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Case Reports
. 2023 Apr;44(4):1419-1422.
doi: 10.1007/s10072-022-06568-w. Epub 2022 Dec 28.

A case of severe increase of liver enzymes in a ATTRv patient after one year of inotersen treatment

Daniele Severi  1 , Giovanni Palumbo  2 , Emanuele Spina  2 , Aniello Iovino  2 , Maria Nolano  2   3 , Fiore Manganelli  2 , Stefano Tozza  2
Affiliations
Case Reports

A case of severe increase of liver enzymes in a ATTRv patient after one year of inotersen treatment

Daniele Severi et al. Neurol Sci. 2023 Apr.

Abstract

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.

Keywords: Adverse event; Hereditary TTR amyloidosis; Inotersen; Liver toxicity.

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Conflict of interest statement

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.

Figures

Fig. 1
Fig. 1
Clinical and laboratorial findings. Figure shows the neurological impairment (NIS) and quality of life (Norfolk) (A), serum transthyretin level (B), platelet count (C), and liver enzymes (D) of the patient during the 12-month period of inotersen treatment
See this image and copyright information in PMC

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