Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Department of Neurology, Indiana University, Indianapolis, Indiana, USA.
Muscle Nerve. 2020 Oct;62(4):502-508. doi: 10.1002/mus.27022. Epub 2020 Aug 13.
Inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, demonstrated significant benefit versus placebo in the modified Neuropathy Impairment Score (NIS) +7 neurophysiologic tests (mNIS+7) in patients with hereditary TTR-mediated amyloidosis (hATTR) with polyneuropathy. This analysis assessed the mNIS+7 components by anatomic location and the lower limb function (LLF) test.
Adults with hATTR in the NEURO-TTR trial (NCT01737398) were randomly assigned to receive weekly doses of subcutaneous inotersen 300 mg or placebo for 65 weeks. The mNIS+7 and LLF were assessed at 35 and 66 weeks.
All major mNIS+7 components (muscle weakness, muscle stretch reflexes, sensation) and the LLF showed significant efficacy in patients receiving inotersen versus placebo; however, NIS-reflexes (upper limb), touch pressure (upper and lower limbs), and heart rate during deep breathing did not show significant effects.
The results of this analysis reinforce the beneficial effect of inotersen on slowing neuropathy progression in patients with hATTR polyneuropathy.
反义寡核苷酸抑制剂转甲状腺素蛋白（TTR）蛋白生产的 inotersen，在遗传性 TTR 介导的淀粉样变性（hATTR）伴多发性神经病患者中，与安慰剂相比，在改良神经病损伤评分（NIS）+7 神经生理测试（mNIS+7）中表现出显著益处。这项分析按解剖部位和下肢功能（LLF）测试评估了 mNIS+7 成分。
在 NEURO-TTR 试验（NCT01737398）中，患有 hATTR 的成年人被随机分配接受每周一次皮下注射 inotersen 300mg 或安慰剂，持续 65 周。在 35 周和 66 周时评估 mNIS+7 和 LLF。
接受 inotersen 治疗的患者的所有主要 mNIS+7 成分（肌肉无力、肌肉拉伸反射、感觉）和 LLF 均显示出显著疗效，而 inotersen 对 NIS-反射（上肢）、触压（上肢和下肢）和深呼吸时的心率没有显示出显著效果。
这项分析的结果加强了 inotersen 对减缓 hATTR 多发性神经病患者神经病变进展的有益作用。
Zotero 插件
