Efficacy and safety of interventions for metastatic castration resistant prostate cancer (mCRPC) patients progressing on androgen receptor-axis-targeted (ARAT) therapy: a systematic literature review.

Background: To review the literature to outline findings from clinical trials assessing interventions for metastatic castration-resistant prostate cancer (mCRPC) in patients who have progressed on androgen receptor-axis-targeted (ARAT) therapies.

Methods: A systematic literature review was performed to identify trials that assessed the efficacy and safety of interventions used in patients that progressed on prior ARAT therapies. A literature search was conducted using the OVID platform that searched the EMBASE, MEDLINE, and CENTRAL bibliographic databases.

Humanistic burden of mucopolysaccharidoses: a systematic literature review.

Objective: To systematically review the literature and summarize the health-related quality-of-life (HRQoL) of patients undergoing treatment for mucopolysaccharidoses (MPS), a rare, hereditary lysosomal storage disorder.

Methods: A systematic review was performed in accordance with PRISMA guidelines to identify research studies that describe the humanistic burden of MPS. A comprehensive literature search was conducted in EMBASE, MEDLINE, and eligible conferences were screened to include applicable abstracts.

Caregiving for Older Adults With Dementia During the Time of COVID-19: A Multi-State Exploratory Qualitative Study.

This qualitative semi-structured interview study explores how 64 family caregivers for older adults with Alzheimer's Disease and related dementias across eight states experienced and executed caregiving decisions before and during the COVID-19 pandemic. First, caregivers experienced challenges communicating with loved ones and healthcare workers in all care settings. Second, caregivers displayed resilient coping strategies in adapting to pandemic restrictions, finding novel strategies to balance risks while preserving communication, oversight, and safety.

Breast Cancer Screening Utilization and Outcomes in Women With Neurofibromatosis Type 1.

Introduction: Women with neurofibromatosis type 1 (NF1) have up to a 5-fold increased risk for breast cancer before age 50 and a 3.5-fold increased risk of breast cancer overall. The purpose of our study was to assess breast cancer screening utilization and outcomes in this population.

An Enhanced Recovery After Surgery protocol for robotic-assisted laparoscopic nephrectomies utilizing a quadratus lumborum block.

Enhanced Recovery After Surgery (ERAS) protocols have been developed in several fields to reduce hospitalization lengths and overall costs. There have also been developments in multimodal analgesia methods to curtail opioid usage after surgery. Herein, we present the results of our initiation of an ERAS protocol for robotic-assisted laparoscopic partial and radical nephrectomies, employing a quadratus lumborum (QL) regional anesthetic block.

Quality of Life in Wilson's Disease: A Systematic Literature Review.

Wilson's disease (WD) is a rare inherited genetic disorder characterized by the progressive accumulation of copper in the brain, liver, and other major organ systems. To date, there have been no comprehensive studies synthesizing evidence pertaining to the quality of life (QOL) in WD. We conducted a systematic literature review to identify and synthesize the evidence on QOL in patients with WD.

Piriformis syndrome: muscle thickness or volume does not correlate with response to CT-guided injection.

Objective: To determine whether size of the piriformis muscle, as characterized by either the coronal width or a segmented volume, correlates with outcomes after CT-guided injections.

Materials And Methods: A consecutive series of 81 patients with piriformis syndrome received CT-guided injections of the sciatic nerve and piriformis muscle. Volume and thickness measurements of the piriformis were taken from T1W and T2W pre-injection images by two readers.

The Impact of Digital Therapeutics on Current Health Technology Assessment Frameworks.

Historically healthcare has been delivered offline (e.g., physician consultations, mental health counseling services).

The RGG domain in the C-terminus of the DEAD box helicases Dbp2 and Ded1 is necessary for G-quadruplex destabilization.

The identification of G-quadruplex (G4) binding proteins and insights into their mechanism of action are important for understanding the regulatory functions of G4 structures. Here, we performed an unbiased affinity-purification assay coupled with mass spectrometry and identified 30 putative G4 binding proteins from the fission yeast Schizosaccharomyces pombe. Gene ontology analysis of the molecular functions enriched in this pull-down assay included mRNA binding, RNA helicase activity, and translation regulator activity.

Pharmacogenetic testing to guide therapeutic decision-making and improve outcomes for children undergoing anthracycline-based chemotherapy.

Anthracyclines are widely used as part of chemotherapeutic regimens in paediatric oncology patients. The most serious adverse drug reaction caused by anthracycline use is cardiotoxicity, a serious condition that can lead to cardiac dysfunction and subsequent heart failure. Both clinical and genetic factors contribute to a patient's risk of experiencing anthracycline-induced cardiotoxicity.

Heterogeneity in renal end points of cardiovascular outcomes trials in Type 2 diabetes.

