Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries


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.

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  1. 1.

    Mendell JR, Leslie ND, Flanigan KM, al-Dahhak R et al (2012) Evidence-based path to newborn. Ann Neurol 71:304–313. doi:10.1002/ana.23528

    CAS  Article  PubMed  Google Scholar 

  2. 2.

    Eagle M, Bourke J, Bullock R, Gibson M et al (2007) Managing Duchenne muscular dystrophy–the additive effect of spinal surgery and home nocturnal ventilation in improving survival. Neuromuscul Disord 17:470–475. doi:10.1016/j.nmd.2007.03.002

    Article  PubMed  Google Scholar 

  3. 3.

    Goemans N, Buyse G (2014) Current treatment and management of dystrophinopathies. Curr Treat Options Neurol 16:287. doi:10.1007/s11940-014-0287-4

    Article  PubMed  Google Scholar 

  4. 4.

    Bushby K, Finkel R, Birnkrant DJ, Case LE et al (2010) Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 9:177–189. doi:10.1016/S1474-4422(09)70272-8

    CAS  Article  PubMed  Google Scholar 

  5. 5.

    World Health Organization (2001) International Classification of Functioning, Disability and Health: ICF. World Health Organization, Geneva

    Google Scholar 

  6. 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 Accessed 12 Jan 2017

  7. 7.

    Deshpande PR, Rajan S, Sudeepthi BL, Abdul Nazir CP (2011) Patient-reported outcomes: a new era in clinical research. Perspect Clin Res 2(4):137–144. doi:10.4103/2229-3485.86879

    Article  PubMed  PubMed Central  Google Scholar 

  8. 8.

    Centers for Disease Control and Prevention (2000) Measuring Healthy Days. CDC, Atlanta, Georgia. Accessed 12 Jan 2017

  9. 9.

    International Society for Quality of Life Research (2016) What is Health-Related Quality of Life Research? ISOQOL. Accessed 10 Jan 2017

  10. 10.

    Ravens-Sieberer U, Erhart M, Wille N, Wetzel R et al (2006) Generic health-related quality-of-life assessment in children and adolescents. Methodological considerations. Pharmacoeconomics 24:1199–1220. doi:10.2165/00019053-200624120-00005

    Article  PubMed  Google Scholar 

  11. 11.

    Dey M, Mohler-Kuo M, Landolt MA (2012) Health-Related Quality of Life among children with mental health problems: a population-based approach. Health Qual Life Outcomes 10:73. doi:10.1186/1477-7525-10-73

    Article  PubMed  PubMed Central  Google Scholar 

  12. 12.

    Solans M, Pane S, Estrada M-D, Serra-Sutton V et al (2008) Health-Related Quality of Life measurement in children and adolescents: a systematic review of generic and disease-specific instruments. Value Health 11:742–764. doi:10.1111/j.1524-4733.2007.00293.x

    Article  PubMed  Google Scholar 

  13. 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

    Article  PubMed  Google Scholar 

  14. 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

    CAS  Article  PubMed  Google Scholar 

  15. 15.

    Uzark K, King E, Cripe L, Spicer R et al (2012) Health-Related Quality of Life in children and adolescents with Duchenne muscular dystrophy. Pediatrics 130:e1559–e1566. doi:10.1542/peds.2012-0858

    Article  PubMed  Google Scholar 

  16. 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

    Article  PubMed  PubMed Central  Google Scholar 

  17. 17.

    Baiardini I, Minetti C, Bonifacino S, Porcu A et al (2011) Quality of Life in Duchenne muscular dystrophy: the subjective impact on children and parents. J Child Neurol 26:707. doi:10.1177/0883073810389043

    Article  PubMed  Google Scholar 

  18. 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/

    Google Scholar 

  19. 19.

    Bray P, Bundy AC, Ryan MM, North KN, Everett A (2010) Health related quality of life in boys with Duchenne muscular dystrophy: agreement between parents and their sons. J Child Neurol 25:1188–1194. doi:10.1177/0883073809357624

    Article  PubMed  Google Scholar 

  20. 20.

    Wei Y, Speechley KN, Zou G, Campbell C (2016) Factors associated with Health-Related Quality of Life in children with Duchenne muscular dystrophy. J Child Neurol 31(7):879–886. doi:10.1177/0883073815627879

    Article  PubMed  Google Scholar 

  21. 21.

