Open-label prospective trial to study efficacy, safety and tolerability of intravenous immunoglobulin (IVIG) once monthly for 6 months in children and adolescents with PANS. Number of subjects: 10. Age range: 4-17 years. Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a recently defined research diagnosis describing an abrupt, dramatic onset of neuropsychiatric symptoms including obsessions/compulsions and/or food restriction in children. Immunologic mechanisms are suspected, but treatment trials are few.
Open-label prospective trial to study efficacy, safety and tolerability of intravenous immunoglobulin (IVIG) once monthly for 6 months in children and adolescents with PANS (including the subgroup PANDAS). Number of subjects: 10. Age range: 4-17 years. Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a recently defined research diagnosis describing an abrupt, dramatic onset of neuropsychiatric symptoms including obsessions/compulsions and/or food restriction in children. Immunologic mechanisms are suspected, but treatment trials are few. The primary objective of this study is to evaluate the efficacy of intravenous immunoglobulin (IVIG), 2 g/kg given every 4 weeks for 6 months, to patients with post-infectious PANS/PANDAS, in improving neuropsychiatric symptoms and impairment. The secondary objectives of this study are to evaluate changes from baseline to follow-up at 3 months, 6 months and 12 months in: * Obsessive Compulsive Disorder (OCD) symptoms * adaptive functioning * quality of life * cognitive functioning * for patients with baseline inflammation signs on cerebral Magnetic Resonance Imaging (MRI) to evaluate changes in these measures after IVIG therapy after 6 months. * number of days of work/school/daily activities missed per subject year due to PANS/PANDAS before and after IVIG therapy * parental care load, e.g. need for sick leave, before and after IVIG therapy * Immunoglobulin (IgG, IgM and IgA) levels at baseline, 3 months, 6 months and 12 months * sustainability of any improvement at 12 months after initiation of IVIG measured with the PANS scale, CGI-S and CGI-I To assess the safety and tolerability of high dose IVIG therapy * Clinical signs and symptoms (nausea, headache, local reactions) * ALAT * Hemoglobin, complete blood count including leucocyte differential Investigational product: Intravenous immunoglobulin IVIG (Privigen), 2 g/kg BW every 4th week for 6 months (= 6 infusions).
Study Type
INTERVENTIONAL
Allocation
NA
Privigen, 2 g/kg BW every 4th week for 6 months (= 6 infusions).
Gillberg Neuropsychiatry Centre
Gothenburg, Sweden
RECRUITINGChange in Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) scale symptoms and impairment
Investigator-rated scale measuring symptom severity and impairment in Pediatric Acute-onset Neuropsychiatric Syndrome (PANS), score range 0-50, lower is better
Time frame: 6 months
Change in CGI-S
CGI-S (Clinical Global Impression-Severity) measures global symptom severity, score range 1-7, lower is better
Time frame: 6 months
CGI-I
CGI-I (Clinical Global Impression-Improvement) measures global improvement, score range 1-7, lower is better
Time frame: 6 months
Change in CY-BOCS scale
CY-BOCS scale measures Obsessive Compulsive Disorder (OCD) Symptoms, investigator-rated, score range 0-40, lower is better
Time frame: 6 months
Change on ABAS (Adaptive Behavior Assessment System) II scale
Measures level of functioning in everyday life, parent- and teacher-rated, score range 40-160, higher is better
Time frame: 6 months
Change on Child Health Inventory (CHIP-CE) scale
Measures quality of life, parent-rated, standardized T-score, mean 50, standard deviation 10, higher score is better
Time frame: 6 months
Change on 5-15 scale
Measures neuropsychiatric symptoms, score range 1-543, lower is better
Time frame: 6 months
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Purpose
TREATMENT
Masking
NONE
Enrollment
10
Change in neuromotor functioning
Neuromotor examination by investigator
Time frame: 6 months
Change in cognitive function
Working memory test from Wechsler scale, standardized scores age norms, mean 100, standard deviation 10, higher is better
Time frame: 6 months
Change in School-PANS (Pediatric Acute-onset Neuropsychiatric Syndrome) Scale
Short version of PANS-scale rated by teacher or school assistant, score range 0-50, lower is better
Time frame: 6 months
School absence
Days absent from school past 3 months or since last visit
Time frame: 6 months
Parental care load
Sick leave number of days
Time frame: 6 months
Change in laboratory assessments
Inflammatory measures as specified in CRF, lower is better
Time frame: 6 months
Adverse events
Parent/child report
Time frame: 6 months
Magnetic Resonance Imaging (MRI)
In a subgroup who can tolerate MRI without general anaesthesia. Specially developed protocol for measuring inflammation signs
Time frame: 6 months