Publications

Factors associated with financial toxicity in patients with transthyretin amyloidosis: results from Amyloidosis Research Consortium’s treatment affordability patient and caregiver survey

Rebello, S., Hsu, K., Nativi-Nicolau, J., Karam, C., Grogan, M., Lousada, I., & Maurer, M. S. (2025). Factors associated with financial toxicity in patients with transthyretin amyloidosis: results from Amyloidosis Research Consortium’s treatment affordability patient and caregiver survey. Amyloid, 1–10. https://doi.org/10.1080/13506129.2025.2462541

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Hereditary ATTR Amyloidosis

Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis

Signorovitch J, Zhang J, Brown D, Dunnmon P, Xiu L, Done N, Hsu K, Barbachano Y, Lousada I. Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis. Ther Innov Regul Sci. 2024 Jul;58(4):600-609. doi: 10.1007/s43441-024-00641-6. Epub 2024 Apr 17. PMID: 38632158; PMCID: PMC11169055.

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Renal Response Criteria for Clinical Trials in Amyloid Light Chain Amyloidosis

Leung N, Comenzo R, Gillmore J, Havasi A, Kastritis E, Guthrie S, Signorovitch J, Heath D, Lousada I; Amyloidosis Forum Working Group Participants. Renal Response Criteria for Clinical Trials in Amyloid Light Chain Amyloidosis. Kidney Int Rep. 2024 Apr 4;9(7):1986-1994. doi: 10.1016/j.ekir.2024.03.033. PMID: 39081759; PMCID: PMC11284377.

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The Transthyretin Amyloidosis – Quality of Life (ATTR-QOL) Questionnaire: Development of a Conceptual Model and Disease-Specific Patient-Reported Outcome Measure

O'Connor, Hsu, Broderick, McCausland, LaGasse, Rebello, Carty, Lousada (2023)

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Peripheral Nervous, Hepatic, and Gastrointestinal Endpoints for AL Amyloidosis Clinical Trials: Report from the Amyloidosis Forum Multi-organ System Working Group

Mauermann, M.L., Clarke, J.O., Litchy, W.J. et al. Adv Ther (2023)

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Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group

Rizio AA, White MK, D’Souza A, Hsu K, Schmitt P, Quock TP, Signorovitch J, Lousada I, Sanchorawala V. Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group. Patient Relat Outcome Meas. 2023 May 18;14:153-169. doi: 10.2147/PROM.S399658. PMID: 37229285; PMCID: PMC10202704.

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Evaluation of NT-proBNPas surrogate endpoint in AL amyloidosis: development of a platform for federated, multi-institution meta-analysis of randomized trials

Angela Dispenzieri, James Signorovtich, Mathew S. Maurer, Xianghua Huang, Vaishali Sanchorawala, Preston Dunnmon, Catherine Klersy, Giulia Gambini, David Brown, Yolanda Barbachano, Rosalyn Adigun, Kristen Hsu, Isabelle Lousada, Liang Xiu, Douglas V. Faller, Richard Labotka, Giampaolo Merlini, Giovanni Palladini. XVIII Meeting of the International Society of Amyloidosis, 2022

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Proposed Cardiac End Points for Clinical Trials in Immunoglobulin Light Chain Amyloidosis: Report From the Amyloidosis Forum Cardiac Working Group

Mathew S Maurer, Preston Dunnmon, Mariana Fontana, Christina Candida Quarta, Krishna Prasad, Ronald M. Witteles, Claudio Rapezzi, James Signorovitch, Isabelle Lousada, Giampaolo Merlini, Circulation: Heart Failure, 121.009038, March 2022

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ARC Compass: A Newsletter for Amyloidosis Research Updates

Compass is a newsletter for amyloidosis research updates with the goal of keeping the amyloidosis community informed of ARC’s research and initiatives. The June 2021 edition focuses on the recently completed Amyloidosis Research Consortium Cardiac Amyloidosis Survey.

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[Poster] The Amyloidosis Forum: A Public-Private Partnership to Advance Drug Development in AL Amyloidosis

Isabelle Lousada, Vaishali Sanchorawala, Angela Dispenzieri, Kristen Hsu, Robyn Himick, Preston Dunnmon, Mathew Maurer. International Society of Amyloidosis, Annual Meeting 2020

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The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis

Lousada and The Inaugural Amyloidosis Forum Panelists, Orphanet Journal of Rare Diseases (2020) 15:268

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ARC’s European Health Technology Assessments (HTA) Toolkit

ARC's comprehensive European Health Technology Assessments (HTA) Toolkit was produced to give information about how treatments are evaluated and approved for use in the various member state healthcare systems. The Toolkit guides patients and patient groups on how they can participate in the process to ensure that those treatments that are developed for their amyloidosis can be made available in their countries.

