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肌肉萎缩症监测、跟踪和研究网络中杜氏肌营养不良症患者的临床和人口统计学特征及全因死亡率的选择。

Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network.

机构信息

Division of Birth Defects and Infant Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, MS S106-3, 4770 Buford Hwy, Chamblee, GA 30341-3717, United States of America.

McKing Consulting Corporation Consultant to Centers for Disease Control and Prevention, 2900 Chamblee Tucker Rd. Building 10, Ste. 100. Atlanta, GA 30341, United States of America.

出版信息

Neuromuscul Disord. 2022 Jun;32(6):468-476. doi: 10.1016/j.nmd.2022.04.008. Epub 2022 Apr 30.

Abstract

Population-based estimates of survival among individuals with Duchenne muscular dystrophy (DMD) living in the United States are lacking. It is also unclear whether the association between glucocorticoid use and all-cause mortality persists in the context of other common treatments (cardiac medication, cough-assist, bilevel positive airway pressure, and scoliosis surgery) observed to delay mortality. Among 526 individuals identified by the Muscular Dystrophy Surveillance, Tracking, and Research Network, the estimated median survival time from birth was 23.7 years. Current glucocorticoid users had a lower hazard of mortality than non-users. Individuals who ever had scoliosis surgery had a lower hazard of mortality than individuals who did not have scoliosis surgery. Individuals who ever used cough assist had a lower hazard of mortality than individuals who never used cough assist. Non-Hispanic Black individuals had a higher hazard of mortality than non-Hispanic White individuals. No differences in hazards of mortality were observed between ever versus never use of cardiac medication and ever versus never use of bilevel positive airway pressure. The glucocorticoid observation is consistent with the 2018 Care Considerations statement that glucocorticoid use continues in the non-ambulatory phase. Our observations may inform the clinical care of individuals living with DMD.

摘要

目前,美国尚缺乏对杜氏肌营养不良症(DMD)患者的生存情况进行人群基础评估。此外,在其他常见治疗方法(心脏药物治疗、咳嗽辅助、双水平气道正压通气和脊柱侧弯手术)被观察到可延迟死亡的情况下,糖皮质激素的使用与全因死亡率之间的关联是否仍然存在,目前也并不清楚。在肌肉萎缩症监测、跟踪和研究网络(Muscular Dystrophy Surveillance,Tracking, and Research Network)确定的 526 名个体中,从出生起估计的中位生存时间为 23.7 年。目前使用糖皮质激素的患者的死亡率风险低于未使用者。曾接受过脊柱侧弯手术的个体的死亡率风险低于未接受过脊柱侧弯手术的个体。曾使用过咳嗽辅助的个体的死亡率风险低于从未使用过咳嗽辅助的个体。非西班牙裔黑人个体的死亡率风险高于非西班牙裔白人个体。使用心脏药物治疗和使用双水平气道正压通气治疗的患者在死亡率风险方面无差异。糖皮质激素的观察结果与 2018 年的护理考虑声明一致,即糖皮质激素的使用会持续到非运动阶段。我们的观察结果可能为 DMD 患者的临床护理提供信息。

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