Evaluation of Intravenous Immunoglobulin in Pediatric Acute-Onset Neuropsychiatric Syndrome
Isaac Melamed, Roger H. Kobayashi, Maeve O’Connor, Ai Lan Kobayashi, Andrew Schechterman, Melinda Heffron, Sharon Canterberry, Holly Miranda, and Nazia Rashid.Journal of Child and Adolescent Psychopharmacology.
A new study published in the Journal of Child and Adolescent Psychopharmacology aims to evaluate the effectiveness of Intravenous Immunoglobulin (IVIG) in Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS) patients. This particular study was designed to include the “on-site administration of IVIG [Octagam 5%] at a dosage of 1 g/kg of body weight every 21 days (±3 days) for a total of six infusions (cycles) over a period of 18 weeks.” According to the study results, patients “demonstrated statistically significant reductions in symptoms from baseline to end of treatment in all six assessments measured.”
The protocol used in this study differs from the IVIG treatment protocol currently recommended by the PANDAS Physicians Network and the PANS Research Consortium. However, we acknowledge that alternate treatment protocols may be investigated and considered for a patient’s unique needs.
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 (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.
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.