Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods

ObjectiveBased on concerns over existing patient reported outcome measures (PROMs) for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the mixed methods development of a new QoL PROM for use in boys and men with DMD: the DMD-QoL.MethodsThe DMD-QoL was developed in 3 stages. First, draft items were generated from 18 semi-structured qualitative interviews with boys and men with DMD, analysed using framework analysis. Second, cognitive debriefing interviews with patients (n = 10), clinicians (n = 8), and patients' parents (n = 10) were undertaken and a reduced item set selected and refined. Third, psychometric data on the draft items from a cross-sectional online survey (n = 102), and stakeholder input from patients and patients' parents, was used to produce the final questionnaire. Patient and public involvement and engagement was embedded throughout the process.ResultsFrom an initial draft of 47 items, a revised set of 27 items was produced at stage 2, and this set was further refined at stage 3 to generate the DMD-QoL, a 14-item QoL PROM. The DMD-QoL is designed for use from 7 years of age by proxy report and from 10 years of age by self or proxy report. The final measure showed good psychometric properties.ConclusionsThe DMD-QoL is a new 14-item QoL PROM for boys and men with DMD, with demonstrable content and face validity.

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Producing a preference-based quality of life measure for people with Duchenne muscular dystrophy: a mixed-methods study protocol

BMJ Open ◽

10.1136/bmjopen-2018-023685 ◽

2019 ◽

Vol 9 (3) ◽

pp. e023685 ◽

Cited By ~ 1

Author(s):

Philip A Powell ◽

Jill Carlton ◽

Donna Rowen ◽

John E Brazier

Keyword(s):

Quality Of Life ◽

Mixed Methods ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Mixed Methods Study ◽

Health State ◽

Open Access Journals ◽

Life Years ◽

Descriptive System

IntroductionPreference-based measures (PBMs) of health-related quality of life (HRQoL) are used to generate quality-adjusted life years, which are necessary for cost-effectiveness evaluations of health interventions via cost–utility analysis. These measures of health can be generic (ie, pandiagnostic) or condition specific. No condition-specific PBM of HRQoL in Duchenne muscular dystrophy (DMD) exists, yet there are concerns that standard generic measures lack the specificity to assess aspects of HRQoL that are especially important to people with DMD. This study has been designed to produce a condition-specific PBM of HRQoL in DMD.Methods and analysisThis mixed-methods study proceeds through three stages. In the first stage (concept elicitation), semistructured interviews will be conducted with boys and men diagnosed with DMD, and analysed with framework to produce a draft health state descriptive system for HRQoL in DMD. In the second stage (refining the descriptive system), patients, clinicians and primary caregivers of people with DMD will assess the face validity of the descriptive system. This will be followed by a quantitative survey on a larger sample of patients, which will be analysed with psychometric analyses to produce a refined descriptive system. In the third stage (valuation and econometric modelling), an online discrete choice experiment with duration will be administered to a general public sample to generate utility values for the new measure.Ethics and disseminationThis study has received ethical approval from the National Health Service (REC reference: 18/SW/0055). The primary output of this research will be a condition-specific PBM (or ‘bolt-on’ to an existing generic PBM) in people with DMD and an associated value set. Results will be disseminated through international conferences and open-access journals.

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Health- related quality of life in patients with Duchenne Muscular Dystrophy

Neuropediatrics ◽

10.1055/s-0031-1273950 ◽

2011 ◽

Vol 42 (S 01) ◽

Cited By ~ 1

Author(s):

B Geers ◽

S Elsenbruch ◽

J Schmid ◽

U Schara

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Health Related Quality ◽

Related Quality ◽

Health Related

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Faculty Opinions recommendation of Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.8172.465661 ◽

2006 ◽

Author(s):

Jean-Paul Praud

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Physical Disability ◽

Respiratory Impairment

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Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

Value in Health ◽

10.1016/j.jval.2021.05.016 ◽

2021 ◽

Vol 24 (10) ◽

pp. 1490-1498

Author(s):

Erik Landfeldt ◽

Joel Iff ◽

Erik Henricson ◽

V. Vishwanathan ◽

S. Chidambaranathan ◽

...

