Objective:

We aimed to compare the safety of different pharmacological treatments currently administered in boys with Duchenne muscular dystrophy (DMD).

Background:

DMD is a severe, progressive neuromuscular disorder affecting male individuals, leading to significant morbidity and early mortality. Current management of DMD is primarily symptomatic, employing various pharmacological agents aimed at slowing disease progression and alleviating associated complications. However, the safety profiles of these medications, particularly in pediatric populations characteristic of DMD, remain a critical concern.

Design/Methods:

PubMed, Ovid, Embase, Web of Science, and Cochrane Library were searched from inception through October 17, 2023 to identify randomized controlled trials (RCTs) assessing different pharmacological therapies in boys with DMD. The outcome analyzed were severe adverse events (SAEs) with Bayesian random models, performed by R software with gemtc package.

Results:

After reviewing 552 studies, 12 RCTs were included with 1,195 patients and a mean age of 7.9 years. The pharmacological treatments reviewed included prednisone, deflazacort, viltolarsen, vamorolone, tamoxifen, ciclosporin A, ataluren, tadalafil and drisapersen. In SAEs, drisapersen (IRR 0.65, 95% CrI 0.43 to 0.97) and vamorolone (IRR 0.80, 95% CrI 0.52 to 1.23) demonstrated a promising safety profile. Deflazacort showed a comparatively lower (IRR 1.20, 95% CrI 0.85 to 1.68) and prednisone (IRR 1.65, 95% CrI 1.22 to 2.24), viltolarsen (IRR 2.4, 95% CrI 1.3 to 3.8), tamoxifen (IRR 2.0, 95% CrI 1.5 to 2.7), ciclosporin A (IRR 1.50, 95% CrI 1.05 to 2.15), ataluren (IRR 1.5, 95% CrI 0.8 to 2.5) and tadalafil (IRR 1.9, 95% CrI 1.0 to 4.1) had a higher probability of SAEs.

Conclusions:

Our study indicates a differential safety profile among the analyzed treatments for boys with DMD, drisapersen and vamorolone exhibit a promising safety profile.