Parents of children with arthritis know the dilemma all too well. While biologic response modifiers offer unparalleled benefits in the short term, they have the potential for unsettling long-term side effects. At least we think so – although biologics have been approved for use with kids since 1999, there’s no national database that tracks patients’ progress with the disease and their response to various medications.
Until now, that is. This summer saw the debut of a database specifically designed to gather such details. As the project’s principal investigator, Laura Schanberg, MD, co-chief of Duke University’s division of pediatric rheumatology, has overseen infrastructure development and rollout via institutions that are part of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), a national network of pediatric rheumatology researchers.
Dr. Schanberg reports that CARRA-net, as the database is named, is up and running at six sites and adding more patients every week toward its initial goal of 20,000. At present, participation is open to kids with JIA, lupus, vasculitis, dermatomyositis, fibromyalgia, systemic scleroderma, localized scleroderma and sarcoidosis who are being treated at a CARRA-affiliated facility (most are university health care centers or pediatric health care facilities). Enrollment consists of filling out consent forms and questionnaires, and takes about 15 minutes, Dr. Schanberg says. Patient health information is entered into the database and updated every six months. The plan is to make participation available at all 60 CARRA sites nationwide, then to all pediatric rheumatologists nationwide, and then to non-specialists who, due to location wind up treating kids with rheumatic conditions.
That’s important because kids in many states may not have access to a pediatric rheumatologist, says Bernard Murphy, chair of the Arthritis Foundation’s Juvenile Arthritis Leadership Group, which directs the Juvenile Arthritis Alliance. “They’re likely to be treated by an adult rheumatologist or even a general practitioner – someone who may not be up to speed on the JA disease model and treatment options.”
As the leader of “open-mic” nights at JIA advocacy summits, during which parents are encouraged to share their concerns about biologics, specifically those known as tumor necrosis factor (TNF) inhibitors, Murphy says he’s come across one question time and again: Will this treatment shorten my child’s life expectancy or put them at risk of developing cancer?
CARRA-net is the first step toward answering that difficult question. “You get that diagnosis, and there will be a system in place that says ‘This is the best course of treatment that we know of based on the fact that we’ve been looking at kids over time who are on different therapies,’” says Amy Melnick, the Arthritis Foundation’s vice president of public policy. “There’s so much more potential to improve care when you can aggregate this data.”
Expanding the reach
The idea isn’t new, Melnick says. The FDA convened a public workshop that started the ball rolling in the spring of 2009.
Initial funding for CARRA-net came last year in the form of $7 million in stimulus funds from the National Institutes of Health. Prior to its development, pharmaceutical companies monitored patients who were taking their medications, but that tracking stopped once a patient changed therapies – something that happens quite often with arthritis patients of all ages.
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