Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials.

Introduction: Fabry disease (FD) is a rare lysosomal storage disorder that is associated with pain and progressive damage to the renal, cardiac, and cerebrovascular systems. Enzyme replacement therapy (ERT) is one of the treatment options for FD and the most recently approved ERT agent, pegunigalsidase alfa, has shown clinical efficacy in three phase 3 clinical trials of adults with FD: BALANCE, BRIDGE, and BRIGHT. Recent published guidelines support the mapping of health utility state data to the EuroQol-5 Dimension-3 Level (EQ-5D-3L) index to align with the preferred methodology used by the National Institute for Health and Care Excellence (NICE).

Population health outcomes of blood-based screening for colorectal cancer in comparison to current screening modalities: insights from a discrete-event simulation model incorporating longitudinal adherence.

Aim: Insufficient adherence to colorectal cancer (CRC) screening impedes individual and population health benefits, with about one-third of individuals non-adherent to available screening options. The impact of poor adherence is inadequately considered in most health economics models, limiting the evaluation of real-world population-level screening outcomes. This study introduces the CAN-SCREEN (Colorectal cANcer SCReening Economics and adherENce) model, utilizing real-world adherence scenarios to assess the effectiveness of a blood-based test (BBT) compared to existing strategies.

Cost-Effectiveness of Plasma Microbial Cell-Free DNA Sequencing When Added to Usual Care Diagnostic Testing for Immunocompromised Host Pneumonia.

Introduction: Immunocompromised host pneumonia (ICHP) is an important cause of morbidity and mortality, yet usual care (UC) diagnostic tests often fail to identify an infectious etiology. A US-based, multicenter study (PICKUP) among ICHP patients with hematological malignancies, including hematological cell transplant recipients, showed that plasma microbial cell-free DNA (mcfDNA) sequencing provided significant additive diagnostic value.

Aim: The objective of this study was to perform a cost-effectiveness analysis (CEA) of adding mcfDNA sequencing to UC diagnostic testing for hospitalized ICHP patients.

Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma.

Objectives: The goal of the research was to assess the quantitative relationship between median progression-free survival (PFS) and median overall survival (OS) specifically among patients with relapsed/refractory multiple myeloma (RRMM) based on published randomized controlled trials (RCTs).

Methods: Two bibliographic databases (PubMed and Embase, 1970-2017) were systematically searched for RCTs in RRMM that reported OS and PFS, followed by an updated search of studies published between 2010 and 2022 in 3 databases (Embase, MEDLINE, and EBM Reviews, 2010-2022). The association between median PFS and median OS was assessed using the nonparametric Spearman rank and parametric Pearson correlation coefficients.

Boundary Integrity Testing of Containment Level 3 (Biological Safety Level 3) Laboratories.

Background: Containment Level 3 (CL3) laboratories may require boundary integrity testing. Existing guidelines for CL3 room leakage are often subjective, lacking a definitive standard for what constitutes a "sealed" room.

Methods: This study reviews global biocontainment guidelines and standards, and it compares multiple test results from global CL3 facilities by standardizing test data to an equivalent test pressure of 250 Pa.

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In 2015, in partnership with a research team, the PROFAN program was initiated by a group of peers to counter the increasing number of overdose-related deaths in Montreal. As of 2018, they have offered, in collaboration with the Association des intervenants en dépendance du Québec (AIDQ) (Quebec Association of Addiction Workers), over 160 training sessions, and have connected with 500 Québec organizations, resulting in a total of over 2,000 participants. A participative evaluation process was undertaken with respect to the expansion of the program.

Comparison of the burden of familial hypercholesterolemia between two cohorts of French Canadians hospitalized 25 years apart for coronary heart disease.

Background: Familial hypercholesterolemia (FH) is associated with lifelong elevated plasma concentrations of low-density lipoprotein cholesterol (LDL-C) and high risk of premature coronary heart disease (CHD). Clinical recommendations and treatments have emerged to facilitate the management of FH patients. Their impact on the burden of FH is however not well documented.

