Pediatric acute-onset neuropsychiatric syndrome, or PANS, has been a challenging disorder to both diagnose and treat. It causes sudden onset, debilitating and difficult to treat obsessive-compulsive disorder as well as other cognitive and behavioral issues, and it affects children’s ability to function both in school and at home. SmartBrief spoke with pediatric physicians Sydney Rice and Michael Daines, co-directors of the Children’s Postinfectious Autoimmune Encephalopathy Center of Excellence at the University of Arizona, to find out about current treatment options for children with PANS. Daines is principal investigator of the IVIG trial and the CPAE Center of Excellence is a participating trial site.
Can you describe PANS and why it has been challenging to diagnose and treat?
PANS is a disorder that affects children and adolescents and causes sudden onset of psychiatric and neurological symptoms, including obsessive-compulsive behavior, tics, irritability, anxiety, mood swings and academic decline. It is believed to be caused by an abnormal immune response, triggered by infections, that affects the brain and causes inflammation. PANS is challenging to treat because it is a clinical diagnosis with no definitive diagnostic test, which makes it difficult to determine the exact cause of the symptoms. Additionally, the symptoms of PANS can mimic those of other psychiatric and neurological conditions, making it challenging to differentiate from other disorders.
How can physicians determine if one of their patients might have PANS?
A physician can determine if a patient has PANS by conducting a thorough evaluation, including a medical and psychiatric history, physical examination and neurological and behavioral assessment. The physician may also order blood tests, imaging studies and other tests to rule out other causes of the symptoms. A diagnosis of PANS is made based on the presence of specific symptoms and the exclusion of other conditions that could cause similar symptoms.
What are the available treatment options for patients with PANS?
There are a variety of treatment options available for PANS, including medications, behavioral therapy and immunomodulatory treatments. Medications, such as antibiotics and anti-inflammatory drugs, can be used to treat underlying infections and reduce inflammation in the brain. Behavioral therapy can help children and adolescents with PANS manage their symptoms and improve their quality of life. Immunomodulatory treatments, such as intravenous immunoglobulin, can also be used to help reduce inflammation and improve symptoms in some patients with PANS.
Are there currently clinical trial options for children with PANS?
ProPANS is a clinical research study designed to investigate the effectiveness of an immunomodulatory treatment for children and adolescents recently diagnosed with PANS. The study drug has been approved by the FDA for other conditions, but not yet for PANS. Participation involves eight visits to a study clinic. The study drug and placebo are given as an IV infusion, and each infusion lasts at least three hours. Every participant receives the study drug for nine weeks and then the placebo for nine weeks, or vice versa, on a randomized basis. There is no cost to study participants.
How can physicians determine if one of their patients may be a good fit for this trial?
To participate, a child must be age 6 to 17 and recently diagnosed with PANS. The family must be willing to visit the study clinic every three weeks over an 18-week period to receive IV infusions of the study drug or placebo. Each visit lasts for two days, and the child will receive an IV infusion each day.
The search for safe and effective therapies for PANS has been difficult, but there is strong evidence that immunomodulation can mitigate or cure this disease. This Phase 3 trial with PANZYGA should help define the role of intravenous administration of immunoglobulins for the management of children with PANS.
If you feel you may have a patient with PANS/PANDAS that would like more information on the ProPANS study, please direct them to the following link: https://pacefoundation4kids.org/ivig-study/
Dr. Daines is an associate professor of pediatrics at the University of Arizona. He is a pediatric allergist-immunologist with a strong interest in improving health outcomes in children with inflammatory diseases. As co-director of the Children’s Postinfectious Encephalopathy (CPAE) Center of excellence at the University of Arizona, he has dedicated research efforts in understanding the postinfectious processes that lead to immune dysregulation and neuroinflammation that alter mood, movement and behavior in children.
Dr. Rice is a professor of pediatrics at the University of Arizona. She is an active clinician caring for children with disabilities, including PANS/pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection (PANDAS). She participates in multiple research teams investigating each of these conditions and is the co-director of the Childhood Postinfectious Autoimmune Encephalopathy (CPAE) Center of Excellence at the University of Arizona.