Familial amyloidosis medical therapy
Familial amyloidosis Microchapters
Familial amyloidosis medical therapy On the Web
American Roentgen Ray Society Images of Familial amyloidosis medical therapy
The optimal therapy for familial amyloidosis is preventing further organ damage and correcting the effects of organ failure. The mainstay of treatment for TTR amyloidosis is liver transplant. We may also use tafamidis, patisiran, Inoteresen, diflunisal, and epigallocathechin-3-gallate.
- The mainstay of treatment for familial amyloidosis is removal of the source of abnormal TTR production.
- Newer therapies have been studied and assessed in clinical trials. These therapies may slow or halt progression of familial ATTR amyloidosis.
- 2-(3,5–dichloro–phenyl)-benzox-azole-6-carboxylic acid is an orally administered drug that acts to stabilize the TTR tetramer through its affinity for the T4-binding site, and it does not carry the risks associated with nonsteroidal anti-inflammatory drug use.
- Recently approved for familial amyloid polyneuropathy (FAP) in Europe.
- This agent is being tested in ongoing trials for other forms of ATTR.
- Patisiran and Inoteresen
- TTR gene silencers.
- FDA recently approved their use for ATTRm amyloidosis with peripheral neuropathy.
- Genetic counseling is recommended for individuals with hereditary amyloidosis and their family members.
- Bulawa CE, Connelly S, Devit M, Wang L, Weigel C, Fleming JA; et al. (2012). "Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade". Proc Natl Acad Sci U S A. 109 (24): 9629–34. doi:10.1073/pnas.1121005109. PMC 3386102. PMID 22645360.
- 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.
- Ferreira N, Cardoso I, Domingues MR, Vitorino R, Bastos M, Bai G; et al. (2009). "Binding of epigallocatechin-3-gallate to transthyretin modulates its amyloidogenicity". FEBS Lett. 583 (22): 3569–76. doi:10.1016/j.febslet.2009.10.062. PMID 19861125.