keyword
https://read.qxmd.com/read/38657265/exagamglogene-autotemcel-for-transfusion-dependent-%C3%AE-thalassemia
#1
JOURNAL ARTICLE
Franco Locatelli, Peter Lang, Donna Wall, Roland Meisel, Selim Corbacioglu, Amanda M Li, Josu de la Fuente, Ami J Shah, Ben Carpenter, Janet L Kwiatkowski, Markus Mapara, Robert I Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Rupert Handgretinger, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William E Hobbs, Haydar Frangoul
BACKGROUND: Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of the erythroid-specific enhancer region of BCL11A in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). METHODS: We conducted an open-label, single-group, phase 3 study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β0 /β0 , β0 /β0 -like, or non-β0 /β0 -like genotype...
April 24, 2024: New England Journal of Medicine
https://read.qxmd.com/read/38657227/an-integrated-mhealth-app-for-smoking-cessation-in-black-smokers-with-hiv-protocol-for-a-randomized-controlled-trial
#2
RANDOMIZED CONTROLLED TRIAL
Andre Bizier, Arielle Jones, Michael Businelle, Krista Kezbers, Bettina B Hoeppner, Thomas P Giordano, Jessica M Thai, Jacqueline Charles, Audrey Montgomery, Matthew W Gallagher, Marshall K Cheney, Michael Zvolensky, Lorra Garey
BACKGROUND: Black adults who smoke and have HIV experience immense stressors (eg, racial discrimination and HIV stigma) that impede smoking cessation success and perpetuate smoking-related health disparities. These stressors also place Black adults who smoke and have HIV at an increased risk of elevated interoceptive stress (eg, anxiety and uncomfortable bodily sensations) and smoking to manage symptoms. In turn, this population is more likely to smoke to manage interoceptive stress, which contributes to worse HIV-related outcomes in this group...
April 24, 2024: JMIR Research Protocols
https://read.qxmd.com/read/38657209/impact-of-preventive-substrate-catheter-ablation-on-implantable-cardioverter-defibrillator-interventions-in-patients-with-ischaemic-cardiomyopathy-and-infarct-related-coronary-chronic-total-occlusion
#3
JOURNAL ARTICLE
David Žižek, Miha Mrak, Matevž Jan, Anja Zupan Mežnar, Maja Ivanovski, Tadej Žlahtič, Nina Kajdič, Bor Antolič, Luka Klemen, Rafael Skale, Jurij Avramovič Gregorič, Jernej Štublar, Andrej Pernat, Matjaž Šinkovec
BACKGROUND: Primary prevention patients with ischemic cardiomyopathy and chronic total occlusion of an infarct-related coronary artery (IRA-CTO) are at a particularly high risk of implantable cardioverter-defibrillator (ICD) therapy occurrence. AIM: To evaluate the efficacy of preventive CTO-related substrate ablation strategy in ischaemic cardiomyopathy patients undergoing primary prevention ICD implantation. METHODS: The PREVENTIVE VT study was a prospective, multicenter, randomized trial including ischaemic patients with ejection fraction ≤40%, no documented VAs, and evidence of scar related to the coronary CTO...
April 24, 2024: Europace: European Pacing, Arrhythmias, and Cardiac Electrophysiology
https://read.qxmd.com/read/38656937/laryngeal-recalibration-therapy-improves-laryngopharyngeal-symptoms-in-patients-with-suspected-laryngopharyngeal-reflux-disease
#4
JOURNAL ARTICLE
Erin Walsh, Amanda J Krause, Madeline Greytak, Alexander M Kaizer, Philip A Weissbrod, Kelli Liu, Tiffany Taft, Rena Yadlapati
BACKGROUND: Laryngopharyngeal symptoms such as cough, throat clearing, voice change, paradoxic vocal fold movement or laryngospasm are hyper-responsive behaviors resulting from local irritation (e.g., refluxate) and heightened sympathetic tone. Laryngeal recalibration therapy (LRT) guided by a speech-language pathologist (SLP) provides mechanical desensitization and cognitive recalibration to suppress hyper-responsive laryngeal patterns. This study aimed to assess symptom response to LRT among patients with chronic laryngopharyngeal symptoms symptoms undergoing evaluation of gastroesophageal reflux disease (GERD)...
