Health-related quality of life in children with Duchenne muscular dystrophy: a follow-up study

Background: Improvement of health-related quality of life (HRQOL) is a major goal in chronic disease management and HRQOL has become an important outcome in clinical trials. Longitudinal data on HRQOL are needed to elucidate change over time and to assess effectiveness of interventions; such research is lacking in the paediatric Duchenne Muscular Dystrophy (DMD) population. Methods: We followed up participants from our initial HRQOL study in 2013 a year and a half later. Multidimensional generic and disease-specific measures from the Pediatric Quality of Life Inventory were used to assess HRQOL from child and parent perspectives. Mean changes in HRQOL were calculated. Results: Data collection is ongoing and currently, data from 16 families (out of the initial 98) are available. Preliminary results indicated that by both child and parent reports, there were declines in all domains of HRQOL except for social function, in which there was a slight improvement. Mean decline in HRQOL scores ranged from 1.6 to 8.6 for child reports; and 3.3 to 7.7 for parent reports. Conclusion: HRQOL of boys with DMD deteriorates over time. Our results may be helpful in interpreting patient reported outcomes in forthcoming clinical trials and determining minimally clinically important changes in this population.

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P.078 Health-related quality of life and fatigue in children with Duchenne muscular dystrophy: A three-year longitudinal study

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

10.1017/cjn.2018.180 ◽

2018 ◽

Vol 45 (s2) ◽

pp. S37-S37

Author(s):

G Bhullar ◽

Y Wei ◽

B El-Aloul ◽

K Speechley ◽

M Miller ◽

...

Keyword(s):

Quality Of Life ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Parent Report ◽

Health Related Quality ◽

Related Quality ◽

Health Related ◽

Pearson Correlations ◽

Over Time

Background: Longitudinal data on health-related quality of life (HRQOL) and fatigue in paediatric Duchenne muscular dystrophy (DMD) are limited. Recently, fatigue was reported to be the greatest predictor of poor HRQOL in paediatric DMD. Understanding the trajectory of HRQOL and its relationship with fatigue may facilitate the development of improved therapeutic strategies. Our objective was to describe three-year changes in HRQOL and fatigue in children with DMD. Methods: Patients identified via the Canadian Neuromuscular Disease Registry received mailed questionnaires (2013–2016). HRQOL was assessed using the PedsQLTM GCS and NMM domains, and fatigue was assessed using the MFS domain (patient- and parent-report). Mean three-year change in scores were computed. Pearson correlations were computed between three-year change in HRQOL and fatigue. Results: Mean decline in MFS scores for patient- and parent-reports were 1.03 and 1.19, respectively. Mean decline in GCS scores for patient- and parent-report were 1.75 and 4.13, respectively. Mean change in NMM scores for patient- and parent-report were 0.72 and -8.36, respectively. Change in MFS score was associated with changes in GCS (r=0.72, p<0.001) and NMM scores (r=0.84, p<0.001) by patient-report. Conclusions: Children with DMD experience worse fatigue and HRQOL over time. Parents perceive a greater decline in HRQOL over time compared to patients.

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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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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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