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Quality-of-Life Measurement in Epidermolysis Bullosa. Position Statement of the European Academy of Dermatology and Venereology Task Force on Quality of Life and Patient-Oriented Outcomes and External Experts.
Int J Dermatol
January 2025
Department of Dermatology, Zealand University Hospital, Roskilde, Denmark.
In this paper, the European Academy of Dermatology and Venereology (EADV) Task Force on Quality of Life (QoL) and Patient-Oriented Outcomes presents its position statements on health-related (HR) QoL assessment in epidermolysis bullosa (EB). The EADV TF on QoL and Patient-Oriented Outcomes recommends the use of the EB-specific instrument QOLEB in patients over the age of 10 years and, in addition to the QOLEB, the use of iscorEB-p in moderate-to-severe EB; the IntoDermQoL proxy instrument with its EB-specific module should be used in children aged under 5 years. The EB-specific instrument iscorEB-p, and the dermatology-specific instrument CDLQI may measure HRQoL in children with EB aged from 5 to 10 years.
View Article and Find Full Text PDFUnlabelled: Transparent and accurate reporting in early phase dose-finding (EPDF) clinical trials is crucial for informing subsequent larger trials. The SPIRIT statement, designed for trial protocol content, does not adequately cover the distinctive features of EPDF trials. Recent findings indicate that the protocol contents in past EPDF trials frequently lacked completeness and clarity.
View Article and Find Full Text PDFUnlabelled: Early phase dose-finding (EPDF) trials are key in the development of novel therapies, with their findings directly informing subsequent clinical development phases and providing valuable insights for reverse translation. Comprehensive and transparent reporting of these studies is critical for their accurate and critical interpretation, which may improve and expedite therapeutic development. However, quality of reporting of design characteristics and results from EPDF trials is often variable and incomplete.
View Article and Find Full Text PDFThe Role of Multimodality Imaging in Multiple Valvular Heart Diseases. A Clinical Consensus Statement of the European Association of Cardiovascular Imaging (EACVI) of the ESC.
Eur Heart J Cardiovasc Imaging
January 2025
Department of Cardiology, Leiden University Medical Center, Albinusdreef 2, 2300RC Leiden, The Netherlands.
With this document, the European Association of Cardiovascular Imaging (EACVI) provides an Expert Consensus on the role of multi-modality imaging (MMI) in the management of patients with multiple valvular heart disease (MVD). Emphasis is given to the use of MMI to unravel the diagnostic challenges that characterize these patients and to improve risk stratification. Complementing the last European Society of Cardiology and European Association of Cardio-Thoracic Surgery guidelines on valvular heart disease, this Expert Consensus document also outlines how MMI assessment should form an integral part of the multi-disciplinary heart team discussion for patients with MVD to help with complex decision-making regarding the choice and timing of treatment.
View Article and Find Full Text PDFIdentification of Cell Fate Determining Transcription Factors for Generating Brain Endothelial Cells.
Stem Cell Rev Rep
January 2025
Stem Cell Institute, Department of Development and Regeneration, KU Leuven, O&N IV Herestraat 49, Leuven, 3000, Belgium.
Reliable models of the blood-brain barrier (BBB), wherein brain microvascular endothelial cells (BMECs) play a key role in maintenance of barrier function, are essential tools for developing therapeutics and disease modeling. Recent studies explored generating BMEC-like cells from human pluripotent stem cells (hPSCs) by mimicking brain-microenvironment signals or genetic reprogramming. However, due to the lack of comprehensive transcriptional studies, the exact cellular identity of most of these cells remains poorly defined.
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