IgPro20 皮下免疫球蛋白治疗 CIDP 的长期安全性和有效性：PATH 扩展研究。
Long-term safety and efficacy of subcutaneous immunoglobulin IgPro20 in CIDP: PATH extension study.
机构信息
Department of Neurology (I.N.S.), Amsterdam University Medical Centers, University of Amsterdam, The Netherlands; Spaarne Gasthuis (I.N.S.), Haarlem, The Netherlands; CSL Behring (O.M., M.P., B.L.D.), Marburg, Germany and King of Prussia, PA; Department of Medicine (Neurology) (V.B.), University Health Network, University of Toronto, Canada; Medical Statistics (N.G.), Department of Biomedical Data Sciences, Leiden University Medical Center, The Netherlands; Department of Neurology (H.-P.H.), Medical Faculty, Heinrich Heine University, Düsseldorf, Germany; Department of Neurology (R.A.L.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (G.S.), Nagoya University Graduate School of Medicine, Japan; Department of Neurology (D.R.C.), Johns Hopkins University School of Medicine, Baltimore, MD; and Department of Neurology (I.S.J.M.), Maastricht University Medical Center, The Netherlands.
出版信息
Neurol Neuroimmunol Neuroinflamm. 2019 Jul 3;6(5):e590. doi: 10.1212/NXI.0000000000000590. eCollection 2019 Sep.
OBJECTIVE
To investigate the long-term safety and efficacy of weekly subcutaneous IgPro20 (Hizentra, CSL Behring) in chronic inflammatory demyelinating polyneuropathy (CIDP).
METHODS
In a 48-week open-label prospective extension study to the PATH study, patients were initially started on 0.2 g/kg or on 0.4 g/kg weekly and-if clinically stable-switched to 0.2 g/kg weekly after 24 weeks. Upon CIDP relapse on the 0.2 g/kg dose, 0.4 g/kg was (re)initiated. CIDP relapse was defined as a deterioration by at least 1 point in the total adjusted Inflammatory Neuropathy Cause and Treatment score.
RESULTS
Eighty-two patients were enrolled. Sixty-two patients initially received 0.4 g/kg, 20 patients 0.2 g/kg weekly. Seventy-two received both doses during the study. Sixty-six patients (81%) completed the 48-week study duration. Overall relapse rates were 10% in 0.4 g/kg-treated patients and 48% in 0.2 g/kg-treated patients. After dose reduction from 0.4 to 0.2 g/kg, 51% (27/53) of patients relapsed, of whom 92% (24 of 26) improved after reinitiation of the 0.4 g/kg dose. Two-thirds of patients (19/28) who completed the PATH study without relapse remained relapse-free on the 0.2 g/kg dose after dose reduction in the extension study. Sixty-two patients had adverse events (AEs) (76%), of which most were mild or moderate with no related serious AEs.
CONCLUSIONS
Subcutaneous treatment with IgPro20 provided long-term benefit at both 0.4 and 0.2 g/kg weekly doses with lower relapse rates on the higher dose. Long-term dosing should be individualized to find the most appropriate dose in a given patient.
CLASSIFICATION OF EVIDENCE
This study provides Class IV evidence that for patients with CIDP, long-term treatment with SCIG beyond 24 weeks is safe and efficacious.
目的
研究慢性炎症性脱髓鞘性多发性神经病（CIDP）患者皮下注射 IgPro20（Hizentra，CSL Behring）的长期安全性和疗效。
方法
在 PATH 研究的 48 周开放性前瞻性扩展研究中，患者最初以 0.2 g/kg 或 0.4 g/kg 每周起始治疗，如果临床稳定，在 24 周后转换为 0.2 g/kg 每周。当 0.2 g/kg 剂量出现 CIDP 复发时，重新起始 0.4 g/kg 治疗。CIDP 复发定义为总调整后的炎症性神经病病因和治疗评分至少恶化 1 分。
结果
共纳入 82 例患者。62 例患者最初接受 0.4 g/kg，20 例患者接受 0.2 g/kg 每周。研究期间 72 例患者接受了两种剂量。66 例患者（81%）完成了 48 周的研究。0.4 g/kg 治疗患者的总体复发率为 10%，0.2 g/kg 治疗患者为 48%。从 0.4 g/kg 剂量减少至 0.2 g/kg 后，51%（27/53）的患者复发，其中 92%（24/26）在重新起始 0.4 g/kg 剂量后得到改善。完成 PATH 研究且无复发的 28 例患者中的三分之二在扩展研究中剂量减少后仍保持 0.2 g/kg 剂量无复发。62 例患者（76%）发生不良事件（AE），其中大多数为轻度或中度，无相关严重 AE。
结论
皮下注射 IgPro20 治疗 CIDP 患者，0.4 g/kg 和 0.2 g/kg 每周剂量均能提供长期获益，且高剂量的复发率较低。长期剂量应个体化，以在特定患者中找到最合适的剂量。
证据分类
本研究提供 IV 级证据，表明对于 CIDP 患者，24 周以上的 SCIG 治疗是安全有效的。
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