Trial methodology

Bayesian statistics plays a pivotal role in advancing medical science by enabling healthcare companies, regulators, and stakeholders to assess the safety and efficacy of new treatments, interventions, and medical procedures. The Bayesian framework offers a unique advantage over the classical framework, especially when incorporating prior information into a new trial with quality external data, such as historical data or another source of co-data. In recent years, there has been a significant increase in regulatory submissions using Bayesian statistics due to its flexibility and ability to provide valuable insights for decision-making, addressing the modern complexity of clinical trials where frequentist trials are inadequate...

Mass spectrometry (MS) can detect multiple myeloma-derived monoclonal proteins in peripheral blood (PB) with high sensitivity, potentially serving as a PB assay for measurable residual disease (MRD). This study evaluated the significance of PB MS MRD negativity during post-transplant therapy in patients with newly diagnosed multiple myeloma. Serum samples from 138 patients treated in the phase 3 ATLAS trial of post-transplant maintenance with either carfilzomib, lenalidomide, dexamethasone or lenalidomide alone were analyzed using EXENT MS methodology...

BACKGROUND: Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety. OBJECTIVE: Conduct a meta-analysis to assess the efficacy and safety of onasemnogene abeparvovec in patients diagnosed with SMA type 1, based on the available evidence. METHODS: This article results from searches conducted on databases up to November 2022...

OBJECTIVE: This systematic literature review aims to assess the impact of COVID-19 on male fertility. DATA SOURCES: The study draws upon data extracted from PubMed, SciELO, and LILACS databases. STUDY SELECTION: The review incorporates cross-sectional studies, cohort studies, and clinical trials, encompassing investigations related to the subject matter. The studies included were published between June 2020 and March 2023, and encompassed content in English, Portuguese, and Spanish...

BACKGROUND: Surgical wounds that become infected are often debrided because clinicians believe that removal of this necrotic or infected tissue may expedite wound healing. There are numerous methods of debridement available, but no consensus on which one is most effective for surgical wounds. OBJECTIVES: To assess the effects of different methods of debridement on the rate of debridement and healing of surgical wounds. SEARCH METHODS: In October 2021, we searched the Cochrane Wounds Specialised Register, CENTRAL, MEDLINE, Embase, and CINAHL...

The evolving prevalence of anabolic androgenic steroids (AAS) abuse among nonathletes is alarming because of the known harm to an individual's health. Among the adverse effects of AAS abuse, male infertility and sexual dysfunction have been often reported in the literature, but little is known regarding its actual prevalence, possible underpinning mechanisms, and potential treatments either during or post-AAS usage. Thus, the current narrative review summarizes the state-of-art regarding the effects of AAS on male fertility and sexual function...

INTRODUCTION: Skull density ratio (SDR) is the ratio between the mean Hounsfield units of marrow and cortical bone, impacting energy transmission through the skull. Low SDR has been used as an exclusion criterion in major trials of magnetic resonance-guided focused ultrasound (MRgFUS) thalamotomy for medication-refractory essential tremor (ET). However, some studies have suggested that patients with low SDR can safely undergo MRgFUS with favorable outcomes. In this case-matched study, we aim to compare the characteristics, sonication parameters, lesion sizes, and clinical outcomes of patients with low SDR vs...

INTRODUCTION: It is essential to link the theoretical framework of any neurophysiotherapy approach with a detailed analysis of the central motor control mechanisms that influence motor behavior. Vojta therapy (VT) falls within interventions aiming to modify neuronal activity. Although it is often mistakenly perceived as exclusively pediatric, its utility spans various functional disorders by acting on central pattern modulation. This study aims to review the existing evidence on the effectiveness of VT across a wide range of conditions, both in the adult population and in pediatrics, and analyze common therapeutic mechanisms, focusing on motor control modulation...

AIM: This systematic review aimed to assess the effects of exercise training during pregnancy and the postpartum period on maternal vascular health and blood pressure (BP). METHODS: The outcome of interest were pulse wave velocity (PWV), flow-mediated dilation (FMD), and BP from pregnancy until 1-year postpartum. Five databases, including Ovid MEDLINE, EMBASE, CINAHL, Web of Science, and Cochrane Library, were systematically searched from inception to August 2023...

A low FODMAP diet (LFD) is a common restrictive diet to manage the symptoms of irritable bowel syndrome (IBS). However, there is no consensus on the alleviating effects of this diet. Herein, a systematic umbrella review with meta-analysis was conducted to investigate the effect of an LFD on IBS symptoms and its secondary outcomes in patients, which were not reported in previous meta-analyses. We performed a systematic literature search in PubMed, Scopus, and ISI Web of Science up to December 2023. The methodological quality of systematic reviews and their included trials was evaluated using AMSTAR 2 and the Cochrane risk of bias, respectively...

