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JOURNAL ARTICLE
Wango Chaulo, Elias C Nyanza, Moses Asori, Deborah S K Thomas, Florentina Mashuda
Congenital anomalies in Sub-Sahara Africa (SSA) are understudied despite the significant pediatric health burden. This retrospective longitudinal hospital-based study evaluated the records of 326 inpatient children under the age of two years with congenital anomalies at Bugando Medical Centre, a tertiary referral hospital in northwestern Tanzania. Classical logistic regression was used in the analysis of congenital malformation of muscles, gastrointestinal malformation, oral facial clefts, neural tube defects, and skeletal malformations...
2024: PLOS Glob Public Health
#2
JOURNAL ARTICLE
Suzanne L Laboyrie, Maria K Svensson, Sabine Josemans, Birgitta Sigvant, Joris I Rotmans, Gunilla Welander
BACKGROUND: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a leading hereditary cause of end-stage kidney disease (ESKD), often utilising hemodialysis as a form of kidney replacement therapy. Patients with ADPKD may also present with extrarenal manifestations, including arterial aneurysms. The gold standard for hemodialysis access is an arteriovenous vascular access (VA), such fistulas (AVFs) or grafts (AVGs). However, limitations, such as low VA flow and inadequate AVF outward remodelling, impact VA utilization...
May 1, 2024: Kidney360
#3
JOURNAL ARTICLE
Michaella Alexandrou, Deniz Mutlu, Athanasios Rempakos, Ahmed Al Ogaili, James W Choi, Paul Poommipanit, Khaldoon Alaswad, Mir Babar Basir, Rhian Davies, Farouc A Jaffer, Phil Dattilo, Lorenzo Azzalini, Nazif Aygul, Niranjan Reddy, Brian K Jefferson, Sevket Gorgulu, Jaikirshan J Khatri, Laura D Young, Oleg Krestyaninov, Dmitrii Khelimskii, Jarrod Frizzell, Basem Elbarouni, Bavana V Rangan, Olga C Mastrodemos, M Nicholas Burke, Yader Sandoval, Emmanouil S Brilakis
Ranolazine is an anti-anginal medication given to patients with chronic angina and persistent symptoms despite medical therapy. We examined 11 491 chronic total occlusion (CTO) percutaneous coronary interventions (PCI) that were performed at 41 US and non-US centers between 2012 and 2023 in the PROGRESS-CTO Registry. Patients on ranolazine at baseline had more comorbidities, more complex lesions, lower procedural and technical success (based on univariable but not multivariable analysis), and higher incidence of major adverse cardiac events (MACE) (on both univariable and multivariable analysis)...
April 30, 2024: Journal of Invasive Cardiology
#4
JOURNAL ARTICLE
Felix Kluge, Yonatan E Brand, M Encarna Micó-Amigo, Stefano Bertuletti, Ilaria D'Ascanio, Eran Gazit, Tecla Bonci, Cameron Kirk, Arne Küderle, Luca Palmerini, Anisoara Paraschiv-Ionescu, Francesca Salis, Abolfazl Soltani, Martin Ullrich, Lisa Alcock, Kamiar Aminian, Clemens Becker, Philip Brown, Joren Buekers, Anne-Elie Carsin, Marco Caruso, Brian Caulfield, Andrea Cereatti, Lorenzo Chiari, Carlos Echevarria, Bjoern Eskofier, Jordi Evers, Judith Garcia-Aymerich, Tilo Hache, Clint Hansen, Jeffrey M Hausdorff, Hugo Hiden, Emily Hume, Alison Keogh, Sarah Koch, Walter Maetzler, Dimitrios Megaritis, Martijn Niessen, Or Perlman, Lars Schwickert, Kirsty Scott, Basil Sharrack, David Singleton, Beatrix Vereijken, Ioannis Vogiatzis, Alison Yarnall, Lynn Rochester, Claudia Mazzà, Silvia Del Din, Arne Mueller
BACKGROUND: Wrist-worn inertial sensors are used in digital health for evaluating mobility in real-world environments. Preceding the estimation of spatiotemporal gait parameters within long-term recordings, gait detection is an important step to identify regions of interest where gait occurs, which requires robust algorithms due to the complexity of arm movements. While algorithms exist for other sensor positions, a comparative validation of algorithms applied to the wrist position on real-world data sets across different disease populations is missing...
