Wittlieb-Weber Carol A, Villa Chet R, Conway Jennifer, Bock Matthew J, Gambetta Katheryn E, Johnson Jonathan N, Lal Ashwin K, Schumacher Kurt R, Law Sabrina P, Deshpande Shriprasad R, West Shawn C, Friedland-Little Joshua M, Lytrivi Irene D, McCulloch Michael A, Butts Ryan J, Weber David R, Knecht Kenneth R
Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, United States of America.
The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States of America.
Prog Pediatr Cardiol. 2019 Jun;53:11-14. doi: 10.1016/j.ppedcard.2019.01.001. Epub 2019 Jan 11.
As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) improve with glucocorticoid therapy and respiratory advances, the proportion of cardiac deaths is increasing. Little is known about the use and outcomes of advanced heart failure (HF) therapies in this population.
A retrospective cohort study of 436 males with DMD was performed, from January 1, 2005-January 1, 2018, with the primary outcome being use of advanced HF therapies including: implantable cardioverter defibrillator (ICD), left ventricular assist device (LVAD), and heart transplantation (HTX).
Nine subjects had an ICD placed, 2 of whom (22.2%) had appropriate shocks for ventricular tachycardia; 1 and 968 days after implant, and all of whom were alive at last follow-up; median 18 (IQR: 12.5-25.5) months from implant. Four subjects had a LVAD implanted with post-LVAD survival of 75% at 1 year; 2 remaining on support and 1 undergoing HTX. One subject was bridged to HTX with ICD and LVAD and was alive at last follow-up, 53 months after HTX.
Advanced HF therapies may be used effectively in select subjects with DMD. Further studies are needed to better understand risk stratification for ICD use and optimal candidacy for LVAD implantation and HTX, with hopes of improving cardiac outcomes.
随着糖皮质激素治疗和呼吸支持技术的进步，杜氏肌营养不良症（DMD）患者的生存率和神经肌肉功能得到改善，心脏死亡的比例却在增加。对于该人群中晚期心力衰竭（HF）治疗的使用情况和结局知之甚少。
对2005年1月1日至2018年1月1日期间的436例男性DMD患者进行了一项回顾性队列研究，主要结局是使用包括植入式心脏复律除颤器（ICD）、左心室辅助装置（LVAD）和心脏移植（HTX）在内的晚期HF治疗。
9例患者植入了ICD，其中2例（22.2%）因室性心动过速接受了适当的电击；分别在植入后1天和968天，所有患者在最后一次随访时均存活；植入后中位时间为18（IQR：12.5 - 25.5）个月。4例患者植入了LVAD，LVAD植入后1年的生存率为75%；2例仍在接受支持治疗，1例接受了HTX。1例患者通过ICD和LVAD过渡到HTX，在最后一次随访时存活，HTX后53个月。
晚期HF治疗可能在部分DMD患者中有效应用。需要进一步研究以更好地理解ICD使用的风险分层以及LVAD植入和HTX的最佳候选资格，以期改善心脏结局。
