Javed Dawood, Javed Abdullah, Jajja Faran Ahmed, Jahanzeb Ahmed
Department of Internal Medicine, Lahore Medical and Dental College, Lahore, Pakistan.
Department of Internal Medicine, Allama Iqbal Medical College, Lahore, Pakistan.
J Pharm Technol. 2025 Jun 24:87551225251349402. doi: 10.1177/87551225251349402.
This meta-analysis evaluates the efficacy and safety of vamorolone, a dissociative glucocorticoid, compared with traditional glucocorticoids in treating Duchenne muscular dystrophy (DMD), aiming to assess its potential as a safer alternative with comparable therapeutic benefits. A systematic search was conducted in PubMed (MEDLINE), Embase, and Web of Science from inception till June 2024. Search terms included (Vamorolone) AND (Corticosteroids OR Glucocorticoids OR Prednisone) AND (Duchenne Muscular Dystrophy OR related terms). Only randomized controlled trials (RCTs) and observational studies were included, with no language restrictions. From 276 identified reports, 135 were screened after duplicate removal, and 30 underwent full-text review. Five studies (2 RCTs, 2 observational, 1 nonrandomized trial) involving 370 patients were included. Two reviewers independently extracted data on efficacy (TTSTAND, TTRW, TTCLIMB velocities) and safety (height percentile, body mass index-z score, osteocalcin, P1NP) using Microsoft Excel, resolving discrepancies through consensus. Vamorolone significantly improved TTSTAND (MD = -0.03, 95% confidence interval [CI] = -0.06 to -0.004, = .02), TTRW (MD = -0.11, 95% CI = -0.22 to -0.01, = .04), and TTCLIMB velocities (MD = -0.04, 95% CI = -0.08 to -0.003, = .03). Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31-26.25, = .001) and bone biomarkers (osteocalcin: MD = 15.68, < .00001; P1NP: MD = 158.34, < .00001), with no significant body mass index-z score difference. Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management.
本荟萃分析评估了解离型糖皮质激素瓦莫洛龙与传统糖皮质激素相比，在治疗杜氏肌营养不良症（DMD）中的疗效和安全性，旨在评估其作为具有可比治疗益处的更安全替代药物的潜力。从创刊至2024年6月，在PubMed（MEDLINE）、Embase和Web of Science中进行了系统检索。检索词包括（瓦莫洛龙）AND（皮质类固醇或糖皮质激素或泼尼松）AND（杜氏肌营养不良症或相关术语）。仅纳入随机对照试验（RCT）和观察性研究，无语言限制。从276篇已识别的报告中，去除重复项后筛选出135篇，30篇进行了全文审查。纳入了五项研究（2项RCT、2项观察性研究、1项非随机试验），涉及370名患者。两名审查员使用Microsoft Excel独立提取了疗效（站立时间、跑步时间、攀爬时间速度）和安全性（身高百分位数、体重指数z评分、骨钙素、I型前胶原氨基端前肽）的数据，通过协商解决差异。瓦莫洛龙显著改善了站立时间（MD=-0.03，95%置信区间[CI]=-0.06至-0.004，P=.02）、跑步时间（MD=-0.11，95%CI=-0.22至-0.01，P=.04）和攀爬时间速度（MD=-0.04，95%CI=-0.08至-0.003，P=.03）。安全性结果显示身高百分位数增加（MD=16.28，95%CI=6.31-26.25，P=.001）和骨生物标志物增加（骨钙素：MD=15.68，P<.00001；I型前胶原氨基端前肽：MD=158.34，P<.00001），体重指数z评分无显著差异。在DMD中，瓦莫洛龙与传统糖皮质激素具有可比的疗效，在运动功能改善以及生长和骨骼健康方面具有更安全的特征，表明其有可能改变DMD的治疗管理。
