Background:Cardiomyopathy is a common complication among muscular dystrophy (MD) patients and often results in advanced heart failure and premature death. In spite of this, there is hesitancy to consider heart transplantation (HTx). This study describes the HTx outcomes in patients with MD in the United States.Methods and Results:All HTx in the United Network for Organ Sharing database from October 1, 1987, to March 31, 2016, were identified. Two patient groups were created: MD cohort (n=81), and a cohort of all other cardiomyopathies, called cardiomyopathy-unmatched (n=41 317). Propensity score matching (ratio 1:2) was performed on transplant age, gender transplant year, renal function, and inotropic support at transplant to form a cardiomyopathy-matched cohort (n=162). Patient characteristics and posttransplant outcomes were compared. In the 81 patients with MD, Becker was the most common type (42%–52%). All the analyzed preoperative characteristics did not statistically differ between the MD and cardiomyopathy-matched cohorts except ventricular assist device use (16% versus 30%; P=0.017), ventilator support (0% versus 6%; P=0.031), and donor race mismatch (30% versus 55%; P<0.001). Median time on waitlist was not statistically different between the 2 groups (52 versus 59 days; P=0.12). Posttransplant survival of MD cohort was not statistically different compared with cardiomyopathy-matched cohort (P=0.18; hazard ratio [95% CI], 0.71 [0.42–1.18]) and was better than the cardiomyopathy-unmatched cohort (P=0.004; hazard ratio [95% CI], 0.53 [0.34–0.82]). Among the types of MD, no statistical difference was observed in posttransplant survival of Becker MD versus non-Becker MD (P=0.12; hazard ratio [95% CI], 2.17 [0.79–6.01]).Conclusions:Patients with MD undergoing HTx had similar long-term posttransplant survival compared with matched cardiomyopathy-related HTx recipients. HTx appears to be an effective treatment for a select group of muscular dystrophy patients with end-stage heart failure.

中文翻译：

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肌营养不良患者的心脏移植：是否可行？

背景：心肌病是肌肉营养不良（MD）患者的常见并发症，通常会导致晚期心力衰竭和过早死亡。尽管如此，仍不愿考虑进行心脏移植（HTx）。这项研究描述了美国MD患者的HTx结果。方法与结果：确定了1987年10月1日至2016年3月31日期间在美国器官共享网络联合数据库中所有HTx。创建了两个患者组：MD队列（n = 81）和所有其他心肌病的队列，称为无心肌病（n = 41 317）。根据移植年龄，性别移植年份，肾功能和移植时的正性肌力支持进行倾向得分匹配（比率1：2），以形成心肌病匹配的队列（n = 162）。比较患者特征和移植后结果。在81名MD患者中，贝克尔是最常见的类型（42％–52％）。MD和心肌病匹配组之间的所有分析的术前特征在统计学上均无统计学差异，除了使用心室辅助装置外（16％对30％；P = 0.017），呼吸机支撑（0％对6％；P = 0.031）和供体种族不匹配（30％对55％；P <0.001）。两组的等待时间中位数无统计学差异（52天与59天；P = 0.12）。MD队列的移植后存活率与心肌病匹配的队列相比无统计学差异（P = 0.18；危险比[95％CI]，0.71 [0.42-1.18]），并且优于无心肌病的队列（P = 0.004；危险比率[95％CI]，0.53 [0.34-0.82]）。在MD的类型中，Becker MD与非Becker MD的移植后存活率无统计学差异（P= 0.12; 风险比[95％CI]，2.17 [0.79–6.01]。）结论：与匹配的心肌病相关的HTx接受者相比，接受HTx治疗的MD患者的长期移植后生存期相似。HTx对于某些患有晚期心力衰竭的肌营养不良患者似乎是一种有效的治疗方法。