Aug. 13, 2026
Cardiac amyloidosis has historically been a rare, fatal disease with limited treatment options. However, with new therapies in development and updated protocols for heart transplantation, patients with amyloidosis have a chance for a cure.
Jose N. Nativi Nicolau, M.D., a transplant cardiologist at Mayo Clinic in Jacksonville, Florida, is part of an international group of investigators dedicated to developing therapies to treat amyloidosis.
"Just a few years ago, amyloidosis had limited options for therapy. That is not true anymore," Dr. Nativi Nicolau says. "We now have frameworks to support heart transplantation and medications that prevent the progression of the disease — and patients are living longer because of it."
How transplantation benefits patients with amyloidosis
While heart transplantation has not always been an option for patients with amyloidosis, new protocols are helping identify patients who are candidates, shortening wait times for new organs.
Mayo Clinic recently published a consensus statement on heart transplantation for cardiac amyloidosis, led by Melissa A. Lyle, M.D., a transplant cardiologist at Mayo Clinic in Jacksonville, Florida, and Taimur Sher, M.B.B.S., M.D., a hematologist at Mayo Clinic in Jacksonville, Florida. An article published in Mayo Clinic Proceedings provides a framework for Mayo Clinic's selection criteria, multidisciplinary approach, and timing of heart transplantation for patients with cardiac amyloidosis.
لُييفات الداء النشواني في عضلة القلب
يحدث الداء النشواني القلبي عندما يتحول البروتين إلى لُييفات الداء النشواني وتترسب في أنسجة عضلة القلب. ويمكن لهذا الترسب أن يعيق عددًا من الأعضاء عن العمل بطريقة سليمة مع مرور الوقت.
[تُظهر هذه الرسوم المتحركة لُييفات الداء النشواني المترسبة في عضلة القلب. وهي تُعرَض من دون صوت].
"Few centers in the world perform heart transplantation in patients with amyloidosis," Dr. Sher says. "This statement highlights the role that cardiac involvement plays in increasing the severity of amyloidosis, and the necessity of evaluation for heart transplant. Given the lack of formalized guidelines for heart transplantation in cardiac amyloidosis, we propose these recommendations for pretransplant evaluation and post-transplant management."
Dr. Nativi Nicolau is co-author of the 2025 American College of Cardiology concise clinical guidance on transthyretin cardiac amyloidosis. Published in the Journal of the American College of Cardiology, this guidance provides a framework for the evaluation and management of cardiac amyloidosis, focuses on early detection and novel therapies for this condition.
Dr. Lyle also is the primary author on a Mayo Clinic enterprise publication on amyloidosis and heart transplantation published in Clinical Transplantation: The Journal of Clinical and Translational Research.
"In recent years, patients with amyloidosis have been transplanted faster, and their survival has improved," Dr. Lyle says. "Combined with disease-targeted therapy, patients with hereditary amyloidosis have had less need for multiorgan transplant."
For those patients with amyloidosis who still require multiorgan transplant, Mayo Clinic in Florida, a leading solid organ transplant center, can provide liver or kidney transplantation along with heart transplantation.
"Years ago, patients with amyloidosis treated with transplantation did not survive long because of recurrence of the disease," Dr. Nativi Nicolau says. "Transplant was contraindicated for these patients, but that is no longer the case. With advances in diagnostics and therapeutics, now we can transplant select patients successfully, improving both quality and length of life."
New therapeutic options to treat amyloidosis
Beyond transplantation, patients with amyloidosis have new treatment options to address their disease at different stages, improving survival and reducing hospitalization. Existing medications can help stabilize the amyloid precursor protein. New, therapies approved by the Food and Drug Administration (FDA) can target the messenger RNA of hepatocytes, helping them avoid synthesizing the amyloid protein and protecting the organs from further damage.
Dr. Nativi Nicolau also collaborates with international investigators on clinical trials studying gene-editing therapies to modify DNA, as well as others to remove amyloid fibrils from the heart. These therapies have the potential to improve the survival of patients who are not eligible for heart transplantation.
A multidisciplinary approach to treating amyloidosis
Patients with amyloidosis often present with a constellation of cardiac, neurological or musculoskeletal symptoms at different points in their lives, making the disease difficult to pinpoint. The most common symptoms in patients with cardiac amyloidosis include heart failure alongside carpal tunnel syndrome, progressive neuropathy or spinal stenosis.
If a physician suspects a patient has amyloidosis, Mayo Clinic is among the few centers worldwide with a dedicated multidisciplinary clinic, where a team of specialists is focused on diagnosing and treating the disease. The collaborative practice, founded by Dr. Sher, encompasses hematologists, cardiologists, neurologists, nephrologists, pathologists, radiologists and geneticists, among others.
The goals of the multidisciplinary amyloidosis clinic include providing:
- Earlier diagnosis. The clinic aims to provide rapid yet accurate diagnoses for patients, knowing that earlier diagnoses lead to better therapeutic responses. Using a comprehensive battery of tests, the amyloidosis team meets to analyze results, identify the condition and formulate a treatment plan.
- Comprehensive evaluation. Beyond diagnosis, the clinic provides information on how the disease affects each patient.
- Patient-centered care. The clinic offers treatment of the amyloid itself and options for the organs affected, including transplantation. Other advances, including novel imaging techniques such as bone scintigraphy, allow for early diagnosis without biopsy.
- Access to clinical trials. These therapeutics are offered to patients looking for novel agents that could further improve their condition.
For more information
Lyle MA, et al. Amyloidosis and heart transplantation in a new era. Clinical Transplantation: The Journal of Clinical and Translational Research. 2025;39:e70070.
Lyle MA, et al. Heart transplantation for cardiac amyloidosis: Mayo Clinic consensus statement. Mayo Clinic Proceedings. 2025;100:1578.
Kittleson MM, et al. Transthyretin cardiac amyloidosis evaluation and management: 2025 ACC concise clinical guidance. Journal of the American College of Cardiology. 2026;87:549.
Refer a patient to Mayo Clinic.