Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
Light chain amyloidosis and transthyretin amyloidosis are rare protein misfolding disorders characterized by amyloid deposition in organs, varied clinical manifestations, and poor outcomes. Amyloid fibrils trigger various signaling pathways that initiate cellular, metabolic, structural, and functional changes in the heart and other organs. Imaging modalities have advanced to enable detection of amyloid deposits in involved organs and to assess organ dysfunction, disease stage, prognosis, and treatment response. The Amyloidosis Forum hosted a hybrid meeting to focus on the use of imaging endpoints in clinical trials for systemic immunoglobulin light chain amyloidosis and transthyretin amyloidosis. Stakeholders from academia and industry, together with representatives from multiple regulatory agencies reviewed the use of imaging biomarkers with a focus on cardiac amyloidosis, described applications and limitations of imaging in clinical trials, and discussed qualification of imaging as a surrogate clinical outcome. Survey results provided important patient perspectives. This review summarizes the proceedings of the Amyloidosis Forum.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC12161488 | PMC |
| http://dx.doi.org/10.1016/j.jcmg.2024.11.003 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Pharmacotherapy of cardiovascular diseases from herbs and pills to nucleic acids.
Eur Heart J
August 2025
Royal Brompton and Harefield Hospitals, Imperial College London and King's College London, London, UK.
Cardiovascular (CV) diseases continue to cause substantial morbidity and mortality. Risk factors are inadequately controlled, compliance with medication remains suboptimal, and treatments are not sufficient to fully prevent the progression of atherosclerotic CV disease, heart failure, arrhythmias, and valvular heart diseases. An increased understanding of the genetic basis of CV diseases and advances in the technology of therapeutics have led to the development of nucleic acid-based therapies (NATs) for prevention and treatment of CV risk factors and diseases.
View Article and Find Full Text PDFCardiac transthyretin amyloidosis treatment improves outcomes after aortic valve replacement for severe stenosis.
Eur Heart J
June 2025
Barts Heart Centre, KGV Building, St Bartholomew's Hospital, West Smithfield, London EC1A 7BE, UK.
Background And Aims: Concomitant aortic stenosis (AS) and transthyretin-associated cardiac amyloidosis (ATTR-CA) is an increasingly recognized cause of structural heart failure. Aortic valve replacement (AVR) improves prognosis in this population, but the efficacy of ATTR-specific medication remains unclear. This study aimed to investigate the prognostic implications of ATTR-specific medication in patients with dual AS-CA.
View Article and Find Full Text PDFDevelopment of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis: Proceedings of the Amyloidosis Forum.
JACC Cardiovasc Imaging
May 2025
Amyloidosis Research Consortium, Newton, Massachusetts, USA. Electronic address:
Light chain amyloidosis and transthyretin amyloidosis are rare protein misfolding disorders characterized by amyloid deposition in organs, varied clinical manifestations, and poor outcomes. Amyloid fibrils trigger various signaling pathways that initiate cellular, metabolic, structural, and functional changes in the heart and other organs. Imaging modalities have advanced to enable detection of amyloid deposits in involved organs and to assess organ dysfunction, disease stage, prognosis, and treatment response.
View Article and Find Full Text PDFDepressive Symptoms and Amyloid Pathology.
JAMA Psychiatry
March 2025
Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, Sahlgrenska Academy at the University of Gothenburg, Mölndal, Sweden.
Importance: Depressive symptoms are associated with cognitive decline in older individuals. Uncertainty about underlying mechanisms hampers diagnostic and therapeutic efforts. This large-scale study aimed to elucidate the association between depressive symptoms and amyloid pathology.
View Article and Find Full Text PDFAssociation of Resilience-Related Life Experiences on Variability on Age of Onset in Dominantly Inherited Alzheimer Disease.
Neurology
October 2024
From the Department of Nuclear Medicine (H.J.S., Jai-Hyuen Lee), Dankook University College of Medicine, Cheonan, Chung Nam; Department of Nuclear Medicine (J.S.K.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea; Department of Neurology (R.J.B., J.J.L.-G., J.C.M.,
Article Synopsis
- The study examines the link between protective lifestyle factors and the age at symptom onset of autosomal dominant Alzheimer disease (ADAD), which is largely influenced by genetics, focusing on how resilience-related experiences may impact this relationship.
- The researchers analyzed data from the Dominantly Inherited Alzheimer Network, assessing both clinical and lifestyle factors in two groups: one looking at general resilience among people showing cognitive stability despite high pathology and another focusing on specific genetic timelines for ADAD.
- Findings from 320 participants indicate variations in age at onset among carriers based on lifestyle and resilience factors, revealing potential avenues for understanding how these influences might delay the onset of symptoms in genetically predisposed individuals.