INTRODUCTION: Advanced therapies offer unprecedented opportunities for treating rare neurological disorders (RNDs) in children. However, health literacy, perceptions and understanding of novel therapies need elucidation across the RND community. This study explored healthcare professionals' and carers' perspectives of advanced therapies in childhood-onset RNDs. METHODS: In this mixed-methodology cross-sectional study, 20 healthcare professionals (clinicians, genetic counsellors and scientists) and 20 carers completed qualitative semistructured interviews and custom-designed surveys...

OBJECTIVE: To evaluate the efficacy and safety of intrapleural perfusion with hyperthermic chemotherapy (IPHC) in treating malignant pleural effusion (MPE). METHODS: PubMed, Embase, Cochrane Library, Chinese National Knowledge Infrastructure (CNKI), Chinese Biomedical Literature Database (CBM), VIP Chinese Science and Technology Journal Full-text Database (VP-CSJFD), and Wanfang database were searched by computer from database establishment to January 17, 2024. Relevant randomized controlled articles with IPHC as the observational group and intrapleural perfusion chemotherapy (IPC) as the control group for MPE were included...

PURPOSE: To evaluate the body of evidence of the effects of work-directed interventions on return-to-work for people on sick leave due to common mental disorders (i.e., mild to moderate depression, anxiety, adjustment disorders and reactions to severe stress). METHODS: The systematic review was conducted in accordance with an a priori developed and registered protocol (Prospero CRD42021235586). The certainty of evidence was assessed by two independent reviewers using the Grading of Recommendations, Assessment, Development and Evaluations...

Effective interventions that support blood donor retention are needed. Yet, integrating an intervention into the time-pressed and operationally sensitive context of a blood donation center requires justification for disruptions to an optimized process. This research provides evidence that virtual reality (VR) paradigms can serve as a research environment in which interventions can be tested prior to being delivered in blood donation centers. Study 1 (N = 48) demonstrated that 360°-video VR blood donation environments elicit a similar profile of emotional experience to a live donor center...

BACKGROUND: Many clinical trials use systematic methodology to monitor adverse events (AE) and determine grade (severity), expectedness, and relatedness to treatments as determined by clinicians. However, patient perspective in the process remains lacking. OBJECTIVES: To compare clinician versus patient grading of AE severity in a urogynecologic surgical trial. Secondary objectives were to estimate the association between patient grading of AEs with decision-making and quality of life outcomes and to determine if patient perspective changes over time...

PURPOSE: To comprehensively synthesize the existing evidence concerning mHealth interventions for patients with breast cancer (BC). DESIGN: On July 30, 2023, we searched PubMed, PsycINFO, and Google Scholar for articles using the following inclusion criteria: evaluation of mHealth interventions in patients with cancer, at least 30 participants with BC, randomized control trials or prospective pre-post studies, determinants of health (patient-reported outcomes [PROs] and quality of life [QoL]) as primary outcomes, interventions lasting at least 8 weeks, publication after January 2015...

Proprioception is significantly impaired in knee osteoarthritis (KOA), contributing to reduced functionality. Strength training (ST) is essential in KOA by improving muscle strength, although it may also be effective in improving proprioception. The purpose was to determine the effect of ST on knee proprioception in KOA patients. Pubmed, CINAHL, Scopus, WOS, and PEDro were searched for randomized controlled trials (RCTs) (inception to March 2023). Comparisons for ST were physical exercise different from ST, non-exercise-based interventions, and no intervention...

OBJECTIVE: Mobile health applications (apps) have gained significant popularity and widespread utilization among patients with coronary heart disease (CHD). The objective of this study is to evaluate the effects of mHealth apps on clinical outcomes and health behaviors in patients with CHD. METHODS: Databases were searched from inception until December 2023, including Cochrane Library, PubMed, EMBASE, Web of Science, CINAHL, China National Knowledge Infrastructure (CNKI), Chinese BioMedical Literature Service System (SinoMed), Wanfang Data, China Science and Technology Journal Database (VIP), for randomized controlled trials (RCTs) regarding the effectiveness of mHealth apps in patients with CHD...

BACKGROUND: Chile faces a significant postpartum depression prevalence and treatment gap, necessitating accessible interventions. While cognitive-behavioral internet-based interventions have proven effective in high-income countries, this field is underdeveloped in Chile. Based on the country's widespread use of digital technology, a guided 8-week cognitive-behavioral web app intervention named " Mamá, te entiendo " was developed. OBJECTIVE: This study aimed to assess the acceptability and feasibility of " Mamá, te entiendo ", for reducing depressive symptomatology in postpartum women...

BACKGROUND: Current imaging advancements quantify the use of cardiovascular magnetic resonance (CMR) derived T1 and T2 tissue characterization as robust indicators for cardiomyopathies, but limited literature exists on its clinical application in Takotsubo syndrome (TTS). This systematic review evaluated the T1 and T2 parametric mapping to delineate the current diagnostic and prognostic CMR imaging outcomes in TTS. METHODS: A comprehensive literature search until October 2023 was performed on ScienceDirect, PubMed, Web of Science, and Cochrane Library by two independent reviewers adhering to the PRISMA framework...