Evaluating the Impact of Multicenter Clinical Trials on DMD Treatment Outcomes: A Systematic Review

Topic:

Other

Poster Number: P18

Author(s):

Mahmoud M. Elsayed, MD, MME Foundation, Yara Hamdi, Badr University in Cairo, Sherif Elhaig, Newgiza University, Amr Genina, Newgiza University, Mohamed Farghaly, Newgiza University, Amira Maghrabi, Newgiza University, Yara Shokry, Newgiza University

Background:
Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by dystrophin deficiency, leading to progressive muscle degeneration. Multicenter clinical trials have played a pivotal role in advancing DMD treatments by enabling robust data collection across diverse populations. However, variability in study design and implementation warrants systematic evaluation of their impact on treatment outcomes.

Objectives:
This review examines the impact of multicenter clinical trials on DMD treatment outcomes, focusing on their contributions to therapeutic advancements, outcome standardization, and patient care optimization.

Methods:
A systematic search of PubMed, Embase, Cochrane Library, and ClinicalTrials.gov identified multicenter trials on DMD published up to 2024. Trials evaluating pharmacological, gene-based, and symptomatic management strategies were included. Data extraction followed PRISMA guidelines, and study quality was assessed using validated tools.

Results:
Analysis of 30 trials involving over 4,000 participants showed significant advancements in DMD treatment. Exon skipping therapies improved dystrophin restoration (5%–15%) and functional outcomes, with a 30-meter increase in the 6-minute walk test (95% CI: 20–40, p < 0.01). Gene therapy using adeno-associated viral vectors achieved 25%–40% dystrophin restoration and motor function gains. Pharmacological agents, including vamorolone, demonstrated enhanced muscle strength with fewer side effects compared to traditional corticosteroids. Multicenter designs supported diverse cohorts and standardized measures such as the North Star Ambulatory Assessment and serum biomarkers, enhancing generalizability. Challenges included variability in protocol adherence and site-specific management differences. Conclusion: Multicenter clinical trials have driven significant progress in DMD treatment, validating therapies and standardizing outcomes. Continued efforts to harmonize protocols and enhance trial design will further improve their impact, ensuring better care and outcomes for DMD patients.