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

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Abstract

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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Acknowledgements

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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    1. Department of Child and Adolescent Psychiatry, Psychotherapy and Psychosomatics, University Medical Center Hamburg-Eppendorf, Martinistrasse 52, 20246, Hamburg, Germany

      Christiane Otto, Claus Barkmann & Ulrike Ravens-Sieberer

    2. The Danish National Rehabilitation Centre for Neuromuscular Diseases, Kongsvang Allé 23, 8000, Aarhus C, Denmark

      Birgit F. Steffensen, Ann-Lisbeth Højberg, Jes Rahbek & Annette Mahoney

    3. Department of Neuropediatrics and Muscle Disorders, University Medical Center Freiburg, Mathildenstraße 1, 79116, Freiburg, Germany

      Julia Vry, Kathrin Gramsch & Janbernd Kirschner

    4. The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, International Centre for Life, Central Parkway, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, NE1 3BZ, UK

      Rachel Thompson, Sunil Rodger, Kate Bushby & Hanns Lochmüller

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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 (www.treat-nmd.eu/about/governance/tgdoc/). 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, AL. 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). https://doi.org/10.1007/s00415-017-8406-2

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