TY - JOUR
T1 - Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis
T2 - Proceedings of the Amyloidosis Forum
AU - Dorbala, Sharmila
AU - Adigun, Rosalyn
AU - Alexander, Kevin M.
AU - Brambatti, Michela
AU - Cuddy, Sarah A.M.
AU - Dispenzieri, Angela
AU - Dunnmon, Preston
AU - Emdin, Michele
AU - Abou Ezzeddine, Omar F.
AU - Falk, Rodney H.
AU - Fontana, Mariana
AU - Grodin, Justin L.
AU - Guthrie, Spencer
AU - Jerosch-Herold, Michael
AU - Hofling, A. Alex
AU - Hsu, Kristen
AU - Lin, Grace
AU - Masri, Ahmad
AU - Maurer, Mathew S.
AU - Mittmann, Clemens
AU - Prasad, Krishna
AU - Quarta, Cristina C.
AU - Race, Jean Michel
AU - Rajendran, Joseph G.
AU - Ruberg, Frederick L.
AU - Sachdev, Vandana
AU - Sanchorawala, Vaishali
AU - Signorovitch, James
AU - Sirac, Christophe
AU - Soman, Prem
AU - Sorensen, Jens
AU - Sperry, Brett W.
AU - Stephens, Andrew W.
AU - Stockbridge, Norman L.
AU - Vest, John
AU - Wall, Jonathan S.
AU - Wechalekar, Ashutosh
AU - Welsh, Cynthia
AU - Lousada, Isabelle
N1 - Publisher Copyright:
© 2025 The Authors
PY - 2025/5
Y1 - 2025/5
N2 - 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.
AB - 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.
KW - amyloidosis
KW - cardiac imaging
KW - clinical trial endpoints
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U2 - 10.1016/j.jcmg.2024.11.003
DO - 10.1016/j.jcmg.2024.11.003
M3 - Review article
C2 - 39985507
AN - SCOPUS:105000069021
SN - 1936-878X
VL - 18
SP - 602
EP - 617
JO - JACC: Cardiovascular Imaging
JF - JACC: Cardiovascular Imaging
IS - 5
ER -