Clinical Features and Quality of Life in Duchenne and Becker Muscular Dystrophy Patients from A Tertiary Center in Turkey

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Yayıcı Köken Ö., Kucur Ö., Taşkıran C., Öztoprak Ü., Genç Sel Ç., Aksoy E., ...More

GUNCEL PEDIATRI-JOURNAL OF CURRENT PEDIATRICS, vol.19, no.1, pp.15-22, 2021 (Peer-Reviewed Journal) identifier


Introduction: Duchenne Muscular Dystrophy (DMD) and Becker Muscular

dystrophy (BMD), are chronic and progressive and rare genetic disorders that

cause systemic involvement such as progressive muscle deterioration, motor

disability, cardiomyopathy, and respiratory problems, with an increased risk of

cognitive decline and psychological problems They are a group of neuromuscular

diseases in which psychological problems affect negatively on quality of life

(Qol) not only patients but also caregivers. This study demonstrates the clinical

features of patients with DMD/BMD and their caregivers and compares controls

with respects to psychological and social aspects.

Materials and Methods: A total of 20 patients (3 with BMD and 17 with DMD),

aged between 8 and 18 years, and 20 age-matched healthy children were included

in this descriptive and cross-sectional study. The patients were evaluated by the

pediatric neurology, cardiology, and psychiatry departments at the study time. Their

demographic and clinical features were recorded. The Wechsler Intelligence Scale

for Children-Revised (WISC-R), Pediatric Quality of Life Inventory (PedsQL)

and its parent form, and the Strengths and Difficulties Questionnaire (SDQ) were

applied to all of the participants.

Results: The QoL scores were lower in patients with moderate and severe DMD/

BMD and their caregivers. In patients with DMD and in both groups, emotional

symptoms, peer problems and prosocial behavior scores were higher in the subsets

of the SDQ. The PedsQL child-parent scores were lower in all of the subsets with

statistical significance.

Conclusions: DMD and BMD comprise a group of chronic diseases with multiple

complications that are difficult to manage. A QoL equal or close to that of the

healthy children should be targeted. Today, proposed or experimental treatments

for this disease group are assessed based on their ability to enhance QoL. Inquiring

into the QoL and counseling should become routine.