Theodorou Aikaterini, Papagiannopoulou Georgia, Akrivaki Alexandra, Fanouraki Stella, Moschovos Christos, Papadopoulou Marianna, Palaiodimou Lina, Merkouri Foteini, Melanis Konstantinos, Salakou Stauroula, Kitsos Dimitrios, Lambadiari Vaia, Zouvelou Vasiliki, Papadimas Georgios, Giannopoulos Sotirios, Alexandrov Andrei V, Tsivgoulis Georgios
Second Department of Neurology, "Attikon" University Hospital, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.
Department of Physiotherapy, Laboratory of Neuromuscular and Cardiovascular Study of Motion, University of West Attica, Athens, Greece.
Eur J Neurol. 2025 Jun;32(6):e70175. doi: 10.1111/ene.70175.
Limited data exist on the efficacy and safety of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors and inclisiran among patients with neuromuscular disorders and statin-induced myotoxicity and/or hepatotoxicity. We assessed the safety and efficacy of PCSK9 inhibitors and inclisiran in this specific patient subgroup.
We conducted an observational cohort study evaluating patients with available clinical and laboratory data prior to and at prespecified time points following treatment initiation with PCSK9 inhibitors or inclisiran.
Eleven patients with neuromuscular disorders and statin intolerance were included in this study. Median follow-up time after PCSK9 inhibitor or inclisiran initiation was 14 (9-17) months. PCSK9 inhibitors or inclisiran use led to a significant decrease in mean low-density lipoprotein cholesterol levels. Moreover, all patients tolerated these lipid-lowering agents without exacerbation of their underlying myositis, myopathy, neuromuscular junction disorder, and without presenting any adverse event or relapse of myotoxicity and/or hepatotoxicity.
The present real-world study highlights the potential therapeutic value of PCSK9 inhibitors and inclisiran among patients with neuromuscular disorders and statin intolerance. The findings of this study are in accordance with previous reports, showing that PCSK9 inhibitors are safe as a lipid lowering treatment for long-term use among patients with statin-associated immune-mediated necrotizing myopathy (IMNM).
关于前蛋白转化酶枯草溶菌素/kexin 9型（PCSK9）抑制剂和英克西兰在神经肌肉疾病以及他汀类药物诱导的肌毒性和/或肝毒性患者中的疗效和安全性的数据有限。我们评估了PCSK9抑制剂和英克西兰在这一特定患者亚组中的安全性和疗效。
我们进行了一项观察性队列研究，评估在开始使用PCSK9抑制剂或英克西兰治疗之前以及在预先设定的时间点具有可用临床和实验室数据的患者。
本研究纳入了11例患有神经肌肉疾病且不耐受他汀类药物的患者。开始使用PCSK9抑制剂或英克西兰后的中位随访时间为14（9 - 17）个月。使用PCSK9抑制剂或英克西兰导致平均低密度脂蛋白胆固醇水平显著降低。此外，所有患者均耐受这些降脂药物，其潜在的肌炎、肌病、神经肌肉接头疾病未加重，且未出现任何不良事件或肌毒性和/或肝毒性复发。
本项真实世界研究突出了PCSK9抑制剂和英克西兰在神经肌肉疾病和他汀类药物不耐受患者中的潜在治疗价值。本研究结果与先前报告一致，表明PCSK9抑制剂作为他汀类药物相关免疫介导坏死性肌病（IMNM）患者的长期降脂治疗是安全的。