April 24, 2024: American Journal of Gastroenterology
https://read.qxmd.com/read/38656775/effects-of-virtual-reality-therapy-combined-with-conventional-rehabilitation-on-pain-kinematic-function-and-disability-in-patients-with-chronic-neck-pain-randomized-controlled-trial
#5
JOURNAL ARTICLE
Qifan Guo, Liming Zhang, Leo Lianyi Han, Chenfan Gui, Guanghui Chen, Chunyan Ling, Wei Wang, Qiang Gao
BACKGROUND: Neck pain is a common condition that leads to neck motor dysfunction and subsequent disability, with a significant global health care burden. As a newly emerging tool, virtual reality (VR) technology has been employed to address pain and reduce disability among patients with neck pain. However, there is still a lack of high-quality studies evaluating the efficacy of VR therapy combined with conventional rehabilitation for patients with chronic neck pain, particularly in terms of kinematic function...
April 24, 2024: JMIR Serious Games
https://read.qxmd.com/read/38656736/safety-tolerability-and-pharmacokinetics-of-oral-bi-1358894-in-healthy-japanese-male-volunteers
#6
JOURNAL ARTICLE
Jangsoo Yoon, Vikas Sharma, Akiko Harada
UNLABELLED: BACKGROUND AND OBJECTIVES: BI 1358894, a novel small-molecule inhibitor of transient receptor potential canonical ion channels, is under development for treatment of major depressive disorder. Phase I trials assessing the safety and pharmacokinetics of BI 1358894 in Caucasian male healthy volunteers (HVs) have been performed. This Phase I, double-blind, placebo-controlled, parallel-group trial assessed the safety, tolerability and pharmacokinetics of BI 1358894 in Japanese male HVs...
April 24, 2024: Clinical Drug Investigation
https://read.qxmd.com/read/38656693/minimally-invasive-surgical-therapies-for-benign-prostatic-hyperplasia-in-the-geriatric-population-a-systematic-review
#7
REVIEW
Ahmad Abid, Huzaifa Piperdi, Mustufa Babar, Justin Loloi, Ahmed Moutwakil, Umair Azhar, Pedro Maria, Alexander Small
BACKGROUND: Geriatric patients, prone to adverse events (AEs) and low compliance with drugs, may benefit from minimally invasive surgical therapies (MISTs) for managing benign prostatic hyperplasia (BPH). We evaluated the efficacy, safety, and procedural characteristics of MISTs in geriatric patients with BPH. METHODS: PubMed/MEDLINE database was systematically searched for relevant articles through October 1, 2023. Eligible studies focused on geriatric patients (≥65 years) with BPH who were treated with MISTs and evaluated follow-up surgical, micturition, and/or sexual outcomes...
April 24, 2024: Prostate
https://read.qxmd.com/read/38656557/first-in-human-study-of-alpibectir-bvl-gsk098-a-novel-potent-anti-tb-drug
#8
JOURNAL ARTICLE
Michel Pieren, Ana Abáigar Gutiérrez-Solana, Rosa María Antonijoan Arbós, Gary W Boyle, Myriam Davila, Maria Davy, Marc Gitzinger, Lisa Husband, María S Martínez-Martínez, Dolores Ochoa Mazarro, Eleni Pefani, Sophie L Penman, Modesto J Remuiñán, Georgios Vlasakakis, Markus Zeitlinger, Glenn E Dale
BACKGROUND: The clinical candidate alpibectir augments the activity of, and overcomes resistance to, the anti-TB drug ethionamide in vitro and in vivo. OBJECTIVES: A Phase 1, double-blind, randomized, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics (PK) and food effect of alpibectir administered as single and multiple oral doses in healthy volunteers (NCT04654143). METHODS: Eighty participants were randomized...
