Measuring quality of life in muscular dystrophy

Bann, Carla; Abresch, Richard T.; Biesecker, Barbara B.; Conway, Kristin Caspers; Heatwole, Chad; Peay, Holly; Scal, Peter; Strober, Jonathan B.; Uzark, Karen; Wolff, Jodi; Margolis, Marjorie; Blackwell, Angela Glover; Street, Natalie; Montesanti, Angela; Bolen, Julie

doi:10.1212/wnl.0000000000001336

Cited by 24 publications

(34 citation statements)

References 36 publications

(66 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…HRQoL entails multiple dimensions including physical, psychological, and social function. Factors associated with reduced HRQoL in muscular dystrophies include older age, longer disease duration, greater disease severity, and depressed mood . Although several studies have used dysphagia‐specific QoL measures in OPMD, there have been no studies of overall HRQoL and its determinants in a purely OPMD population.…”

mentioning

confidence: 99%

The relationship between physical symptoms and health‐related quality of life in oculopharyngeal muscular dystrophy

Youssof

2016

Muscle and Nerve

View full text Add to dashboard Cite

Introduction Oculopharyngeal muscular dystrophy (OPMD) causes ptosis, dysphagia, and limb weakness. Health-related quality of life (HRQoL) and its relationship to physical symptoms was investigated Methods The Short Form-36 (SF-36) was completed by 89 participants in the US OPMD Registry. Multiple hierarchical regression was used to determine the relative contributions of dysphagia severity and lower extremity functional impairment to the physical (PCS) and mental (MCS) components of the SF-36. Results HRQoL was reduced in OPMD compared with population norms. Lower extremity functional impairment explained a significant proportion of variance in PCS and MCS. Dysphagia symptom severity explained a moderate amount of variance only in MCS. Dysphagia symptom severity had the strongest associations with general health perception and social functioning domains. Discussion Lower extremity functional impairment in OPMD deserves attention due to its large influence on HRQoL. Both generic and dysphagia-specific measures are necessary to assess HRQoL in OPMD.

show abstract

mentioning

confidence: 99%

The relationship between physical symptoms and health‐related quality of life in oculopharyngeal muscular dystrophy

Youssof

2016

Muscle and Nerve

View full text Add to dashboard Cite

show abstract

“…Individuals living with NMD do not perceive physical ability as important in the physical domain of QoL; ease of mobility, vitality, pain and fatigue were identified as more important 55 . These components are captured in the French VSP-A measure utilised by Vuillerot et al 53 , who compared QoL in youth with NMD amongst each other grouped by physical ability.…”

Section: Physical Wellbeing Domains Of Qolmentioning

confidence: 97%

“…Notwithstanding, our checklist identified a need for clearer reporting of recruitment strategies, methods that recruit greater participant numbers and use of age and population validated QoL measures 55 .…”

Section: Limitations and Directions For Future Researchmentioning

confidence: 99%

“…Whilst the DISABKIDS Chronic Generic Measure DCGM-37 was recently appraised as having strongest evidence to support its measurement properties in children with neurodisability 74 , the single study sourced utilising the DISABKIDS 47 presented data in figures only that could not be extracted. Efforts to construct and validate disease and age specific instruments with involvement of patient stakeholders are ongoing 55 , with the PROMIS 75 and SOLE 76 outcome measures being recent examples.…”

Section: Limitations and Directions For Future Researchmentioning

confidence: 99%

See 1 more Smart Citation

Quality of Life and Psychosocial Well-Being in Youth With Neuromuscular Disorders Who Are Wheelchair Users: A Systematic Review

Travlos

Patman

Wilson

et al. 2017

Archives of Physical Medicine and Rehabilitation

View full text Add to dashboard Cite

“…11,12 The complex nature of DM1 results in various levels of disability, and for many, reduced social participation and independence. 15,16 Although the findings regarding HRQoL in DM1 have been diverging, several studies have found reduced HRQoL. 3 International research programmes such as OPTIMISTIC have emerged, aiming to improve clinical practice and quality-of-life standards in the DM1 population.…”

Section: Introductionmentioning

confidence: 99%

Uncovering health and social care needs among myotonic dystrophy patients

et al. 2019

View full text Add to dashboard Cite

Objectives: Myotonic dystrophy type 1 (DM1) is a slowly progressive multisystem disorder. Guidelines recommend multidisciplinary follow-up. We aimed to investigate the presence of unmet health and social care needs among patients with DM1 and whether unmet needs correlated with motor function, cognitive impairments, or quality of life. Material and Methods: Patients were 22 adults with DM1. "Needs and ProvisionsComplexity Scale" (NPCS) was applied to evaluate the individual's needs and provision of health and social services. The Muscular Impairment Rating Scale (MIRS) was used to measure motor function and disease stage. All patients underwent neuropsychological testing. The EQ-5D-3L questionnaire was used to evaluate the patients'health-related quality of life (HRQoL). Results:Median time from diagnosis was 11 years (range: 1-40). Twenty patients had developed needs related to social care, personal care, and rehabilitation that had not been met, whereas need for medical follow-up was largely met. The more pronounced the muscular impairment, the more unmet needs were experienced by DM1 patients (r = 0.50, P = 0.019). Degree of unmet needs did not correlate with full-scale IQ (r = −0.27, P = 0.23) or HRQoL (r = −0.14, P = 0.55). Conclusion:Using NPCS, we discovered that patients with DM1 had unmet needs with respect to social care, personal care, and rehabilitation although their need for medical follow-up was met. Thus, the use of NPCS helped bring our practice in better accordance with guidelines. A higher MIRS grade should alert the clinician to the likelihood of unmet needs. K E Y W O R D Scognitive function, healthcare delivery, myotonic dystrophy type 1, quality of life, social support

show abstract

Measuring quality of life in muscular dystrophy

Abstract: Additional scale development research is needed to enhance assessment of QOL for individuals with MD. Item banking and computerized adaptive assessment would be particularly beneficial by allowing the scale to be tailored to each individual, thereby minimizing respondent burden.

Cited by 24 publications

References 36 publications

The relationship between physical symptoms and health‐related quality of life in oculopharyngeal muscular dystrophy

The relationship between physical symptoms and health‐related quality of life in oculopharyngeal muscular dystrophy

Quality of Life and Psychosocial Well-Being in Youth With Neuromuscular Disorders Who Are Wheelchair Users: A Systematic Review

Uncovering health and social care needs among myotonic dystrophy patients

Contact Info

Product

Resources

About