title: Postapproval Outcomes: The Berlin Heart EXCOR Pediatric in North America.
abstract: The Berlin Heart EXCOR Pediatric Ventricular Assist Device (BH) was approved for use in the United States in December 2011, based on a prospective investigational device exemption (IDE) trial. Strict exclusion criteria for the trial selected a low-risk "ideal" cohort. We sought to determine whether postapproval usage of the BH in a "real world" cohort of recipients would result in similar outcomes. Preimplant diagnostic information was collected for all patients. Efficacy was evaluated by comparison of all children (efficacy group, n = 247) implanted between FDA approval and April 2015 to those in the IDE trial (IDE, n = 48), with regard to achievement of one of four end-states: transplanted, successful weaning, death/unsuccessful weaning, or still-on-device. Safety outcomes were compared between IDE patients and a subset of postapproval patients (safety group, n = 39) for whom adjudicated adverse events were tracked in a regulator-mandated dataset. Diagnostic categories were similar between groups: IDE (congenital 19%, dilated cardiomyopathy/myocarditis/other 81%) versus Efficacy Group (congenital 24%, dilated cardiomyopathy/myocarditis/other 75%). Patients in the IDE cohort were larger (median 14.8 kg, range 3.6-58.1 kg vs. 10.7 kg, 2.9-112.0 kg, p = 0.02). More IDE patients were successfully supported than in the efficacy group cohort (90% vs. 77%, p = 0.05). Proportions with bleeding and stroke were similar between the IDE and safety group cohorts (46% vs. 41%, p = 0.65; 29% vs. 33%, p = 0.68, respectively). With usage of the BH in a less-ideal population, rates of bridge to transplant and weaning have declined slightly, but remain encouragingly high. Bleeding and neurologic event rates have not increased.
authors.affiliation: From the *Duke Children's Hospital, Durham, North Carolina; †The Hospital for Sick Children, Toronto, Ontario, Canada; ‡St. Louis Children's Hospital, St. Louis, Missouri; §Cincinnati Children's Hospital, Cincinnati, Ohio; ¶Stanford University, Palo Alto, California; and ‖Texas Children's Hospital, Houston, Texas.
authors.forename: Robert D B
authors.initials: RD
authors.lastname: Jaquiss
authors.forename: Tilman
authors.initials: T
authors.lastname: Humpl
authors.forename: Charles E
authors.initials: CE
authors.lastname: Canter
authors.forename: David L S
authors.initials: DL
authors.lastname: Morales
authors.forename: David N
authors.initials: DN
authors.lastname: Rosenthal
authors.forename: Charles D
authors.initials: CD
authors.lastname: Fraser
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D018795:Device Approval; D006353:Heart-Assist Devices / Q000009:adverse effects*; D006801:Humans; D007223:Infant; D009656:North America; D011446:Prospective Studies