RT Journal Article
SR Electronic
T1 Benefits of IVIG in Pediatric Acute-Onset Neuropsychiatric Syndrome (2411)
JF Neurology
JO Neurology
FD Lippincott Williams & Wilkins
SP 2411
VO 94
IS 15 Supplement
A1 Melamed, Isaac
A1 Kobayashi, Roger
A1 O’Connor, Maeve
A1 Kobayashi, Ai Lan
A1 Schechterman, Andrew
A1 Heffron, Melinda
A1 Canterberry, Sharon
A1 Miranda, Holly
A1 Rashid, Nazia
YR 2020
UL http://n.neurology.org/content/94/15_Supplement/2411.abstract
AB Objective: To test the hypothesis that pediatric acute-onset neuropsychiatric syndrome (PANS) is related to an autoimmune dysfunction, a multi-site study explored intravenous immunoglobulin (IVIG)[Octagam 5%] for treatment.Background: PANS is a clinical diagnosis in children who have an acute manifestation of varied neuropsychiatric symptoms, including obsessive compulsive disorder (OCD), eating disorders, tics, anxiety, irritability, and problems with attention/concentration. PANS may develop as a result of a post-infectious syndrome. Our hypothesis is that PANS may represent a new form of post-infectious autoimmunity, through molecular mimicry, suggesting a potential mechanism by which the disorder evolves.Design/Methods: The primary endpoint was evaluation of IVIG efficacy over a period of 6 months/infusions based on mean changes in psychological evaluation scores using 6 validated assessments including the Children’s Yale-Brown Obsessive Compulsive Scale (CY-BOCS). Secondary endpoints included evaluation of presenting immune/autoimmune panels and key potential biomarkers.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 (infusion 6) in all 6 assessments measured. CY-BOCS results demonstrated statistically significant reductions in obsessive compulsive symptoms, 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, IVIG [Octagam 5%] successfully ameliorated psychological symptoms and dysfunction, with sustained benefits for at least 8 weeks, and up to 46 weeks, following the final infusion. 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.Disclosure: Dr. Melamed has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Octapharma, IMMUNOe. Dr. Melamed has received research support from Octapharma, Pharming Group. Dr. Kobayashi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Octapharma, Shire/Takeda, UCLA. Dr. O’Connor has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Octapharma, Allergy, Asthma & Immunology Relief. Dr. O’Connor has received research support from Octapharma. Dr. Kobayashi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Octapharma, Midlands Pediatrics. Dr. Kobayashi has received research support from Octapharma. Dr. Schechterman has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Colorado Neurogeriatrics. Dr. Heffron has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with IMMUNOe. Dr. Canterberry has nothing to disclose. Dr. Miranda has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with IMMUNOe. Dr. Rashid has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Dunwoody Consulting.