The greatest myth about arthritis is that it only afflicts older adults. In fact, the disease is a leading cause of acquired disability in the young, with about 300,000 infants and children in the U.S. facing a variety of rheumatologic conditions. These patients face a special set of challenges.
With support from the Rosalind Russell Medical Research Center for Arthritis, UCSF is a national leader in pediatric rheumatology—a relatively new medical sub-specialty for an underserved population—in terms of research, patient care and training physicians to treat patients.
"Patients come here not only to see the pediatric rheumatologist, but also to see our entire team, which includes a nutritionist, a physical or occupational therapist, and a social worker," says Dr. Emily von Scheven, director of pediatric rheumatology at UCSF, a program of the UCSF Department of Pediatrics. "We have a large emphasis on rehabilitative issues because these diseases affect the child and family in many ways."
"We see kids in the hospital when they are sick enough to require hospitalization," she says. "But the majority of our practice is outpatient."
The most common rheumatologic disease among children is JIA, or juvenile idiopathic arthritis, which used to be called JRA—juvenile rheumatoid arthritis. The term was a misnomer because, it turns out, in most cases pediatric arthritis bears little resemblance to adult rheumatoid arthritis. "The second most common disease we see is lupus," says Dr. von Scheven, "but many of our patients have more rare things like scleroderma, Lyme disease, Crohn's disease, vasculitis, and mixed connective tissue disease."
"Before the recent explosion in new drugs, some kids died of some of these diseases. For example, the five-year survival for childhood lupus used to be around 50-60 percent. Now it is approximately 95 percent. And in the past, many children with severe arthritis were crippled, whereas today they can expect to have near normal functioning. And now there is a new focus on normalizing life function and preventing secondary problems, like osteoporosis and poor growth."
One of the biggest challenges facing young patients is that arthritis is frequently misdiagnosed, and incorrectly treated, because neither parents nor primary care physicians expect to see it in children. "There are only approximately 200 board-certified pediatric rheumatologists in the U.S., and none between San Francisco and Oregon," says Dr. von Scheven. UCSF is a major referral center for pediatric rheumatology in Northern California, for the Central Valley and for the entire state of Nevada.
"One of the problems we have in pediatric rheumatology is that most of our medications are not formally studied in children," says Dr. von Scheven. "We have to adapt drugs approved for adults for use in kids, and that is considered 'off-label.' In order to really understand how well drugs work in kids—the risks and benefits—we need to study them directly in children.
"It's not just taking a medication and adjusting the dose for a smaller body. It's much more complicated. First of all, smaller bodies sometimes need proportionately higher medication because their metabolic rates are higher. We use more methotrexate in children than in adults, for example. But more importantly, we have to look at the effect of these drugs on growth and development. The risks of drugs may be different in a growing person than in an adult." Furthermore, in many cases the rheumatic diseases in children are actually different than in adults, thereby responding differently to the medications.
UCSF is at the forefront of conducting clinical trials on the safety and efficacy of drugs in pediatric rheumatology patients.
Because osteoporosis—poor bone density, which is a risk factor for fractured bones—often occurs in children with arthritis as a consequence of taking steroids for their disease, Dr. von Scheven and colleagues conducted the first placebo-controlled study of alendronate (Fosamax®) in children with inflammatory diseases. They found the drug is effective and safe in children between 8 - 18 years old, although it appears that a longer term of treatment may be required than the 18 months in the study.
A current UCSF study is the first to look at the benefits and risks of the cholesterol-fighting drug atorvstatin (Lipitor®) in pediatric lupus patients. Concurrently conducted at more than 20 sites nationwide, the study is important because high cholesterol levels, combined with other lupus-related factors, can increase the risk of heart attack and stroke about fifty times. Although lupus is usually a disease of older individuals, patients with childhood-onset lupus can have heart attacks and strokes in their 20s and 30s.
Across the country, very few doctors learn how to diagnose and treat arthritis in children, whose limping is usually attributed to accidents and whose joint pains are often written off as growing pains. "Only 40 percent of pediatric residents in the United States—the future pediatricians for the country—have any exposure to pediatric rheumatology," says Dr. von Scheven.
UCSF is making a significant difference in this regard. Dr. von Scheven directs the pediatric rheumatology fellowship program, which trains pediatricians in this sub-specialty. It is the only institution in the country to receive support from the National Institutes of Health to train physicians in both adult and pediatric rheumatology. That means physicians at UCSF learning to be pediatric rheumatologists spend some time in the adult rheumatology clinic, and physicians learning to be adult rheumatologists spend time with children in the pediatric rheumatology clinic.
One of Dr. von Scheven's goals is to significantly expand the number of pediatric rheumatologists nationally so that many more children with rheumatic conditions have access to an appropriately trained provider, and to train more physicians to conduct research in these rare diseases. Over time this will result in enhanced care, improved outcome, and ultimately better adult lives for these children.
(Published June 2008 in Arthritis Progress Report, the newsletter of the Rosalind Russell Medical Research Center for Arthritis. To be added to our mailing list, please send us a note with your name and address to
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