Open AccessEvaluation of Intravenous Immunoglobulin in Pediatric Acute-Onset Neuropsychiatric Syndrome
Author(s) -
Isaac Melamed,
Roger H. Kobayashi,
Maeve O’Connor,
Ai Kobayashi,
Andrew Schechterman,
Melinda Heffron,
Sharon Canterberry,
Holly Miranda,
Nazia Rashid
Publication year - 2021
Publication title -
journal of child and adolescent psychopharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.935
H-Index - 84
eISSN - 1557-8992
pISSN - 1044-5463
DOI - 10.1089/cap.2020.0100
Subject(s) - irritability , medicine , clinical global impression , cohort , pandas , anxiety , tics , pediatrics , psychiatry , placebo , alternative medicine , pathology
Objectives: 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. Methods: 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). Results: 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 ( p < 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. Conclusions: 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.