Gillberg Neuropsychiatry Centre, Sahlgrenska Academy, University of Gothenburg, Kungsgatan 12A, 411 19, Gothenburg, Sweden.
Akutläkarna Specialist Clinic, Gothenburg, Sweden.
BMC Psychiatry. 2022 Aug 6;22(1):535. doi: 10.1186/s12888-022-04181-x.
Treatment with intravenous immunoglobulin (IVIG) in children with Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) has for many years been used on clinical indications, but the research evidence for its efficacy is insufficient.
Open-label prospective in-depth trial including ten children (median age 10.3 years) with PANS, who received IVIG treatment 2 g/kg monthly for three months. Primary outcomes were changes in symptom severity and impairment from baseline to first and second follow-up visits one month after first and one month after third treatment, using three investigator-rated scales: Paediatric Acute Neuropsychiatric Symptom (PANS) scale, Clinical Global Impression - Severity and Improvement (CGI-S and CGI-I) scales. Secondary outcomes reported here were changes in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) scores, and side effects.
All ten children received three treatments at one-month intervals according to study plan. From baseline to second follow-up marked reductions were seen in mean total PANS scale scores (p = .005), and CGI-S scores (p = .004). CGI-I ratings showed much to very much global improvement (mean CGI-I 1.8). Nine children had clinical response defined as > 30% reduction in PANS Scale scores. Improvements were also noted for CY-BOCS scores (p = .005), and in school attendance. Three children suffered moderate to severe temporary side effects after the first treatment, and the remaining seven had mild to moderate side effects. Side effects were much less severe after second and third treatments.
Considerable and pervasive improvements in symptoms and clinical impairments were seen in these ten children after three monthly IVIG treatments. Moderate to severe transient side effects occurred in three cases.
EudraCT no. 2019-004758-27, Clinicaltrials.gov no. NCT04609761, 05/10/2020.
静脉注射免疫球蛋白 (IVIG) 治疗儿科急性发作神经精神综合征 (PANS) 已有多年的临床应用，但疗效的研究证据不足。
对 10 名 PANS 儿童（中位年龄 10.3 岁）进行开放性前瞻性深入试验，每月接受 2g/kg 的 IVIG 治疗，共 3 个月。主要结局指标是从基线到首次和第二次随访（首次治疗后一个月和第三次治疗后一个月），使用三种研究者评定量表（儿科急性神经精神症状 (PANS) 量表、临床总体印象-严重程度和改善 (CGI-S 和 CGI-I) 量表）评估症状严重程度和损害的变化。这里报告的次要结局是儿童耶鲁布朗强迫症量表 (CY-BOCS) 评分的变化和副作用。
所有 10 名儿童均按研究计划在一个月的间隔内接受了三次治疗。从基线到第二次随访，平均总 PANS 量表评分（p=0.005）和 CGI-S 评分（p=0.004）显著降低。CGI-I 评分显示有很大或非常大的总体改善（平均 CGI-I 为 1.8）。9 名儿童的 PANS 量表评分降低≥30%，定义为有临床反应。CY-BOCS 评分（p=0.005）和上学出勤率也有改善。首次治疗后 3 名儿童出现中重度短暂性副作用，其余 7 名儿童出现轻中度副作用。第二次和第三次治疗后副作用明显减轻。
这 10 名儿童在接受三次每月 IVIG 治疗后，症状和临床损害均有显著和广泛的改善。三种情况下出现中重度短暂性副作用。
EudraCT 编号 2019-004758-27，Clinicaltrials.gov 编号 NCT04609761，2020 年 5 月 10 日。
