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

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 implantation of isolated left ventricular assist devices between 2014 and 2017, were included. Both cohorts were further stratified into shock and nonshock groups using surrogate markers of shock (extracorporeal membrane oxygenation, temporary ventricular assist device, vasopressor infusions). Survival, transplantation rates, adverse events, 6-minute walk test, and quality-of-life measures were obtained for all 4 groups at 6 and 12 months. RESULTS: Total of 7907 patients were divided into IM1-2 (n = 3909), IM3-5 (n = 3998), shock (n = 3469), and nonshock (n = 3040) groups. Recategorization 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 IM1-2 group (1-year survival: 86% vs 85%; P = .74). Patients in the nonshock group also had similar outcomes to the IM3-5 (1-year survival: 90% vs 90%; P = .43). CONCLUSIONS: The 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 allow reliable comparisons of outcomes and adverse events.

Effects of MetAP2 inhibition on hyperphagia and body weight in Prader-Willi syndrome: A randomized, double-blind, placebo-controlled trial.

AIMS: There are no treatments for the extreme hyperphagia and obesity in Prader-Willi syndrome (PWS). The bestPWS clinical trial assessed the efficacy, safety and tolerability of the methionine aminopeptidase 2 (MetAP2) inhibitor, beloranib. MATERIALS AND METHODS: Participants with PWS (12-65 years old) were randomly assigned (1:1:1) to biweekly placebo, 1.8 mg beloranib or 2.4 mg beloranib injection for 26 weeks at 15 US sites. Co-primary endpoints were the changes in hyperphagia [measured by Hyperphagia Questionnaire for Clinical Trials (HQ-CT); possible score 0-36] and weight by intention-to-treat. ClinicalTrials.gov registration: NCT02179151. RESULTS: One-hundred and seven participants were included in the intention-to-treat analysis: placebo (n = 34); 1.8 mg beloranib (n = 36); or 2.4 mg beloranib (n = 37). Improvement (reduction) in HQ-CT total score was greater in the 1.8 mg (mean difference -6.3, 95% CI -9.6 to -3.0; P = .0003) and 2.4 mg beloranib groups (-7.0, 95% CI -10.5 to -3.6; P = .0001) vs placebo. Compared with placebo, weight change was greater with 1.8 mg (mean difference - 8.2%, 95% CI -10.8 to -5.6; P < .0001) and 2.4 mg beloranib (-9.5%, 95% CI -12.1 to -6.8; P < .0001). Injection site bruising was the most frequent adverse event with beloranib. Dosing was stopped early due to an imbalance in venous thrombotic events in beloranib-treated participants (2 fatal events of pulmonary embolism and 2 events of deep vein thrombosis) compared with placebo. CONCLUSIONS: MetAP2 inhibition with beloranib produced statistically significant and clinically meaningful improvements in hyperphagia-related behaviours and weight loss in participants with PWS. Although investigation of beloranib has ceased, inhibition of MetAP2 is a novel mechanism for treating hyperphagia and obesity.

Incorporating Antiracism Coursework into a Cultural Competency Curriculum.

Creating a socially conscious educational environment is an imperative if health care practitioners are to have a significant impact on health inequities. The effects of practitioner bias, prejudice, and discrimination on health and health outcomes have been well documented in the literature. Individuals being trained to provide health care will be entering into an increasingly diverse world and must be equipped with the appropriate knowledge and skills in order to meet the needs of those seeking their care. Cultural competency training in medical education has evolved over the past 15 years since the Institute of Medicine's 2002 report, Unequal Treatment: Confronting Racial and Ethnic Disparities in Health Care. As research on the effectiveness of this training has emerged, several authors have called for the integration of antiracism training into the cultural competency curriculum, but few have found effective ways of doing so. This article describes the approach of one midwifery program in order to inform clinical education programs across the spectrum of health care practitioners.

Transfer from planned home birth to hospital: improving interprofessional collaboration.

Women's heightened interest in choice of birthplace and increased rates of planned home birth in the United States have been well documented, yet there remains significant public and professional debate about the ethics of planned home birth in jurisdictions where care is not clearly integrated across birth settings. Simultaneously, the quality of interprofessional interactions is recognized as a predictor of health outcomes during obstetric events. When care is transferred across birth settings, confusion and conflict among providers with respect to roles and responsibilities can adversely affect both outcomes and the experience of care for women and newborns. This article reviews findings of recent North American studies that examine provider attitudes toward planned home birth, differing concepts of safety of birthplace as reported by women and providers, and sources of conflict among maternity care providers during transfer from home to hospital. Emerging evidence and clinical exemplars can inform the development of systems for seamless transfer of women and newborns from planned home births to hospital and improve experience and perceptions of safety among families and providers. Three successful models in the United States that have enhanced multidisciplinary cooperation and coordination of care across birth settings are described. Finally, best practice guidelines for roles, communication, and mutual accommodation among all participating providers when transfer occurs are introduced. Research, health professional education, and policy recommendations for incorporation of key components into existing health care systems in the United States are included.