Perspectives on Everyday Life Challenges of Danish Young People With Duchenne Muscular Dystrophy (DMD) on Corticosteroids

Handberg, Charlotte; Werlauff, Ulla; Højberg, Ann-Lisbeth

doi:10.1177/23333936221094858

Cited by 6 publications

(4 citation statements)

References 36 publications

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“…Granted, these differences are not consistently statistically different throughout the study period. Previous studies of individuals with DMD and their parents reported how parents perceive the challenges faced by individuals with DMD as more severe than the individuals themselves [ 9 ]. It is likely that similar worries influence siblings, which in turn result in lower academic performances.…”

Section: Discussionmentioning

confidence: 99%

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Burden of Disease of Duchenne Muscular Dystrophy in Denmark – A National Register-Based Study of Individuals with Duchenne Muscular Dystrophy and their Closest Relatives

Rudolfsen,

Vissing,

Werlauff

et al. 2024

Journal of Neuromuscular Diseases

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Background: Duchenne Muscular Dystrophy (DMD) is a progressive genetic disease with a prevalence of 1 per 3,600–6,000 male births. Individuals with DMD are typically diagnosed at age 4–7 years; median survival is 30 years. They require multidisciplinary care, personal assistance, and often special education. Objective: The aim was to assess the burden of disease in DMD in Denmark. This includes incidence, prevalence, use of healthcare services, labour market participation, educational outcomes, and overall attributable costs due to DMD. Impact on the closest relatives (siblings and parents) was also investigated. Methods: The comprehensive Danish national health and administrative registers were used to assess the burden of disease following individuals with DMD and closest relatives from five years before, and up to 20 years after DMD diagnosis. Individuals with DMD (and relatives) from 1994–2021 were included. All outcomes were compared to matched control groups without the disease drawn from the Danish population. Results: 213 unique individuals with DMD were identified. They had lower grades in school, required more special education and more healthcare and home care compared to their control group. The extra costs of special education summed to EUR 180,900 over the course of 11 years elementary school. They had an annual average productivity loss of EUR 20,200 between the age of 18 to 30. The extra healthcare costs of DMD in the 20 years after diagnosis were estimated to EUR 1,524,000. If an individual with DMD lives to be 30, total extra costs sum to EUR 2,365,800. Conclusions: Using national register data this study presented detailed results on the burden of disease of DMD, including impact on closest relatives. With 60 additional hospital admissions and 200 extra outpatient contacts in 20 years healthcare costs, but also costs of home care and special education, increases as disease progresses.

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Section: Discussionmentioning

confidence: 99%

“…Additionally, many individuals have behaviour-and learning difficulties. As a result, individuals with DMD have reduced life expectancy with a median survival age of about 30 years [5][6][7][8] and impaired quality of life and an existential worry about life [9].…”

Section: Introductionmentioning

confidence: 99%

Burden of Disease of Duchenne Muscular Dystrophy in Denmark – A National Register-Based Study of Individuals with Duchenne Muscular Dystrophy and their Closest Relatives

Rudolfsen,

Vissing,

Werlauff

et al. 2024

Journal of Neuromuscular Diseases

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“…Is it better for children to be involved in their parents' hopes for their future when this hope entails a cure that will make a fundamental change in the child – hinting that a life without disability is preferable? Does it harm the child to introduce a little escapism and “dream big” as one parent puts it when the child's disability seems to be in the way of the life they want to lead (Handberg et al, 2022)? Or should parents rather from an early age support the children in forming strong positive disability identities, installing the belief in them that they can live a perfectly full life with their disease?…”

Section: Discussionmentioning

confidence: 99%

New medicine for neuromuscular diseases: An evolving paradox for patient and family hopes and expectations

