Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

The Muscle Study Group, and TREAT-NMD

Research output: Contribution to journalArticlepeer-review

Abstract

Knowledge of health related quality of life (HRQOL) in the immediate phase following DMD diagnosis has not been well-characterized. It is important to understand HRQOL early in disease for both clinical care and studies of treatment. The relationship between parent-proxy and child self-report HRQOL and their associations with medical, psycho-social and behavioral symptoms deserve study. In this study HRQOL was measured using the PedsQL inventory in parent/caregiver and corticosteroid-naïve boys (ages 4 to 7 years) participating in the FOR-DMD study. Agreement between the parent-proxy report and the boys’ self-report HRQOL was measured using intraclass correlation coefficients (ICCs). Factors associated with HRQOL, including standardized psychosocial and behavioral measures in this cross-sectional sample, were explored using correlations. The results showed that the level of agreement between 70 dyads of child self-report and parent-proxy ratings of HRQOL was poor for the generic PedsQL total score (ICC=0.48, 95% CI (0.23, 0.66)) and its subscale scores, and was similarly low for the neuromuscular disease module (ICC=0.24, 95% CI (0.00, 0.45)). Parents rated their child's HRQOL as poorer than the children rated themselves in all scales. Psychosocial outcome measures were more highly associated with HRQOL measures than disease severity or patient demographic variables. In the early phases of DMD, child and parent-proxy HRQOL ratings were discordant. In early DMD, psychosocial and behavioral aspects appear to be more relevant to HRQOL than disease severity factors.

Original languageEnglish (US)
JournalNeuromuscular Disorders
DOIs
StateAccepted/In press - 2021
Externally publishedYes

Bibliographical note

Funding Information:
Research reported in this publication was supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Numbers U01NS061799 and U01NS061795 . The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Support for this research was also provided by Sarepta Therapeutics, PTC Therapeutics, Muscular Dystrophy Association (MDA), Parent Project Muscular Dystrophy (PPMD) and Telethon Italy. We acknowledge the patient and family organizations including Action Duchenne, Muscular Dystrophy UK, and Muscular Dystrophy Canada for their promotion of the study. The FOR-DMD Steering Committee and the study Site Investigators (listed at bottom) are members of the Muscle Study Group. Michela Guglieri is part of the Medical Research Council (UK) and TREAT-NMD. Finally, we are grateful to the boys participating in the study, as well as their families, all site investigators, research nurses, physiotherapists and study coordinators for their commitment and contribution to the study.

Funding Information:
Research reported in this publication was supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Numbers U01NS061799 and U01NS061795. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Support for this research was also provided by Sarepta Therapeutics, PTC Therapeutics, Muscular Dystrophy Association (MDA), Parent Project Muscular Dystrophy (PPMD) and Telethon Italy. We acknowledge the patient and family organizations including Action Duchenne, Muscular Dystrophy UK, and Muscular Dystrophy Canada for their promotion of the study. The FOR-DMD Steering Committee and the study Site Investigators (listed at bottom) are members of the Muscle Study Group. Michela Guglieri is part of the Medical Research Council (UK) and TREAT-NMD. Finally, we are grateful to the boys participating in the study, as well as their families, all site investigators, research nurses, physiotherapists and study coordinators for their commitment and contribution to the study. Volker Straub MD, PhD, John Walton Muscular Dystrophy Research Center, Newcastle University, UK. Anne-Marie Childs MRCP FRCPCH, Leeds Teaching Hospitals, UK. Emma Ciafaloni MD, University of Rochester Medical Center. Perry B. Shieh MD, PhD, UCLA. Stefan Spinty MRCPCH MMedSc, Alder Hey Children's Hospital, Liverpool, UK. Russell J. Butterfield MD, PhD, University of Utah. Iain Horrocks MRCPCH, Greater Glasgow and Clyde NHS Yorkhill Hospital. Helen Roper, MD, FRCPCH, University Hospitals Birmingham NHS Foundation Trust, UK. Lorenzo Maggi MD, Neurological Institute "Carlo Besta" Milan, Italy. Giovanni Baranello, MD, PhD, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy. Kevin M. Flanigan MD, Nationwide Children's Hospital, Columbus. Nancy L. Kuntz MD, Ann & Robert H. Lurie Children's Hospital, Chicago. Adnan Y. Manzur FRCPCH, Great Ormond Street Hospital, London. Basil T. Darras MD, Boston Children's Hospital. Peter Kang, MD, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA. Jean K. Mah MD MSc, University of Calgary, Canada. Tiziana Mongini MD, University of Turin, Italy. Federica Ricci, MD, Neuromuscular Center, AOU Citt? della Salute e della Scienza, University of Turin, Italy. Leslie Morrison MD, University of New Mexico. Monika Krzesniak-Swinarska, University of New Mexico. Maja von der Hagen MD, Children's Hospital, Technical University Dresden, Germany. Richard S. Finkel MD, Nemours Children's Hospital, Orlando; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee. Ashutosh Kumar MD, Milton S. Hershey Medical Center. Matthew Wicklund MD, Milton S. Hershey Medical Center; Department of Neurology, University of Colorado School of Medicine, Aurora, Colorado. Craig M. McDonald MD, UC Davis Medical Center, Sacramento. Erik K. Henricson, PhD, MPH, UC Davis Medical Center, Sacramento. Ulrike Schara-Schmidt, MD, PhD, University of Essen, Germany. Ekkehard Wilichowski MD, Children's University Hospital, G?ttingen, Germany. Richard J. Barohn MD, University of Missouri, Columbia, Missouri. Jeffrey Statland MD, University of Kansas Medical Center. Janbernd Kirschner MD, University Medical Center, Freiburg, Germany; Department of Neuropediatrics, University Hospital Bonn, Germany. Giuseppe Vita MD, University of Messina AOU Policlinico Gaetano Martino, Italy. Gian Luca Vita, MD, PhD, University of Messina AOU Policlinico Gaetano Martino, Italy. James F. Howard Jr. MD, University of North Carolina Medical Center at Chapel Hill. Imelda Hughes MB FRCPCH, Royal Manchester Children's Hospital, UK. Hugh J. McMillan MD MSc, Children's Hospital of Eastern Ontario, Ottawa, Canada. Elena Pegoraro MD PhD, University of Padua, Italy. Luca Bello, MD, PhD, ERN Neuromuscular Unit, Department of Neuroscience, University of Padua, Italy. W. Bryan Burnette MD, Vanderbilt Children's Hospital, Nashville. Mathula Thangarajh MD PhD, Department of Neurology, Virginia Commonwealth University, Richmond, Virginia. Taeun Chang, MD, Children's National Medical Center, Washington, DC.

Publisher Copyright:
© 2021 Elsevier B.V.

Keywords

  • Duchenne muscular dystrophy
  • Health related quality of life
  • Psychosocial

PubMed: MeSH publication types

  • Journal Article

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