Research, Clinical Care Combine to Improve Childhood Arthritis


When children with multiple swollen joints come to the Center for Childhood Arthritis and Rheumatic Disease for treatment, and then a few weeks later they are back on the field playing sports, their energy is contagious for pediatric rheumatologist Pamela Weiss, MD MSCE.

A clinician, researcher, and mentor at The Children’s Hospital of Philadelphia, Dr. Weiss received the Spondylitis Association of America Bruckel Young Investigator Award in honor of her contributions to the understanding of juvenile spondyloarthritis (JSpA).

Children with JSpA experience inflammation and stiffness of joints that can result in growth disturbances and loss of range of motion if not controlled properly. They tend to develop arthritis of lower extremities and are as at risk of developing arthritis of the lower back or spine. JSpA is associated with more frequent and higher intensity pain and poorer quality of life than other categories of juvenile arthritis.

“One of the questions we always get is, ‘Do a lot of kids actually have arthritis?’” said Dr. Weiss, clinical research director for CHOP’s division of rheumatology. “Actually, our clinic is full of these children. Our treatments are so effective that unless our patients chose to share that they have a chronic condition, most of the time no one knows there’s anything wrong with them.”

Juvenile arthritis is the most common rheumatologic disease among children, ranging from one to four cases per 1,000 children, which is about the same prevalence as Type 1 diabetes mellitus and cystic fibrosis in children. JSpA accounts for about 20 percent of childhood arthritis.

Dr. Weiss’ research concentrates on characterizing the disease features of children with JSpA, with a focus on enthesitis (inflammation of the tendon insertions) and arthritis of the lower back. She has an ongoing project evaluating the uses and effects of drugs for this condition. Recently she led an international effort to develop and validate a disease activity score for children with JSpA based on input from more than 100 physicians.

“This disease activity score is a really important metric because we need to gauge whether our patients are getting better on treatment or not,” Dr. Weiss said. “Prior to the development of this score, no metrics existed for these kids.”

Dr. Weiss also conducted a study that determined using ultrasound is more reliable than physical exams for accurate diagnosis of enthesitis. This is crucial because detection of enthesitis influences disease classification and subsequent treatment decisions.

In July, Dr. Weiss published results from another study that focused on determining how many children with newly diagnosed JSpA develop early lower back arthritis. Her team found that 20 percent of children had imaging evidence of lower back arthritis within six months of diagnosis, and that the majority of these children did not have the traditional symptoms used to screen for this condition, including back pain.

“Looking at kids at diagnosis to see if they have early arthritis of the lower back could impact the rapidity with which we start medications and could potentially alter their entire disease course,” Dr. Weiss said.

Currently, Dr. Weiss is analyzing data from a large database she created that includes children with enthesitis-related arthritis from five pediatric rheumatology practices, including one in Italy. She is exploring the effectiveness of treatment strategies for newly diagnosed patients.

When she is not improving children’s outcomes through research, Dr. Weiss has leadership roles within several national organizations dedicated to finding treatments and improving care for pediatric rheumatic diseases. One of her major projects was leading an update of the American College of Rheumatology’s juvenile arthritis treatment guidelines published in 2013. She is a member of the literature review team for the American College of Rheumatology’s spondyloarthritis treatment guidelines that will be published later in 2015.

Dr. Weiss also is the principal investigator at CHOP for a new national registry launching this summer by the Childhood Arthritis & Rheumatology Research Alliance (CARRA). About 60 sites are participating, and the data collection will be geared toward monitoring the long-term safety of medications prescribed for pediatric patients.

“I want to make kids better, but with the least amount of side effects,” said Dr. Weiss, who is co-chair of the Juvenile Idiopathic Arthritis Research Committee for CARRA. “In rheumatology, there are so many new drugs coming on the market, yet pediatrics is always behind adult medicine in terms of approval for the drugs. So there is a lot of work to be done to make sure the new medications are both effective and safe for our kids.”

Dr. Weiss shares her passion for rheumatology by acting as a mentor for fellows who are interested in pursuing research in this area, and she presents lectures to CHOP residents on pediatric rheumatology topics.

“By doing a combination of research and clinical care, I feel like I’m doing something different and meaningful every single day, and I love all aspects of it,” Dr. Weiss said.

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