The quality of life in boys with Duchenne muscular dystrophy

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Highlights

  • Boys with Duchenne muscular dystrophy have a relatively satisfactory quality of life.

  • These patients have positive attitude toward life despite their progressive disease.

  • Social support could improve parents' perception about quality of life.

Abstract

We conducted a study to evaluate the quality of life in boys with Duchenne muscular dystrophy aged 8–18 years, compared with that in matched healthy controls. A total of 85 boys with Duchenne muscular dystrophy aged 8–18 years and 136 age, sex and living place matched healthy controls were included in this study. Patients and one of their parents separately completed the 27-item Persian version of KIDSCREEN questionnaire (child and adolescent version and parent version). From the children's perspective, the quality of life in patients was found to be lower in two subclasses: “physical activities and health” (p < 0.001) and “friends” (p = 0.005). Parental estimation of their sick child's quality of life was significantly lower than children's own assessment in two subclasses: “physical activities and health” (p < 0.001) and “general mood and feelings” (p < 0.001). Our results indicate that boys with Duchenne muscular dystrophy have quite a satisfactory quality of life. A happier and more hopeful life can be promoted through increasing social support and improving the parental knowledge regarding their child's more positive life perspective.

Introduction

Duchenne muscular dystrophy (DMD) is the most common hereditary progressive muscular disease in children, with a prevalence of 1 in every 4000 boys [1]. Despite advances in diagnosis and treatment measures, psychological and environmental aspects of patients have received less attention in daily practice [2].

Health-related quality of life has a multidimensional definition and explains the effect of the health status of individuals including physical, mental, and social domains on the quality of life [3]. Quality of life assessment has become an inseparable part of the evaluation of treatment trials and the US Food and Drug Administration recommends using quality of life assessment tools [4].

Health-related quality of life is used for the assessment of the health requirements of the society, evaluation of the social impact of diseases, identification of individuals at risk, execution of appropriate health policies proportionate to these issues, and allocation of resources in the health sector [5].

Health-related quality of life assessment is well-recognized in adults, but its application in children does not have a long history. Previous studies have shown that children can report their well-being with a high degree of certainty using age appropriate tools. However, if the child is not cooperative due to the lack of lingual and cognitive skills or the presence of a severe disease, the parent's participation in the assessment of their child's health-related quality of life is essential and vital [6], [7]. In such cases, however, assessment may be inaccurate because parents answer the questions from their own perspective and viewpoint [8].

Previous studies on the effect of DMD on the quality of life in boys have reported different results. In one study, the quality of life in these patients has showed a decrease only in the physical subclass when compared to controls, whereas other studies have shown a decrease in all quality of life related subclasses when compared with age matched controls [9], [10], [11], [12], [13]. Very few studies have investigated the level of agreement between children's self-reports and parents' reports of the health-related quality of life in children with DMD. In two separate studies, Bray et al. and Davis et al. have shown that parental estimation of their child's quality of life in the subclass “general mood and feelings” was lower than children's own self-reports [2], [13].

To our knowledge, no study has assessed the health-related quality of life in children and adolescents with DMD in Iran. Therefore, we aimed to evaluate the health-related quality of life in boys with DMD aged 8–18 years compared with matched healthy controls by using the standard 27-item KIDSCREEN questionnaires for child self-assessment and parent assessment.

Section snippets

Patients

This study was performed on 85 boys with DMD aged 8–18 years who lived in Tehran and were monitored in the Pediatric Neurology Department of Children's Medical Center and Iran Muscular Dystrophy Association. The reason for considering this age group was that both patients and their parents were adapted to the disease.

Inclusion criteria were patient's age (8–18 years), residence in Tehran, clinical picture of disease, high levels of muscle enzymes, and DMD confirmed via biopsy or genetic

Sample description

The patient group comprised 85 boys with DMD aged 8–18 years (mean age 12.6 ± 3.34 years) and the control group included 136 age-matched healthy children aged 8–18 years (mean age 12.1 ± 2.51 years).

Comparative analysis of the health-related quality of life in healthy and affected children

The quality of life in healthy children and boys with DMD from the perspective of the children and adolescents and the perspective of their parents was assessed by using the KIDSCREEN-27. From the children's and adolescents' perspective, significantly lower scores were observed in the patient group

Discussion

The present study showed that from the perspective of children and adolescents, the quality of life was similar to boys with DMD when compared to that in healthy controls, except for the subclasses “physical activity and health” and “friends”. The scores in the subclasses “general mood and feeling”, “family and free time”, and “school and learning” were similar in patients and controls, indicating that these subclasses were not affected by DMD. In a study by Opstal et al. on 40 patients, the

Conclusions

In conclusion, our results indicate that boys with DMD have quite a satisfactory quality of life. In addition, boys with DMD have a positive attitude toward life despite the progressive nature of the disease and limitations in physical activities and relationship with friends. Therefore, in order to maintain this positive attitude, the health system should create and implement a plan to improve the social support for boys with DMD and their families and to facilitate their participation in

Acknowledgements

We wish to thank Mrs. Ramak Heidari and Mrs. Neda Anisi of Iranian Muscular Dystrophy Association for their invaluable assistance in data gathering. We are really grateful to Dr. Peyman Jafari and Mrs. Sara Garshad for their help in data scoring. The authors are grateful to all parents and patients for their participation in this study. We are extremely indebted to the authorities of the Research Deputy of Tehran University of Medical Sciences (Grant number: 10010273). for their financial and

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