Prospective Trial of a Pediatric Ventricular Assist Device

Abstract

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.).

Favorable Waitlist and Posttransplant Outcomes in Children and Adolescent Patients Supported With Durable Continuous-Flow Ventricular Assist Devices

“…In the present study, we found that overall waitlist survival of patients on CFVADs at 30 (95%), 90 (92%), and 180 days (88%) compares favorably with the aggregated pediatric pulsatile VAD experience (2,3,22). Of note, unlike adult patients, pediatric patients remain status 1a on the transplant waiting list, even if they are discharged home.…”

“…Multivariable analysis was not performed in light of the low event numbers. For the same reason, caution must be applied in interpreting these predictors, although the requirement for biventricular support has been consistently observed as a risk factor in the pediatric pulsatile-flow VAD literature (2,3,23,24).…”

“…This may mean that the early risk associated with device implantation is not fully captured in these data, a period that coincides with greatest hazard in many VAD outcome studies (3,24). Finally, many important comorbidities are not captured in this data set, and the large proportions of missing data precluded detailed analysis of neurological outcomes in these patients-a point that would be of great interest, given the prevalence of these comorbidities in pediatric patients implanted with a pulsatile VAD (2,24). Many of these comorbidity concerns will be addressed specifically through analysis of the Pedimacs registry.…”

Funding Grants for Rare Diseases at the Food and Drug Administration

“…One last example is of a pivotal, multicenter study that evaluated the safety and probable benefit of the Berlin Heart Pediatric Ventricular Assist Device (EXCOR Pediatric System) as a bridge to heart transplant or successful weaning in 48 children with severe heart failure [9]. Although heart transplantation offers relief from symptoms of severe heart failure, there are fewer pediatric-sized donor hearts available for transplantation than for adults, thereby prolonging the waiting period in children until transplantation can occur [10].…”

“…Although heart transplantation offers relief from symptoms of severe heart failure, there are fewer pediatric-sized donor hearts available for transplantation than for adults, thereby prolonging the waiting period in children until transplantation can occur [10]. This study compared children with severe end-stage heart failure requiring mechanical support as a bridge to transplant who underwent implantation of EXCOR, with a similar group of historical controls who received circulatory support with extracorporeal membrane oxygenation (ECMO) [9]. Survival in each of the two cohorts (cohorts based on size: < 0.7 m 2 , and ‡ 0.7 m 2 < 1.5 m 2 ) receiving EXCOR mechanical support exceeded 88% [9].…”