A Framework for the Use and Likelihood of Regulatory Acceptance of Single-Arm Trials.

Background: Single-arm clinical trials (SAT) are common in drug and biologic submissions for rare or life-threatening conditions, especially when no therapeutic options exist. External control arms (ECAs) improve interpretation of SATs but pose methodological and regulatory challenges.

Objective: Through narrative reviews and expert input, we developed a framework for considerations that might influence regulatory use and likelihood of regulatory acceptance of an SAT, identifying non-oncology first indication approvals as an area of interest.

Skeletal muscle density performance for screening frailty in older adults with cancer and the impact of diabetes: The CARE Registry.

Introduction: Skeletal muscle density (SMD) measurements from imaging scans identify myosteatosis and could screen patients for geriatric assessment. We assessed SMD performance as a screening tool to identify older adults with cancer likely to be frail and who could benefit from in-depth assessment; we compared performance by sex and diabetes status.

Materials And Methods: We analyzed patients in the Cancer & Aging Resilience Evaluation (CARE) Registry.

Tendon Transfer to Treat Radial Nerve Palsy Following COVID-19 Infection.

Multiple manifestations have been associated with the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) virus. Among them are mononeuritis multiplex (MNM) and other neurological complications, whose connection to coronavirus disease 2019 (COVID-19) is still unclear. One of the most common sites of nerve injury is the radial nerve, which can be treated with both nerve or tendon transfer.

Geriatric Assessment Impairment Profiles and Mortality in Older Adults With Gastrointestinal Cancers: Latent Class Analysis of the CARE Registry.

Background: Many older adults with cancer have ≥2 impairments on geriatric assessment, which affects present and future frailty status, treatment tolerability, and outcomes. Our objective was to identify and describe distinct geriatric assessment impairment classes using latent class analysis (LCA) in older patients with gastrointestinal malignancies and assess 1-year mortality.

Methods: We used the Cancer & Aging Resilience Evaluation (CARE) Study, a registry of older adults (≥60 years) at University of Alabama at Birmingham.

Epidemiology and clinical characteristics of interstitial lung disease in patients with rheumatoid arthritis from the JointMan database.

Interstitial lung disease (ILD) is a progressive fibrotic disease associated with rheumatoid arthritis (RA); real-world data for evaluating RA-associated ILD (RA-ILD) are limited. We evaluated prevalence, time to onset, clinical characteristics and prognostic factors in patients diagnosed with RA (n = 8963) in the Discus Analytics JointMan database (2009-2019) with and without ILD. ILD prevalence was 4.

Suboptimal Clinical and Quality of Life Outcomes in Patients with Psoriasis Undertreated with Oral Therapies: International Physician and Patient Survey.

Introduction: Psoriasis is a chronic, inflammatory, immune-mediated disease. This study assessed the time at which patients switched from a conventional oral systemic treatment to a biologic therapy; patient clinical and quality of life (QoL) outcomes associated with oral systemic treatments; and the proportion of patients who persisted on oral therapy (nonswitchers), despite reported suboptimal clinical and QoL outcomes.

Methods: This data analysis used the Adelphi Real World Psoriasis Disease Specific Programme, a non-interventional, retrospective, cross-sectional survey conducted in the USA, France, Germany, and United Kingdom (August 2018-April 2019).

Real-world treatment patterns and healthcare costs in patients with psoriasis taking systemic oral or biologic therapies.

Background: Psoriasis is a chronic, immune-mediated, systemic inflammatory disorder associated with high costs. This study evaluated real-world treatment patterns and associated costs in patients in the United States with psoriasis initiating systemic oral or biologic treatments.

Methods: This retrospective cohort study used IBM (now Merative™) MarketScan Commercial and Medicare claims (1 January 2006-31 December 2019) to evaluate patterns of switching, discontinuation, and nonswitching in two cohorts of patients initiating oral or biologic systemic therapy.

Serious Cardiovascular Adverse Events Associated with Hydroxychloroquine/Chloroquine Alone or with Azithromycin in Patients with COVID-19: A Pharmacovigilance Analysis of the FDA Adverse Event Reporting System (FAERS).

Background: The use of hydroxychloroquine or chloroquine (HCQ/CQ) as monotherapy or combined with azithromycin for the treatment of coronavirus disease 2019 (COVID-19) may increase the risk of serious cardiovascular adverse events (SCAEs).

Objective: Our objective was to describe and evaluate the risk of SCAEs with HCQ/CQ as monotherapy or combined with azithromycin compared with that for therapeutic alternatives.

Methods: We performed a disproportionality analysis and descriptive case series using the US FDA Adverse Event Reporting System.

