Duchenne Muscular Dystrophy—Parental Perceptions

Abstract

Quality of life and availability of services are important for boys with Duchenne muscular dystrophy (DMD) and their families. Families attending our neuromuscular clinic completed a questionnaire on parental perception regarding the importance of services, health issues, and quality of life issues both "now" and "in the future." Eighty-nine percent of the families (31/35) completed questionnaires. Services and health issues related to prolonging ambulation were most important, especially for the parents of younger boys. Mental health issues such as social isolation, anger, and depression were very important, particularly for the families of older boys and were anticipated to be more important in the future. Pediatricians should be aware of both the immediate needs of families to meet the physical and emotional challenges of DMD and the increasing requirement to address the social needs of these patients and their families as the boys become older.

British Thoracic Society guideline for respiratory management of children with neuromuscular weakness

“…Studies show that parental decision-making regarding treatment is directly related to perception of QOL 234 235. Understanding HRQOL from the perspective of the patient is essential to facilitate shared decision-making with healthcare professionals and parents or carers.…”

Health-Related Quality of Life in Children and Adolescents With Duchenne Muscular Dystrophy

“…2 Studies evaluating QoL in children with DMD are few and rarely examine selfperceptions in children and adolescents. Past studies have often relied on parental report [3][4][5] and/or are limited by small sample size 5,6 or have focused predominantly on end-of-life decisionmaking in adults. 7 In the absence of standardized measurements, health care providers often fail to recognize problems in patients' health-related QoL.…”

Recent advances in the management of Duchenne muscular dystrophy

“…With the increasing lifespan seen in this patient group, more research studies on quality-of-life issues are emerging. It is well known that children with DMD do not experience their life as unhappy, which is probably due to the fact that they have physical difficulties from an early age and consider this to be normal for them 29. Some studies among adults with DMD appear to confirm these views,30 whereas others paint a less positive picture 31 32.…”