Comparative effectiveness of disease modifying antirheumatic drugs for patients with cardiac sarcoidosis.

Objectives: We aimed to evaluate the comparative efficacy of disease modifying antirheumatic drugs (DMARDs) for patients with cardiac sarcoidosis.

Methods: We performed a retrospective cohort study of new users of methotrexate, mycophenolate, or azathioprine for sarcoidosis using the US-based TriNetX electronic health records database from 2008-2023. Hazard ratios were calculated using inverse probability of treatment weighted Cox proportional hazards regressions to compare the efficacy of DMARDs with respect to delaying major adverse cardiac events among patients with cardiac sarcoidosis and preventing cardiac sarcoidosis from developing among patients with non-cardiac sarcoidosis.

Barriers to CAR T-cell therapy in rheumatology.

Chimeric antigen receptor (CAR) T cells have recently shown remarkable promise in treating rheumatic diseases, including systemic lupus erythematosus (SLE), idiopathic inflammatory myopathies, and systemic sclerosis. Currently, there are 37 clinical trials registered for CAR T-cell therapy in rheumatic diseases and many more are being planned. Much of this enthusiasm is justifiable, but widespread adoption of CAR T-cell therapy in rheumatology faces several barriers.

Incidence of Pneumonia and Prophylaxis-Associated Adverse Events Among Patients With Systemic Lupus Erythematosus.

Objective: pneumonia (PJP) is an opportunistic infection that may affect patients with systemic lupus erythematosus (SLE). The objective of this project was to describe the incidence of PJP among patients with SLE.

Methods: A retrospective cohort analysis of the TriNetX database was conducted.

Accuracy of continuous glucose monitoring in the hospital setting: an observational study.

Aims/hypothesis: Continuous glucose monitoring (CGM) improves glycaemic outcomes in the outpatient setting; however, there are limited data regarding CGM accuracy in hospital.

Methods: We conducted a prospective, observational study comparing CGM data from blinded Dexcom G6 Pro sensors with reference point of care and laboratory glucose measurements during participants' hospitalisations. Key accuracy metrics included the proportion of CGM values within ±20% of reference glucose values >5.

Advances in diabetes technology to improve the lives of people with cystic fibrosis.

People with cystic fibrosis (CF) are at risk for dysglycaemia caused by progressive beta cell dysfunction and destruction due to pancreatic exocrine disease and fibrosis. CF-related diabetes (CFRD) is a unique form of diabetes that has distinctive features from both type 1 and type 2 diabetes. Recent advances in diabetes technology may be of particular benefit in this population given the complex, multi-system organ involvement and challenging health issues that people with CFRD often face.

The State of Patient-Reported Outcome Measures in Rheumatology.

Objective: We sought to evaluate the quality and timeliness of patient-reported outcome (PRO) measure reporting, which have not been previously studied.

Methods: Clinical trials that informed new US Food and Drug Administration (FDA) approvals for the first rheumatological indication between 1995 and 2021 were identified. Data were recorded to determine whether collected PROs were published, met minimum clinically important difference (MCID) or statistical significance ( < 0.

Proximal femur fracture detection on plain radiography via feature pyramid networks.

Hip fractures exceed 250,000 cases annually in the United States, with the worldwide incidence projected to increase by 240-310% by 2050. Hip fractures are predominantly diagnosed by radiologist review of radiographs. In this study, we developed a deep learning model by extending the VarifocalNet Feature Pyramid Network (FPN) for detection and localization of proximal femur fractures from plain radiography with clinically relevant metrics.

Impact of Glucocorticoid Dose on Complete Response, Serious Infections, and Mortality During the Initial Therapy of Lupus Nephritis: A Systematic Review and Meta-Analysis of the Control Arms of Randomized Controlled Trials.

Objective: Our objective was to evaluate the effect of glucocorticoid regimens on renal response, infections, and mortality among patients with lupus nephritis (LN).

Methods: We performed a systematic review and meta-analysis of the control arms of randomized clinical trials (RCTs). We included RCTs of biopsy-proven LN that used a protocolized regimen of glucocorticoids in combination with mycophenolic acid analogs or cyclophosphamide and reported the outcomes of complete response (CR), serious infections, and death.

Risk of Neuroinflammatory Diseases Among New Recipients of Biologic and Targeted Synthetic Disease-Modifying Antirheumatic Drugs.

Objective: Neuroinflammatory adverse events have been observed among new users of tumor necrosis factor (TNF) inhibitors. No studies to date have compared the real-world risk of TNFs with other new users of biologic or targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs). The objective of this study is to describe the risk of neuroinflammatory disease after initiation b/tsDMARDs.

Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force.

The COVID-19 pandemic necessitated a rapid shift in clinical research to perform virtual visits and remote endpoint assessments, providing a key opportunity to optimize the use of remote endpoints for clinical trials in cystic fibrosis. The use of remote endpoints could allow more diverse participation in clinical trials while minimizing participant burden but must be robustly evaluated to ensure adequate performance and feasibility. In response, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplemental Table 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members tasked to better understand the current and future use of remote endpoints for clinical research.

Continuous glucose monitoring and advanced glycation endproducts for prediction of clinical outcomes and development of cystic fibrosis-related diabetes in adults with CF.

Introductions: Cystic fibrosis-related diabetes (CFRD) is associated with pulmonary decline, compromised nutritional status, and earlier mortality. Onset is often insidious, so screening for early detection of glycemic abnormalities is important. Continuous glucose monitoring (CGM) has been validated in people with CF and has been shown to detect early glycemic variability otherwise missed on 2-hour oral glucose tolerance testing (OGTT).

Association between biological immunotherapy for psoriasis and time to incident inflammatory arthritis: a retrospective cohort study.

Background: Targeted biological immunotherapies have been highly effective in controlling skin disease in patients with psoriasis, but whether therapy delays progression to inflammatory arthritis is unclear. The aim of this study was to compare the time to incident inflammatory arthritis among patients newly receiving biological therapies for psoriasis.

Methods: In this retrospective cohort study, we obtained data on a national sample of patients in the USA from the electronic health records database of the US-based TriNetX network.

Unintentional unblinding in rheumatic disease trials.

The practice of blinding treatment assignment in randomised controlled trials mitigates important biases in observational studies. Unblinding, whereby study participants or investigators become aware of treatment assignments, is an important threat to the validity of trial results. Rheumatology studies might be particularly susceptible to unblinding because rheumatic disease therapies often cause high rates of idiosyncratic side-effects and frequently rely on subjective endpoints.

Fragility of randomised controlled trials for systemic lupus erythematosus and lupus nephritis therapies.

Objective: We aimed to evaluate the robustness of phase III randomised controlled trials (RCTs) for SLE and lupus nephritis (LN) using the fragility index (FI), the reverse FI (RFI) and the fragility quotient (FQ).

Methods: We searched for phase III RCTs that included patients with active SLE or LN. Data on primary endpoints, total participants and the number of events for each arm were obtained.

Nutritional status of adults with phenylketonuria on pegvaliase: A 15-month prospective study.

Background: Pegvaliase has allowed many adults with phenylketonuria (PKU) to achieve acceptable blood Phe control while eating an unrestricted diet. However, little is known about potential differences in nutritional status and eating behaviors after transitioning from a phenylalanine (Phe)-restricted to an unrestricted diet. Here we assessed change in nutritional status in adults with early-treated PKU who were consuming a Phe-restricted diet (intact protein ≤0.