Background:
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder profoundly impacting the lives of patients and their families. Understanding patient perspectives on treatments is crucial for designing interventions that align with their needs and priorities, ultimately improving quality of life and treatment adherence.
Objectives: This systematic review synthesizes qualitative studies exploring patient and caregiver perspectives on DMD treatments, focusing on their experiences, preferences, and unmet needs.
Methods:
A systematic search of PubMed, Embase, PsycINFO, and Cochrane Library identified qualitative studies published up to 2024 that examined patient or caregiver perspectives on DMD treatments. Thematic synthesis was used to identify recurring themes and key insights.
Results:
Analysis of 20 studies revealed three dominant themes: treatment efficacy, quality of life, and psychosocial impact. Patients and caregivers prioritized therapies that improved functional independence and slowed disease progression, with a strong preference for non-invasive and convenient treatment options. Emotional and financial burdens of treatment were significant concerns, underscoring the need for holistic care approaches. Participants emphasized the importance of communication with healthcare providers, seeking clear information on treatment outcomes and side effects. Gaps in access to advanced therapies and support services were frequently highlighted.
Conclusion:
Patient perspectives on DMD treatments reveal a strong focus on improving functional outcomes, minimizing burdens, and enhancing overall quality of life. These findings emphasize the need for patient-centered care strategies, integrating preferences and psychosocial support into treatment plans to address the comprehensive needs of DMD patients and their families.