Background: Alternative care models seek to improve the quality or efficiency of care, or both, and thus optimise patient health outcomes. They provide the same health care but change how, when, where, or by whom health care is delivered and co-ordinated. Examples include care delivered via telemedicine versus in-person care or care delivered to groups versus individual patients.
View Article and Find Full Text PDFBackground: Facioscapulohumeral muscular dystrophy (FSHD) is a hereditary muscle disease without an available cure. The first trials with potentially disease-modifying therapies have started, including a phase ll open-label study and a phase lll double-blind randomized placebo-controlled trial assessing the safety and efficacy of losmapimod. Having a more in-depth understanding of the patient's experience of these trials will further enhance the design and recruitment of future trials.
View Article and Find Full Text PDFPortal vein stenosis (PVS) is a relatively frequent vascular complication after pediatric liver transplantation (pLT) that may result in portal hypertension. The aim of this study was to provide an overview of various diagnostic methods and imaging criteria used to diagnose PVS and to report their diagnostic accuracy. Until August 2024, PubMed and Embase were searched for English-language manuscripts with >5 patients and radiologic features of PVS.
View Article and Find Full Text PDFIntroduction: Risk prediction models (RPMs) are statistical tools that predict outcomes on the basis of clinical characteristics and can thereby support (shared) decision-making. With the shift toward personalized medicine, the number of RPMs has increased exponentially, including in multimodal sarcoma care. However, their integration into routine soft-tissue sarcoma (STS) care remains largely unknown.
View Article and Find Full Text PDFBackground And Objectives: Mitochondrial disorders are multiorgan disorders resulting in significant morbidity and mortality. We aimed to characterize death-associated factors in an international cohort of deceased individuals with mitochondrial disorders.
Methods: This cross-sectional multicenter observational study used data provided by 26 mitochondrial disease centers from 8 countries from January 2022 to March 2023.