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.
METHODS: 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.
CONCLUSIONS: 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.
METHODS: 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.
CONCLUSIONS: 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.
摘要：
背景：Inotersen是一种用于治疗遗传性转甲状腺素蛋白淀粉样变性（ATTRv）的反义寡核苷酸。最常见的药物相关不良反应（AE）包括血小板减少症和肾小球肾炎。肝脏损伤是罕见的，但是肝酶监测是强制性的.
方法：一名接受inotersen治疗的70岁ATTRv（Val30Met）患者出现转氨酶严重升高，胆红素和胆碱酯酶水平正常，迫使我们停止治疗.同时,其他获得性肝炎的原因被排除，药物中断后40天，肝酶正常化支持了inotersen相关肝毒性的假设。
结论：我们的案例表明，1年inotersen治疗可以稳定神经功能缺损，甚至改善生活质量，并建议仔细监测肝酶，以避免inotersen相关的肝功能障碍。
方法：一名接受inotersen治疗的70岁ATTRv（Val30Met）患者出现转氨酶严重升高，胆红素和胆碱酯酶水平正常，迫使我们停止治疗.同时,其他获得性肝炎的原因被排除，药物中断后40天，肝酶正常化支持了inotersen相关肝毒性的假设。
结论：我们的案例表明，1年inotersen治疗可以稳定神经功能缺损，甚至改善生活质量，并建议仔细监测肝酶，以避免inotersen相关的肝功能障碍。