May 1, 2024: JMIR Formative Research
#5
JOURNAL ARTICLE
Wakako Yoshioka, Harumasa Nakamura, Mari Oba, Yoshihiko Saito, Ichizo Nishino, Madoka Mori-Yoshimura
BACKGROUND: GNE myopathy is an ultra-rare autosomal recessive distal myopathy caused by pathogenic variants of the GNE gene, which encodes a key enzyme in sialic acid biosynthesis. The present study aimed to examine the long-term progression of GNE myopathy, genotype-phenotype correlations, and complications to provide useful information for predicting patient progression and designing clinical trials using a large collection of registry data over a 10-year period. METHODS: We analyzed 220 Japanese patients with GNE myopathy from a national registry in Japan...
May 1, 2024: Journal of Neurology
#6
JOURNAL ARTICLE
Elaheh Malakan Rad, Sara Momtazmanesh, Sahar Saeedi Moghaddam, Negar Rezaei, Nazila Rezaei, Hamidreza Jamshidi, Mohsen Naghavi, Bagher Larijani, Farshad Farzadfar
BACKGROUND: Infective endocarditis (IE), a severe and economically impactful condition, lacks substantial epidemiological data in the North Africa and Middle East (NAME) region. This study focused on analyzing the trends and burden of IE in NAME from 1990 to 2019, taking into account factors like age, gender, and socio-demographic index (SDI). METHODS: The Global Burden of Disease data from 1990 to 2019 was retrieved from the Institute for Health Metrics and Evaluation (IHME) website...
May 1, 2024: Archives of Iranian Medicine
#7
JOURNAL ARTICLE
Chinonso C Opara, Roy H Lan, Joselyn Rwebembera, Emmy Okello, David A Watkins, Andrew Y Chang, Chris T Longenecker
BACKGROUND: Atrial fibrillation (AF) is a common complication of rheumatic heart disease (RHD) and is challenging to treat in lower-resourced settings in which RHD remains endemic. OBJECTIVE: We characterized demographics, treatment outcomes, and factors leading to care retention for participants with RHD and AF in Uganda. METHODS: We conducted a retrospective analysis of the Uganda national RHD registry between June 2009 and May 2018. Participants with AF or atrial flutter were included...
April 2024: Heart rhythm O2
#8
REVIEW
Savvas Symeonidis, Ioannis Mantzoros, Elissavet Anestiadou, Orestis Ioannidis, Panagiotis Christidis, Stefanos Bitsianis, Vasiliki Bisbinas, Konstantinos Zapsalis, Trigona Karastergiou, Dimitra Athanasiou, Stylianos Apostolidis, Stamatios Angelopoulos
Laparoscopic cholecystectomy (LC) remains one of the most commonly performed procedures in adult and paediatric populations. Despite the advances made in intraoperative biliary anatomy recognition, iatrogenic bile duct injuries during LC represent a fatal complication and consist an economic burden for healthcare systems. A series of methods have been proposed to prevent bile duct injury, among them the use of indocyanine green (ICG) fluorescence. The most commonly reported method of ICG injection is the intravenous administration, while literature is lacking studies investigating the direct intragallbladder ICG injection...
April 27, 2024: World Journal of Gastrointestinal Surgery
#9
JOURNAL ARTICLE
Elise Hoover, Vanessa Holliday, Nicole Merullo, Dorothee Oberdhan, Ronald D Perrone, Chris Rusconi, Meyeon Park, Milind A Phadnis, Nadeesha Thewarapperuma, Neera K Dahl
RATIONALE & OBJECTIVE: Autosomal dominant polycystic kidney disease (ADPKD) affects health-related quality of life (HRQoL) including pain, discomfort, fatigue, emotional distress, and impaired mobility. Stakeholders prioritized kidney cyst-related pain as an important core outcome domain in clinical trials, leading to the development of disease-specific assessment tools. STUDY DESIGN: The ADPKD Registry is hosted online with multiple disease-specific patient-reported outcomes modules to characterize the patient experience in the United States...
