Background And Objectives: In the rare disease primary hyperoxaluria type 1, overproduction of oxalate by the liver causes kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. Lumasiran, an RNA interference therapeutic, suppresses glycolate oxidase, reducing hepatic oxalate production. The objective of this first-in-human, randomized, placebo-controlled trial was to evaluate the safety, pharmacokinetic, and pharmacodynamic profiles of lumasiran in healthy participants and patients with primary hyperoxaluria type 1.
View Article and Find Full Text PDFBackground: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate that leads to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. Lumasiran, an investigational RNA interference (RNAi) therapeutic agent, reduces hepatic oxalate production by targeting glycolate oxidase.
Methods: In this double-blind, phase 3 trial, we randomly assigned (in a 2:1 ratio) patients with PH1 who were 6 years of age or older to receive subcutaneous lumasiran or placebo for 6 months (with doses given at baseline and at months 1, 2, 3, and 6).
Background: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.
Methods: In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with polyneuropathy, in a 2:1 ratio, to receive intravenous patisiran (0.3 mg per kilogram of body weight) or placebo once every 3 weeks.
Background And Objective: Despite the increasing use of data derived from randomized controlled trials (RCTs) to perform observational studies, little is known about the validity of this approach. We compared inferences from studies that were performed using Global Utilization of Streptokinase and t-PA for Occluded Coronary Arteries (GUSTO) RCT data with those derived from studies using data from the population-based Cooperative Cardiovascular Project (CCP).
Methods: We performed a systematic review.
Background: Many hospitals use critical pathways to attempt to reduce postoperative length of stay (PLOS) for diverse conditions and procedures.
Objective: To evaluate whether critical pathways were associated with reductions in postoperative PLOS after accounting for prepathway trends in PLOS.
Research Design: Retrospective cohort study, from 1988 to 1998.
Background: Underuse of coronary angiography is common among patients with acute myocardial infarction (AMI) and the magnitude of underuse varies across geographic areas.
Objectives: To examine the influence of patient demographic, clinical and hospital characteristics on underuse of coronary angiography, and the contribution of these factors to variation in underuse across geographic regions.
Research Design: Cohort study using data from the Cooperative Cardiovascular Project.
While the public and policy-makers place a priority on equity in the organ allocation process, several studies suggest that women may be less likely than men to receive a renal transplant. However, the cause of this disparity and whether it exists among children with end-stage renal disease (ESRD) are unknown. To address these issues, two nationally representative cohorts of incident patients were examined: (1) 7594 adults with ESRD onset between 1986 and 1993 for whom detailed data were available from the medical record on health status; and (2) 3217 patients <20 yr old who developed ESRD between 1988 and 1993.
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