Publications

2025 Amyloidosis Forum Annual Meeting Report

Amyloidosis Research Consortium. 2025 Amyloidosis Forum Annual Meeting: Cross Stakeholder Working Group Projects to Accelerate Drug Development in AL and ATTR Amyloidosis. Newton (MA): Amyloidosis Research Consortium; 2026.

Prognostic Factors and Progression Biomarkers in AL Amyloidosis: Mapping Current Knowledge and Critical Gaps

Rajshekhar Chakraborty, Yevgeniy Brailovsky, Mazen Hanna, Ronald Witteles, Joban Vaishnav, James E. Hoffman, Jan Marie Griffin, Pablo Garcia-Pavia, David Wolinsky, Chafic Karam, Helen J Lachmann, Morie A. Gertz, Brian C Boursiquot, Dimitrios Bampatsias, Kristen Hsu, Phaedra Theodora Johnson, Jamie L Zigterman, Ana Carolina Kazemzadeh, Mathew S. Maurer, Ashutosh D. Wechalekar; Prognostic Factors and Progression Biomarkers in AL Amyloidosis: Mapping Current Knowledge and Critical Gaps. Blood 2026; blood.2025031651. doi: https://doi.org/10.1182/blood.2025031651

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

Dorbala, S, Adigun, R, Alexander, K. et al. Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis: Proceedings of the Amyloidosis Forum. J Am Coll Cardiol Img. null2025, 0 (0) . https://doi.org/10.1016/j.jcmg.2024.11.003

Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings

Maurer MS, Soman P, Hernandez A, Garcia-Pavia P, Signorovitch J, Wei LJ, Hanna M, Ruberg FL, Kittleson M, Kazi D, Dorbala S, Hsu K, Lousada I, Adigun R, Dunnmon P, Kelly J, Gillmore J; Amyloidosis Forum Meeting Panelists. Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings. Adv Ther 41, 2723–2742 (2024). https://doi.org/10.1007/s12325-024-02891-0

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.

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.

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.

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

Mauermann ML, Clarke JO, Litchy WJ, Obici L, Lousada I, Gertz MA; Amyloidosis Forum Working Group Participants. Peripheral Nervous, Hepatic, and Gastrointestinal Endpoints for AL Amyloidosis Clinical Trials: Report from the Amyloidosis Forum Multi-organ System Working Group. Adv Ther. 2023 Nov;40(11):4695-4710. doi: 10.1007/s12325-023-02637-4. Epub 2023 Sep 2. PMID: 37658177; PMCID: PMC10567953.

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

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

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

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