Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries

Abstract

Duchenne muscular dystrophy (DMD) is a progressive, genetically determined neuromuscular disease that affects males and leads to severe physical disability in early teenage years. Over the last decades, patient-reported outcomes such as Health-Related Quality of Life (HRQoL) gained great interest in clinical research. However, little is known about factors affecting HRQoL in boys with DMD. Data from the multi-center CARE-NMD project of boys with DMD from six European countries collected between 2011 and 2012 were analyzed (8-17 years old; n = 321). HRQoL was measured using the KIDSCREEN-10 index, the Pediatric Quality of Life Inventory (PedsQL) and the Neuromuscular Module of the PedsQL (NMM). Linear regression models served to examine influences of socio-demographic, disease- and treatment-specific as well as participation- and environment-related factors on overall and disease-specific HRQoL. Proportions of explained variance varied across models using different outcomes (18-34%). Overall HRQoL according to the KIDSCREEN-10 index was associated with household income, the frequency of attending a clinic with specialized staff, the number of days spent outside home, and the attitude of the local community, but no significant association with age occurred. Overall HRQoL according to the generic PedsQL and disease-specific HRQoL were both positively associated with age and influenced by the country of residence, the disease stage, number of days spent outside home, and the attitude of the local community. Our results may be relevant for clinical practice and planning interventions for this population, but should be confirmed by future research. Further questions for future studies on boys with DMD are proposed.

Duchenne and Becker muscular dystrophy in adolescents: current perspectives

“… 42 , 45 – 47 While the initial loss of ambulation significantly detracts from QOL, QOL appears to improve again with age, which has been termed the “well-being paradox”. 48 , 49 The same pattern has been found in parents of individuals with DMD, who feel better equipped to manage their child’s condition once ambulation ceases and they concentrate their hopes on the progression of their child as an individual, rather than the progression of the condition. 50 Furthermore, equipment typically prescribed due to functional deterioration is seen as a positive improvement, because it reduces other impediments to QOL, such as illness, hospitalizations, or limited access to care locations.…”

“… 57 , 58 This finding is likely associated with the well-being paradox previously described: the longer the period from initial diagnosis and initial loss of ambulation, the lower the impact illness has on their psychosocial well-being. 48 , 49 , 57 Other data have shown an increased prevalence of depression (29%) and anxiety (27%) in adolescents and adults with DMD. 28 , 59 Interviews with adults in advanced stages of DMD indicated that while overall their depression and anxiety was low, they did experience heightened anxiety during critical disease-progression milestones, including full-time wheelchair use and the initiation of mechanical ventilation, which may be one possible explanation for the variable findings on depression and anxiety in the literature.…”

Developing multidisciplinary clinics for neuromuscular care and research

“…A nascent literature has already begun to document that multidisciplinary care is more effective than the traditional alternative as it has been associated with increased adherence to clinical care guidelines and more efficient resource utilization. [10][11][12][13][14][15]17,18 There is also some evidence to suggest that multidisciplinary care is associated with reduced hospital admissions/emergency-room visits, increased survival, and improved quality of life. [10][11][12][13][14][15]17,18 Ideally, one would measure and track patientcentered outcome data to document the impact of multidisciplinary care on health utility.…”

“…Patients and clinicians in multidisciplinary clinics believe that the model is preferable to uncoordinated care . Furthermore, studies have shown that the quality of care delivered in multidisciplinary care is high and that both quality of life and survival are better in neuromuscular populations treated in multidisciplinary clinics than in isolated neurological clinics …”

“…9 Furthermore, studies have shown that the quality of care delivered in multidisciplinary care is high and that both quality of life and survival are better in neuromuscular populations treated in multidisciplinary clinics than in isolated neurological clinics. [10][11][12][13][14][15] The evolution of the health care system in the United States presents both an opportunity and a challenge to neuromuscular clinicians as they strive to establish and run multidisciplinary clinics. The recent focus on improving the quality of care through patient-centered care and care coordination aligns with both the goals and demonstrable results of multidisciplinary care.…”