Neurobiologic, Immunologic, and Rheumatologic Markers in Youth With PANS

This study is an investigation of the neurologic, immunologic, and rheumatologic markers of Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS). PANS is a condition characterized by the abrupt, dramatic onset of obsessive compulsive disorder (OCD) and/or eating restriction accompanied by equally abrupt and severe co-morbid neuropsychiatric symptoms, which include anxiety, emotional lability, depression, irritability, aggression, oppositionality, deterioration in school performance, behavioral (developmental) regression, sensory amplification, movement abnormalities, sleep disturbance, and urinary frequency. PANS is thought to be caused by infection, inflammation, or alternate triggers that is associated with a brain response that leads to these symptoms. The purpose of this study is to examine specific neurologic, immunologic, rheumatologic, and genomic, components in children with the acute-onset of psychiatric symptoms. This research may begin to uncover a much larger story of autoimmune processes that are involved in psychiatric disorders of childhood. By better understanding the etiologic components of psychiatric phenomenon, future treatments may be better targeted to underlying causes.

Study Overview

• Status: Recruiting
• Conditions: PANDAS; PANS; Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections; Pediatric Acute-Onset Neuropsychiatric Syndrome

Detailed Description

The investigators will recruit 500 children, 1-18 years old at onset with PANS/PANDAS. They will be treatment naive and within one month of onset/exacerbation. The 500 children with PANS will be gender- and age-matched to 100 healthy children, to allow examination of immunologic, neurologic, genomic, and behavioral differences between these two groups of children.

• Study Type: Observational
• Enrollment (Anticipated): 500

Contacts and Locations

• Study Contact: Name: Joanne Cheung; Email: pansresearch@stanford.edu
• Study Contact Backup: Name: Ellen Spartz

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94305-5906
      • Recruiting
      • Stanford University
      • Contact: Joanne Cheung; Email: pansresearch@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

The investigators to enroll 500 children with PANS/PANDAS and 100 healthy controls. Children's parents will also be enrolled to report information about their children as well as their own caregiver burden. The 500 patients with PANS will meet diagnostic criteria. These patients are targeted because this study is seeking to specifically assess the immunologic and neurological markers of PANS.

Description

Inclusion Criteria:

• Children with PANS

  1. Age 1-18 at onset of PANS
  2. Diagnosis of PANS: abrupt onset of OCD or food restriction, and at least two of the following associated symptoms: frequent urination, worsening handwriting/cognition, inattention, anorexia, separation anxiety, oppositionality, irritability/rage outbursts, and emotional lability.
  3. Patients must live within 90 miles of Stanford University and have a new onset of PANS illness
  4. Patients must have an established pediatrician within 90 miles of Stanford University for 3 years.

• Healthy Controls

  1. Age 4-18
  2. No psychiatric diagnosis

Exclusion Criteria:

• Any neuropsychiatric illness that may obscure the clear diagnosis of PANS

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
PANS group; 500 children, 1-18 years old at onset with a strict diagnosis of PANS/PANDAS will be recruited
Health Controls; 100 healthy children age- and gender- matched to the PANS group will be recruited

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cerebral blood flow	The investigators will report results of altered cerebral blood flow from patients with PANS.	Through study completion, up to 12 years
EEG patterns	The investigators will report results of abnormal EEG patterns from patients with PANS. All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Through study completion, up to 12 years
Rapid Eye Movement (REM) motor disinhibition	The investigators will report results of REM motor disinhibition from polysomnography studies. All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Through study completion, up to 12 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Global Impairment Scores	All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Every 2-4 weeks for up to 12 years
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)	All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Every 2-4 weeks for up to 12 years
Columbia Impairment Scale	All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Every 2-4 weeks for up to 12 years
Caregiver Burden Inventory	All data will be obtained through the review of medical records, which are created during the routine clinical care of patients.	Every 2-4 weeks for up to 12 years
Neurological findings	The investigators will report data from neurological exam findings, including milk maid grip, chorea, choreiform movements of arms and legs, apraxia, overflow dystonia, truncal instability, piano-playing fingers, glabellar sign, etc. All data will be obtained through the review of medical records, which are created during the routine clinical care of patients. These results will be aggregated to report the number of participants with abnormal neurological findings.	Every 2-4 weeks for up to 12 years

Collaborators and Investigators

• Sponsor: Stanford University

Publications and helpful links

General Publications

• Chang K, Frankovich J, Cooperstock M, Cunningham MW, Latimer ME, Murphy TK, Pasternack M, Thienemann M, Williams K, Walter J, Swedo SE; PANS Collaborative Consortium. Clinical evaluation of youth with pediatric acute-onset neuropsychiatric syndrome (PANS): recommendations from the 2013 PANS Consensus Conference. J Child Adolesc Psychopharmacol. 2015 Feb;25(1):3-13. doi: 10.1089/cap.2014.0084. Epub 2014 Oct 17.
• Swedo SE, Leckman JF, Rose NR (2012) From Research Subgroup to Clinical Syndrome: Modifying the PANDAS Criteria to Describe PANS (Pediatric Acute-onset Neuropsychiatric Syndrome). Pediatr Therapeut 2:113. doi: 10.4172/2161-0665.1000113
• Murphy TK, Gerardi DM, Leckman JF. Pediatric acute-onset neuropsychiatric syndrome. Psychiatr Clin North Am. 2014 Sep;37(3):353-74. doi: 10.1016/j.psc.2014.06.001.

Study record dates

Study Major Dates

• Study Start: March 1, 2013
• Primary Completion (Anticipated): March 1, 2028
• Study Completion (Anticipated): March 1, 2028

Study Registration Dates

• First Submitted: August 22, 2016
• First Submitted That Met QC Criteria: August 30, 2016
• First Posted (Estimate): September 5, 2016

Study Record Updates

• Last Update Posted (Estimate): October 6, 2016
• Last Update Submitted That Met QC Criteria: October 5, 2016
• Last Verified: October 1, 2016

More Information

Terms related to this study

• Keywords: PANS; PANDAS
• Additional Relevant MeSH Terms: Infections; Bacterial Infections; Bacterial Infections and Mycoses; Gram-Positive Bacterial Infections; Streptococcal Infections
• Other Study ID Numbers: 26922

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Data is available upon request as allowed by the Stanford Institutional Review board.