    Vry J, Gramsch K, Rodger S, Thompson R et al (2016) European cross-sectional survey of current practices for Duchenne muscular dystrophy reveals regional and age-dependent differences. J Neuromuscul Dis 3(4):517–527. doi:10.3233/JND-160185

    Article  PubMed  PubMed Central  Google Scholar 

  22. 22.

    Rodger S, Woods KL, Bladen CL, Stringer A et al (2015) Adult care for Duchenne muscular dystrophy in the UK. J Neurol 262:629–641. doi:10.1007/s00415-014-7585-3

    Article  PubMed  Google Scholar 

  23. 23.

    Bladen CL, Rafferty K, Straub V, Monges S et al (2013) The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia. Hum Mutat 34:1449–1457. doi:10.1002/humu.22390

    Article  PubMed  Google Scholar 

  24. 24.

    Ravens-Sieberer U, The KIDSCREEN Group Europe (2006) The KIDSCREEN Questionnaires. Quality of Life questionnaires for children and adolescents—Handbook. Papst Science Publisher, Lengerich

  25. 25.

    Ravens-Sieberer U, Herdman M, Devine J, Otto C et al (2014) The European KIDSCREEN approach to measure quality of life and well-being in children: development, current application, and future advances. Qual Life Res 23:791–803. doi:10.1007/s11136-013-0428-3

    Article  PubMed  Google Scholar 

  26. 26.

    Varni JW, Seid M, Kurtin PS (2001) PedsQL 4.0 TM: reliability and validity of the Pediatric Quality of Life InventoryTM version 4.0 Generic Core Scales in healthy and patient populations. Med Care 39:800–812

    CAS  Article  PubMed  Google Scholar 

  27. 27.

    eurostat (2011) Average and median household income in European countries. Accessed 16 Feb 2011 and 20 March 2016

  28. 28.

    Dickinson HO, Parkinson KN, Ravens-Sieberer U, Schirripa G et al (2007) Self-reported quality of life of 8–12-year-old children with cerebral palsy: a cross-sectional European study. The Lancet 369(9580):2171–2178. doi:10.1016/S0140-6736(07)61013-7

    Article  Google Scholar 

  29. 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

    Article  PubMed  PubMed Central  Google Scholar 

  30. 30.

    Varni JW, Burwinkle TM, Seid M (2006) The PedsQLTM 4.0 as a school population health measure: feasibility, reliability, and validity. Qual Life Res 15:203–215. doi:10.1077/s11136-005-1388-z

    Article  PubMed  Google Scholar 

  31. 31.

    Varni JW (2016) The PedsQL™—Measurement Model for the Pediatric Quality of Life Inventory™—PedsQL™ translations. Accessed 10 Jan 2017

  32. 32.

    Stevanovic D, Atilola O, Balhara YPS, Avicenna M et al (2015) The Relationships between alcohol/drug use and quality of life among adolescents: an International, Cross-Sectional Study. J Child Adolesc Subst Abuse 24(4):177–185. doi:10.1080/1067828X.2013.773864

    Article  Google Scholar 

  33. 33.

    Idorn L, Jensen AS, Juul K, Overgaard D et al (2013) Quality of life and cognitive function in Fontan patients, a population-based study. Int J Cardiol 168(4):3230–3235. doi:10.1016/j.ijcard.2013.04.008

    Article  PubMed  Google Scholar 

  34. 34.

    Felder-Puig R, Frey E, Proksch K, Varni JW et al (2004) Validation of the German version of the Pediatric Quality of Life Inventory (PedsQL) in childhood cancer patients off treatment and children with epilepsy. Qual Life Res 13(1):223–234

    CAS  Article  PubMed  Google Scholar 

  35. 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

    Article  Google Scholar 

  36. 36.

    Buresova G, Veleminsky M, Veleminsky M (2008) Health related quality of life of children and adolescents with type 1 diabetes. Neuro Endocrinol Lett 29(6):1045–1053

    PubMed  Google Scholar 

  37. 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. Accessed 10 Jan 2017

  38. 38.

    Kline R (2000) Reliability of tests: practical issues. The handbook of psychological testing, 2nd edn. Routledge, London, pp 7–16

    Google Scholar 

  39. 39.

    de Vaus D (2002) How to assess reliability. Analysing social science data. Sage Ltd, London, pp 17–21

    Google Scholar 

  40. 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 75–130

    Google Scholar 

  41. 41.

    Field A (2015) Regression. Discovering statistics using IBM SPSS Statistics, 4th edn. Sage Ltd, London, pp 293–356

    Google Scholar 

  42. 42.

