McCulloch Michael A, Lal Ashwin K, Knecht Kenneth, Butts Ryan J, Villa Chet R, Johnson Jonathan N, Conway Jennifer, Bock Matthew J, Schumacher Kurt R, Law Sabrina P, Friedland-Little Joshua M, Deshpande Shriprasad R, West Shawn C, Lytrivi Irene D, Gambetta Katheryn E, Wittlieb-Weber Carol A
Division of Pediatric Cardiology, University of Virginia Children's Hospital, PO Box 800386, Charlottesville, VA, 22903, USA.
Primary Children's Hospital, University of Utah, Salt Lake City, UT, USA.
Pediatr Cardiol. 2020 Jun;41(5):925-931. doi: 10.1007/s00246-020-02336-9. Epub 2020 Mar 10.
Duchenne muscular dystrophy (DMD) is characterized by myocardial fibrosis and left ventricular (LV) dysfunction. Implantable cardioverter defibrillator (ICD) use has not been characterized in this population but is considered for symptomatic patients with severe LV dysfunction (SLVD) receiving guideline-directed medical therapy (GDMT). We evaluated ICD utilization and efficacy in patients with DMD. Retrospective cohort study of DMD patients from 17 centers across North America between January 2, 2005 and December 31, 2015. ICD use and its effect on survival were evaluated in patients with SLVD defined as ejection fraction (EF) < 35% and/ or shortening fraction (SF) < 16% on final echocardiogram. SLVD was present in 57/436 (13.1%) patients, of which 12 (21.1%) died during the study period. Of these 12, (mean EF 20.9 ± 6.2% and SF 13.7 ± 7.2%), 8 received GDMT, 5 received steroids, and none received an ICD. ICDs were placed in 9/57 (15.8%) patients with SLVD (mean EF 31.2 ± 8.5% and SF 10.3 ± 4.9%) at a mean age of 20.4 ± 6.3 years; 8/9 received GDMT, 7 received steroids, and all were alive at study end; mean ICD duration was 36.1 ± 26.2 months. Nine ICDs were implanted at six different institutions, associated with two appropriate shocks for ventricular tachycardia in two patients, no inappropriate shocks, and one lead fracture. ICD use may be associated with improved survival and minimal complications in DMD cardiomyopathy with SLVD. However, inconsistent GDMT utilization may be a significant confounder. Future studies should define optimal indications for ICD implantation in patients with DMD cardiomyopathy.
杜氏肌营养不良症（DMD）的特征是心肌纤维化和左心室（LV）功能障碍。植入式心脏复律除颤器（ICD）在该人群中的使用情况尚未明确，但对于接受指南指导药物治疗（GDMT）的重度左心室功能障碍（SLVD）的症状性患者会考虑使用。我们评估了DMD患者中ICD的使用情况和疗效。对2005年1月2日至2015年12月31日期间来自北美17个中心的DMD患者进行回顾性队列研究。对定义为最终超声心动图射血分数（EF）<35%和/或缩短分数（SF）<16%的SLVD患者评估ICD的使用情况及其对生存的影响。57/436（13.1%）例患者存在SLVD，其中12例（21.1%）在研究期间死亡。在这12例患者中（平均EF 20.9±6.2%，SF 13.7±7.2%），8例接受了GDMT，5例接受了类固醇治疗，无1例接受ICD治疗。9/57（15.8%）例SLVD患者（平均EF 31.2±8.5%，SF 10.3±4.9%）植入了ICD，平均年龄为20.4±6.3岁；8/9例接受了GDMT，7例接受了类固醇治疗，研究结束时均存活；平均ICD使用时长为36.1±26.2个月。9台ICD在6个不同机构植入，2例患者因室性心动过速接受了2次恰当电击，无不恰当电击，1例发生导线断裂。在伴有SLVD的DMD心肌病中，使用ICD可能与生存率提高及并发症最少相关。然而，GDMT使用不一致可能是一个显著的混杂因素。未来的研究应明确DMD心肌病患者ICD植入的最佳指征。
