Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis: Proceedings of the Amyloidosis Forum.

IF 12.8 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

JACC. Cardiovascular imaging Pub Date : 2025-02-20 DOI:10.1016/j.jcmg.2024.11.003

Sharmila Dorbala, Rosalyn Adigun, Kevin M Alexander, Michela Brambatti, Sarah A M Cuddy, Angela Dispenzieri, Preston Dunnmon, Michele Emdin, Omar F Abou Ezzeddine, Rodney H Falk, Mariana Fontana, Justin L Grodin, Spencer Guthrie, Michael Jerosch-Herold, A Alex Hofling, Kristen Hsu, Grace Lin, Ahmad Masri, Mathew S Maurer, Clemens Mittmann, Krishna Prasad, Cristina C Quarta, Jean-Michel Race, Joseph G Rajendran, Frederick L Ruberg, Vandana Sachdev, Vaishali Sanchorawala, James Signorovitch, Christophe Sirac, Prem Soman, Jens Sorensen, Brett W Sperry, Andrew W Stephens, Norman L Stockbridge, John Vest, Jonathan S Wall, Ashutosh Wechalekar, Cynthia Welsh, Isabelle Lousada

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引用次数: 0

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.

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来源期刊

JACC. Cardiovascular imaging CARDIAC & CARDIOVASCULAR SYSTEMS-RADIOLOGY, NUCLEAR MEDICINE & MEDICAL IMAGING

CiteScore

24.90

自引率

5.70%

发文量

330

审稿时长

4-8 weeks

期刊介绍： JACC: Cardiovascular Imaging, part of the prestigious Journal of the American College of Cardiology (JACC) family, offers readers a comprehensive perspective on all aspects of cardiovascular imaging. This specialist journal covers original clinical research on both non-invasive and invasive imaging techniques, including echocardiography, CT, CMR, nuclear, optical imaging, and cine-angiography. JACC. Cardiovascular imaging highlights advances in basic science and molecular imaging that are expected to significantly impact clinical practice in the next decade. This influence encompasses improvements in diagnostic performance, enhanced understanding of the pathogenetic basis of diseases, and advancements in therapy. In addition to cutting-edge research,the content of JACC: Cardiovascular Imaging emphasizes practical aspects for the practicing cardiologist, including advocacy and practice management.The journal also features state-of-the-art reviews, ensuring a well-rounded and insightful resource for professionals in the field of cardiovascular imaging.