@article{731a122baf8247af9ca118e9155ab1fe,
title = "TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients",
abstract = "Objective: Patients with hereditary transthyretin (TTR) amyloidosis (hATTR) often experience disease progression after orthotopic liver transplant (POLT) due in part to wild type ATTR amyloid deposition. The management strategy is not defined. We propose that TTR gene silencing with an antisense oligonucleotide or a small interfering ribonucleic acid may be a treatment for these patients. Methods: We reviewed the charts of hATTR patients POLT treated with a TTR gene silencing agent at 7 different Amyloid Clinics between 2018–2020. Results: Nine hATTR patients with POLT were treated with TTR gene silencing therapy (Inotersen). The median age was 61 years. The median time from OLT to initiation of TTR gene silencing therapy was 7.5 years. The median duration of therapy was 12 months. Neuropathy impairment score remained stable or improved in all patients. Five patients stopped treatment: 3 because of thrombocytopenia, 2 because of reversible liver rejection. Three patients who discontinued treatment subsequently experienced worsening of their neuropathy. Conclusion: TTR gene silencing therapy in hATTR patients with POLT could be a treatment option. Vigilant monitoring of renal, liver and bone marrow functions is necessary because of frequent complications. Further studies are needed to determine efficacy.",
keywords = "Inotersen, TTR gene silencing, antisense oligonucleotide, hATTR amyloidosis, liver transplant",
author = "Orly Moshe-Lilie and Diana Dimitrova and Heitner, {Stephen B.} and Brannagan, {Thomas H.} and Sasha Zivkovic and Mazen Hanna and Ahmad Masri and Michael Polydefkis and Berk, {John L.} and Gertz, {Morie A.} and Chafic Karam",
note = "Funding Information: Stephen Heitner has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Akcea, Pfizer and Eidos. Thomas H Brannagan III has received personal compensation for serving on scientific advisory boards or speaking with Akcea, Alnylam and Pfizer. Sasha Zivkovic has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Akcea, Alnylam, and Argenx. He has received research support from Akcea, Alnylam and Pfizer. Mazen Hanna has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Akcea, Alnyalm, Eidos, and Pfizer. John L Berk has received personal compensation for serving on scientific advisory boards or speaking with Akcea, Alnylam, Corino and Intellia. Michael Polydefkis has received personal compensation for serving on scientific advisory boards or speaking with Alnylam, Akcea, Vertex and Pfizer. Morie A Gertz has received personal compensation for Ionis/Akcea, Alnylam, Prothena, Celgene, Janssen, Annexon, Appellis, Amgen, Medscape, Physicians Education Resource, Data Safety Monitoring board from Abbvie, Research to Practice. He also received speaker fees from Teva, Johnson and Johnson, Medscape, DAVA oncology, as well as personal compensation and grants from Spectrum. He also served on advisory board of Pharmacyclics, Proclara outside the submitted work. Dr Gertz also received royalties from Springer Publishing, grant funding from Amyloidosis Foundation and International Waldenstrom Foundation, grant NCI SPORE MM SPORE 5P50 CA186781-04, Educational Programme development i3Health. Chafic Karam has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Akcea, Alexion, Alnyalm, Argenx, Biogen, CSL Behring and Sanofi Genzyme. Dr. Karam has received research support from Akcea and Sanofi Genzyme. The other authors report no conflicts of interest. Publisher Copyright: {\textcopyright} 2020 Informa UK Limited, trading as Taylor & Francis Group.",
year = "2020",
month = dec,
doi = "10.1080/13506129.2020.1784134",
language = "English (US)",
volume = "27",
pages = "250--253",
journal = "Amyloid",
issn = "1350-6129",
publisher = "Informa Healthcare",
number = "4",
}