April 24, 2024: Journal of Antimicrobial Chemotherapy
https://read.qxmd.com/read/38656437/automatic-segmentation-of-tumour-and-organs-at-risk-in-3d-mri-for-cervical-cancer-radiation-therapy-with-anatomical-variations
#9
JOURNAL ARTICLE
Sze-Nung Leung, Shekhar S Chandra, Karen Lim, Tony Young, Lois Holloway, Jason A Dowling
Cervical cancer is a common cancer in women globally, with treatment usually involving radiation therapy (RT). Accurate segmentation for the tumour site and organ-at-risks (OARs) could assist in the reduction of treatment side effects and improve treatment planning efficiency. Cervical cancer Magnetic Resonance Imaging (MRI) segmentation is challenging due to a limited amount of training data available and large inter- and intra- patient shape variation for OARs. The proposed Masked-Net consists of a masked encoder within the 3D U-Net to account for the large shape variation within the dataset, with additional dilated layers added to improve segmentation performance...
April 24, 2024: Physical and engineering sciences in medicine
https://read.qxmd.com/read/38656428/cognitive-behavior-therapy-vs-mindfulness-in-treatment-of-prolonged-grief-disorder-a-randomized-clinical-trial
#10
JOURNAL ARTICLE
Richard A Bryant, Suzanna Azevedo, Srishti Yadav, Catherine Cahill, Lucy Kenny, Fiona Maccallum, Jenny Tran, Jasmine Choi-Christou, Natasha Rawson, Julia Tockar, Benjamin Garber, Dharani Keyan, Katie S Dawson
IMPORTANCE: Although grief-focused cognitive behavior therapies are the most empirically supported treatment for prolonged grief disorder, many people find this treatment difficult. A viable alternative for treatment is mindfulness-based cognitive therapy. OBJECTIVE: To examine the relative efficacies of grief-focused cognitive behavior therapy and mindfulness-based cognitive therapy to reduce prolonged grief disorder severity. DESIGN, SETTING, AND PARTICIPANTS: A single-blind, parallel, randomized clinical trial was conducted among adults aged 18 to 70 years with prolonged grief disorder, as defined in the International Classification of Diseases, 11th Revision, and assessed by clinical interview based on the Prolonged Grief-13 (PG-13) scale...
April 24, 2024: JAMA Psychiatry
https://read.qxmd.com/read/38656356/significance-of-radiation-therapy-in-the-myxoid-round-cell-liposarcoma-treatment-regimen
#11
JOURNAL ARTICLE
Akiyoshi Shimatani, Hirohisa Katagiri, Hideki Murata, Junji Wasa, Michihito Miyagi, Yosuke Honda, Mitsuru Takahashi, Hiroyuki Harada, Hirofumi Asakura, Tsuyoshi Onoe
BACKGROUND AND PURPOSE: Because myxoid liposarcomas are more radiosensitive than other soft tissue sarcomas, there have been several reports of 50 Gy preoperative radiation therapy combined with surgery, but the wound complication rate is reportedly high. We have performed preoperative irradiation at a reduced dose of 40 Gy and definitive radiation therapy for unresectable cases. This study aimed to report the tumor reduction rate and oncological results with a reduced dose of preoperative irradiation and the outcome of definitive irradiation for unresectable cases...
April 24, 2024: International Journal of Clinical Oncology
https://read.qxmd.com/read/38656292/atrial-fibrillation-ablation-in-heart-failure-with-reduced-vs-preserved-ejection-fraction-a-systematic-review-and-meta-analysis
#12
JOURNAL ARTICLE
Alireza Oraii, William F McIntyre, Ratika Parkash, Krzysztof Kowalik, Ghazal Razeghi, Alexander P Benz, Emilie P Belley-Côté, David Conen, Stuart J Connolly, Anthony S L Tang, Jeff S Healey, Jorge A Wong
IMPORTANCE: Catheter ablation is associated with reduced heart failure (HF) hospitalization and death in select patients with atrial fibrillation (AF) and heart failure with reduced ejection fraction (HFrEF). However, the benefit in patients with HF with preserved ejection fraction (HFpEF) is uncertain. OBJECTIVE: To investigate whether catheter ablation for AF is associated with reduced HF-related outcomes according to HF phenotype. DATA SOURCE: A systematic search of MEDLINE, Embase, and Cochrane Central was conducted among studies published from inception to September 2023...
