April 18, 2025
A new study authored by experts at Mayo Clinic who specialize in amyloidosis found the disease shouldn't preclude transplant consideration.
"Up until a little more than a decade ago, data suggested that amyloid patients may not be as worthy of a heart transplant because they had poorer outcomes than nonamyloid patients," says lead author Julie L. Rosenthal, M.D., a transplant cardiologist at Mayo Clinic in Arizona. "And it's true that clinically, amyloid transplant recipients in the past didn't do as well long-term due to other morbidities."
The study, published in Clinical Transplantation, examined the outcomes of 55 patients with the rare disease. The researchers found that, in the modern cohort of patients with amyloidosis, survival following a heart transplant was similar to that of patients who don't have the condition.
Dr. Rosenthal and her fellow researchers performed the retrospective review of patients with advanced amyloidosis who had a heart transplant at one of Mayo Clinic's three campuses — in Arizona, Florida and Minnesota — between January 2007 and December 2020.
Melissa A. Lyle, M.D., a transplant cardiologist at Mayo Clinic in Florida who co-authored the study, notes, "Historically, there was concern about heart transplant for patients with amyloidosis, due to inferior outcomes, but those outcomes were mostly related to patients with AL amyloidosis (amyloid light chain or primary amyloidosis) prior to advancements in chemotherapy and immunotherapy. Now, in the current era, we found no significant overall post-transplant survival difference in patients with amyloidosis compared with patients who underwent heart transplantation for nonamyloid indications."
Promising signs from new therapies
In addition to the primary finding that amyloidosis is not a contraindication for transplant, the study found a decline in multiorgan transplantation in patients with hereditary amyloidosis over time, corresponding with a rise in the use of disease-targeted interventions.
Although there isn't a cure for amyloidosis, the Food and Drug Administration has approved several targeted therapy drugs in the past 10 years that have been shown to slow the progression of the disease. Dr. Rosenthal and her colleagues are planning a follow-up study to see if transplanting just one organ in combination with the addition of one of the drugs — such as a transthyretin (TTR) protein stabilizer or a silencer — would be effective, and to map those patient outcomes.
"These therapies have just been around for a few years and were never evaluated in any randomized, controlled trial," Dr. Rosenthal says. "No one really has data on using them in transplant recipients."
Dr. Rosenthal also notes that a 2018 change in the organ allocation process — from a three-tiered prioritization system to a six-tiered one — has benefited patients with amyloidosis who need transplant.
"The goal of the allocation system change was to more fairly prioritize patients for transplant based on the severity of their illness," Dr. Rosenthal says. The researchers demonstrated a significant decrease in the median wait time across three different regions.
Dr. Lyle is the first author of a follow-up study currently awaiting publication that explores how to choose which patients with amyloidosis are good candidates for heart transplant.
Cardiac Amyloidosis Clinic
To learn more about how Mayo Clinic diagnoses and treats amyloidosis, or to refer a patient, contact our Cardiac Amyloidosis Clinic:
- Arizona: 480-301-8484
- Florida: 904-953-0859
- Minnesota: 507-284-3994
For more information
Lyle MA, et al. Amyloidosis and heart transplantation in a new era. Clinical Transplant. 2025;39:e70070.
Refer a patient to Mayo Clinic.