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Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey.
Orphanet J Rare Dis
January 2025
Department of Gastroenterology, Hepatology and Infectious Diseases, University Hospital, Heinrich- Heine University, Düsseldorf, Germany.
Background: Patients with Gaucher disease (GD) require continual monitoring; however, lack of specific disease biomarkers was a significant challenge in the past. Glucosylsphingosine (lyso-Gb1) has been shown to be a reliable, key, specific, and sensitive biomarker for diagnosis, prognosis, and treatment response in clinical studies of patients with GD. We evaluated the change in lyso-Gb1 concentration over time following enzyme replacement therapy in patients with confirmed GD using real-world data from the Gaucher Outcome Survey disease registry.
View Article and Find Full Text PDFUsing Pharmacoepidemiology to Examine the Interplay of Sulfonylureas and Infection Risk in Patients With Diabetes Mellitus.
CPT Pharmacometrics Syst Pharmacol
January 2025
School of Medicine, College of Medicine, I-Shou University, Kaohsiung, Taiwan.
Sulfonylureas (SU) are commonly prescribed as oral hypoglycemic agents for the management of diabetes mellitus (DM). We postulated that SU possess antimicrobial properties due to their structural resemblance to the antimicrobial agent sulfamethoxazole. Using data from Taiwan's National Health Insurance Research Database, we enrolled patients diagnosed with DM between 2000 and 2013 and followed them for a three-year period.
View Article and Find Full Text PDFComparative cardiovascular safety of romosozumab versus bisphosphonates in Japanese patients with osteoporosis: a new-user, active comparator design with instrumental variable analyses.
J Bone Miner Res
January 2025
Department of Clinical Epidemiology, Graduate School of Medicine, Fukushima Medical University, Fukushima-city, Fukushima, Japan.
This study analyzed the association of romosozumab, a human monoclonal antibody with bone-forming and bone resorption-inhibiting effects, and bisphosphonates with the development of cardiovascular disease among patients with osteoporosis. A new-user design was employed to address selection bias, and instrumental variable analysis was used to address confounding by indication. Japanese patients aged ≥40 years, diagnosed with osteoporosis or experienced a fragility fracture, were admitted to medical facilities covered by a commercial administrative claims database, and newly prescribed romosozumab or bisphosphonates after the commercialization of romosozumab in Japan (March 4, 2019) were included based on verification of a 180-day washout period.
View Article and Find Full Text PDFAdvancing Principled Pharmacoepidemiologic Research to Support Regulatory and Healthcare Decision Making: The Era of Real-World Evidence.
Clin Pharmacol Ther
January 2025
Center for Observational Research, Amgen Inc., Thousand Oaks, California, USA.
A compilation of factors over the past decade-including the availability of increasingly large and rich healthcare datasets, advanced technologies to extract unstructured information from health records and digital sources, advancement of principled study design and analytic methods to emulate clinical trials, and frameworks to support transparent study conduct-has ushered in a new era of real-world evidence (RWE). This review article describes the evolution of the RWE era, including pharmacoepidemiologic methods designed to support causal inferences regarding treatment effects, the role of regulators and other health authorities in establishing distributed real-world data networks enabling analytics at scale, and the many global guidance documents on principled methods of producing RWE. This article also highlights the growing opportunity for RWE to support decision making by regulators, health technology assessment groups, clinicians, patients, and other stakeholders and provides examples of influential RWE studies.
View Article and Find Full Text PDFDetermining the feasibility of linked claims and vaccination data for a COVID-19 vaccine pharmacoepidemiological study in Germany-RiCO feasibility study protocol.
BMJ Open
December 2024
PMV Research Group, Medical Faculty and University Hospital Cologne, University of Cologne, Koln, Germany
Introduction: In Germany, there has been no population-level pharmacoepidemiological study on the safety of the COVID-19 vaccines. One factor preventing such a study so far relates to challenges combining the different relevant data bodies on vaccination with suitable outcome data, specifically statutory health insurance claims data. Individual identifiers used across these data bodies are of unknown quality and reliability for data linkage.
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