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Expert Consensus Recommendations to Improve Diagnosis of ATTR Amyloidosis with Polyneuropathy

ARC-supported consensus best practice recommendations for the diagnosis of ATTR amyloidosis with polyneuropathy published in the Journal of Neurology, Circulation: Heart Failure.

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Amyloidosis Research Consortium Cardiac Amyloidosis Survey: An International Analysis of the Diagnostic Journey in AL and ATTR Amyloidosis

"Amyloidosis Research Consortium Cardiac Amyloidosis Survey: An International Analysis of the Diagnostic Journey in AL and ATTR Amyloidosis," was presented at the 23rd annual Heart Failure Society of America meeting from September 13-16, 2019 in Philadelphia, PA. This poster presents results from 745 patients and caregivers and highlights regional differences, changes over time, and how we can improve the diagnostic journey of amyloidosis patients.

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Expert Consensus Recommendations for the Suspicion and Diagnosis of Cardiac Transthyretin Amyloidosis

ARC-supported consensus best practices guidelines for the diagnosis of cardiac ATTR amyloidosis published in the journal of the American Heart Association, Circulation: Heart Failure.

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(99m) Technetium-Pyrophosphate Imaging for Transthyretin Cardiac Amyloidosis

These practice points from the American Society of Nuclear Cardiology (ASNC) are to identify the critical components involved in performing (99m) Tc-PYP imaging for the evaluation of cardiac transthyretin amyloidosis (ATTR).

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Bridging the Gap: ARC’s Research Strategy

ARC's comprehensive research strategy promotes a "from the patient's bedside back to bench" model, where patient's needs drive our prioritized portfolio of research. Read in detail our research strategy, and learn how we intend to achieve and measure our goals.

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Hereditary Transthyretin Amyloidosis (hATTR): Burden of Disease and Treatment Perspectives Poster

ARC’s research on Hereditary Transthyretin Amyloidosis (hATTR): Burden of Disease and Treatment Perspectives, was presented at the XII Paulista Congress of Neurology in Brazil. The English version of the poster is translated from Portuguese, and the data can be used by the Brazilian HTA during their evaluation of new treatments for patients with hATTR neuropathy.

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Amyloidois Research Consortium Cardiac Amyloidosis Survey: Results from Patients with AL and ATTR Amyloidosis and their Caregivers

Amyloidosis Research Consortium Cardiac Amyloidosis Survey: Results from Patients with AL and ATTR Amyloidosis and their Caregivers, was presented at the XVIth International Symposium on Amyloidosis from March 26-29, 2018 in Kumamoto, Japan. This poster presents results from 699 patients and caregivers and highlights the complex and burdensome diagnostic journey in amyloidosis.

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Advancing Amyloidosis: A Research Roadmap

The white paper contains the highest priorities and a research framework for the systemic amyloidosis diseases. This consensus document was developed through a rigorous process with the participation of 71 international experts from academic, clinical, regulatory, patient advocacy, health policy, industry, and other key areas. This publication is intended to identify, improve and accelerate a drug development pathway for the systemic amyloidosis diseases.

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Voice of the Patient Report

A comprehensive report submitted to the FDA in 2016 by ARC. This is a detailed summary of the patient testimony presented at the Patient-Focused Drug Development meeting. This report will serve a critical function in communicating to both FDA review staff and industry the improvements that patients want and hope to see in their daily lives.

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Rationale, Application and Clinical Qualification for NT-proBNP as a Surrogate End Point

Published: Leukemia, July 2016 | Author: G Merlini et al.

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Light Chain Amyloidosis Patient Experience Survey

Published: Advances in Therapy, 2015 | Author: I Lousada et al.

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ARC Cardiac Amyloidosis Survey: Results from AL Patients and Caregivers

Amyloidosis Research Consortium Cardiac Amyloidosis Survey: Results from Patients with AL Amyloidosis and Their Caregivers, was presented at the European Hematology Association (EHA) 22nd Annual Congress from June 22 - 25, 2017 in Madrid, Spain. This poster captures results from 137 AL amyloidosis patients and caregivers to help understand the difficult diagnostic journey and experiences of AL amyloidosis patients.

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Guidance for Industry AL Amyloidosis – Developing Drugs for Industry

Developing the guidance involved active participation from all stakeholder groups including patients, medical experts, academics and biopharmaceutical industry representatives. This guidance has been submitted to FDA.

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