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Rasch Analysis ◽

Quality Of Life Inventory ◽

Life Inventory

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A.01 The relationship between fatigue and health-related quality of life in a clinical trial population of Duchenne muscular dystrophy patients

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques ◽

10.1017/cjn.2017.65 ◽

2017 ◽

Vol 44 (S2) ◽

pp. S8-S8

Author(s):

Y Wei ◽

B El-Aloul ◽

C Nguyen ◽

E Zapata-Aldana ◽

C Campbell

Keyword(s):

Quality Of Life ◽

Clinical Trial ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Parent Report ◽

Health Related Quality ◽

Patient Report ◽

Related Quality ◽

Health Related

Background: Fatigue was recently reported to be the largest contributor to poor health-related quality of life (HRQOL) in paediatric Duchenne muscular dystrophy (DMD). Additional studies are necessary to confirm the generalizability of this finding. Our objective was to explore the longitudinal relationship between fatigue and HRQOL in an additional cohort of DMD patients. Methods: We performed a secondary analysis of data from a clinical trial (NCT00592553), which enrolled patients with nonsense mutation DMD, aged 5–20 years, from 37 sites in 11 countries (N=174). Fatigue and HRQOL were assessed using the PedsQLTM Multidimensional Fatigue Scale and Generic Core Scales, respectively, by patient- and parent-report at baseline and over 48 weeks. Results: Patients reported greater fatigue than healthy controls from published data. There was no significant difference between patient- and parent-reported fatigue. Fatigue was significantly correlated with worse HRQOL at baseline, by patient-report (r=0.70, P<0.001) and parent-report (r=0.70, P<0.001); and at 48 weeks, by patient-report (r=0.79, P<0.001) and parent-report (r=0.74, P<0.001). Change in fatigue was significantly correlated with change in HRQOL over 48 weeks, by patient-report (r=0.64, P<0.001) and parent-report (r=0.67, P<0.001). Conclusions: Fatigue is a major contributor to HRQOL in DMD. The strong association between fatigue and HRQOL corroborates previous studies, and suggests that reducing fatigue may improve HRQOL.

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P.072 Agreement between children and their parents’ ratings of the health-related quality of life of children with Duchenne Muscular Dystrophy

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques ◽

10.1017/cjn.2018.174 ◽

2018 ◽

Vol 45 (s2) ◽

pp. S35-S35

Author(s):

S Brar ◽

C Campbell ◽

E McColl ◽

W Martens ◽

M McDermott ◽

...

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Self Report ◽

Health Related Quality ◽

Related Quality ◽

Health Related ◽

Parent Proxy ◽

Level Of Agreement

Background: When measuring young Duchenne Muscular Dystrophy (DMD) patients’ health-related quality of life (HRQoL), parent-proxy reports are heavily relied on. Therefore, it is imperative that the relationship between parent-proxy and child self-report HRQoL is understood. This study examined the level of agreement between children and their parent-proxy rating of the child’s HRQoL. Methods: We used FOR-DMD clinical trial baseline data. HRQoL, measured using the PedsQL inventory, was reported by 178 parent and child (ages 4 to 7 years) dyads. Intracorrelation coefficients (ICC) measured absolute agreement while paired t-tests determined differences in the average HRQoL ratings between groups. Results: The level of agreement between child and parent-proxy ratings of HRQoL was poor for the generic PedsQL scale (ICC: 0.29) and its subscales; and, similarly low for the neuromuscular disease module (ICC:0.16). On average, parents rated their child’s HRQoL as poorer than the children rated themselves in all scales except for psychosocial and school functioning. Conclusions: Child and parent-proxy HRQoL ratings are discordant in this study sample, as occurs in other chronic pediatric diseases. This should be taken into account when interpreting clinical and research HRQoL findings in this population. Future studies should examine reasons for parents’ perception of poorer HRQoL than that reported by their children.

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