A review of quality of life themes in Duchenne muscular dystrophy for patients and carers

Abstract

Duchenne Muscular Dystrophy (DMD) is a severe, life-limiting and incurable condition. However, studies estimating quality of life and those measuring actual quality of life in people living with DMD vary considerably. This discrepancy indicates potential difficulties with assessing quality of life using common generic quality of life instruments in this rare and unique population. This study sought to document the range of themes relevant to quality of life for people with DMD by examining the published literature and additionally to investigate the themes that are relevant to quality of life for carers and the wider family. Eligible studies for the review were primary studies of any study design that reported outcomes or themes relevant to quality of life for either people with DMD, their families, or both. A review of studies identified from searching medical bibliographic sources between 2010 and 2016 found 45 relevant published studies. A thematic framework is proposed to categorise the themes identified into: i. physical; ii. psychological; iii. Social; iv. well-being domains. A final “other” domain was included to encompass themes identified from the literature that are not covered by commonly used quality of life instruments. The rich variety of themes identified from the review highlights that DMD has a complex quality of life profile which is not currently captured by standard quality of life tools that are commonly employed in the healthcare setting. The findings also highlight that the resulting impact on the quality of life of carers and wider family of people with DMD requires consideration.Electronic supplementary materialThe online version of this article (10.1186/s12955-018-1062-0) contains supplementary material, which is available to authorized users.

Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review

“…The impact of these aspects on HRQoL may be profound given that the loss of ambulation tends to occur around the time that a DMD patient's peers would be gaining independence. Therefore, there may be justification for the use of disease-specific preference-based instruments to capture the full range of effects that DMD can have on both patients and their caregivers [32,33]. However, their value for decision-making has yet to be understood [34].…”

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

“…Yet, as the respiratory muscles continue to deteriorate, all patients eventually need assistance to breathe also during the daytime to survive [5]. Due to the morbidity and mortality caused by the disease, DMD has been shown to be associated with a substantial burden on affected patients [6], informal caregivers [7], and society [8].…”

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

“…No restrictions on date or language were applied to the search. Search A terms are illustrated in full in Additional file 1 and included: (I) Duchenne muscular dystrophy (and Duchenne*) AND ((II) a search filter provided by the PROM group at the University of Oxford to identify PROMs (available online [20] and in Additional file 1) OR (III) PROMs known to be used in people with DMD based on a prior rapid review of the literature [21].…”