[Utilization of remote psychosocial intervention with men: What is the state of knowledge?].

Background Remote psychosocial intervention has been used by most health care organizations since the beginning of COVID-19 pandemic. However, the rapid introduction of this type of practice generates new methods of intervention that raise many questions, particularly about men who, in general, use face-to-face psychosocial intervention less than women. This documentary research aims to report on current knowledge on remote psychosocial intervention with men.

Economic Evaluation Associated With Clinical-Grade Mobile App-Based Digital Therapeutic Interventions: Systematic Review.

Background: Digital therapeutics (DTx), a class of software-based clinical interventions, are promising new technologies that can potentially prevent, manage, or treat a spectrum of medical disorders and diseases as well as deliver unprecedented portability for patients and scalability for health care providers. Their adoption and implementation were accelerated by the need for remote care during the COVID-19 pandemic, and awareness about their utility has rapidly grown among providers, payers, and regulators. Despite this, relatively little is known about the capacity of DTx to provide economic value in care.

Brief communication: global temporal trends in the efficacy of clarithromycin-based regimens for the treatment of infection.

Background: eradication rates achieved with clarithromycin-based triple therapies are declining due to antibiotic resistance, but data regarding temporal changes in efficacy with these eradication therapies are scarce.

Objective: To evaluate the efficacy of clarithromycin-based triple eradication regimens over time.

Design: A comprehensive literature review and time-trend analysis.

Impact of age on the Quadrella index assessing oncological and functional results after prostate brachytherapy: A 6-year analysis.

Purpose: As the oncological results of prostate brachytherapy (BT) are excellent for low-risk (LR) or favorable intermediate-risk (FIR) prostate cancer (PCa), evaluating the side effects has become a major issue, especially for young men. The objective of the study was to compare the oncologic and functional results of BT using Quadrella index for patients aged 60 or less compared with older patients.

Material And Methods: From June, 2007 to June, 2017, 222 patients, including 70 ≤ 60 years old and 152 > 60 years old, underwent BT for LR-FIR PCa, with good erectile function at baseline according to International Index of Erectile Function-5 (IIEF-5) > 16.

Overall and complete response rates as potential surrogates for overall survival in relapsed/refractory multiple myeloma.

The correlation between response and survival has not been well-studied in relapsed or refractory multiple myeloma (RRMM). A systematic literature review of Medline, Embase and Cochrane databases (2010-06/2020) and relevant congresses (2018-2020) was performed to identify randomized clinical trials in RRMM reporting median overall survival (mOS), progression-free survival and response end points. The relationship between mOS and response end points was analyzed using Pearson's product-moment correlation.

Cost-Effectiveness Analysis of Nefecon versus Best Supportive Care for People with Immunoglobulin A Nephropathy (IgAN) in the United States.

Purpose: To estimate the cost-effectiveness of Nefecon in addition to the best supportive care (BSC) vs BSC in a hypothetical cohort of commercially insured adult patients with primary immunoglobulin A nephropathy (IgAN) from a United States (US) societal perspective.

Methods: A lifetime horizon, semi-Markov model was developed that consisted of nine health states: chronic kidney disease (CKD) stage 1, 2, 3a, 3b, 4, end-stage renal disease (ESRD) with dialysis, ESRD without dialysis, post-kidney transplant, and death. Health state occupancy was estimated from individual patient-level data from the Phase 3 randomized controlled trial NefIgArd Part A (NCT03643965).

Engineering Controls and Technologies to Enhance Safety in the COVID-19 Pandemic Landscape: Lessons for Laboratories and Non-Laboratories.

Introduction: The global SARS-CoV-2 pandemic ushered in a new way of life in a short time, with many lasting impacts that have yet to be fully realized. This pandemic threat landscape resulted in massive efforts to increase safety, minimize person-to-person transmission, and rethink how society approaches personal and collective health issues. The buildings and environments in which we live, work, and learn now became environments that pose new risks.

