Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, 3333 Burnet Ave, MLC 2003, Cincinnati, OH, 45229, USA.
Department of Pediatrics, Division of Cardiology, University of Colorado, Denver Anschutz Medical Campus, Children's Hospital Colorado Aurora, Aurora, CO, USA.
Pediatr Cardiol. 2022 Jun;43(5):977-985. doi: 10.1007/s00246-021-02807-7. Epub 2022 Jan 13.
Cardiac disease has emerged as a leading cause of mortality in Duchenne muscular dystrophy in the current era. This survey sought to identify the diagnostic and therapeutic approach to DMD among pediatric cardiologists in Advanced Cardiac Therapies Improving Outcomes Network. Pediatric cardiology providers within ACTION (a multi-center pediatric heart failure learning network) were surveyed regarding their approaches to cardiac care in DMD. Thirty-one providers from 23 centers responded. Cardiac MRI and Holter monitoring are routinely obtained, but the frequency of use and indications for ordering these tests varied widely. Angiotensin converting enzyme inhibitor and aldosterone antagonist are generally initiated prior to onset of systolic dysfunction, while the indications for initiating beta-blocker therapy vary more widely. Seventeen (55%) providers report their center has placed an implantable cardioverter defibrillator in at least 1 DMD patient, while 11 providers (35%) would not place an ICD for primary prevention in a DMD patient. Twenty-three providers (74%) would consider placement of a ventricular assist device (VAD) as destination therapy (n = 23, 74%) and three providers (10%) would consider a VAD only as bridge to transplant. Five providers (16%) would not consider VAD at their institution. Cardiac diagnostic and therapeutic approaches vary among ACTION centers, with notable variation present regarding the use of advanced therapies (ICD and VAD). The network is currently working to harmonize medical practices and optimize clinical care in an era of rapidly evolving outcomes and cardiac/skeletal muscle therapies.
在当前时代，心脏病已成为杜氏肌营养不良症患者死亡的主要原因。本调查旨在确定先进心脏治疗改善预后网络（ACTION）中的儿科心脏病专家对 DMD 的诊断和治疗方法。ACTION 中的儿科心脏病学提供者就他们在 DMD 中的心脏护理方法接受了调查。来自 23 个中心的 31 名提供者做出了回应。心脏 MRI 和 Holter 监测通常是常规进行的，但这些检查的使用频率和适应证差异很大。血管紧张素转换酶抑制剂和醛固酮拮抗剂通常在收缩功能障碍出现之前开始使用，而开始β受体阻滞剂治疗的适应证则更为广泛。17 名（55%）提供者报告其中心至少在 1 名 DMD 患者中放置了植入式心脏复律除颤器，而 11 名（35%）提供者不会为 DMD 患者的一级预防放置 ICD。23 名（74%）提供者会考虑将心室辅助装置（VAD）作为终末期治疗（n=23，74%），3 名（10%）提供者仅会考虑将 VAD 作为心脏移植的桥接。5 名（16%）提供者不会考虑在他们的机构中使用 VAD。ACTION 中心的心脏诊断和治疗方法存在差异，在使用先进治疗方法（ICD 和 VAD）方面存在明显差异。该网络目前正在努力协调医疗实践，优化在快速发展的治疗方法和心脏/骨骼肌治疗时代的临床护理。