Composite renal end points and end stage renal disease (ESRD) are frequently included as prespecified secondary end points in the cardiovascular outcomes trials (CVOTs) of diabetes medications. We examined the heterogeneity in the definitions of composite renal end point and ESRD in CVOTs. Five criteria (macroalbuminuria, doubling of serum creatinine, estimated glomerular filtration rate [GFR], ESRD and renal death), were considered for the renal composite end point across the trials.

Piriformis syndrome: pain response outcomes following CT-guided injection and incremental value of botulinum toxin injection.

Purpose: Piriformis syndrome is a common pain condition affecting the buttock and posterior hip with or without radiation to the leg, and management of the condition involves many treatments. In this study, we hypothesize that a CT-guided injection with botulinum toxin is more effective in providing pain relief than a CT-guided injection without Botox.

Methods: Overall, 97 consecutive patients with piriformis syndrome presented for a CT-guided injection of the piriformis muscle and perineural injection of the sciatic nerve.

Poly(A)-Binding Protein Regulates the Efficiency of Translation Termination.

Multiple factors influence translation termination efficiency, including nonsense codon identity and immediate context. To determine whether the relative position of a nonsense codon within an open reading frame (ORF) influences termination efficiency, we quantitate the production of prematurely terminated and/or readthrough polypeptides from 26 nonsense alleles of 3 genes expressed in yeast. The accumulation of premature termination products and the extent of readthrough for the respective premature termination codons (PTCs) manifest a marked dependence on PTC proximity to the mRNA 3' end.

Can Standard Health Technology Assessment Approaches Help Guide the Price of Orphan Drugs in Canada? A Review of Submissions to the Canadian Agency for Drugs and Technologies in Health Common Drug Review.

Orphan drugs have high acquisition costs and when standard health technology assessment (HTA) approaches are used to assess their cost-effectiveness, they often appear not cost-effective. The Canadian Patented Medicine Review Board (PMPRB), through new regulations, will apply HTA assessment results from the Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d'excellence en santé et en services sociaux (INESSS) when setting the maximum price that can be charged for Category I patented medicines (treatments with an annual cost exceeding 150% of GDP per capita of Canada or with expected annual market size >$50M). Through these regulations, PMPRB has also established a willingness-to-pay threshold of CAD$200,000 or CAD$150,000 per quality adjusted life year (QALY) for medications with a prevalence of no more than 1 in 2000 across all approved indications.

Epidemiology of pediatric multiple sclerosis: A systematic literature review and meta-analysis.

Background: Multiple sclerosis (MS) is a debilitating immune disease leading to demyelination, neurodegeneration, and chronic inflammation of the central nervous system. Pediatric MS is a rare form of the disease and effects approximately 2-10% of individuals with MS. Diagnostic criteria and therapies are continuously evolving, thus it is imperative to further understand the epidemiology and subsequently global and regional disease burden of pediatric MS.

Two Decades of Nursing Home Compare: What Have We Learned?

Approximately two decades ago, federally mandated public reporting began for U.S. nursing homes through a system now known as Nursing Home Compare.

Saccharomyces cerevisiae Mus81-Mms4 prevents accelerated senescence in telomerase-deficient cells.

Alternative lengthening of telomeres (ALT) in human cells is a conserved process that is often activated in telomerase-deficient human cancers. This process exploits components of the recombination machinery to extend telomere ends, thus allowing for increased proliferative potential. Human MUS81 (Mus81 in Saccharomyces cerevisiae) is the catalytic subunit of structure-selective endonucleases involved in recombination and has been implicated in the ALT mechanism.

Epidemiology of Atypical Hemolytic Uremic Syndrome: A Systematic Literature Review.

Atypical hemolytic uremic syndrome (aHUS) is a rare but severe disorder that frequently has a genetic component and results from the overactivation of the alternative complement pathway. As research moves toward improved diagnosis and therapy of aHUS, it will be important to better understand its epidemiology. Our objective was to conduct a systematic literature review to assess the incidence and prevalence estimates of aHUS globally.

Reslizumab and mepolizumab for moderate-to-severe poorly controlled asthma: an indirect comparison meta-analysis.

A systematic literature review and network meta-analysis assessed the efficacy and safety of reslizumab 3.0 mg/kg and mepolizumab 100 mg. Eligible studies evaluated reslizumab and mepolizumab in patients with inadequately-controlled severe eosinophilic asthma.

Advanced MR imaging of bone marrow: quantification of signal alterations on T1-weighted Dixon and T2-weighted Dixon sequences in red marrow, yellow marrow, and pathologic marrow lesions.

Objectives: To quantify and compare signal intensity (SI) changes on T1-weighted (W) and T2W Dixon imaging in yellow marrow, red marrow, and bone marrow lesions.

Materials And Methods: A total of 141 patients (77 controls, 64 lesions-33 benign, 31 malignant) between January 2016 and December 2017 were retrospectively identified. For the control group, fixed 2-cm region of interests (ROI) were drawn at L5, bilateral ilium and femurs on in-phase and opposed-phase T1W and T2W Dixon images.