目的:本研究评估了假定的自身免疫性体位性心动过速综合征(POTS)对静脉注射免疫球蛋白(IVIG)的反应。
背景:POTS可能与自身免疫性疾病有关,血清自身抗体,或最近的感染。未控制的病例研究表明IVIG对治疗自身免疫POTS有益。以前没有进行过随机对照试验。
方法:这项单中心随机对照试验比较了IVIG和静脉白蛋白输注。白蛋白比较器确保盲化和控制体积膨胀的影响。符合条件的POTS患者的COMPASS-31总加权评分≥40,符合提示自身免疫的预定标准。超过12周,参与者接受了8次输液(每次0.4gm/kg).每周进行四次输液,然后每隔一周进行四次输液。主要结果指标是最终输注后2周COMPASS-31的改善。
结果:共有50名参与者同意;30名符合纳入标准并接受研究药物(16名IVIG和14名白蛋白;29名女性)。组基线特征匹配良好;27名参与者完成了治疗方案。COMPASS-31的变化在组间没有差异(中位数变化[IQR];IVIG:-5.5[-23.3,2.5]与白蛋白:-10.6[-14.1,-4.7];p值=0.629)。IVIG组有较高的反应率(46.7%对38.5%),但这没有统计学意义。不良事件是常见的,但通常是轻度的,治疗组之间没有差异。
结论:这项在POTS中使用IVIG的小型随机对照试验发现,与白蛋白输注相比,反应没有统计学差异。两组均显示出可能与体积膨胀或其他影响有关的改善,从而掩盖了组差异。这些发现为POTS未来免疫调节临床试验的发展提供了依据。
This
study assesses response to intravenous immunoglobulin (IVIG) in presumed autoimmune postural orthostatic tachycardia syndrome (POTS).
POTS may be associated with autoimmune disorders, serum autoantibodies, or recent infection. Uncontrolled case studies suggest that IVIG is beneficial for treating autoimmune POTS. No previous randomized controlled trials have been conducted.
This single-site randomized controlled
trial compared IVIG with intravenous albumin infusions. Albumin comparator ensured blinding and control for effects of volume expansion. Eligible patients with POTS had COMPASS-31 total weighted score ≥ 40 and met predetermined criteria suggesting autoimmunity. Over 12 weeks, participants received eight infusions (0.4 gm/kg each). Four infusions were given weekly followed by four infusions every other week. Primary outcome measure was improvement in COMPASS-31 2 weeks after final infusion.
A total of 50 participants consented; 30 met inclusion criteria and received
study drug (16 IVIG and 14 albumin; 29 female). Group baseline characteristics were well matched; 27 participants completed treatment protocol. Change in COMPASS-31 did not differ between groups (median change [IQR]; IVIG: -5.5 [-23.3, 2.5] versus albumin: -10.6 [-14.1, -4.7]; p-value = 0.629). The IVIG group had a higher response rate (46.7% versus 38.5%), but this was not statistically significant. Adverse events were common but usually mild and did not differ between treatment groups.
This small randomized controlled
trial of IVIG in POTS found no statistical difference in response compared with albumin infusion. Both groups showed improvement possibly related to volume expansion or other effects obscuring group differences. These findings inform development of future immunomodulatory clinical trials in POTS.