Statistical considerations for cardiovascular clinical trials straddling the continuum of pandemic phases.

In 2020, the NIH and FDA issued guidance documents that laid the foundation for human subject research during an unprecedented pandemic. To bridge these general considerations to actual applications in cardiovascular interventional device trials, the PAndemic Impact on INTErventional device ReSearch (PAIINTERS) Working Group was formed in early 2021 under the Predictable And Sustainable Implementation Of National CardioVascular Registries (PASSION CV Registries). The PAIINTER's Part I report, published by Rymer et al.

Elexacaftor-Tezacaftor-Ivacaftor Therapy for Chronic Sinus Disease in Cystic Fibrosis.

Importance: Cystic fibrosis (CF) is a multiorgan genetic disease with progressive upper and lower airway involvement. The effects of CF transmembrane conductance regulator (CFTR) modifier therapies on CF-related upper airway disease, specifically chronic rhinosinusitis (CRS), are not characterized.

Objective: To determine the outcome of elexacaftor-tezacaftor-ivacaftor (ETI) on CRS as measured by changes in sinus computed tomography (CT) metrics and on clinical parameters in individuals with CF.

Impact of Elexacaftor-Tezacaftor-Ivacaftor on lung disease in cystic fibrosis.

Background: In people with cystic fibrosis (pwCF), the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, such as Elexacaftor-Tezacaftor-Ivacaftor (ETI), on structural changes in the lungs is unclear.

Objective: To determine the impact of ETI on clinical parameters and on structural lung disease as measured by the changes in the chest computed tomography (CT) scans in pwCF.

Methods: Percent predicted forced expiratory volume in one second (ppFEV1), body mass index (BMI), and microbiologic data were collected at initiation and 3-month intervals for 1 year.

Impact of chronic medication de-escalation in patients with cystic fibrosis taking elexacaftor, tezacaftor, ivacaftor: A retrospective review.

Background: This single-center, retrospective study evaluated the effects of de-escalating cystic fibrosis (CF) supportive therapies in patients on elexacaftor/tezacaftor/ivacaftor (ETI). For many with CF, the clinical benefit of ETI exceeds that of supportive therapies. Therefore, we anticipated patients would desire to discontinue many of their supportive therapies, leading to the creation of a de-escalation algorithm.

Clinical change 2 years from start of elexacaftor-tezacaftor-ivacaftor in severe cystic fibrosis.

Rationale: Limited published research is available on the impact of elexacaftor/tezacaftor/ivacaftor (ETI) beyond the initial few months postdrug initiation, especially for those who initiated therapy via individual investigational new drug application. The experiences of patients with cystic fibrosis (CF) experiencing severe lung disease were reviewed for significant improvements in clinical symptoms and quality of life.

Objectives: To examine clinical outcomes 2 years post-ETI in patients with CF and advanced lung disease.

Accuracy of Three Commercial Home-Use Hemoglobin A1c Tests.

The COVID-19 pandemic and the rapid expansion of telemedicine have increased the need for accurate and reliable capillary hemoglobin A1c (HbA1c) testing. Nevertheless, validation studies of commercially available products suitable for home use have been in short supply. Three commercial home-use capillary blood sample HbA1c tests (Home Access, CoreMedica, and A1cNow+) were evaluated in 219 participants with type 1 or type 2 diabetes (4-80 years years of age, HbA1c 5.

An Evaluation of Two Capillary Sample Collection Kits for Laboratory Measurement of HbA1c.

The COVID-19 pandemic has impacted the conduct of clinic visits. We conducted a study to evaluate two academic laboratories' fingerstick capillary blood collection kits suitable for home use for laboratory measurement of HbA1c. Four clinical sites recruited 240 participants (aged 4-80 years, HbA1c 5.

Evaluating Glucose Control With a Novel Composite Continuous Glucose Monitoring Index.

Objective: The objective was to describe a novel composite continuous glucose monitoring index (COGI) and to evaluate its utility, in adults with type 1 diabetes, during hybrid closed-loop (HCL) therapy and multiple daily injections (MDI) therapy combined with real-time continuous glucose monitoring (CGM).

Methods: COGI consists of three key components of glucose control as assessed by CGM: Time in range (TIR), time below range (TBR), and glucose variability (GV) (weighted by 50%, 35% and 15%). COGI ranges from 0 to 100, where 1% increase of time <3.

