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.

Conclusion:

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.

Plain Language Summary

Current State of Care for People With Amyloidosis at Specialized U.S. Centers: What Works and What We Need to Improve

Why was this study done?

Amyloidosis is a rare disease that can affect several organs in the body. Because it is complex and not well-known, people with amyloidosis often have challenges getting the right diagnosis and care. In recent years, specialized amyloidosis centers (SACs) have been set up across the United States by hospitals to provide appropriate care to these patients. This study aimed to understand how these centers work, what they do well, and what challenges they face.

What did the researchers do?

The research team spoke with 77 people across the U.S., including doctors, nurses, patients, referring physicians, and leaders of patient advocacy groups. All participants were involved in amyloidosis care. The interviews explored how SACs provide care, how patients access services, and what could be improved.

What did the research find?

Participants described several best practices at SACs, such as coordinated care by teams of different specialists, fast intake for high-risk patients, and strong communication with patients. Most SACs also track data on patients and many are involved in clinical trials. Patients valued the compassion and responsiveness of SAC staff.

However, some challenges remain. Patients and doctors still see a lot of delays in diagnosis. Travel distance to centers, insurance barriers, and limited emotional support were common issues. Many patients said they received little information at the time of diagnosis. Telemedicine was helpful for some but not ideal for all situations.

What do these findings mean?

This study shows that SACs offer many benefits for people with amyloidosis, but improvements are still needed–especially in education, emotional support, and coordination with local care providers. The researchers and participants hope that by sharing their perspectives, newer SACs and doctors, as well as patients, can be better informed about providing and receiving care that centers the patient experience.