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TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients
- Source :
- Amyloid. 27:250-253
- Publication Year :
- 2020
- Publisher :
- Informa UK Limited, 2020.
-
Abstract
- 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.We reviewed the charts of hATTR patients POLT treated with a TTR gene silencing agent at 7 different Amyloid Clinics between 2018-2020.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.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.
- Subjects :
- Adult
Male
Oligonucleotides
030204 cardiovascular system & hematology
03 medical and health sciences
0302 clinical medicine
Internal Medicine
Humans
Prealbumin
Medicine
Gene silencing
Gene Silencing
Aged
Amyloid Neuropathies, Familial
biology
business.industry
Amyloidosis
Disease progression
Wild type
nutritional and metabolic diseases
Orthotopic Liver Transplant
Middle Aged
Oligonucleotides, Antisense
medicine.disease
Liver Transplantation
Transthyretin
Treatment Outcome
Amyloid deposition
Disease Progression
biology.protein
Cancer research
Female
business
030217 neurology & neurosurgery
Attr amyloidosis
Subjects
Details
- ISSN :
- 17442818 and 13506129
- Volume :
- 27
- Database :
- OpenAIRE
- Journal :
- Amyloid
- Accession number :
- edsair.doi.dedup.....d27cb88d7b6be2f3a7cc49783cdd9501