Accuracy, Utilization, and Effectiveness Comparisons of Different Continuous Glucose Monitoring Systems.

Background: Accuracy and feature sets of continuous glucose monitoring (CGM) systems may influence device utilization and outcomes. We compared clinical trial accuracy and real-world utilization and effectiveness of two different CGM systems.

Materials And Methods: Separately conducted accuracy studies of a fifth-generation and a sixth-generation CGM system involved 50 and 159 adults, respectively.

Performance of a Factory-Calibrated, Real-Time Continuous Glucose Monitoring System in Pediatric Participants With Type 1 Diabetes.

Background: The perceived value and consistent use of continuous glucose monitoring (CGM) systems depends in part on their accuracy. We assessed the performance of a sixth-generation CGM system (Dexcom G6) in children and adolescents.

Methods: Forty-nine participants (6-17 years of age, mean ± SD of 13.

Resistance to Acetaminophen Interference in a Novel Continuous Glucose Monitoring System.

Acetaminophen (APAP) can cause erroneously high readings in real-time continuous glucose monitoring (rtCGM) systems. APAP-associated bias in an investigational rtCGM system (G6) was evaluated by taking the difference in glucose measurements between rtCGM and YSI from 1 hour before to 6 hours after a 1-g oral APAP dose in 66 subjects with type 1 or type 2 diabetes. The interference effect was defined as the average post-dose (30-90 minutes) bias minus the average baseline bias for each subject.

Assessment of occupational exposure to heat stress and solar ultraviolet radiation among groundskeepers in an eastern North Carolina university setting.

Groundskeepers spend most of the year working outdoors, exposing them to heat and solar ultraviolet (UV) radiation and increasing their risk to related adverse health effects. Various studies on heat and UV exposures in different occupations have been published, but those on groundskeepers are rare. The purpose of this study was to assess the exposure to heat stress and solar UV radiation among groundskeepers in an eastern North Carolina university setting.

Computed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation.

Background: Ivacaftor corrects the cystic fibrosis transmembrane conductance regulator (CFTR) gating defect associated with G551D mutation and is quickly becoming an important treatment in patients with cystic fibrosis (CF) due to this genetic mutation.

Methods: A single-center study was performed in CF patients receiving ivacaftor to evaluate the usefulness of high resolution computed tomography (HRCT) of the chest as a way to gauge response to ivacaftor therapy.

Results: Ten patients with CF were enrolled for at least one year before and after starting ivacaftor.

Wilcoxon's signed-rank statistic: what null hypothesis and why it matters.

In statistical literature, the term 'signed-rank test' (or 'Wilcoxon signed-rank test') has been used to refer to two distinct tests: a test for symmetry of distribution and a test for the median of a symmetric distribution, sharing a common test statistic. To avoid potential ambiguity, we propose to refer to those two tests by different names, as 'test for symmetry based on signed-rank statistic' and 'test for median based on signed-rank statistic', respectively. The utility of such terminological differentiation should become evident through our discussion of how those tests connect and contrast with sign test and one-sample t-test.

Trends in the treatment of localized prostate cancer using supplemented cancer registry data.

Objective: To conduct an analysis of localized prostate cancer treatment in the USA between 1998 and 2002.

Patients And Methods: Results from the National Cancer Institute's Patterns of Care study from 10 regional cancer registries in 1998 and 14 registries in 2002 were compared using univariate and multivariate statistical methods.

Results: Patients with localized prostate cancer in 2002 were younger, had lower prostate-specific antigen values, and higher Gleason scores compared with those diagnosed in 1998.

Racial/Ethnic differences in functional outcomes in the 5 years after diagnosis of localized prostate cancer.

Purpose: We investigated racial/ethnic differences in functional outcomes up to 5 years after diagnosis among men with aggressively treated localized prostate cancer.

Patients And Methods: Patients were from the Prostate Cancer Outcomes Study, a population-based cohort study that surveyed patients at 6, 12, 24, and 60 months after diagnosis. Analyses were stratified by primary treatment.

Developing and implementing lifestyle management programs© with people with multiple sclerosis.

SUMMARY This paper describes the development and use of Lifestyle Management Programs© (LMPs) by occupational therapists at the Multiple Sclerosis Society of New South Wales, Australia. A case study is used to demonstrate how the program is applied in an individual situation. LMPs are used with people with MS, or their support people, to enable management of the impact of fluctuating and interactive physical, sensory and cognitive symptoms.