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By KRISTA CONGER Aches and pains are an unfortunate yet often unavoidable byproduct of aging. But unlike older adults, the nearly 300,000 children in this country with arthritis or other debilitating rheumatic diseases don’t have the solace of a lifetime of memories to help them battle joint pain and stiffness, movement difficulties and muscle weakness. To add insult to injury, a dearth of pediatric rheumatologists, coupled with an under-recognition of the problem, conspire to deny many young sufferers access to medical treatment that could tremendously improve their lives. "It’s not unusual to discover someone out there for a year or two who’s just in horrible shape. It’s distressing how many patients don’t get to someone who can take care of them," said Christy Sandborg, MD, chief of pediatric rheumatology at Lucile Packard Children’s Hospital and associate professor pediatrics at the School of Medicine. "But when we do get the patients, we can make such a dramatic change in how they’re doing by recognizing and treating the condition correctly." Sandborg was recently named the first Kirkwood Distinguished Packard Fellow. Distinguished Packard fellowships are awarded to physicians in medical sub-specialties that are not well-supported by today’s health-care systems. Although the specialists at Packard Children’s Hospital provide ongoing care for about 700 children, many others slip through the cracks, going undiagnosed or receiving inappropriate treatment. Sandborg estimates that more than two-thirds of children with rheumatic diseases in the Bay Area are not being treated by physicians experienced in pediatric rheumatology. Rheumatic disorders can strike children of all ages and backgrounds. The most common is juvenile rheumatoid arthritis, or JRA. It can strike as early as age 1, causing joint pain and swelling and difficulty walking. JRA and other rheumatic disorders can also cause skin rashes and fevers, muscle weakness or other systemic illness and, in severe cases, compromise the heart, lungs and kidneys. If left untreated, rheumatic disorders would be the most common cause of acquired disability in childhood. Despite the prevalence of the conditions, however, pediatric rheumatology became a sub-specialty only about 20 years ago, and there are fewer than 200 board-certified pediatric rheumatologists in the country. Packard Children’s Hospital’s pediatric rheumatology program cares for children from throughout the state, as well as Nevada and Arizona — states without a single practicing pediatric rheumatologist. It is also one of 21 centers in the country dedicated to training new fellows in pediatric rheumatology. "Adult rheumatologists often don’t understand what it takes to treat kids," said Sandborg, citing a recent example of a newly arrived patient. "The previous doctor didn’t understand the risk of continued steroid treatment in a child." Because steroids can inhibit growth, the 11-year-old patient was the size of a 5 year old. In addition to considering a child’s special nutritional and development needs, Sandborg and her colleagues are cultivating a greater understanding about how the disease affects their young patients. Pediatric rheumatologist Elizabeth Mellins, MD, is using microarray technology to study why some children with JRA are more severely affected than others and why patients with JRA don’t always respond in the same way to identical treatments. "Our goal is to keep all of our patients functioning to the best of their ability. Ultimately we’d like to control and put their diseases into remission," said Sandborg. "If we can understand why some children have more severe disease or respond to certain medications, we may be able one day to predict within the first couple of months after diagnosis the safest and most efficacious treatment plan for each patient." Rheumatic disorders in childhood fall under a variety of labels. In addition to JRA, the list includes systemic lupus erythematosus juvenile dermatomyositis, juvenile ankylosing spondylitis, and scleroderma. Despite the complicated names, most of these devastating disorders have a common thread. "They’re nearly all autoimmune diseases," said Sandborg, "in which some of your immune cells have gone haywire and are attacking your own tissues." Treatment may include modified steroid regimens to reduce inflammation and even partial-strength chemotherapy treatment to tame the out-of-control immune cells. To help patients stay in school and grapple with family issues during their treatment, Sandborg and her colleagues rely on a team that includes, among others, nurse specialists, social workers and physical and occupational therapists. The team is skilled in guiding patients through the transition from adolescence to adulthood: a time when many teens might be tempted to give up on their treatment. "We try to help them understand their disease enough to take control of it as an adult," said Sandborg. "We watch our patients grow up and, in some cases, have kids of their own. Often, after we’ve helped them through their childhood and teenage years they say ‘Thank you so much for not giving up on me.’ "
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Lucile Packard Children’s Hospital New approach to genetic screening may rapidly identify immune diseases (5/14/03)
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Stanford Report, May 21, 2003
