背景:没有FDA批准的左心室辅助装置(LVAD)用于允许常规出院的较小儿童。接受LVAD支持的较小的儿童通常会住院数月,等待心脏移植,这是家庭的主要负担,也是医院的挑战。我们描述了Jarvik2015的初始结果,这是一种小型化的植入式连续流LVAD,在NHLBI资助的儿童泵中,婴儿,和新生儿(PumpKIN)研究,用于心脏桥移植.
方法:在美国7个中心招募了体重8至30公斤的重度收缩性心力衰竭且未通过最佳药物治疗的儿童。排除严重右心衰竭和单心室先天性心脏病患者。主要可行性终点为无严重卒中或非操作设备失效的存活30天。
结果:在植入的7名儿童中,中位年龄为2.2岁(范围0.7,7.1岁),中位体重10公斤(8.2~20.7);86%患有扩张型心肌病;29%为INTERMACS谱1.Jarvik2015年支持的中位持续时间为149天(范围5至188天),所有7名儿童均存活,包括5名接受心脏移植的儿童。1恢复,和1转换为近邻设备。在心肌恢复的情况下,一名患者在装置支持的第53天经历了缺血性中风。一名患者需要ECMO支持治疗顽固性室性心律失常,并最终从体外双室VAD支持中移植。中值泵速度为1600RPM,功率范围为1-4瓦。在第7、30、90和180天或外植体时间,血浆游离血红蛋白中位数分别为19、30、19和30mg/dL,分别。所有患者均达到主要可行性终点。患者报告的使用该装置的结果在参与所有活动方面是有利的。由于与制造商的财务问题,本研究在第8例患者同意后暂停.
结论:Jarvik2015LVAD作为可支持出院的较小儿童的植入式连续流装置似乎具有重要的前景。FDA已批准该设备进行22个主题的关键试验。由于寻求开发儿科医疗设备的行业面临的财务挑战,该设备是否会存活到商业化仍不清楚。(由NIH/NHLBIHHS合同N268201200001I支持,clinicaltrials.gov02954497)。
BACKGROUND: There is no FDA-approved left ventricular assist device (LVAD) for smaller children permitting routine hospital discharge. Smaller children supported with LVADs typically remain hospitalized for months awaiting heart transplant-a major burden for families and a challenge for hospitals. We describe the initial outcomes of the Jarvik 2015, a miniaturized implantable continuous flow LVAD, in the NHLBI-funded Pumps for Kids, Infants, and Neonates (PumpKIN) study, for bridge-to-heart transplant.
METHODS: Children weighing 8 to 30 kg with severe systolic heart failure and failing optimal medical therapy were recruited at 7 centers in the United States. Patients with severe right heart failure and single-ventricle congenital heart disease were excluded. The primary feasibility endpoint was survival to 30 days without severe stroke or non-operational device failure.
RESULTS: Of 7 children implanted, the median age was 2.2 (range 0.7, 7.1) years, median weight 10 (8.2 to 20.7) kilograms; 86% had dilated cardiomyopathy; 29% were INTERMACS profile 1. The median duration of Jarvik 2015 support was 149 (range 5 to 188) days where all 7 children survived including 5 to heart transplant, 1 to recovery, and 1 to conversion to a paracorporeal device. One patient experienced an ischemic stroke on day 53 of device support in the setting of myocardial recovery. One patient required ECMO support for intractable ventricular arrhythmias and was eventually transplanted from paracorporeal biventricular VAD support. The median pump speed was 1600 RPM with power ranging from 1-4 Watts. The median plasma free hemoglobin was 19, 30, 19 and 30 mg/dL at 7, 30, 90 and 180 days or time of explant, respectively. All patients reached the primary feasibility endpoint. Patient-reported outcomes with the device were favorable with respect to participation in a full range of activities. Due to financial issues with the manufacturer, the study was suspended after consent of the eighth patient.
CONCLUSIONS: The Jarvik 2015 LVAD appears to hold important promise as an implantable continuous flow device for smaller children that may support hospital discharge. The FDA has approved the device to proceed to a 22-subject pivotal trial. Whether this device will survive to commercialization remains unclear because of the financial challenges faced by industry seeking to develop pediatric medical devices. (Supported by NIH/NHLBI HHS Contract N268201200001I, clinicaltrials.gov 02954497).