Children's National Heart Institute, Children's National Hospital, Washington, District of Columbia, USA
D2A, Los Angeles, California, USA.
Open Heart. 2021 Mar;8(1). doi: 10.1136/openhrt-2021-001592.
Cardiac disease is now the leading cause of death in Duchenne muscular dystrophy (DMD). Clinical evaluations over time have demonstrated asymptomatic cardiac troponin elevations and acute elevations are associated with symptoms and cardiac dysfunction in DMD. Clinicians require a better understanding of the relationship of symptoms, troponin levels and progression of cardiac disease in DMD. As clinical trials begin to assess novel cardiac therapeutics in DMD, troponin levels in DMD are important for safety monitoring and outcome measures. The Parent Project Muscular Dystrophy convened an expert panel of cardiologists, scientists, and regulatory and industry specialists on 16 December 2019 in Silver Spring, Maryland and reviewed published and unpublished data from their institutions. The panel recommended retrospective troponin data analyses, prospective longitudinal troponin collection using high-sensitivity cardiac troponin I assays, inclusion of troponin in future clinical trial outcomes and future development of clinical guidelines for monitoring and treating troponin elevations in DMD.
心脏疾病目前是杜氏肌营养不良症（DMD）的主要致死原因。随着时间的推移，临床评估显示无症状性心肌肌钙蛋白升高，急性升高与 DMD 中的症状和心功能障碍有关。临床医生需要更好地了解 DMD 中心律失常症状、肌钙蛋白水平和疾病进展之间的关系。随着临床试验开始评估新型 DMD 心脏治疗药物，DMD 中的肌钙蛋白水平对于安全性监测和结果测量非常重要。肌营养不良症父母项目组织心脏病专家、科学家、监管和行业专家于 2019 年 12 月 16 日在马里兰州银泉召开了一次专家小组会议，审查了他们机构的已发表和未发表的数据。专家组建议进行回顾性肌钙蛋白数据分析，前瞻性纵向采集使用高敏心肌肌钙蛋白 I 检测的肌钙蛋白，将肌钙蛋白纳入未来的临床试验结果中，并为监测和治疗 DMD 中心肌钙蛋白升高制定临床指南。
