View more of the CPAE experience of Holland and her family at https://www.youtube.com/watch?v=45tYYK5TAaA&feature=youtu.be.
The University of Arizona Steele Children's Research Center has established a new "center of excellence" that is the first of its kind in the U.S. to treat and conduct research into a family of acute-onset neuropsychiatric disorders that historically have been misdiagnosed or undiagnosed in children.
Patient services will be provided at Banner Children's at Diamond Children's Medical Center, and research will be conducted at the Steele Center.
As part of the UA Health Sciences, the Children's Postinfectious Autoimmune Encephalopathy Center of Excellence at the UA Steele Children's Research Center is the first to implement a model of clinical care, teaching and research to treat and potentially cure a spectrum of postinfectious autoimmune encephalopathies, such as Pediatric Acute-Onset Neuropsychiatric Syndrome, or PANS, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Strep, or PANDAS, and Sydenham's chorea.
Children's Postinfectious Autoimmune Encephalopathy
Children's postinfectious autoimmune encephalopathies, like other autoimmune disorders, are on the rise in children in the Western world. These diseases occur when a child's immune system, while fighting off a virus or infection, mistakenly targets or disrupts a part of the child's own body. In CPAE, the child's immune system attacks the brain, causing a range of neuropsychiatric symptoms.
Symptoms typically occur suddenly — sometimes overnight — and can include:
- Obsessive-compulsive disorder
- Tics (motor and phonic)
- Severe anxiety
- Attention deficit hyperactivity disorder
- Restrictive eating
- Depression and mood changes
- Irritability and aggression
- Changes in handwriting
- Separation anxiety
- Poor academic performance
- Frequent urination
- Sensory sensitivities
From Despair to Healing
Karen and her husband, Charles, of Scottsdale, know the utter despair parents feel having a very sick child and no answers.
Their 10-year-old daughter, Holland, suffered for more than two years with debilitating symptoms.
"What happened to Holland was every parent's worst nightmare," Karen recalled. "One day she was a healthy, happy, vibrant child, and the next day she woke up with terrible anxieties, a horrible headache and excruciating abdomen pain. She was afraid to eat and afraid to go to school, to name just a few of her horrible symptoms."
What happened next was an exhausting procession of specialist after specialist, test after test, medication after medication, and long hospitalizations.
"We found no answers and Holland continued to get worse," Karen said, noting that her daughter's weight dropped to 47 pounds. "Finally, I demanded she be hospitalized until a diagnosis was made. But, still no answers."
Holland finally was diagnosed with PANS/PANDAS, by way of a specialized test known as the Cunningham Panel.
"We then went on an exhaustive search to find a specialist who could actually treat PANS/PANDAS, but couldn't find any in Arizona and started looking to other states," Karen said.
"We life-flighted Holland to a specialist in the San Francisco Bay area, but she was too sick to travel for follow-up care. Then, a friend told us about the new service she heard about in Tucson," Charles said.
Holland was brought to Diamond Children's and met Dr. Fayez K. Ghishan, professor and director of the UA Steele Center and physician-in-chief of Diamond Children's, and his medical team.
Once stabilized, Holland received her first IVIG treatment, a drug used to reduce the inflammation in her brain.
"That was an absolute miracle to watch happen," Karen said. "Before the IVIG treatment, Holland wasn't speaking or eating, couldn't walk or tolerate light, was bedridden and in constant pain. Soon after treatment, she was walking, talking, singing, dancing and playing. We were amazed.
"We were so grateful to find this new center in Tucson. The UA/Banner medical team gave us our daughter back."
CPAE Center: First in US to Implement New Model
The UA Steele Center and Banner – Diamond Children's are now part of the Autoimmune Encephalopathy PANS/PANDAS Clinical and Research Network, a consortium recently established by the National Institutes of Health/National Institutes of Mental Health of 14 universities dedicated to CPAE-like disorders.
"With this solid foundation, there is no doubt that the UA and Banner Health will lead the nationwide network of clinicians and investigators," said Dr. Sue Swedo, investigator and chief of the NIMH Pediatrics & Developmental Neuroscience Branch.
"As the leading public research university in the Southwest, the UA and Banner Health are in a uniquely valuable position to contribute to clinical care and research on PANS, PANDAS and related disorders. Moreover, the UA Health Sciences network is ideally suited to training health professionals."
"Treatment for this complex spectrum of disorders requires a team-based approach," Ghishan said. "Banner – Diamond Children's has the state-of-the art clinical facility to take care of these children, and the clinical team includes specialists in developmental and behavioral pediatrics, immunology, gastroenterology and nutrition, sleep medicine and psychiatry to provide these children with the comprehensive care they need."
Basic laboratory research, coupled with clinical research, is vital to understanding the disease. The first application for an NIH RO1 grant has been submitted by Steele Center researchers. The study will focus on understanding the mechanisms in the brain that cause some children to develop these disorders. Clinical research projects, including IVIG therapies, are planned as well.
Educating physicians is key to ensuring that the medical community is alert to this emerging family of diseases. More than 50 current pediatric residents will have hands-on training, and UA College of Medicine – Tucson students will receive education as part of their pediatrics clerkships.
Origins and Supporters
The effort to create a center in Arizona was spearheaded by the Phoenix-based PACE Foundation, a national nonprofit organization dedicated to helping individuals with postinfectious autoimmune encephalopathies.
"Our goal was to establish a center in Arizona that would diagnose, treat and research disorders like PANS/PANDAS," said Paul Ryan, PACE Foundation founder. "We wanted to partner with the University of Arizona Health Sciences because of its world-class research and faculty."
The UA effort, led by Ghishan, began to formulate a Center of Excellence team, in collaboration with their partners at Banner Health and the NIH. The Arizona Legislature passed an "Awareness Day," set up a House Ad Hoc Committee on Pediatric Autoimmune Neurological Disorders and passed legislation that authorized $250,000 for research into these disorders. The Arizona Department of Health Services launched an awareness effort to educate pediatricians throughout the state. The PACE Foundation enlisted the support of Camelback Toyota in Phoenix and Option Care to support future clinical research projects.
"This center came about by the tireless efforts of many dedicated individuals and organizations working together," Ghishan said. "We could not have established this Center of Excellence without the support from Banner Health, the Arizona Legislature, the Arizona Department of Health Services, the PACE Foundation, the UA and UA Health Sciences, the NIH/NIMH and countless others. We're excited about providing hope and healing to children suffering from these devastating disorders through clinical care, research and education."