PMS84 PSYCHOMETRIC ANALYSIS OF THE PEDIATRIC QUALITY OF LIFE GENERIC CORE SCALE AND NEUROMUSCULAR MODULE IN DUCHENNE MUSCULAR DYSTROPHY: IMPLICATIONS FOR DETERMINING PATIENT-PERCEIVED TREATMENT BENEFIT

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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Quality of Life in Duchenne Muscular Dystrophy: The Subjective Impact on Children and Parents

Journal of Child Neurology ◽

10.1177/0883073810389043 ◽

2011 ◽

Vol 26 (6) ◽

pp. 707-713 ◽

Cited By ~ 36

Author(s):

Ilaria Baiardini ◽

Carlo Minetti ◽

Simona Bonifacino ◽

Anna Porcu ◽

Catherine Klersy ◽

...

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Children And Parents

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P294 – 1740 A prospective natural history study of the progression of physical impairment, activity limitation and quality of life in Duchenne muscular dystrophy

European Journal of Paediatric Neurology ◽

10.1016/s1090-3798(13)70473-2 ◽

2013 ◽

Vol 17 ◽

pp. S134

Author(s):

N Goemans ◽

E Mercuri ◽

A Morgan ◽

M Eagle ◽

F Lawrence ◽

...

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Natural History ◽

Activity Limitation ◽

Physical Impairment ◽

Natural History Study

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P.7.4 Quality of life of adult patients with Duchenne muscular dystrophy

Neuromuscular Disorders ◽

10.1016/j.nmd.2013.06.485 ◽

2013 ◽

Vol 23 (9-10) ◽

pp. 774 ◽

Cited By ~ 1

Author(s):

M. Kobayashi ◽

K. Obara ◽

E. Abe ◽

C. Wada ◽

I. Toyoshima

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Adult Patients

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Quality Of Life And Coping In Parents Raising Children With Duchenne Muscular Dystrophy (DMD) In Nepal

Journal of Psychiatrists Association of Nepal ◽

10.3126/jpan.v9i2.36282 ◽

2020 ◽

Vol 9 (2) ◽

pp. 30-41

Author(s):

M Rana ◽

R Prajapati ◽

J Chaudhari ◽

B Gautam ◽

B Gurung

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Coping Strategies ◽

Psychological Health ◽

World Health ◽

Life Questionnaire ◽

Raising Children ◽

And Coping

Introduction: Quality of Life (QoL) and Coping are important components of mental health, especially important to parents raising children with Duchenne Muscular Dystrophy (DMD). Children with DMD face difficulties due to their disability and create special challenges for parents. Material And Method: The study aimed to assess QoL, demographic variables and coping strategies of parents raising children with DMD in Nepal. A descriptive cross-sectional design was adopted to collect data from 32 parents (mothers = 15, 47%; fathers =17, 53%) through purposive sampling. Socio-demographic pro-forma, World Health Organization's Quality of Life Questionnaire-BREF (WHOQOL-BREF) and Brief COPE Inventory were assessed. Results: The results revealed parents scores were below cutoff <60, indicating probable poor quality of life. QOL was moderately positively correlated with coping strategies and highly positively correlated with emotion-focused coping. There were significant differences in psychological health of parents in reference to their occupation, and in reference to their hospital born children. Parents in government job had better psychological health and parents into private business significantly used emotion-focused coping. Mutual caregivers had significantly better psychological health. Parents without health related problems significantly adopted problem focused coping and emotion focused coping. Conclusion: These findings will help stakeholders to understand DMD children and their impact on QOL and coping of parents and will facilitate to develop appropriate psychosocial intervention programs for the parents for the effective management of their children and their psycho-social wellbeing.

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