STS INTERMACS Database: The Key to Conduct Single-arm Trials in Advanced Heart Failure Patients.

Abstract Background Safe introduction of novel mechanical circulatory support (MCS) devices into clinical practice is a challenging process. Single-arm trials using a control arm from existing database is an effective alternative that could be applied for regulatory approval. This study analyzes the capability of the Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS) database to establish objective performance criteria and select patient population that could be used for future single-arm MCS trials. Methods Patients with INTERMACS profiles IM1-2 and IM3-5, who underwent implant of isolated Left Ventricular Assist Devises between 2014-2017 were included. Both cohorts were further stratified into Shock and Non-Shock groups using surrogate markers of shock (ECMO, temporary VAD, vasopressor infusions). Survival, transplant rates, adverse events, 6 min walk test and quality of life measures were obtained for all 4 groups at 6 and 12 months. Results Total of 7,907 patients were divided into IM1-2(n=3,909), IM3-5(n=3,998), Shock(n=3,469) and Non-Shock(n=3,040). Re-categorization occurred in 11% of patients from the IM3-5 group into the Shock group. Overall, patients in the Shock group had similar outcomes to the IM 1-2 group (1-year survival: 86% vs 85%, p=0.74). Patients in the Non-Shock group also had similar outcomes to the IM 3-5 (1-year survival: 90% vs 90%, p=0.43). Conclusions INTERMACS database can successfully establish objective performance criteria and concurrent control group for single-arm trials that could be used to support regulatory approval of new, less invasive MCS. INTERMACS data allows reliable comparisons of outcomes and adverse events.