Background:
Duchenne muscular dystrophy (DMD) is a progressive genetic disorder that profoundly affects quality of life (QoL) for patients and caregivers. Despite advancements in therapeutic options, understanding and addressing patient-centered outcomes such as physical, emotional, and social well-being remain critical for improving care.
Objectives:
This systematic review evaluates patient-centric approaches in DMD research, focusing on QoL outcomes and identifying strategies to enhance holistic care.
Methods: A systematic search of PubMed, Embase, and Cochrane Library was conducted to identify studies published up to 2024. Eligible studies included randomized controlled trials, observational studies, and qualitative research reporting QoL outcomes in DMD. Data were synthesized qualitatively to identify trends and areas for intervention.
Results:
Analysis of 32 studies revealed that patients with DMD experience significantly lower QoL compared to healthy peers, particularly in physical and emotional domains. Caregivers reported high levels of psychological distress, with 40% meeting clinical thresholds for anxiety or depression. Interventions such as multidisciplinary care, mental health counseling, and community support improved QoL metrics for both patients and caregivers. Early intervention correlated with better outcomes, highlighting the importance of timely psychosocial support.
Conclusion:
Patient-centric approaches are essential for optimizing QoL in DMD management. Comprehensive care models that integrate psychosocial, emotional, and physical support for patients and caregivers can significantly enhance overall well-being. These findings underscore the need for ongoing research to refine and expand patient-centered strategies in DMD care.