Akademik Veri Yönetim Sistemi
GUNCEL PEDIATRI-JOURNAL OF CURRENT PEDIATRICS, cilt.19, sa.1, ss.15-22, 2021 (Hakemli Dergi)
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.