Fast Five Quiz: Treatment of Hereditary (Familial) Amyloid Transthyretin-Related (hATTR) Amyloidosis

Jefferson R. Roberts, MD; Victoria M.F. Mank, MD


May 24, 2021

Liver transplantation was the first therapeutic approach for patients with hATTR amyloidosis polyneuropathy, as TTR is the site of more than 95% of TTR production. Transplant replaces the main source of variant TTR. Liver transplantation can lead to disease stabilization, especially the stabilization of neurologic complications. Referral for liver transplantation ideally should occur as early in the disease course as possible, preferably before significant neurologic disability has set in. However, current guidelines propose therapeutic strategies that include medications like TTR stabilizers, NSAIDs, and newer gene-silencing therapies.

Carpal tunnel release can be performed in patients with carpal ligament involvement. Kidney and heart transplants may be performed in patients with renal and cardiac involvement, respectively. Although these treatments may address disease-related dysfunction, they do not address the underlying cause of the amyloidosis (TTR-associated amyloid formation).

Learn more about surgical interventions for patients with hATTR amyloidosis.


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