GBS/CIDP Center of Excellence, University of South Florida, Tampa, Florida, USA.
Muscle Nerve. 2021 Sep;64(3):351-357. doi: 10.1002/mus.27345. Epub 2021 Jun 15.
INTRODUCTION/AIMS: Intravenous immunoglobulin (IVIg) is a common therapy for patients with chronic inflammatory demyelinating polyneuropathy (CIDP). IVIg may cause systemic adverse events (AEs); therefore, infusion of subcutaneous immunoglobulin (SCIg) may be preferred by some patients. In this study we document the experiences of patients transitioning from IVIg to SCIg.
Transitioning subjects with CIDP were followed in a 6-month prospective, open-label study. The primary endpoint was percentage of subjects who withdrew for any reason (including significant AEs). The secondary endpoint was symptom progression or relapse requiring a change in management. Quality of life (QOL) and treatment satisfaction were assessed using the Short Form 36-item Health Survey (SF-36), Treatment Satisfaction Questionnaire for Medication (TSQM), and Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI). Efficacy was assessed using the Inflammatory Rasch-built Overall Disability Scale, hand-held dynamometry, limb motor strength testing (LMST), and timed 25-ft walk (T25-FW).
Fifteen CIDP subjects transitioned from IVIg to SCIg. Of these, three (20%) met the primary endpoint and one (7%) met the secondary endpoint. The SF-36 showed a statistically significant improvement for the domain of role limitations-physical after 24 weeks (P = .03), with no significant differences observed in other domains. TSQM and CAP-PRI showed significant differences in favor of SCIg (P = .003 and .02, respectively). No significant differences were observed in efficacy after 24 weeks, except for LMST, which favored SCIg (P = .003). Eight of the 12 study completers (67%) continued with SCIg.
Transition to SCIg was associated with maintained efficacy and improved QOL.
简介/目的：静脉注射免疫球蛋白（IVIg）是治疗慢性炎症性脱髓鞘性多发性神经病（CIDP）患者的常用疗法。IVIg 可能会引起全身性不良反应（AE）；因此，一些患者可能更倾向于皮下注射免疫球蛋白（SCIg）。在这项研究中，我们记录了从 IVIg 转为 SCIg 的患者的经验。
在一项为期 6 个月的前瞻性、开放标签研究中对患有 CIDP 的转用患者进行随访。主要终点是因任何原因（包括严重 AE）退出的患者比例。次要终点是需要改变治疗方案的症状进展或复发。使用 36 项简明健康调查问卷（SF-36）、药物治疗满意度问卷（TSQM）和慢性获得性多发性神经病患者报告指数（CAP-PRI）评估生活质量（QOL）和治疗满意度。使用炎症 Rasch 构建整体残疾量表、手持式测力计、肢体运动力量测试（LMST）和 25 英尺步行计时测试（T25-FW）评估疗效。
15 例 CIDP 患者从 IVIg 转为 SCIg。其中，3 例（20%）达到主要终点，1 例（7%）达到次要终点。SF-36 在 24 周后身体角色受限领域的评分有统计学显著改善（P=.03），而其他领域未见显著差异。TSQM 和 CAP-PRI 对 SCIg 的评分明显有利（P=.003 和.02）。24 周后疗效无显著差异，除 LMST 外，SCIg 更有利（P=.003）。12 例研究完成者中有 8 例（67%）继续使用 SCIg。
转为 SCIg 与维持疗效和改善 QOL 相关。
