Session to Provide Update on CNS Angiitis in Children

Primary angiitis or vasculitis of the central nervous system (PACNS) is not an everyday event, particularly not in children. Diagnosis is challenging and there is a paucity of robust epidemiological data, but up to half of pediatric strokes may be related to inflammation of blood vessels.

“People think the incidence of childhood arterial stroke could be approximately three to seven cases per 100,000 children, and perhaps 25­–60% may be related to CNS vasculitis,” said Eyal Muscal, MD, MS, Chief and the Pawelek Endowed Chair of Pediatric Rheumatology at Texas Children’s Hospital, and Professor of Pediatric Rheumatology at Baylor College of Medicine. “Adult PACNS, while still rare, may be at least 10 times greater than in children. Even if you’re in a hospital that serves 200,000 children, you might not see more than a couple of cases a year. Diagnosing and treating rare diseases may be quite a challenge.”

Pediatric PACNS is so rare there are no prospective clinical trial data. But PACNS working groups throughout the world have come to broad agreement on diagnostic schema and treatment regimens that can support rheumatologists and other clinicians in recognizing and treating the disease more efficiently.

Dr. Muscal will review clinical features and novel biomarkers during Cerebral Crossroads: Primary Central Nervous System Angiitis in Children Explained on Tuesday, Nov. 19, from 8:15–9:15 a.m. ET in Room 144ABC of the Walter E. Washington Convention Center. Heather Van Mater, MD, MS, Chief of Rheumatology and Associate Professor of Pediatrics at Duke University, will discuss multidisciplinary management of pediatric PACNS. The session will be available on demand within 48 hours for registered ACR Convergence 2024 participants.

Signs and symptoms of PACNS can be nonspecific and may overlap with a host of pediatric neurologic conditions and syndromes, Dr. Muscal said. Common features may include strokes, cognitive deficits, sometimes behavioral and psychiatric issues, or significant developmental regression that is acute in a previously healthy child. Rheumatologists should be involved early with any child who has a newly acquired focal or diffuse neurologic deficit so PACNS can stay on their team’s radar.

MRI and other neuroimaging techniques, as well as emerging clinical and laboratory biomarkers, correlate well with underlying pathologic processes in the brain.

“Treating providers have to get to thinking of a possible vascular injury or inflammation pretty quickly,” Dr. Muscal said. “If you only think about PACNS weeks after the neurologic event, you may have missed the opportunity to dampen inflammation and may not be able to head off occlusion of a blood vessel or ischemia to specific regions of the brain.”

Children with PACNS may present in the emergency department, neurology, intensive care, or other settings, he explained. One of the keys to early diagnosis is breaking down silos of care to speed multidisciplinary assessments to rule out PACNS mimics. Novel imaging and lab biomarkers may assist in early diagnosis and better-targeted immunomodulation.

Outcomes can be good, although at times guarded, with early diagnosis and prompt treatment, Dr. Muscal said. Delayed diagnosis and severe presentation carry the potential for significant motor, cognitive, and behavioral deficits.

“We want to crystalize the phenotypes that should start attendees thinking of primary angiitis or vasculitis of the CNS in children,” he said. “And we want to help rheumatologists become more comfortable discussing the phenotype and diagnostic algorithms with colleagues in other specialties. Rheumatologists are the experts in systemic inflammation. We encourage rheumatologists to be collaborative and engaged with other central nervous system disease experts. We should be figuring out how to be vital members of teams when patients like these arise.”