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CHOP Rheumatologist Recognized for Dedication to Research

Published on
Dec 29, 2020
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“Clinical research allows me to take what we accept as the standard of care and make it better for these kids,” says Dr. Weiss.

shafere1 [at] email.chop.edu (By Emily Shafer)

A Grand Rounds presentation during residency featuring lupus sparked the rheumatology career for CHOP pediatric rheumatologist Pamela Weiss, MD, MSCE. Since then, her passion for research in the field, particularly in juvenile arthritis, continues to be fervent.

That dedication led to an exciting year for Dr. Weiss, who is clinical research director in the Division of Rheumatology. It included a prestigious research award and a significant grant for an exciting new project in spondyloarthritis.

“Clinical research allows me to take what we accept as the standard of care and make it better for these kids,” said Dr. Weiss, who is also associate professor of Pediatrics at Perelman School of Medicine of the University of Pennsylvania. “Spondyloarthritis in particular has been understudied. There is so much more that we could do in terms of better understanding all of the manifestations of the disease and tailoring our treatments to improve outcomes for these kids.”

“Pamela

Pamela Weiss, MD, MSCE

Dr. Weiss is the recipient of this year’s American College of Rheumatology (ACR) Henry Kunkel Early Career Investigator Award. This award acknowledges an early career physician-scientist who is 12 years post-Rheumatology certifying examination eligibility and has made outstanding and promising independent contributions to basic, translational, or clinical research in the field of rheumatology. This award is not often given to pediatric rheumatologists — Dr. Weiss is the fourth to receive the award in its 32-year history.

A faculty member of the Center for Pediatric Clinical Effectiveness at CHOP, Dr. Weiss credits her colleagues in that group for much of her success in research, particularly the grant-writing team. She also appreciates the “unwavering support and enthusiasm” from colleagues in the Division of Rheumatology and the “invaluable collaborations” within the Division of Radiology.

Some of the spondyloarthritis research projects that Dr. Weiss is most proud of involve demonstrating that the use of IV contrast during a pelvic MRI adds no value to the procedure or ability to diagnose arthritis at the sacroiliac joint, and that arthritis at the sacroiliac joint actually is prevalent at diagnosis. These studies impacted standard of care for these children at CHOP and other pediatric institutions in the U.S.

Dr. Weiss is continuing her research in spondyloarthritis, thanks to a new grant from the Patient-Centered Outcomes Research Institute (PCORI). For this project, Dr. Weiss and her team will be evaluating whether children with spondyloarthritis who reach “quiet disease” after treatment with tumor necrosis factor (TNF) inhibitors, can take less medication, or even stop it all together.

“This is an important question to ask because many patients and families would like to stop taking these medications,” Dr. Weiss said. “Most of them are injectable medications, they have side effects, and they’re really expensive. If kids could come off the medications, even for a year or two, and still do really well, that’s a big win.”

The researchers will evaluate how patients with inactive spondyloarthritis on TNF inhibitors respond to three different treatment strategies: continuing TNF inhibitor medication; spacing out timing of the doses; and stopping the medication completely. They will also analyze factors that might predict disease flares after reducing the medication, and whether remission can be recaptured quickly after restarting therapy.

The study will leverage the PEDSnet research network to enroll 198 children with spondyloarthritis from 20 participating healthcare centers across the country. Patients are eligible for the study if they are receiving TNF inhibitor therapy and have had quiet disease for at least six months. The children will be randomly assigned to one of the three treatment strategies. If patients experience a flare, their study participation will end, and they will work to get back to quiet disease. The researchers will track disease flares via electronic health record data after the trial participation ends.

“It would be fantastic if we can show that kids who have a sustained remission can come off medication and do really well,” Dr. Weiss said. “The next best thing would be, if we can’t stop the medications completely, to show that we can space the injections out further, and kids can stay in remission with less medication.”