May 2024: Kidney medicine
#10
JOURNAL ARTICLE
Dorothy Liu, Mark M Youssef, Josephine A Grace, Marie Sinclair
BACKGROUND: De novo malignancy is a leading cause of late morbidity and mortality in liver transplant recipients. Cumulative immunosuppression has been shown to contribute to post-transplant malignancy (PTM) risk. There is emerging evidence on the differential carcinogenic risk profile of individual immunosuppressive drugs, independent of the net effect of immunosuppression. Calcineurin inhibitors such as tacrolimus may promote tumourigenesis, whereas mycophenolic acid (MPA), the active metabolite of mycophenolate mofetil, may limit tumour progression...
April 27, 2024: World Journal of Hepatology
#11
JOURNAL ARTICLE
Gwendolyn A Ung, Kevin H Nguyen, Alvin Hui, Nathan D Wong, Elizabeth H Dineen
UNLABELLED: High cardiorespiratory fitness (CRF) is associated with decreased mortality in people with pre-diabetes (pre-DM) and diabetes mellitus (DM); however, the degree to which CRF attenuates the risk of cardiovascular disease (CVD)-related and all-cause mortality is unclear. STUDY OBJECTIVE: We examined the impact of CRF status on CVD-related morbidity and all-cause mortality in non-DM, Pre-DM, and DM populations. DESIGN AND SETTING: 13,968 adults from the Third US National Health and Nutrition Examination Survey (NHANES III) were stratified into non-DM, pre-DM, or DM groups based on HbA1c levels...
June 2024: American heart journal plus: cardiology research and practice
#12
JOURNAL ARTICLE
Ivan Lylyk, Esteban Scrivano, Javier Lundquist, Carlos Bleise, Nicolas Perez, Pedro Nicolas Lylyk, Rodolfo Nella-Castro, Pedro Lylyk
BACKGROUND: The PEDESTRIAN registry demonstrated high rates of complete long-term occlusion and good clinical outcomes among patients with intracranial aneurysms treated with the pipeline embolization device. The pipeline flex embolization device with shield technology was introduced to minimize thromboembolic complications. In this study, we investigated the safety and effectiveness of pipeline embolization device with shield technology among all patients treated for intracranial aneurysms at our center...
April 30, 2024: Interventional Neuroradiology
#13
JOURNAL ARTICLE
Madoka Mori-Yoshimura, Keiko Ishigaki, Yuko Shimizu-Motohashi, Naoko Ishihara, Atushi Unuma, Sumiko Yoshida, Harumasa Nakamura
BACKGROUND: Little is known about the social difficulties and health care needs of adult Duchenne muscular dystrophy (DMD) patients in Japan, as well as the financial and physical stress experienced by their caregivers. This study aimed to clarify the social circumstances surrounding adult DMD patients and assess the degree of involvement of family members in their care and the associated economic burden of the disorder in Japan. METHODS: Adult DMD patients were identified through the Registry of Muscular Dystrophy (Remudy) in Japan and invited to complete a questionnaire together with a caregiver...
April 30, 2024: Orphanet Journal of Rare Diseases
#14
RANDOMIZED CONTROLLED TRIAL
Halima I Hassan, Bashir Kaka, Fatima Bello, Francis Fatoye, Aminu A Ibrahim
BACKGROUND: Diabetes mellitus is one of the fastest-growing health challenges of the twenty-first century with multifactorial impact including high rates of morbidity and mortality as well as increased healthcare costs. It is associated with musculoskeletal complications, with frozen shoulder being commonly reported. While low-level laser therapy (LLLT) and muscle energy technique (MET) are commonly used to manage this condition, there remains a lack of agreement on the most effective approach, with limited research available on their comparative efficacy...
April 30, 2024: Journal of Orthopaedic Surgery and Research
#15
REVIEW
Ana Jovanovic, Eve Miller-Hodges, Felicia Castriota, Shweta Takyar, Heena Howitt, Olulade Ayodele
BACKGROUND: Fabry disease (FD) is a rare lysosomal storage disease associated with glycolipid accumulation that impacts multiple physiological systems. We conducted a systematic literature review (SLR) to characterize the humanistic (quality of life [QoL]) and economic burden of FD. METHODS: Searches were conducted in the Embase, MEDLINE® , and MEDLINE® In-Process databases from inception to January 19, 2022. Conference abstracts of specified congresses were manually searched...
