Conway Kristin M, Thomas Shiny, Neyaz Tahereh, Ciafaloni Emma, Mann Joshua R, Staron-Ehlinger Michelle, Beasley Gary S, Romitti Paul A, Mathews Katherine D
Department of Epidemiology, College of Public Health, The University of Iowa, Iowa City, Iowa, USA.
New York State Department of Health, Albany, New York, USA.
Muscle Nerve. 2025 Apr;71(4):574-582. doi: 10.1002/mus.28353. Epub 2025 Jan 24.
INTRODUCTION/AIMS: Prophylactic treatment of left ventricular dysfunction (LVD) in Duchenne muscular dystrophy (DMD) delays onset of LVD, but there is limited data showing impact on survival. Our aim was to describe survival among treated and untreated individuals with DMD.
Retrospective, population-based surveillance data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet) were used. We analyzed 327 males with DMD born between 1982 and 2009 who were at least 6 years old at the last visit and who initiated cardiac prophylactic medication before age 14 years. Death status was ascertained through vital record linkages and medical record review. Prophylaxis was defined as cardiac medication use at least 1 year before LVD onset (ejection fraction < 55% or shortening fraction < 28%). Age at first visit, corticosteroid use, scoliosis surgery, initiation of noninvasive ventilation, and loss of ambulation were also coded. Cox Proportional Hazard modeling with time-varying covariates describes associations.
Prophylactic cardiac treatment was documented for 27.7% (n = 90); corticosteroids were used by 60.9% (n = 157). Adjusting for age at first visit and MD STARnet site, prophylactic treatment was associated with a 54% lower hazard of death (HR = 0.46, 95% CI = 0.22-0.93) compared to no prophylaxis. Adjusting for selected clinical covariates did not appreciably change the estimate (HR = 0.46, 95% CI = 0.22-0.99).
Initiation of cardiac medication when left ventricular function is normal was associated with prolonged survival in this study of males with DMD. Only one-quarter of individuals received this treatment, however, indicating a topic of focus for improving care.
引言/目的：对杜氏肌营养不良症（DMD）患者的左心室功能障碍（LVD）进行预防性治疗可延迟LVD的发病，但显示对生存影响的数据有限。我们的目的是描述接受治疗和未接受治疗的DMD患者的生存情况。
使用来自肌肉萎缩症监测、追踪和研究网络（MD STARnet）的基于人群的回顾性监测数据。我们分析了1982年至2009年间出生的327例DMD男性患者，他们在最后一次就诊时至少6岁，且在14岁之前开始使用心脏预防性药物。通过生命记录关联和病历审查确定死亡状态。预防性治疗定义为在LVD发病（射血分数<55%或缩短分数<28%）前至少1年使用心脏药物。首次就诊年龄、使用皮质类固醇、脊柱侧弯手术、开始使用无创通气以及失去行走能力也进行了编码。使用具有时变协变量的Cox比例风险模型描述关联。
有27.7%（n = 90）的患者记录了心脏预防性治疗；60.9%（n = 157）的患者使用了皮质类固醇。在对首次就诊年龄和MD STARnet站点进行调整后，与未进行预防性治疗相比，预防性治疗使死亡风险降低了54%（HR = 0.46，95%CI = 0.22 - 0.93）。对选定的临床协变量进行调整后，估计值没有明显变化（HR = 0.46，95%CI = 0.22 - 0.99）。
在这项对DMD男性患者的研究中，在左心室功能正常时开始使用心脏药物与延长生存期相关。然而，只有四分之一的患者接受了这种治疗，这表明这是改善护理的一个重点关注话题。