    Erhart M, Ottova V, Gaspar T, Jericek H et al (2009) Measuring mental health and well-being of school-children in 15 European countries using the KIDSCREEN-10 index. Int J Public Health 54:S160–S166. doi:10.1007/s00038-009-5407-7

    Article  Google Scholar 

  43. 43.

    Cohen J (1988) Multiple regression and correlation analyses. Statistical power analysis for the behavioral sciences, 2nd edn. Erlbaum, Hillsdale, pp 407–466

    Google Scholar 

  44. 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 2017

  45. 45.

    Fayed N, de Camargo OK, Kerr E, Rosenbaum P et al (2012) Generic patient-reported outcomes in child health research: a review of conceptual content using World Health Organization definitions. Dev Med Child Neurol 54:1085–1095. doi:10.1111/j.1469-8749.2012.04393.x

    Article  PubMed  Google Scholar 

  46. 46.

    Jelenova D, Prasko J, Ociskova M, Latalova K et al (2016) Quality of life and parental styles assessed by adolescents suffering from inflammatory bowel diseases and their parents. Neuropsychiatr Dis Treat 23(12):665–672. doi:10.2147/NDT.S104260

    Google Scholar 

  47. 47.

    Rueden U, Gosch A, Rajmil L, Bisegger C et al (2006) Socioeconomic determinants of health related quality of life in childhood and adolescence: results from a European study. J Epidemiol Community Health 60:130–135. doi:10.1136/jech.2005.039792

    Article  Google Scholar 

  48. 48.

    Sentenac M, Gavin A, Gabhainn SN, Molcho M et al (2013) Peer victimization and subjective health among students reporting disability or chronic illness in 11 western countries. Eur J of public health 23:421–426. doi:10.1093/eurpub/cks073

    Article  Google Scholar 

  49. 49.

    Michel G, Bisegger C, Fuhr DC, Abel T, KIDSCREEN group (2009) Age and gender differences in Health-Related Quality of Life of children and adolescents in Europe: a multilevel analysis. Qual Life Res 18:1147–1157. doi:10.1007/s11136-009-9538-3

    Article  PubMed  Google Scholar 

  50. 50.

    Landfeldt E, Lindgren P, Bell CF, Schmitt C et al (2015) Compliance to Care Guidelines for Duchenne Muscular Dystrophy. J Neuromuscul Dis 2:63–72. doi:10.3233/JND-140053

    PubMed  PubMed Central  Google Scholar 

  51. 51.

    Landfeldt E, Lindgren P, Bell CF, Guglieri M et al (2015) Health related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study. Dev Med Child Neurol. doi:10.1111/dmcn.12938

    PubMed  PubMed Central  Google Scholar 

  52. 52.

    Eiser C, Morse R (2001) A review of measures of quality of life for children with chronic illness. Arch Dis Child 84:205–211. doi:10.1136/adc.84.3.205

    CAS  Article  PubMed  PubMed Central  Google Scholar 

  53. 53.

    Rahbek J, Werge B, Madsen A, Marquardt J et al (2005) Adult life with Duchenne muscular dystrophy: observations among an emerging and unforeseen patient population. Pediatr Rehabil 8:17–28. doi:10.1080/10.1080/13638490400010191

    Article  PubMed  Google Scholar 

  54. 54.

    Colver AF, Dickinson HO, Parkinson K, Arnaud C et al (2011) Access of children with cerebral palsy to the physical, social and attitudinal environment they need: a cross-sectional European study. Disabil Rehabil 33:28–35. doi:10.3109/09638288.2010.485669

    Article  PubMed  Google Scholar 

  55. 55.

    UNICEF Office of Research (2013) Child Well-being in Rich Countries: A comparative overview, Innocenti Report Card 11. UNICEF Office of Research, Florence. Accessed 12 Dec 2016

  56. 56.

    TREAT-NMD neuromuscular network (2007) Family Guide. Newcastle University. Accessed 12 Jan 2017

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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.

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Correspondence to Birgit F. Steffensen.

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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 ( All persons gave their informed consent prior to their inclusion in the study.

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B. F. Steffensen and C. Otto contributed equally to this manuscript.

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Otto, C., Steffensen, B.F., Højberg, A. et al. Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries. J Neurol 264, 709–723 (2017).

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  • Health-Related Quality of Life
  • Psychosocial health
  • Duchenne muscular dystrophy
  • Children and adolescents
  • Europe
  • Linear regression