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Best Practices and Key Barriers for Amyloidosis Patient Care at US Specialized Amyloidosis Centers: An Analysis by ARC-ASPIRE
Publication Details
Clinical Medicine Insights: Cardiology
April 2026
Author(s)
Jose Nativi-Nicolau1, Johana Fajardo2, Alyssa Galloway3 4, Sami Khella5, Nisith Kumar4 6, Michaela Liedtke7, Isabelle Lousada8, Tiffany Patrick 4 9, Colleen Moffitt4 9, Lori Klein10, Gabriela Tamariz4 9, Karthikeyan Veeraraghavalu4 11, Alexandra Haddad-Angulo4 6
Affiliations
1Mayo Clinic, FL, USA; 2Duke University Hospital, NC, USA; 3BridgeBio, CA, USA; 4Amyloidosis Reserach Consortium (ARC) - Amyloidosis Stakeholder Partnerships for Impact, Reach & Equity (ASPIRE), MA, USA; 5University of Pennsylvania School of Medicine, PA, USA; 6Pfizer Inc, NY, USA; 7Stanford Health Care, CA, USA; 8Amyloidosis Research Consortium (ARC), MA, USA; 9Alnylam Pharmaceuticals, MA, USA; 10Putnam Associates, MA, USA; 11Prothena Biosciences Inc, CA, USA
Abstract
Objectives:
Amyloidosis is a group of rare diseases that often manifest in multi-organ symptoms requiring an effective multidisciplinary team of care providers to manage. This study aimed to identify the best practices and key barriers to patient-centric care at US specialized amyloidosis centers (SACs) which have emerged as central hubs for multidisciplinary amyloidosis care.
Methods:
We conducted and analyzed interviews with 77 stakeholders of amyloidosis diseases, including specialist physicians from 17 SACs across the US, patients, patient advocacy group representatives, and referring physicians.
Results:
The most commonly mentioned best practices were patient-centric multidisciplinary care, timely intake of new high-risk patients, and responsiveness to patient questions. Expanding local physician education was noted as critical to earlier diagnosis. Fifteen out of 17 SACs are actively involved in clinical trials for amyloidosis treatments. Key barriers to care identified were patient distance to SACs, limited medical records and data sharing, and insurance hurdles. Most SACs keep track of metrics, such as patient volume, patient demographics, time to first appointment, as part of internal or in external registries to measure progress.
Conclusions:
These findings offer practical insights for optimizing amyloidosis care and highlight the structural and informational gaps that must be addressed to improve access and outcomes.
PMID
42077643
DOI
10.1177/11795468261444669
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