April 30, 2024: Orphanet Journal of Rare Diseases
#16
JOURNAL ARTICLE
Niels Hendrickx, France Mentré, Andreas Traschütz, Cynthia Gagnon, Rebecca Schüle, Matthis Synofzik, Emmanuelle Comets
The aim of this study was to develop a model to predict individual subject disease trajectories including parameter uncertainty and accounting for missing data in rare neurological diseases, showcased by the ultra-rare disease Autosomal-Recessive Spastic Ataxia Charlevoix Saguenay (ARSACS). We modelled the change in SARA (Scale for Assessment and Rating of Ataxia) score versus Time Since Onset of symptoms using non-linear mixed effect models for a population of 173 patients with ARSACS included in the prospective real-world multicenter Autosomal Recessive Cerebellar Ataxia (ARCA) registry...
April 30, 2024: AAPS Journal
#17
JOURNAL ARTICLE
Jannis Müller, Izanne Roos, Tomas Kalincik, Johannes Lorscheider, Edoardo Galli, Pascal Benkert, Sabine Schädelin, Sifat Sharmin, Maximilian Einsiedler, Peter Hänni, Jürg Schmid, Jens Kuhle, Tobias Derfuss, Cristina Granziera, Tjalf Ziemssen, Timo Siepmann, Özgür Yaldizli
BACKGROUND: In patients with relapsing remitting multiple sclerosis (RRMS) on low-efficacy disease modifying therapies (DMT), the optimal strategy on how to escalate treatment once needed, remains unknown. METHODS: We studied RRMS patients on low-efficacy DMTs listed in the Swiss National Treatment Registry, who underwent escalation to either medium- or high-efficacy DMTs. Propensity score-based matching was applied using 12 clinically relevant variables. Both groups were also separately matched with control subjects who did not escalate therapy...
May 2024: Brain and Behavior
#18
JOURNAL ARTICLE
Anne-Laure Faucon, Sigrid Lundberg, Stefania Lando, Julia Wijkström, Mårten Segelmark, Marie Evans, Juan-Jesús Carrero
BACKGROUND & HYPOTHESIS: KDIGO recommends proteinuria <1 g/d as a treatment target in patients with immunoglobulin A nephropathy (IgAN) because of high-risk of progression to kidney failure. However, long-term kidney outcomes in patients with low-grade proteinuria remain insufficiently studied. METHODS: We enrolled patients with biopsy-proven primary IgAN from the Swedish Renal Registry and analyzed associations between urine albumin-to-creatinine ratio (uACR, in categories < 0...
April 30, 2024: Nephrology, Dialysis, Transplantation
#19
JOURNAL ARTICLE
Nynne Nyboe Andersen, Signe Wildt, Aske Thorn Iversen, Gry Poulsen, Tine Jess, Lars Kristian Munck, Christian Borup
OBJECTIVE: Bile acid diarrhoea is a common cause of chronic diarrhoea. Increased levels of potentially carcinogenic bile acids in faeces, theoretically, may increase the risk of colorectal cancer in particular, but the long-term disease course is unknown. We aimed to investigate the overall and site-specific cancer risk in bile acid diarrhoea. DESIGN: Adult patients with bile acid diarrhoea were identified using nationwide Danish registries from 2003 to 2020 by a diagnostic gold-standard 75-selenium tauroselcholic acid procedure followed within 6 months by sequestrant prescription...
April 30, 2024: BMJ Open Gastroenterology
#20
MULTICENTER STUDY
Jennifer Scott, Arthur White, Cathal Walsh, Louis Aslett, Matthew A Rutherford, James Ng, Conor Judge, Kuruvilla Sebastian, Sorcha O'Brien, John Kelleher, Julie Power, Niall Conlon, Sarah M Moran, Raashid Ahmed Luqmani, Peter A Merkel, Vladimir Tesar, Zdenka Hruskova, Mark A Little
OBJECTIVE: ANCA-associated vasculitis (AAV) is a relapsing-remitting disease, resulting in incremental tissue injury. The gold-standard relapse definition (Birmingham Vasculitis Activity Score, BVAS>0) is often missing or inaccurate in registry settings, leading to errors in ascertainment of this key outcome. We sought to create a computable phenotype (CP) to automate retrospective identification of relapse using real-world data in the research setting. METHODS: We studied 536 patients with AAV and >6 months follow-up recruited to the Rare Kidney Disease registry (a national longitudinal, multicentre cohort study)...
April 30, 2024: RMD Open
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