Modeling cystic fibrosis patient prognosis: Nomograms to predict lung transplantation and survival prior to highly effective modular therapy.

Background: The duration of time a person with cystic fibrosis (pwCF) spends on the lung transplant waitlist is dependent on waitlist and post-transplant survival probabilities and can extend up to 2 years. Understanding the characteristics involved with lung transplant and survival prognoses may help guide decision making by the patient, the referring CF Center and the transplant team.

Methods: This study seeks to identify clinical predictors of lung transplant and survival of individuals with CF using 29,847 subjects from 2003-2014 entered in the Cystic Fibrosis Foundation Patient Registry (CFFPR).

Growth and Symmetry of the Levator Veli Palatini Muscle Within the First Two Years of Life.

Objective: To quantify differences in levator veli palatini (LVP) muscle dimensions based on age, sex, and race and determine the typical range of asymmetry between the left and right sides of the LVP under age 2.

Design: Retrospective cohort study.

Setting: Children's tertiary care hospital.

Craniofacial and Velopharyngeal Dimensions in Infants 0-12 Months: Between- and Within-Group Differences Based on Age and Sex.

Purpose: The purpose of the present study is to (a) provide quantitative data on the growth of levator veli palatini (LVP), velopharyngeal (VP), and craniofacial dimensions in children under 12 months while controlling for corrected age and sex and (b) compare variability within age and sex groups.

Method: Magnetic resonance imaging scans of 75 infants between 0 and 12 months were measured and divided into four age groups. These data were obtained as part of a larger retrospective study.

Reducing Central Nervous System-Active Medications to Prevent Falls and Injuries Among Older Adults: A Cluster Randomized Clinical Trial.

Importance: High-risk medications that contribute to adverse health outcomes are frequently prescribed to older adults. Deprescribing interventions reduce their use, but studies are often not designed to examine effects on patient-relevant health outcomes.

Objective: To test the effect of a health system-embedded deprescribing intervention targeting older adults and their primary care clinicians for reducing the use of central nervous system-active drugs and preventing medically treated falls.

Engaging staff to improve COVID-19 vaccination response at long-term care facilities (ENSPIRE): A cluster randomized trial of co-designed, tailored vaccine promotion materials.

Background: COVID-19 vaccination rates among long-term care center (LTCC) workers are among the lowest of all frontline health care workers. Current efforts to increase COVID-19 vaccine uptake generally focus on strategies that have proven effective for increasing influenza vaccine uptake among health care workers including educational and communication strategies. Experimental evidence is lacking on the comparative advantage of educational strategies to improve vaccine acceptance and uptake, especially in the context of COVID-19.

Expectancy for Adderall influences subjective mood and drug effects regardless of concurrent caffeine ingestion: A randomized controlled trial.

Rationale: Nonmedical prescription stimulant use (NPS; use without a prescription or in ways other than prescribed) is common among college students. Despite the potential for negative consequences, students continue engaging in NPS for cognitive enhancement purposes, which may be maintained by expectancy and placebo effects.

Objectives: This study examined if a placebo administered under the guise of Adderall influenced subjective mood/drug effects and cognitive performance.

Growth Effects on Velopharyngeal Anatomy Within the First 2 Years of Life.

Purpose: Limited quantitative data exist regarding growth of the velopharynx within the first 2 years of life. The purpose of this study was to (a) quantify changes in velopharyngeal structures due to growth during the first 2 years of life, (b) examine the impact of sex and race within this age range, and (c) provide normative measures for comparison to individuals with cleft palate.

Method/description: A retrospective chart review was completed of all patients up to 24 months of age that underwent magnetic resonance imaging of the head for medical necessity within the past 18 months using a three-dimensional fluid-attenuated inversion recovery sequence.

Developing crash prediction models using parametric and nonparametric approaches for rural mountainous freeways: A case study on Wyoming Interstate 80.

Interstate 80 in Wyoming is one of the busiest freight corridors that is characterized with harsh winter conditions and challenging mountainous roadway sections. The fatality rates in Wyoming are always typically higher than the national level. The 402-mile I-80 corridor in Wyoming was selected by the USDOT FHWA for piloting connected vehicle technology to improve the safety and mobility of heavy trucks.

Analysis of a large cohort of cystic fibrosis patients with severe liver disease indicates lung function decline does not significantly differ from that of the general cystic fibrosis population.

Previous reports of lung function in cystic fibrosis (CF) patients with liver disease have shown worse, similar, or even better forced expiratory volume in 1 second (FEV1), compared to CF patients without liver disease. Varying definitions of CF liver disease likely contribute to these inconsistent relationships reported between CF lung function and liver disease. We retrospectively evaluated spirometric data in 179 subjects (62% male; 58% Phe508del homozygous) with severe CF liver disease (CFLD; defined by presence of portal hypertension due to cirrhosis).

Shared parameter models for joint analysis of longitudinal and survival data with left truncation due to delayed entry - Applications to cystic fibrosis.

Many longitudinal studies observe time to occurrence of a clinical event such as death, while also collecting serial measurements of one or more biomarkers that are predictive of the event, or are surrogate outcomes of interest. Joint modeling can be used to examine the relationship between the biomarker and the event, and also as a way of adjusting analyses of the biomarker for non-ignorable dropout. In settings such as registry studies, an additional complexity is caused when follow-up of subjects is delayed, referred to as left-truncation of follow-up in the survival analysis setting.

Balancing statistical and ethical considerations in planning clinical trials: recommendations for response-adaptive randomization urn designs.

During a clinical trial, balancing statistical and ethical considerations are important. Response-adaptive randomization methods use the information from past patients to increase the probability of the next patient receiving the better treatment while avoiding the statistical concern of selection bias. We compared three response-adaptive randomization urn designs, Randomized Play-the-Winner, Modified Play-the-Winner, and Birth-and-Death Urn with Immigration, to the traditional equal allocation design with respect to power and allocation of patients to the better treatment.

Jointly modeling the relationship between longitudinal and survival data subject to left truncation with applications to cystic fibrosis.

Numerous methods for joint analysis of longitudinal measures of a continuous outcome y and a time to event outcome T have recently been developed either to focus on the longitudinal data y while correcting for nonignorable dropout, to predict the survival outcome T using the longitudinal data y, or to examine the relationship between y and T. The motivating problem for our work is in joint modeling of the serial measurements of pulmonary function (FEV1% predicted) and survival in cystic fibrosis (CF) patients using registry data. Within the CF registry data, an additional complexity is that not all patients have been followed from birth; therefore, some patients have delayed entry into the study while others may have been missed completely, giving rise to a left truncated distribution.