Texas Children's Hospital and Baylor College of Medicine, Houston, Texas 77030, USA.
N Engl J Med. 2012 Aug 9;367(6):532-41. doi: 10.1056/NEJMoa1014164.
Options for mechanical circulatory support as a bridge to heart transplantation in children with severe heart failure are limited.
We conducted a prospective, single-group trial of a ventricular assist device designed specifically for children as a bridge to heart transplantation. Patients 16 years of age or younger were divided into two cohorts according to body-surface area (cohort 1, <0.7 m(2); cohort 2, 0.7 to <1.5 m(2)), with 24 patients in each group. Survival in the two cohorts receiving mechanical support (with data censored at the time of transplantation or weaning from the device owing to recovery) was compared with survival in two propensity-score-matched historical control groups (one for each cohort) undergoing extracorporeal membrane oxygenation (ECMO).
For participants in cohort 1, the median survival time had not been reached at 174 days, whereas in the matched ECMO group, the median survival was 13 days (P<0.001 by the log-rank test). For participants in cohort 2 and the matched ECMO group, the median survival was 144 days and 10 days, respectively (P<0.001 by the log-rank test). Serious adverse events in cohort 1 and cohort 2 included major bleeding (in 42% and 50% of patients, respectively), infection (in 63% and 50%), and stroke (in 29% and 29%).
Our trial showed that survival rates were significantly higher with the ventricular assist device than with ECMO. Serious adverse events, including infection, stroke, and bleeding, occurred in a majority of study participants. (Funded by Berlin Heart and the Food and Drug Administration Office of Orphan Product Development; ClinicalTrials.gov number, NCT00583661.).
在患有严重心力衰竭的儿童中，作为心脏移植桥接的机械循环支持选择有限。
我们进行了一项针对专为儿童设计的心室辅助装置作为心脏移植桥接的前瞻性、单组试验。根据体表面积将 16 岁或以下的患者分为两组（队列 1，<0.7 m2；队列 2，0.7 至<1.5 m2），每组 24 例患者。接受机械支持（数据在因恢复而移植或从装置脱机时被截短）的两组患者（与每个队列的两个匹配历史对照组）的生存情况（每个队列一个）与体外膜氧合（ECMO）的生存情况进行了比较。
对于队列 1 的参与者，中位生存时间未达到 174 天，而在匹配的 ECMO 组中，中位生存时间为 13 天（对数秩检验 P<0.001）。对于队列 2 和匹配的 ECMO 组的参与者，中位生存时间分别为 144 天和 10 天（对数秩检验 P<0.001）。队列 1 和队列 2 的严重不良事件包括大出血（分别为 42%和 50%的患者）、感染（分别为 63%和 50%）和中风（分别为 29%和 29%）。
我们的试验表明，与 ECMO 相比，心室辅助装置的生存率显著提高。大多数研究参与者发生了严重的不良事件，包括感染、中风和出血。（由柏林心脏和食品和药物管理局孤儿产品开发办公室资助；ClinicalTrials.gov 编号，NCT00583661）。