Mahoney¹,

Handberg

2022

Nursing Inquiry

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Recent developments in novel therapies for neuromuscular diseases offer parents new perspectives on their affected children's future. This article examines how the emergence of new therapies impacts the lives of parents of children with Duchenne muscular dystrophy or spinal muscular atrophy type 2, two genetic neuromuscular disorders characterized by progressive muscle degeneration. Aiming for a firstperson perspective, fieldwork was conducted utilizing participant observation, semistructured interviews, and several internet sources. Six families with a total of 12 persons, all living in Denmark, were included in the interviews. Two types of parents were identified who were at opposite ends in dealing with the new therapies-the cure optimists and the cure pragmatists. Different hopes resulted in different narratives for their children's futures. The article raises questions about how and when children with chronic diseases should be involved in their parent's hopes for a cure and highlights the dilemmas facing health professionals working in the field of children with chronic diseases for which the prospects of a cure are improving. We conclude that health professionals must find a way to carefully balance guidance and information about experimental medicines, including the fact that experimental medicine sometimes fails, does not work as well as hoped for, or does not become available, with sustaining parental hopes for their children's future.

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“…The questionnaire was set up in SurveyXact ® , an online secure survey platform (Ramboll Denmark), and developed by the study group based on the results from three semi-structured focus groupinterviews (in review elsewhere) [16] with ambulant and non-ambulant women with NMD. The aim of the focus-group-interviews were to identify problem areas in relation to urinating when not at home and to target questions for the questionnaire.…”

Section: Questionnairementioning

confidence: 99%

HAP-PEE: A Danish National Study of Challenges Related to Urinating When Away from Home in Women with Neuromuscular Diseases, Impact on Activity and Participation and Prevalence of Lower Urinary Tract Symptoms

Werlauff,

Handberg,

Kristensen

et al. 2024

Journal of Neuromuscular Diseases

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BACKGROUND: Little is known about the challenges faced by women with a neuromuscular disease (NMD) when having to go to the toilet in other places than home; a topic that is highly important for participation and bladder health. OBJECTIVE: The aim was to investigate whether women with NMD have problems in going to the toilet when not at home, the problems’ impact on their social activities, education, and working life, which strategies they use to manage the problems, and the prevalence of lower urinary tract symptoms (LUTS). METHODS: A national survey containing questions on type of NMD, mobility, impacts on social activities, education, working life, and bladder health was developed by women with NMD and researchers. LUTS were assessed by the International Consultation on Incontinence Questionnaire Female Lower Urinary Tract Symptoms Modules (ICIQ-FLUTS). Female patients≥12 years (n = 1617) registered at the Danish National Rehabilitation Centre for Neuromuscular Diseases were invited. RESULTS: 692 women (43% ) accepted the invitation; 21% were non-ambulant. 25% of respondents avoided going to the toilet when not at home. One third of respondents experienced that problems in going to the toilet impacted their social life. 43% of respondents refrained from drinking to avoid voiding when not at home, 61% had a low frequency of urinating, 17% had experienced urinary tract infections, and 35% had experienced urine incontinence. Problems were seldom discussed with professionals, only 5% of participants had been referred to neuro-urological evaluation. CONCLUSION: The results highlight the difficulties in urinating faced by women with NMD when not at home and how these difficulties impact functioning, participation, and bladder health. The study illustrates a lack of awareness of the problems in the neuro-urological clinic. It is necessary to address this in clinical practice to provide supportive treatment and solutions that will enable participation for women with NMD.∥\keywords \KWDtitle KeywordsNeuromuscular diseases\sep women’s health\sep toilet facilities\sep urinary tract infection\sep participation\sep rehabilitation \end contstartabstract.

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Perspectives on Everyday Life Challenges of Danish Young People With Duchenne Muscular Dystrophy (DMD) on Corticosteroids

Cited by 6 publications

References 36 publications

Burden of Disease of Duchenne Muscular Dystrophy in Denmark – A National Register-Based Study of Individuals with Duchenne Muscular Dystrophy and their Closest Relatives

Burden of Disease of Duchenne Muscular Dystrophy in Denmark – A National Register-Based Study of Individuals with Duchenne Muscular Dystrophy and their Closest Relatives

New medicine for neuromuscular diseases: An evolving paradox for patient and family hopes and expectations

HAP-PEE: A Danish National Study of Challenges Related to Urinating When Away from Home in Women with Neuromuscular Diseases, Impact on Activity and Participation and Prevalence of Lower Urinary Tract Symptoms

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