Evaluation of FDA Labeling Changes Related to PREA Safety-Waivers.

Purpose: The Pediatric Research Equity Act (PREA) gives the US Food and Drug Administration (FDA) authority to require pediatric studies for drug and biologics products under certain circumstances and to waive this requirement in some, or all, pediatric ages. When studies are waived for safety, PREA stipulates the safety issue must be described in labeling. This study assessed the rate of including waiver-related safety information in labeling.

Model-Informed Approach Supporting Approval of Nexviazyme (Avalglucosidase Alfa-ngpt) in Pediatric Patients with Late-Onset Pompe Disease.

In August 2021, the US Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease (LOPD). The effectiveness and safety were studied in patients with LOPD and patients with infantile-onset Pompe disease (IOPD). The dosage(s) tested in clinical trials was 20 mg/kg every other week (qow) in patients with LOPD and 20 mg/kg and 40 mg/kg qow in patients with IOPD.

Noninferiority Trials to Evaluate Drug Effects in Rheumatoid Arthritis.

Objective: The increased availability of highly effective treatments in rheumatoid arthritis (RA) necessitates a reexamination of study designs evaluating new treatments. We undertook this study to discuss possible specifications and considerations of noninferiority (NI) trials assessing drug effects in RA.

Methods: We focused on the use of approved tumor necrosis factor inhibitors (TNFi) as potential active controls and reviewed previous placebo-controlled studies.

Temporal relationship between uric acid concentration and risk of diabetes in a community-based study population.

Some observational studies have identified elevated uric acid concentration as a risk factor for diabetes, while others have found an inverse relationship. We examined both the association of uric acid level with incident diabetes and the change in uric acid concentration after a diabetes diagnosis. We analyzed data from the Atherosclerosis Risk in Communities (ARIC) Study and quantified the independent association between uric acid level and incident diabetes via Cox proportional hazards models.

Racial differences in gout incidence in a population-based cohort: Atherosclerosis Risk in Communities Study.

We examined racial differences in gout incidence among black and white participants in a longitudinal, population-based cohort and tested whether racial differences were explained by higher levels of serum urate. The Atherosclerosis Risk in Communities Study is a prospective, US population-based cohort study of middle-aged adults enrolled between 1987 and 1989 with ongoing annual follow-up through 2012. We estimated the adjusted hazard ratios and 95% confidence intervals of incident gout by race among 11,963 men and women using adjusted Cox proportional hazards models.

Risk factors for incident hyperuricemia during mid-adulthood in African American and white men and women enrolled in the ARIC cohort study.

Background: Increased serum urate levels are associated with poor outcomes including but not limited to gout. It is unclear whether serum urate levels are the sole predictor of incident hyperuricemia or whether demographic and clinical risk factors also predict the development of hyperuricemia. The goal of this study was to identify risk factors for incident hyperuricemia over 9 years in a population-based study, ARIC.

Hypertension and the risk of incident gout in a population-based study: the atherosclerosis risk in communities cohort.

The authors quantified the impact of hypertension on gout incidence in middle-aged white and African American men and women. The Atherosclerosis Risk in Communities Study (ARIC) was a prospective population-based cohort that recruited patients between 1987 and 1989 from 4 US communities. Using a time-dependent Cox proportional hazards model, the authors estimated the adjusted hazard ratio (HR) of incident gout by time-varying hypertension and tested for mediation by serum urate level.

Anemia and the onset of gout in a population-based cohort of adults: Atherosclerosis Risk in Communities study.

Introduction: There is a growing prevalence of gout in the US and worldwide. Gout is a recognized risk factor for cardiovascular disease (CVD). It is unclear whether other risk factors for CVD are also associated with increased risk of gout.

A urate gene-by-diuretic interaction and gout risk in participants with hypertension: results from the ARIC study.

Objective: To test for a urate gene-by-diuretic interaction on incident gout.