Bayesian hierarchical model-based network meta-analysis to overcome survival extrapolation challenges caused by data immaturity.

This research evaluated standard Weibull mixture cure (WMC) network meta-analysis (NMA) with Bayesian hierarchical (BH) WMC NMA to inform long-term survival of therapies. Four trials in previously treated metastatic non-small-cell lung cancer with PD-L1 >1% were used comparing docetaxel with nivolumab, pembrolizumab and atezolizumab. Cure parameters related to a certain treatment class were assumed to share a common distribution.

Polarimetric multiple scattering LiDAR model based on Poisson distribution.

Multiple scattering is always present in LiDAR measurements. It is one of the major causes of LiDAR signal depolarization when detecting backscattering from water clouds. For a given probing wavelength, the LiDAR signal is a function of the aerosol size distribution, cloud range, and optical depth, and of the LiDAR field of view (FoV).

Economic Evaluation of Cost and Time Required for a Platform Trial vs Conventional Trials.

Importance: Platform trial design allows the introduction of new interventions after the trial is initiated and offers efficiencies to clinical research. However, limited guidance exists on the economic resources required to establish and maintain platform trials.

Objective: To compare cost (US dollars) and time requirements of conducting a platform trial vs a series of conventional (nonplatform) trials using a real-life example.

Potassium-Competitive Acid Blocker and Proton Pump Inhibitor-Based Regimens for First-Line Eradication: A Network Meta-Analysis.

Background And Aims: Effective acid suppression is a crucial component of () eradication regimens. Approved treatments include dual, triple, and quadruple therapies composed of certain antibiotics in combination with proton pump inhibitors (PPIs). Vonoprazan, a potassium-competitive acid blocker, provides more potent and durable acid suppression than PPIs.

Response rates and minimal residual disease outcomes as potential surrogates for progression-free survival in newly diagnosed multiple myeloma.

Progression-free survival (PFS) is a common primary endpoint in newly diagnosed multiple myeloma (NDMM). Patients with NDMM typically have longer PFS and are more likely to achieve minimal residual disease (MRD) or complete response (CR) compared to patients with relapsed or refractory multiple myeloma. Response-based surrogate endpoints may hold value given the longer follow-up time required to evaluate PFS in NDMM.

Humanistic and Economic Burden of Conversion Therapy Among LGBTQ Youths in the United States.

Importance: Sexual orientation and gender identity change efforts (SOGICE), also called conversion therapy, is a discredited practice attempting to convert lesbian, gay, bisexual, transgender, queer, or questioning (LGBTQ) individuals to be heterosexual and/or cisgender.

Objectives: To identify and synthesize evidence on the humanistic and economic consequences of SOGICE among LGBTQ youths in the US.

Design, Setting, And Participants: This study, conducted from December 1, 2020, to February 15, 2021, included a systematic literature review and economic evaluation.

Effectiveness of crizotinib versus entrectinib in -positive non-small-cell lung cancer using clinical and real-world data.

To compare clinical trial results for crizotinib and entrectinib in -positive non-small-cell lung cancer and compare clinical trial data and real-world outcomes for crizotinib. We analyzed four phase I-II studies using a simulated treatment comparison (STC). A STC of clinical trial versus real-world evidence compared crizotinib clinical data to real-world outcomes.

CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy.

Discovery of the CRISPR-Cas (clustered regularly interspaced short palindromic repeat, CRISPR-associated) system a decade ago has opened new possibilities in the field of precision medicine. CRISPR-Cas was initially identified in bacteria and archaea to play a protective role against foreign genetic elements during viral infections. The application of this technique for the correction of different mutations found in the Duchenne muscular dystrophy (DMD) gene led to the development of several potential therapeutic approaches for DMD patients.