PMID- 36576645
OWN - NLM
STAT- MEDLINE
DCOM- 20230321
LR  - 20230321
IS  - 1590-3478 (Electronic)
IS  - 1590-1874 (Print)
IS  - 1590-1874 (Linking)
VI  - 44
IP  - 4
DP  - 2023 Apr
TI  - A case of severe increase of liver enzymes in a ATTRv patient after one year of 
      inotersen treatment.
PG  - 1419-1422
LID - 10.1007/s10072-022-06568-w [doi]
AB  - BACKGROUND: Inotersen is an antisense oligonucleotide used to treat hereditary 
      transthyretin amyloidosis (ATTRv). The most common drug-related adverse effects 
      (AEs) include thrombocytopenia and glomerulonephritis. Hepatic damage is rare, 
      but liver enzyme monitoring is mandatory. CASE REPORT: A 70-year-old man with 
      ATTRv (Val30Met) treated with inotersen developed a severe increase of 
      transaminases, with normal bilirubin and cholinesterase levels, that forced us to 
      stop therapy. At the same time, other causes of acquired hepatitis were excluded, 
      and the hypothesis of an inotersen-related hepatic toxicity was supported by the 
      normalization of liver enzymes after 40 days from the drug interruption. 
      DISCUSSION: Our case showed that 1-year inotersen treatment can stabilize 
      neurological impairment and even improve quality of life and suggests to 
      carefully monitor liver enzymes in order to avoid an inotersen-related hepatic 
      dysfunction.
CI  - (c) 2022. Fondazione Societa Italiana di Neurologia.
FAU - Severi, Daniele
AU  - Severi D
AUID- ORCID: 0000-0003-3131-032X
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy. 
      d.severi.92@gmail.com.
FAU - Palumbo, Giovanni
AU  - Palumbo G
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
FAU - Spina, Emanuele
AU  - Spina E
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
FAU - Iovino, Aniello
AU  - Iovino A
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
FAU - Nolano, Maria
AU  - Nolano M
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
AD  - Neurology Department, Skin Biopsy Laboratory, Istituti Clinici Scientifici 
      Maugeri IRCCS, Telese Terme, Italy.
FAU - Manganelli, Fiore
AU  - Manganelli F
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
FAU - Tozza, Stefano
AU  - Tozza S
AD  - Department of Neuroscience, Reproductive Sciences and Odontostomatology, 
      University of Naples "Federico II", Via Sergio Pansini 5, 80131, Naples, Italy.
LA  - eng
PT  - Case Reports
PT  - Journal Article
DEP - 20221228
PL  - Italy
TA  - Neurol Sci
JT  - Neurological sciences : official journal of the Italian Neurological Society and 
      of the Italian Society of Clinical Neurophysiology
JID - 100959175
RN  - 0IEO0F56LV (Inotersen)
RN  - 0 (Oligonucleotides)
RN  - 0 (Oligonucleotides, Antisense)
RN  - 0 (Prealbumin)
SB  - IM
MH  - Male
MH  - Humans
MH  - Aged
MH  - *Quality of Life
MH  - Oligonucleotides/adverse effects
MH  - Oligonucleotides, Antisense
MH  - *Amyloid Neuropathies, Familial/drug therapy
MH  - Liver
MH  - Prealbumin
PMC - PMC9795110
OTO - NOTNLM
OT  - Adverse event
OT  - Hereditary TTR amyloidosis
OT  - Inotersen
OT  - Liver toxicity
COIS- D.S. has received a travel grant to attend scientific meetings from SOBI. F.M. 
      received personal fees for scientific events from Alfa-Sigma, Alnylam 
      Pharmaceuticals, and Akcea Therapeutics, and has received a travel grant to 
      attend scientific meetings from CSL Behring. S.T. received personal fees for 
      scientific events from Alnyalm Pharmaceuticals and Amicus Therapeutics, and 
      travel grants to attend scientific meetings from Akcea Therapeutics.
EDAT- 2022/12/29 06:00
MHDA- 2023/03/22 06:00
PMCR- 2022/12/28
CRDT- 2022/12/28 11:18
PHST- 2022/11/13 00:00 [received]
PHST- 2022/12/15 00:00 [accepted]
PHST- 2022/12/29 06:00 [pubmed]
PHST- 2023/03/22 06:00 [medline]
PHST- 2022/12/28 11:18 [entrez]
PHST- 2022/12/28 00:00 [pmc-release]
AID - 10.1007/s10072-022-06568-w [pii]
AID - 6568 [pii]
AID - 10.1007/s10072-022-06568-w [doi]
PST - ppublish
SO  - Neurol Sci. 2023 Apr;44(4):1419-1422. doi: 10.1007/s10072-022-06568-w. Epub 2022 
      Dec 28.