IMMUNOe Research Center, Centennial, Colorado, USA.
Pediatric Immunology & Allergy, University of California, Los Angeles School of Medicine, Los Angeles, California, USA.
J Child Adolesc Psychopharmacol. 2021 Mar;31(2):118-128. doi: 10.1089/cap.2020.0100. Epub 2021 Feb 18.
Pediatric acute-onset neuropsychiatric syndrome (PANS) is a clinical diagnosis in children who have an acute manifestation of varied neuropsychiatric symptoms, including obsessive compulsive disorder, eating disorders, tics, anxiety, irritability, and problems with attention/concentration. PANS may develop as a result of a postinfectious syndrome and may represent a new form of postinfectious autoimmunity. To test the hypothesis that multiple, consecutive infusions of intravenous immunoglobulin (IVIG) for PANS can be efficacious, a multisite, open-label study was designed. The primary endpoint was evaluation of the efficacy of IVIG [Octagam 5%] in PANS over a period of 6 months (six infusions) based on mean changes in psychological evaluation scores using 6 different assessments, including the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS), Clinical Global Impression of Severity, and the Parent-Rated Pediatric Acute Neuropsychiatric Symptom Scale (PANS Scale). The final cohort consisted of 21 subjects (7 per site) with moderate to severe PANS. The mean age was 10.86 years (range: 4-16 years). Results demonstrated statistically significant reductions in symptoms from baseline to end of treatment in all six assessments measured. CY-BOCS results demonstrated statistically significant reductions in obsessive compulsive symptoms ( < 0.0001), resulting in >50% improvement sustained for at least 8 weeks after the final infusion and up to 46 weeks in a subset of subjects. In PANS, which may be associated with an underlying immune dysregulation, sequential infusions of IVIG [Octagam 5%] successfully ameliorated psychological symptoms and dysfunction, with sustained benefits for at least 8 weeks, and up to 46 weeks in a subset of subjects. In addition, baseline immune and autoimmune profiles demonstrated significant elevations in a majority of subjects, which requires further evaluation, characterization, and study to clarify the potential immune dysfunction by which PANS manifests and progresses.
儿科急性发作的神经精神综合征（PANS）是一种儿童临床诊断，其表现为各种神经精神症状的急性发作，包括强迫症、饮食障碍、抽搐、焦虑、易怒和注意力/集中力问题。PANS 可能是感染后综合征的结果，也可能代表一种新的感染后自身免疫形式。为了检验 PANS 多次连续静脉注射免疫球蛋白（IVIG）治疗有效的假说，设计了一项多中心、开放标签研究。主要终点是根据 6 种不同评估工具（包括儿童耶鲁-布朗强迫症量表（CY-BOCS）、临床总体印象严重程度和父母评定的儿童急性神经精神症状量表（PANS 量表））心理评估得分的平均变化，评估 6 个月（6 次输注）期间 IVIG[Octagam 5%]治疗 PANS 的疗效。最终队列包括 21 名（每个中心 7 名）中度至重度 PANS 患者。平均年龄为 10.86 岁（范围：4-16 岁）。结果表明，所有 6 项评估均显示治疗结束时症状较基线显著减轻。CY-BOCS 结果显示强迫症症状显著减轻（ < 0.0001），至少 8 周后最后一次输注后持续改善，在亚组中可持续长达 46 周。在可能与潜在免疫失调相关的 PANS 中，连续输注 IVIG[Octagam 5%]成功改善了心理症状和功能障碍，至少持续 8 周，在亚组中可持续长达 46 周。此外，大多数患者的基线免疫和自身免疫特征显著升高，这需要进一步评估、特征描述和研究，以阐明 PANS 表现和进展的潜在免疫功能障碍。
