Familial amyloidosis medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Roukoz A. Karam, M.D.[2]
Overview
Medical Therapy
- The optimal therapy for familial amyloidosis is removal of the source of abnormal TTR production.
- The liver is the dominant source of transthyretin; hence, in patients with less advanced disease, liver transplant may be performed.
- Patients with severe heart involvement may benefit from a heart transplant.
- In addition, multiple clinical trials are now testing medications that may slow or halt progression of familial ATTR amyloidosis.
- Genetic counseling is recommended for individuals with hereditary amyloidosis and their family members.
References
- ↑ Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M; et al. (2018). "Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy". N Engl J Med. 379 (11): 1007–1016. doi:10.1056/NEJMoa1805689. PMID 30145929.
- ↑ Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK; et al. (2018). "Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis". N Engl J Med. 379 (1): 22–31. doi:10.1056/NEJMoa1716793. PMID 29972757.