Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
11 Jan 2024
Historique:
medline: 10 1 2024
pubmed: 10 1 2024
entrez: 10 1 2024
Statut: ppublish

Résumé

Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR and leads to more than 90% stabilization across the dosing interval as measured ex vivo. In this phase 3, double-blind trial, we randomly assigned patients with transthyretin amyloid cardiomyopathy in a 2:1 ratio to receive acoramidis hydrochloride at a dose of 800 mg twice daily or matching placebo for 30 months. Efficacy was assessed in the patients who had an estimated glomerular filtration rate of at least 30 ml per minute per 1.73 m A total of 632 patients underwent randomization. The primary analysis favored acoramidis over placebo (P<0.001); the corresponding win ratio was 1.8 (95% confidence interval [CI], 1.4 to 2.2), with 63.7% of pairwise comparisons favoring acoramidis and 35.9% favoring placebo. Together, death from any cause and cardiovascular-related hospitalization contributed more than half the wins and losses to the win ratio (58% of all pairwise comparisons); NT-proBNP pairwise comparisons yielded the highest ratio of wins to losses (23.3% vs. 7.0%). The overall incidence of adverse events was similar in the acoramidis group and the placebo group (98.1% and 97.6%, respectively); serious adverse events were reported in 54.6% and 64.9% of the patients. In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo. Adverse events were similar in the two groups. (Funded by BridgeBio Pharma; ATTRibute-CM ClinicalTrials.gov number, NCT03860935.).

Sections du résumé

BACKGROUND BACKGROUND
Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR and leads to more than 90% stabilization across the dosing interval as measured ex vivo.
METHODS METHODS
In this phase 3, double-blind trial, we randomly assigned patients with transthyretin amyloid cardiomyopathy in a 2:1 ratio to receive acoramidis hydrochloride at a dose of 800 mg twice daily or matching placebo for 30 months. Efficacy was assessed in the patients who had an estimated glomerular filtration rate of at least 30 ml per minute per 1.73 m
RESULTS RESULTS
A total of 632 patients underwent randomization. The primary analysis favored acoramidis over placebo (P<0.001); the corresponding win ratio was 1.8 (95% confidence interval [CI], 1.4 to 2.2), with 63.7% of pairwise comparisons favoring acoramidis and 35.9% favoring placebo. Together, death from any cause and cardiovascular-related hospitalization contributed more than half the wins and losses to the win ratio (58% of all pairwise comparisons); NT-proBNP pairwise comparisons yielded the highest ratio of wins to losses (23.3% vs. 7.0%). The overall incidence of adverse events was similar in the acoramidis group and the placebo group (98.1% and 97.6%, respectively); serious adverse events were reported in 54.6% and 64.9% of the patients.
CONCLUSIONS CONCLUSIONS
In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo. Adverse events were similar in the two groups. (Funded by BridgeBio Pharma; ATTRibute-CM ClinicalTrials.gov number, NCT03860935.).

Identifiants

DOI: 10.1056/NEJMoa2305434 PMID: 38197816
pubmed: 38197816
doi: 10.1056/NEJMoa2305434
doi:

Banques de données

ClinicalTrials.gov
['NCT03860935']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

132-142

Investigateurs

Christopher Hayward (C)
Noemi Horvath (N)
Dariusz Korczyk (D)
Kaitlyn Lam (K)
David Russell (D)
Stephen Bek Ngie Ting (SBN)
Philippe Debonnaire (P)
Riet Dierckx (R)
Matthias Dupont (M)
Philippe Jr Timmermans (PJ)
Johan Van Cleemput (J)
Fabio Fernandes (F)
Paulo Leães (P)
Roberto Sant'Anna (R)
Pedro Schwartzmann (P)
Tonnison Silva (T)
Fabian Alejandro Azzari (FA)
Margot Davis (M)
Diego Delgado (D)
Anique Ducharme (A)
Nowell Fine (N)
Doug Hayami (D)
Gordon Moe (G)
Frederic Poulin (F)
Francois Tournoux (F)
Shelley Zieroth (S)
Jan Krejci (J)
Milos Kubanek (M)
Tomas Palecek (T)
Steen Hvitfeldt Poulsen (SH)
Efstathios Kastritis (E)
Emer Joyce (E)
Kenneth McDonald (K)
Michael Arad (M)
Arthur Pollak (A)
Leonardo Bolognese (L)
Francesco Cappelli (F)
Samuela Carigi (S)
Michele Emdin (M)
Laura Obici (L)
Christian Knackstedt (C)
Marish Oerlemans (M)
Peter van der Meer (P)
Hugh Goodman (H)
Timothy Sutton (T)
Ewa Jankowska (E)
Jacek Grzybowski (J)
João Agostinho (J)
Teresa Coelho (T)
Antoni Bayes-Genis (A)
Pablo Garcia-Pavia (P)
José Gonzalez-Juanatey (J)
Ramón Lecumberri Villamediana (R)
Julio Nuñez (J)
Tomás Ripoll Vera (T)
Dong-Ju Choi (DJ)
Seung-Pyo Lee (SP)
Julian Gillmore (J)
Marianna Fontana (M)
Jorg Taubel (J)
Olakunle Akinboboye (O)
Craig Alpert (C)
Amrut Ambardekar (A)
John Berk (J)
Kunal Bhatt (K)
Mirnela Byku (M)
Richard Cheng (R)
Noel Dasgupta (N)
Brian Drachman (B)
J Emanuel Finet (JE)
Mazen Hanna (M)
Cesia Gallegos (C)
Robert Gordon (R)
Justin Grodin (J)
Martha Grogan (M)
Jason Guichard (J)
James Hoffman (J)
Sandeep Jani (S)
Michael Johnstone (M)
Daniel Judge (D)
Saurabh Kapoor (S)
Michel Khouri (M)
Catherine Marti (C)
Ahmad Masri (A)
Mathew Maurer (M)
Joshua Mitchell (J)
Sumeet Mitter (S)
Deirdre Mooney (D)
Jignesh Patel (J)
Alex Reyentovich (A)
Nitasha Sarswat (N)
John Schmedtje (J)
Keyur Shah (K)
Sanjiv Shah (S)
Farooq Sheikh (F)
Prem Soman (P)
Brett Sperry (B)
Josef Stehlik (J)
David Stern (D)
James Tauras (J)
Richard Wright (R)
Alejandra Gonzalez-Duarte (A)
Matthew S Maurer (MS)
Renato Lopes (R)
Simon Gibbs (S)

Informations de copyright

Copyright © 2024 Massachusetts Medical Society.

Auteurs

Julian D Gillmore (JD)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Daniel P Judge (DP)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Francesco Cappelli (F)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Marianna Fontana (M)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Pablo Garcia-Pavia (P)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Simon Gibbs (S)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Martha Grogan (M)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Mazen Hanna (M)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

James Hoffman (J)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Ahmad Masri (A)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Mathew S Maurer (MS)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).
ORCID: 0000-0001-5400-5008

Jose Nativi-Nicolau (J)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Laura Obici (L)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Steen Hvitfeldt Poulsen (SH)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Frank Rockhold (F)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Keyur B Shah (KB)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Prem Soman (P)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Jyotsna Garg (J)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Karen Chiswell (K)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Haolin Xu (H)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Xiaofan Cao (X)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Ted Lystig (T)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Uma Sinha (U)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Jonathan C Fox (JC)

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Classifications MeSH