Roger Kobayashi, United States of America
University of California, Los Angeles School of MedicinePresenter of 1 Presentation
PEDIATRIC ACUTE-ONSET NEUROPSYCHIATRIC SYNDROME (PANS) AS A POST-INFECTIOUS AUTOIMMUNE DISEASE: BENEFITS OF INTRAVENOUS IMMUNOGLOBULIN (IVIG)
Abstract
Background and Aims
Recent findings in PANS suggest a relationship between a post-infectious response and behavioral changes, which may be a form of autoimmunity, through molecular mimicry, resulting in various neurological symptoms. We’ve identified several common immune defects in PANS patients which suggests a new immune deficiency. Given our findings, we hypothesized that an immune memory defect is the underlying mechanism leading to PANS (Melamed I. Immunother Open Acc. 2016;2:2).
Methods
Based on this hypothesis, we proposed a study to explore the efficacy of IVIG [Octagam 5%] for PANS treatment. The primary objective was evaluation of IVIG efficacy over a period of 6 months (6 infusions) based on scores of 6 different assessments, including the Children’s Yale-Brown Obsessive Compulsive Scale (CY-BOCS). Secondary objectives included evaluation of the immunomodulatory effect of IVIG on key biomarkers associated with PANS, including assessment of toll-like receptor (TLR) functionality at baseline and following IVIG.
Results
Statistically significant reductions in symptoms from baseline to end of study were seen in all 6 assessments measured. CY-BOCS results demonstrated statistically significant reductions in obsessive compulsive symptoms, representing > 50% improvement, sustained 8 weeks after the final infusion. In addition, there was a statistically significant upregulation of TLR function in response to IVIG treatment, which we believe may occur to better address inflammatory processes.
Conclusions
In patients presenting with PANS, which may be associated with an underlying immune deficiency mechanism, IVIG [Octagam 5%] successfully mitigated psychological symptoms and upregulated TLR function.