level multilevel regression model. In: Marcoulides GA (ed) Multilevel and longitudinal modeling with IBM SPSS, 2nd edn. Routledge, New York, pp 75–130Google Scholar
- 41.Field A (2015) Regression. Discovering statistics using IBM SPSS Statistics, 4th edn. Sage Ltd, London, pp 293–356Google Scholar
- 43.Cohen J (1988) Multiple regression and correlation analyses. Statistical power analysis for the behavioral sciences, 2nd edn. Erlbaum, Hillsdale, pp 407–466Google Scholar
- 44.European Medicines Agency (2015) Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. EMA/CHMP/732154/20131, Committee for Medicinal Products for Human Use (CHMP), London, UK. Accessed 16 Jan 2017Google Scholar
- 55.UNICEF Office of Research (2013) Child Well-being in Rich Countries: A comparative overview, Innocenti Report Card 11. UNICEF Office of Research, Florence. http://www.unicef-irc.org/publications/pdf/rc11_eng.pdf. Accessed 12 Dec 2016
- 56.TREAT-NMD neuromuscular network (2007) Family Guide. Newcastle University. http://www.treat-nmd.eu/care/dmd/family-guide/. Accessed 12 Jan 2017