Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy; Pneumology Unit - University Hospital Pediatric Department, Bambino Gesù Children Hospital, IRCCS, Rome, Italy; Cardiovascular Science, Cardio-Thoracic Surgery Unit, Catholic University, A. Gemelli Hospital, Rome, Italy.
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
J Thorac Cardiovasc Surg. 2017 Mar;153(3):669-674. doi: 10.1016/j.jtcvs.2016.08.016. Epub 2016 Aug 28.
We report our experience with the use of a left ventricular assist device (LVAD) as destination therapy (DT) for the management of patients with cardiac end-stage dystrophinopathies.
From February 2011 to February 2016, 7 patients with dystrophinopathies and dilated cardiomyopathy (DCM) were treated with LVADs at our institution. Median age at surgery was 16.5 years (range, 14.2-23.4 years). All patients were preoperatively evaluated by a multidisciplinary team approach.
All patients survived to hospital discharge. The early postoperative course was characterized by abdominal bleeding (1 patient) and retropharyngeal bleeding (1 patient). Because of abdominal or retropharyngeal bleeding, both required postoperative heparin infusion discontinuation for 35 and 33 days, respectively. Among the late complications, 1 child developed osteolysis and infection at the pedestal site of the device, which required surgical displacement; 1 patient required gastrostomy as a result of poor feeding, and another had a cerebral stroke, which was treated with percutaneous thrombus aspiration. The other 2 patients did not show early or late complications. At a median follow-up time of 21.7 months (range, 3-45 months) there have been 3 deaths: 1 patient died of a lung infection after 45 months, 1 died of tracheal bleeding after 29 months, and 1 died of cerebral hemorrhage after 14 months.
Our experience indicates that the use of an LVAD as DT in patients with dystrophinopathies with end-stage DCM is feasible, suggesting that it may be suitable as a palliative therapy for the treatment of these patients with no other therapeutic options.
我们报告使用左心室辅助装置（LVAD）作为终末期肌营养不良症患者的心脏的治疗终点（DT）的经验。
从 2011 年 2 月至 2016 年 2 月，我们机构对 7 例肌营养不良症合并扩张型心肌病（DCM）患者采用 LVAD 进行治疗。手术时的中位年龄为 16.5 岁（范围，14.2-23.4 岁）。所有患者均采用多学科团队方法进行术前评估。
所有患者均存活至出院。术后早期病程的特点是腹部出血（1 例）和咽后出血（1 例）。由于腹部或咽后出血，这两例患者均需要分别中断术后肝素输注 35 天和 33 天。在晚期并发症中，1 例患儿因设备基座部位发生骨溶解和感染而需要手术移位；1 例患儿因喂养不良而需要进行胃造口术，另 1 例患儿发生脑卒，经皮血栓抽吸治疗。另外 2 例患者未出现早期或晚期并发症。在中位随访时间 21.7 个月（范围，3-45 个月）内有 3 例死亡：1 例患者在 45 个月后死于肺部感染，1 例患者在 29 个月后死于气管出血，1 例患者在 14 个月后死于脑出血。
我们的经验表明，LVAD 作为终末期 DCM 肌营养不良症患者的 DT 使用是可行的，这表明它可能适合作为这些无其他治疗选择的患者的姑息性治疗。