A systematic review of the patient burden of Crohn's disease-related rectovaginal and anovaginal fistulas.

Background: Crohn's disease (CD)-related rectovaginal fistulas (RVFs) and anovaginal fistulas (AVFs) are rare, debilitating conditions that present a substantial disease and treatment burden for women. This systematic literature review (SLR) assessed the burden of Crohn's-related RVF and AVF, summarizing evidence from observational studies and highlighting knowledge gaps.

Methods: This SLR identified articles in PubMed and Embase that provide data and insight into the patient experience and disease burden of Crohn's-related RVF and AVF.

Antioxidant Response Activating nanoParticles (ARAPas) localize to atherosclerotic plaque and locally activate the Nrf2 pathway.

Atherosclerotic disease is the leading cause of death world-wide with few novel therapies available despite the ongoing health burden. Redox dysfunction is a well-established driver of atherosclerotic progression; however, the clinical translation of redox-based therapies is lacking. One of the challenges facing redox-based therapies is their targeted delivery to cellular domains of redox dysregulation.

Evaluation of rivaroxaban-, apixaban- and dabigatran-associated hemorrhagic events using the FDA-Adverse event reporting system (FAERS) database.

Background Rivaroxaban, apixaban and dabigatran are non-vitamin K antagonist oral anticoagulants (NOACs) that are widely used for treatment or prevention of venous thromboembolism and stroke in patients with atrial fibrillation. Objective To estimate and compare hemorrhagic events report of rivaroxaban, apixaban and dabigatran. Setting FDA Adverse Event Reporting System (FAERS) database.

Increased risk of incident dementia following use of anticholinergic agents: A systematic literature review and meta-analysis.

Background/rationale: Long-term treatment with anticholinergic agents may increase the risk of cognitive impairment or dementia. This systematic literature review and meta-analysis aimed to assess the impact of ≥3 months of exposure to anticholinergics as a class on the risk of dementia, mild cognitive impairment, and change in cognitive function. The impact of anticholinergic agents specifically used to treat overactive bladder was also evaluated.

Dipeptidyl peptidase-4 inhibitors and risk of venous thromboembolism: data mining of FDA adverse event reporting system.

Background: A recent study is raising concerns that dipeptidyl peptidase 4 inhibitors are associated with increased risk of venous thromboembolism.

Objective: We aimed to assess the association between dipeptidyl peptidase-4 inhibitors and venous thromboembolism using the US Food and Drug Administration Adverse Event Reporting System database.

Methods: We searched the venous thromboembolism cases related to dipeptidyl peptidase-4 inhibitors from 2004 first quarter to 2018 first quarter.

Traditional Chinese medicine and drug-induced anaphylaxis: data from the Beijing pharmacovigilance database.

Background Traditional Chinese medicine (TCM) is one of the major triggers for drug-induced anaphylaxis (DIA). Objective We aimed to use the Beijing pharmacovigilance database (BPD) to analyze TCM-induced DIAs in Beijing, China. Setting Drug allergy case reports from the BPD provided by the Beijing Center for Adverse Drug Reaction Monitoring.

Controlling for Frailty in Pharmacoepidemiologic Studies of Older Adults: Validation of an Existing Medicare Claims-based Algorithm.

Background: Frailty is a geriatric syndrome characterized by weakness and weight loss and is associated with adverse health outcomes. It is often an unmeasured confounder in pharmacoepidemiologic and comparative effectiveness studies using administrative claims data.

Methods: Among the Atherosclerosis Risk in Communities (ARIC) Study Visit 5 participants (2011-2013; n = 3,146), we conducted a validation study to compare a Medicare claims-based algorithm of dependency in activities of daily living (or dependency) developed as a proxy for frailty with a reference standard measure of phenotypic frailty.

Development and validation of an algorithm to identify drug-induced anaphylaxis in the Beijing Pharmacovigilance Database.

Background Pharmacovigilance databases are utilized to identify serious adverse drug events (ADEs). In China, very few studies have evaluated the validity of using pharmacovigilance databases to identify drug-induced anaphylaxis (DIA). Objective We aimed to develop and validate an algorithm to identify DIA using the Beijing Pharmacovigilance Database (BPD).

Messages that matter: Age differences in affective responses to framed health messages.

Age differences in responses to framed health messages-which can influence judgments and decisions-are critical to understand yet relatively unexplored. Age-related emotional shifts toward positivity would be expected to differentially impact the affective responses of older and younger adults to framed messages. In this study, we measured the subjective and physiological affective responses of older and younger adults to gain- and loss-framed exercise promotion messages.