Elevating Research with Broad-Based Collaborations in Pediatric Rheumatology
A little more than 20 years ago, Karen Brandt Onel, MD, began her medical career as an assistant attending pediatrician and assistant scientist in the Division of Rheumatology at Hospital for Special Surgery. After nine years, Dr. Onel accepted a position at the University of Chicago as Program Director of the Pediatric Rheumatology Training Program and then Section Chief of Pediatric Rheumatology. In December 2016, Dr. Onel returned to HSS as Chief of Pediatric Rheumatology, having learned some important truths along the way.
“Hospital for Special Surgery is a remarkable institution for pediatric rheumatology,” she says. “When I left I wanted an opportunity to spend some time in a children’s hospital, and I had a wonderful 12 years and some great and unusual experiences that were distinctive to working on the South Side of Chicago. The most important thing that I learned by leaving HSS is that in a field like pediatric rheumatology where the diseases are relatively unusual, you can forget what patients bring to the equation. When you stay in one place you have a feeling you know the right way to do things, but you forget that this is affected by your location, the kinds of patients that you treat, the staff you work with, and the resources that are available to you.”
“The patients I treated in Chicago were very different from those I’d treated in New York,” continues Dr. Onel. “My time in Chicago made me much more focused on collaborative research because that is the only way that you can address the variabilities that patients bring — from different sites and different races, ethnicities, and socioeconomic classes. For example, some of our diseases vary in severity to some extent by ethnicity. It was a very rewarding experience.”
“Chronic recurrent multifocal osteomyelitis is a rare inflammatory bone disease that affects 1 in 1,000,000. It’s poorly understood, very distinctive in pediatrics, and is too rare for any one institution to get to the bottom of it. So, we are participating in a working group across the country to study this disease.”
— Dr. Karen B. Onel
Collaboration is Key for Rare Diseases
Dr. Onel says that she has returned to New York “much more invested in collaboration. When you realize there is so much to do, working as a group is the only way we are going to take care of children with rare diseases. There is no question about that.”
In returning to HSS, Dr. Onel is also coming back to the roots of her medical education at NewYork-Presbyterian. After earning her medical degree at Weill Cornell Medicine, Dr. Onel pursued residency training in pediatrics at NewYork-Presbyterian/Columbia University Irving Medical Center. She then went on to complete a clinical fellowship in pediatric rheumatology at HSS.
Dr. Onel’s clinical expertise focuses on children and young adults with lupus, juvenile inflammatory arthritis, vasculitis, uveitis, chronic non-infectious osteomyelitis, dermatomyositis, and the periodic fever syndromes. Through her research, she is gaining a greater understanding of the causes of rheumatic illnesses, as well as evaluating the safety and tolerability of new treatments. To that end, she partners with other institutions to define evidence-based best treatment practices, particularly for children with juvenile idiopathic arthritis, systemic lupus erythematosus, and recurrent non-infectious osteomyelitis. She has authored numerous papers, reviews, and book chapters on pediatric rheumatology. In addition, she serves as a reviewer for several scientific journals, including Arthritis and Rheumatism, Lupus, Seminars in Arthritis and Rheumatism, and Pediatric Rheumatology.
“I’ve always been very interested in clinical and basic translational research,” says Dr. Onel, who serves on the Finance and Ethics Committees and Chairs the Systemic Juvenile Arthritis Study Group of the Childhood Arthritis and Rheumatology Research Alliance (CARRA). “The truth is we rise together, or we stand alone. We need to do more research, more studies, more clinical trials, and publish more papers. We also need to be open to trying new paths. In addition, we need to foster more studies nationwide.”
Key CARRA trials underway across the country include STOP-JIA (Start Time Optimization of Biologic Therapy in Polyarticular JIA), FROST (FiRst-line Options for Systemic JIA Treatment) and the CARRA Registry. The common denominator in all of these trials is collaboration, says Dr. Onel. “We have been able to enroll more than 400 children into STOP-JIA from around the country. In the past, these numbers would never have been possible and the opportunity to ask and answer these very important questions would have been lost.”
Dr. Onel is also a member of the Advisory Council of the Pediatric Rheumatology Collaborative Study Group (PRCSG). “The PRCSG Advisory Council reviews all of the clinical trials proposals,” says Dr. Onel. “The Advisory Council decides what trials are appropriate scientifically and feasible for our network.” This past July, the PRCSG Advisory Council had an overview review published in the Pediatric Rheumatology Online Journal of the strategies employed by the study group to achieve drug and biologic approvals for children with pediatric rheumatic diseases, particularly juvenile idiopathic arthritis. The novel trial designs utilized for more efficient testing of innovative drug candidates have all been developed or co-developed by the PRCSG research network.
“One of my goals is to collaborate more with colleagues at Weill Cornell and at Columbia,” adds Dr. Onel. “Dr. Alexis Boneparth, a pediatric rheumatologist at Columbia, and I have already reached out to each other.”
The proximity of HSS to Memorial Sloan Kettering Cancer Center also offers opportunities to share discoveries. “The overlap with cancer and inflammatory disease is interesting,” says Dr. Onel. “We are looking at some of the children who are receiving transplants and are immunodeficient. This is a group with a lot of rheumatic disease overlap. Some of the new drugs that they are using for adults, the checkpoint inhibitors, may have implications for children. It has not necessarily been the case yet in children, but we suspect that the use of immunomodulators is going to increase.”
Dr. Onel is also studying chronic recurrent multifocal osteomyelitis, a rare inflammatory bone disease that affects 1 in 1,000,000. “The numbers are too small. If we are going to do this by ourselves, we are never going to get to the answer of what people actually need,” she says. “At HSS we’ve started doing whole body MRI, which is standard of care now, and we are participating in a working group across the country to understand this disease. It’s poorly understood, very distinctive in pediatrics, and is too rare for any one institution to get to the bottom of it.”
Another area of interest for Dr. Onel is juvenile arthritis. “We are going to work with the Sports Medicine physicians at HSS to formally look at children with juvenile arthritis who are treated and feeling well to see if participation in athletics can produce an effect that is good, bad, or otherwise,” she says. “For instance, if a child has wrist arthritis, is it appropriate for him or her to play volleyball after treatment? What we want to do is leverage the strengths that we have here at HSS to answer questions that really matter for kids and their families.”
Brunner HI, Rider LG, Kingsbury DJ, Co D, Schneider R, Goldmuntz E, Onel KB, Giannini EH, Lovell DJ; PRCSG Advisory Council. Pediatric Rheumatology Collaborative Study Group — over four decades of pivotal clinical drug research in pediatric rheumatology. Pediatric Rheumatology Online Journal. 2018. Jul 11;16(1):45.
For More Information
Dr. Karen B. Onel | [email protected]