Ellen & Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University Health Network, University of Toronto, Toronto, Canada.
Department of Neurology, UKD and Center for Neurology and Neuropsychiatry, LVR Klinikum, Medical Faculty, Heinrich Heine University, Düsseldorf, Germany; Brain and Mind Centre, University of Sydney, Australia; Medical University of Vienna, Austria.
Clin Neurophysiol. 2021 Sep;132(9):2184-2190. doi: 10.1016/j.clinph.2021.05.018. Epub 2021 Jun 12.
To assess axonal function prior to subcutaneous immunoglobulin (SCIG) therapy or placebo in relation to relapse in chronic inflammatory demyelinating polyneuropathy (CIDP) to determine whether axonal damage can predict therapy response.
Relapse rates in patients from the Polyneuropathy and Treatment with Hizentra (PATH) study, where patients were treated with placebo or SCIG (IgPro20), were analyzed by baseline (post-intravenous immunoglobulin stabilization) axonal damage (≤1 mV peroneal compound muscle action potential) status.
In patients with non-axonal damage, relapses were significantly higher with placebo (73.0%) than IgPro20 (0.2 g/kg: 39.1%, 0.4 g/kg: 19.2%). In patients with axonal damage, IgPro20 had no effect on relapse (placebo: 25.0%, IgPro20: 0.2 g/kg: 30.0%, 0.4 g/kg: 19.4%). Patients with axonal damage relapsed significantly less on placebo versus non-axonal damage, but they also demonstrated higher baseline disability.
Axonal damage may correspond to relapse upon treatment withdrawal; patients with axonal damage relapse less, possibly reflecting poor response to immunoglobulin therapy, while non-axonal damage patients may experience more relapse, perhaps indicating better treatment response.
In CIDP patients with axonal loss, immunoglobulin therapy may not be as effective. Assessing axonal damage could help guide therapy, with immunoglobulins ideally used before substantial axonal damage arises.
评估慢性炎症性脱髓鞘性多发性神经病（CIDP）患者皮下免疫球蛋白（SCIG）治疗或安慰剂治疗前的轴突功能与复发的关系，以确定轴突损伤是否可以预测治疗反应。
分析 Polyneuropathy and Treatment with Hizentra（PATH）研究中患者的复发率，这些患者接受安慰剂或 SCIG（IgPro20）治疗，根据基线（静脉内免疫球蛋白稳定后）轴突损伤（≤1 mV 腓肠肌复合肌肉动作电位）状态分析。
在无轴突损伤的患者中，安慰剂的复发率明显高于 IgPro20（0.2 g/kg：39.1%，0.4 g/kg：19.2%）（73.0%）。在有轴突损伤的患者中，IgPro20 对复发无影响（安慰剂：25.0%，IgPro20：0.2 g/kg：30.0%，0.4 g/kg：19.4%）。与无轴突损伤相比，有轴突损伤的患者在安慰剂治疗时复发明显减少，但他们的基线残疾程度也更高。
轴突损伤可能与停药后复发相对应；有轴突损伤的患者复发较少，可能反映对免疫球蛋白治疗的反应较差，而无轴突损伤的患者可能会经历更多的复发，也许表明治疗反应更好。
在有轴突丢失的 CIDP 患者中，免疫球蛋白治疗可能效果不佳。评估轴突损伤可以帮助指导治疗，理想情况下在出现大量轴突损伤之前使用免疫球蛋白。
