Evidence map›Paper›PMID 39985507›Full record

ReviewJACC. Cardiovascular imaging2025

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

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 and 29 more

Abstract readReview
In one paragraph

Review in JACC. Cardiovascular imaging, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

39 authors.

Sharmila DorbalaBrigham and Women's Hospital, Boston, Massachusetts, USA.
Rosalyn AdigunU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Kevin M AlexanderStanford University, Stanford, California, USA.
Michela BrambattiIonis Pharmaceuticals, Inc, Carlsbad, California, USA.
Sarah A M CuddyBrigham and Women's Hospital, Boston, Massachusetts, USA.
Angela DispenzieriMayo Clinic, Rochester, Minnesota, USA.
Preston DunnmonJanssen Research and Development, Raritan, New Jersey, USA.
Michele EmdinScuola Superiore Sant'Anna, Fondazione G. Monasterio, Pisa, Italy.
Omar F Abou EzzeddineMayo Clinic, Rochester, Minnesota, USA.
Rodney H FalkHarvard Medical School, Boston, Massachusetts, USA.
Mariana FontanaUniversity College London, London, England, United Kingdom.
Justin L GrodinUniversity of Texas Southwestern Medical Center, Dallas, Texas, USA.
Spencer GuthrieAttralus, Inc, San Francisco, California, USA.
Michael Jerosch-HeroldBrigham and Women's Hospital, Boston, Massachusetts, USA.
A Alex HoflingU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Kristen HsuAmyloidosis Research Consortium, Newton, Massachusetts, USA.
Grace LinMayo Clinic, Rochester, Minnesota, USA.
Ahmad MasriOregon Health and Science University, Portland, Oregon, USA.
Mathew S MaurerColumbia University Irving Medical Center, New York, New York, USA.
Clemens MittmannFederal Institute for Drugs and Medical Devices, Bonn, Germany.
Krishna PrasadUK Medicines and Healthcare Products Regulatory Agency, London, England, United Kingdom.
Cristina C QuartaAlexion Pharmaceuticals, Inc, Boston, Massachusetts, USA.
Jean-Michel RaceAgence nationale de sécurité du médicament et des produits de santés, Saint Denis, France.
Joseph G RajendranU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Frederick L RubergBoston University Chobanian and Avedisian School of Medicine, Boston, Massachusetts, USA.
Vandana SachdevNational Heart, Lung, and Blood Institute, Bethesda, Maryland, USA.
Vaishali SanchorawalaBoston University Chobanian and Avedisian School of Medicine, Boston, Massachusetts, USA.
James SignorovitchAnalysis Group, Boston, Massachusetts, USA.
Christophe SiracUniversity of Limoges, Limoges, France.
Prem SomanUniversity of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA.
Jens SorensenUppsala University, Uppsala, Sweden.
Brett W SperrySaint Luke's Mid America Heart Institute, Kansas City, Missouri, USA.
Andrew W StephensLife Molecular Imaging, Berlin, Germany.
Norman L StockbridgeU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
John VestAlnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Jonathan S WallUniversity of Tennessee Graduate School of Medicine, Knoxville, Tennessee, USA.
Ashutosh WechalekarUniversity College London, London, England, United Kingdom.
Cynthia WelshU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Isabelle LousadaAmyloidosis Research Consortium, Newton, Massachusetts, USA. Electronic address: ILousada@arci.org.

Funding

SCAN-MP (Screening for Cardiac Amyloidosis with Nuclear imaging in Minority Populations) COVID-19 SuppplementR01HL139671 · NHLBI · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI MAURER, MATHEW S, RUBERG, FREDERICK LIEF · 2019 to 2023
$7.4M
IDENTIFYING SUBCLINICAL TRANSTHYRETIN CARDIAC AMYLOIDOSIS IN ASYMPTOMATIC CARRIERS OF THE V122I TTR ALLELER01HL160892 · NHLBI · UT SOUTHWESTERN MEDICAL CENTER · PI Justin Lee Grodin · 2022 to 2026
$3.6M
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac AmyloidosisR01AG081582 · NIA · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI MATHEW S MAURER · 2023 to 2026
$2.8M
Personalized Exercise Training to Improve Functional Capacity in Transthyretin Cardiac AmyloidosisK23HL166686 · NHLBI · BRIGHAM AND WOMEN'S HOSPITAL · PI Sarah AM Cuddy · 2023 to 2026
$756k
NHLBI NIH HHS K23 HL166686NHLBI NIH HHS R01 HL139671NHLBI NIH HHS R01 HL160892NIA NIH HHS R01 AG081582
6 · The paper itself

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.

Indexed as

Amyloid Neuropathies, FamilialCardiomyopathiesClinical Trials as TopicDiagnostic ImagingEndpoint DeterminationImmunoglobulin Light-chain AmyloidosisAmyloidosisBiomarkersHumansPredictive Value of TestsPrognosisBiomarkersamyloidosiscardiac imagingclinical trial endpoints

Identifiers

PMID39985507
PMCPMC12161488

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.