April 24, 2024: JAMA Cardiology
https://read.qxmd.com/read/38656204/microbiological-diagnosis-of-pleural-infections-a-comparative-evaluation-of-a-novel-syndromic-real-time-pcr-panel
#13
JOURNAL ARTICLE
Øyvind Kommedal, Tomas Mikal Eagan, Øystein Fløtten, Truls Michael Leegaard, William Siljan, Hilde Fardal, Bjørnar Bø, Fredrik Grøvan, Kjersti Wik Larssen, Arne Kildahl-Andersen, Reidar Hjetland, Rune Tilseth, Sølvi Kristine Øyen Hareide, Marit Tellevik, Ruben Dyrhovden
Current microbial diagnostics for pleural infections are insufficient. Studies using 16S targeted next-generation sequencing report that only 10%-16% of bacteria present are cultured and that 50%-78% of pleural fluids containing relevant microbial DNA remain culture negative. As a rapid diagnostic alternative suitable for clinical laboratories, we wanted to explore a PCR-based approach. Based on the identification of key pathogens, we developed a syndromic PCR panel for community-acquired pleural infections (CAPIs)...
April 24, 2024: Microbiology Spectrum
https://read.qxmd.com/read/38656011/are-depression-and-poor-sleep-quality-a-major-problem-in-turkish-women-receiving-chemotherapy-for-breast-cancer
#14
JOURNAL ARTICLE
Mahmut Buyuksimsek, Ahmet Gulmez, Okan Pirinci, Mert Tohumcuoglu, Mehmet Mutlu Kidi
OBJECTIVE: The aim of this study was to evaluate depression and sleep quality in Turkish women receiving neoadjuvant or adjuvant chemotherapy for breast cancer and investigate their relationship. METHODS: This cross-sectional, descriptive, and analytical study included 183 patients who received chemotherapy for non-metastatic breast cancer. Data were collected using the Beck Depression Inventory-II, the Pittsburgh Sleep Quality Index, and a disease-related/sociodemographic information form...
2024: Revista da Associação Médica Brasileira
https://read.qxmd.com/read/38656000/mobile-app-for-pelvic-floor-muscle-training-for-urinary-incontinence-during-the-coronavirus-disease-2019-pandemic-clinical-trial
#15
RANDOMIZED CONTROLLED TRIAL
Camila Carvalho de Araujo, Luiz Gustavo Oliveira Brito, Andrea Marques, Marcela Bardin, Cássia Raquel Teatin Juliato
OBJECTIVE: The objective of this study was to evaluate the effects of home-based pelvic floor muscle training in women with urinary incontinence, addressing the difficulties arising from social isolation due to the coronavirus disease 2019 pandemic by utilizing a specialized mobile app. METHODS: This randomized, single-group clinical trial aimed to assess the efficacy of pelvic floor muscle training guided by a mobile app (Diario Saúde) in women with stress urinary incontinence...
2024: Revista da Associação Médica Brasileira
https://read.qxmd.com/read/38655933/efficacy-of-a-hospital-discharge-transition-plan-in-the-care-competence-of-patients-with-chronic-conditions-and-their-family-caregivers-a-clinical-trial
#16
RANDOMIZED CONTROLLED TRIAL
Yuliana Valentina Rincón Estrada, Astrid Nathalia Páez Esteban, Maria Stella Campos de Aldana, Erika Yurley Durán Niño, Juan Sebastian Rincón Contreras, Maria Del Pilar Castillo Galvis, Valeria Arias Pinzón, Sergio Andrés García Arenas
OBJECTIVE: to assess the efficacy of a Hospital Discharge Transition Plan in the care competence and in adherence to the therapy of dyads comprised by patients with non-communicable chronic diseases and their caregivers. METHOD: a controlled and randomized clinical trial; the sample was comprised by 80 dyads of patients with chronic conditions and their caregivers, randomly allocated as follows: 40 to the control group and another 40 to the intervention group...
