TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients

Orly Moshe-Lilie, Diana Dimitrova, Stephen B. Heitner, Thomas H. Brannagan, Sasha Zivkovic, Mazen Hanna, Ahmad Masri, Michael Polydefkis, John L. Berk, Morie A. Gertz, Chafic Karam

Research output: Contribution to journalArticlepeer-review

5 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)250-253
Number of pages4
JournalAmyloid
Volume27
Issue number4
DOIs
StatePublished - Dec 2020

Keywords

  • Inotersen
  • TTR gene silencing
  • antisense oligonucleotide
  • hATTR amyloidosis
  • liver transplant

ASJC Scopus subject areas

  • Internal Medicine

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