Physician-Researchers Will Explore Emerging Advances in the Understanding of Pediatric ANCA-Associated Vasculitis

While it is a relatively rare condition, pediatric antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis is potentially fatal, with significant risk associated with medication exposure and organ damage. Pediatric rheumatologists are challenged with limited treatment options and a scarcity of pediatric-specific data to guide them.

On Monday, Nov. 18, the session Pediatric ANCA-Associated Vasculitis, which will take place from 1–2 p.m. ET in Room 204ABC of the Walter E. Washington Convention Center, will feature a pair of experienced physician-researchers who will discuss the latest study findings, current and future research directions, and emerging therapeutic strategies in treating ANCA-associated vasculitis in children. The session will be available on demand within 48 hours for registered ACR Convergence 2024 participants.

“Because of its rarity, ANCA-associated vasculitis in children has been very difficult to study,” said Kimberly Morishita, MD, Clinical Associate Professor at the University of British Columbia, Canada, a pediatric rheumatologist at BC Children’s Hospital in Vancouver whose research focus is in pediatric vasculitis and translational medicine, with particular interest in outcomes research in ANCA-associated vasculitis.

Although ANCA-associated vasculitis research in the adult world has flourished in the past 10 to 20 years, Dr. Morishita said that progress in the pediatric world has lagged behind due to the lack of needed patient numbers to do meaningful research.

“A lot of information that we do have has been extrapolated from adult studies,” she said. “That leaves us to question whether this disease is actually the same in children, and whether it’s appropriate to use the adult literature and adult guidelines when we counsel families of children diagnosed with ANCA-associated vasculitis.”

Dr. Morishita hopes that a growing emphasis on pediatric-specific research will help to answer those questions. In her presentation, she will discuss ongoing research efforts at her center and other centers looking at organ-specific outcomes in pediatric ANCA-associated vasculitis.

“In this disease, major organs are typically involved, often the kidneys and the lungs. There may be non-major organ involvement as well, though we tend to focus on those major organs because they typically guide our treatment,” Dr. Morishita said. “The bulk of research recently has been on renal disease and renal outcomes, less on some of the other organs, but as we get more patient data and more patient numbers, we’re increasingly able to look more specifically at how each organ is doing.”

She is hopeful that collaborative research efforts and a growing amount of pediatric-specific patient data becoming available through international registries will continue to drive research and ultimately lead to the improvement of specific guidance on the diagnosis, treatment, and management of the disease in pediatric patients, she said.

“Through the combined efforts of groups from around the world, we’ve accrued a large number of pediatric patients, which has enabled us to begin to reach more pediatric-specific conclusions and do more meaningful research,” Dr. Morishita said. “We need to do larger, more prospective studies to truly understand if what we’re seeing in these early studies holds true, but we’re making a lot of progress.”

Also during this session, Peter Merkel, MD, MPH, Chief of Rheumatology and Professor of Medicine and Epidemiology at the University of Pennsylvania, will discuss how some of the lessons learned from studies of ANCA-associated vasculitis in adults have translated to studying and treating the condition in children.