2024: Revista Latino-americana de Enfermagem
https://read.qxmd.com/read/38655859/from-the-randomized-ambora-trial-to-clinical-practice-comparison-of-medication-errors-in%C3%A2-oral-antitumor-therapy
#17
JOURNAL ARTICLE
Lisa Cuba, Pauline Dürr, Frank Dörje, Martin F Fromm, Katja Schlichtig
The randomized AMBORA trial showed that medication errors are frequent in patients treated with oral antitumor therapeutics and that they can be substantially reduced by an intensified clinical pharmacological/pharmaceutical care program. While randomized controlled trials are essential to generate clinical evidence, their generalizability in real-world is not always given. The AMBORA care program was implemented in clinical routine within the AMBORA Competence and Consultation Center (AMBORA Center) at the Comprehensive Cancer Center Erlangen-EMN, allowing a thorough comparison of medication error frequencies and characteristics...
April 24, 2024: Clinical Pharmacology and Therapeutics
https://read.qxmd.com/read/38655665/association-between-variation-in-hemoglobin-a1c-levels-and-diabetes-therapy-related-quality-of-life-in-patients-with-diabetes
#18
JOURNAL ARTICLE
Dan Imai, Emi Ushigome, Ryosuke Sakai, Noriyuki Kitagawa, Masahide Hamaguchi, Masahiro Yamazaki, Michiaki Fukui
AIMS/INTRODUCTION: We aimed to investigate the association between glycemic variability and quality of life (QOL) in patients with diabetes, which has not been studied previously. MATERIALS AND METHODS: Patients who were undergoing treatment at the Kyoto Prefectural University of Medicine Hospital and Kameoka City Hospital participated in the KAMOGAWA-DM study, and completed the diabetes therapy-related (DTR)-QOL questionnaire from January 2016 to July 2020 were included in this study...
April 24, 2024: Journal of Diabetes Investigation
https://read.qxmd.com/read/38655537/evaluation-of-an-ultrasound-guided-freeze-core-biopsy-system-for-canine-and-feline-brain-tumors
#19
JOURNAL ARTICLE
Brian S Adams, Dominic J Marino, Catherine A Loughin, Leonard J Marino, Teresa Southard, Martin L Lesser, Meredith Akerman, Patrick Roynard
OBJECTIVE: To determine if a single brain biopsy utilizing a freeze-core needle harvest system Cassi II under ultrasound guidance provides a diagnostic sample; to evaluate the technique's efficacy in procuring diagnostic samples in comparison with "open" surgical biopsies; and to describe intraoperative complications associated with the technique. STUDY DESIGN: Experimental clinical study. ANIMALS: Seventeen dogs and four cats with magnetic resonance imaging (MRI) diagnoses of readily surgically accessible intracranial masses...
2024: Frontiers in Veterinary Science
https://read.qxmd.com/read/38655186/safety-and-efficacy-of-east-asian-herbal-medicine-for-iron-deficiency-anemia-in-children-and-adolescents-a-systematic-review-and-meta-analysis
#20
Yoon Kyoung Jeong, Jae Hyun Kim, Sun Haeng Lee, Miran Bang, Gyu Tae Chang
BACKGROUND: This systematic review and meta-analysis aimed to evaluate the efficacy and safety of East Asian herbal medicine (HM) for iron deficiency anemia (IDA) in children and adolescents. METHODS: Twelve electronic databases were searched in 28 May 2023 for randomized controlled trials (RCTs) evaluating the efficacy of HM in children with IDA. The primary outcome measures for treatment included blood hemoglobin and serum ferritin levels, whereas the secondary outcomes included the total effective rate (TER), incidence of adverse events (AEs), average healing time, and hematologic indicators related to IDA...
2024: Frontiers in Pharmacology
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