Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data.

Objective: The natural history of Friedreich ataxia is being investigated in a multi-center longitudinal study designated the Friedreich ataxia Clinical Outcome Measures Study (FACOMS). To understand the utility of this study in analysis of clinical trials, we performed a propensity-matched comparison of data from the open-label MOXIe extension (omaveloxolone) to that from FACOMS.

Methods: MOXIe extension patients were matched to FACOMS patients using logistic regression to estimate propensity scores based on multiple covariates: sex, baseline age, age of onset, baseline modified Friedreich Ataxia Rating scale (mFARS) score, and baseline gait score.

Effects of donation frequency on U.S. source plasma donor health.

Background: Plasma-derived medicinal products (PDMPs) are essential, life-saving medicines manufactured from plasma donated by healthy human volunteers. PDMPs are used to treat a range of rare, serious, and chronic conditions, often genetic in origin. Approximately 70% of the Source Plasma (SP) used for PDMP manufacturing comes from United States (US).

A Multicenter Study of the Causes and Consequences of Optimistic Expectations About Prognosis by Surrogate Decision-Makers in ICUs.

Objectives: Optimistic expectations about prognosis by surrogate decision-makers in ICUs are common, but there are few data about the causes and clinical consequences. Our objective was to determine the causes of optimistic expectations about prognosis among surrogates and whether it is associated with more use of life support at the end of life.

Design: Prospective, multicenter cohort study from 2009 to 2012.

A Randomized Trial of a Family-Support Intervention in Intensive Care Units.

Background: Surrogate decision makers for incapacitated, critically ill patients often struggle with decisions related to goals of care. Such decisions cause psychological distress in surrogates and may lead to treatment that does not align with patients' preferences.

Methods: We conducted a stepped-wedge, cluster-randomized trial involving patients with a high risk of death and their surrogates in five intensive care units (ICUs) to compare a multicomponent family-support intervention delivered by the interprofessional ICU team with usual care.

Procalcitonin-Guided Use of Antibiotics for Lower Respiratory Tract Infection.

Background: The effect of procalcitonin-guided use of antibiotics on treatment for suspected lower respiratory tract infection is unclear.

Methods: In 14 U.S.