Methods: The Atherosclerosis Risk in Communities Study is a prospective population-based cohort of 15 792 participants recruited from four US communities (1987-1989). Participants with hypertension and available single nucleotide polymorphism (SNP) genotype data were included.

Incident gout in women and association with obesity in the Atherosclerosis Risk in Communities (ARIC) Study.

Background: We hypothesized that women with early- and mid-adult life obesity, as well as high mid-adult life waist-to-hip ratios, and high weight gain during adulthood, experience a greater incidence of gout.

Methods: We examined the incidence of gout in the Atherosclerosis Risk in Communities Study, a population-based biracial cohort comprised of individuals aged 45-65 years at baseline (1987-1989). A total of 6263 women without prior history of gout were identified.

Low socioeconomic status is associated with cardiovascular risk factors and outcomes in systemic lupus erythematosus.

Objective: Accelerated atherosclerosis is a major cause of death in systemic lupus erythematosus (SLE), yet little is known about the effect of socioeconomic status. We investigated whether education or income levels are associated with cardiovascular risk factors and outcomes in SLE.

Methods: Our study involved a longitudinal cohort of all patients with SLE enrolled in the Hopkins Lupus Cohort from 1987 through September 2011.

Diuretic use, increased serum urate levels, and risk of incident gout in a population-based study of adults with hypertension: the Atherosclerosis Risk in Communities cohort study.

Objective: To quantify the role of diuretic use in gout development in an adult population with hypertension.

Methods: The Atherosclerosis Risk in Communities study, a prospective population-based cohort from 4 US communities, consisted of 4 visits over a 9-year period. Participants were included in this analysis if they answered a query about gout, were free of gout at baseline, and had hypertension (defined as taking medication to treat hypertension or having blood pressure of ≥140/90 mm Hg).

Obesity and younger age at gout onset in a community-based cohort.

Objective: Obesity is associated with gout risk. It is unclear whether obesity is associated with a younger age at gout onset. We examined whether obesity is related to age at gout onset and quantified the risk of incident gout by obesity status in the Campaign Against Cancer and Heart Disease (CLUE II) study, a longitudinal community-based cohort.

Reliability and sensitivity of the self-report of physician-diagnosed gout in the campaign against cancer and heart disease and the atherosclerosis risk in the community cohorts.

Objective: gout is often defined by self-report in epidemiologic studies. Yet the validity of self-reported gout is uncertain. We evaluated the reliability and sensitivity of the self-report of physician-diagnosed gout in the Campaign Against Cancer and Heart Disease (CLUE II) and the Atherosclerosis Risk in the Community (ARIC) cohorts.

Testicular pain followed by microscopic hematuria, a renal mass, palpable purpura, polyarthritis, and hematochezia.

We present the instructive case of a man who developed progressively severe testicular pain. He sought the medical care of several physicians, including general medicine, urology, and rheumatology. What began with focal testicular pain evolved over the ensuing weeks to a multisystem disorder affecting at least 3 additional organ systems beyond the genitourinary tract.

Vitamin D treatment for the prevention of falls in older adults: systematic review and meta-analysis.

Objectives: To systematically review and quantitatively synthesize the effect of vitamin D therapy on fall prevention in older adults.

Design: Systematic review and meta-analysis.

Setting: MEDLINE, CINAHL, Web of Science, EMBASE, Cochrane Library, LILACS, bibliographies of selected articles, and previous systematic reviews through February 2009 were searched for eligible studies.

Secondary Sjogren's syndrome in systemic lupus erythematosus defines a distinct disease subset.

Objective: Sjögren's syndrome (SS) may occur in patients with systemic lupus erythematosus (SLE). We sought to determine whether the presence of SS in a large cohort of patients with SLE defines a subset with distinctive sociodemographic, clinical, and laboratory features.

Methods: The Johns Hopkins Lupus Cohort was divided into 2 groups, based on the presence or absence of SS, defined by the presence of an objective measure of sicca or an abnormal minor salivary gland biopsy in a patient with sicca symptoms.