The consequences of a child receiving insufficient or inappropriate treatment for a rheumatic disease will last a lifetime — a lifetime that, statistically, will be significantly longer than an adult patient receiving comparably inadequate care. With fewer than 350 pediatric rheumatologists practicing in the United States, children with rheumatic diseases are often relegated to rheumatologists who typically treat adults and, in the absence of a pediatric specialist, do their best to provide care to pediatric patients.
More than 300,000 U.S. children are diagnosed with arthritis and other rheumatic conditions, and the nationwide scarcity of subspecialists and the unmet needs of this vulnerable population should be driving residents toward pediatric rheumatology. Despite widespread efforts to spread awareness of this unmet need, the pediatric rheumatologist shortage persists — the question is why.
A simple answer pertains to the structure of medical education, according to Sangeeta Sule, MD, PhD, chief of pediatric rheumatology at Children’s National Medical Center. “One reason is that the fellowships for pediatric rheumatology are 3 years as opposed to 2 years,” she said in an interview with Healio Rheumatology. “Another reason is that rheumatology is primarily an outpatient field, but, in clinics, most residents are exposed to patients through inpatient training. Pediatrics residents are not exposed to it, so they do not even see it to become excited about it or ultimately enter the field.”
With fewer than 350 pediatric rheumatologists practicing in the United States, children with rheumatic diseases are often relegated to rheumatologists who typically treat adults.
Heather Ann Van Mater, MD, associate professor of pediatrics at Duke University Medical Center, built on this point. “Residents who do see pediatric rheumatology patients are seeing the inpatients, who are generally the sickest patients with lupus or systemic onset juvenile arthritis, and they are therefore getting a skewed idea of what it means to treat these children,” she said. “Residents come away thinking that all pediatric rheumatology patients are just treated with high-dose IV steroids, which is what we frequently use in the inpatient setting, and that is it. They don’t have the opportunity to see the real spectrum of patients, and how new, exciting new therapies are altering the course of these diseases.”
The American College of Rheumatology has taken strides to rectify the numbers problem. “In 2001, the ACR started a pediatric rheumatology residence program, in which residents are paired with a mentor, to try to boost those who may be thinking about it,” Sule said. “Over the last 17 years, as many as 73% of residents who went through the program chose pediatric rheumatology training.”
A similar residency program at Duke also demonstrated benefits, according to Van Mater. “We have seen more residents going into the field, and, interestingly, most of them were not expecting to go into pediatric rheumatology when they started,” she said. “Seeing the breadth of inpatient and outpatient care has a really big impact. When you change the system just a little bit, you can see benefits.”
Despite these gains, the shortage is likely to persist, if not worsen. In the meantime, a significant proportion of rheumatologists who primarily treat adults will be forced to see pediatric patients. It is for this reason that pediatric specialists are often eager to spread the word about the subspecialty, and offer some guidance along the way.
Treating ‘Small People’
Karen Brandt Onel, MD, chief of the department of pediatric rheumatology at Hospital for Special Surgery, and professor of clinical pediatrics at Weill Cornell Medicine, laid out the particulars of dealing with pediatric patients. “Performing a joint exam on a 2-year-old is extremely difficult unless you have spent a significant amount of time doing it,” she said. “Prescribing medications for these children can also be problematic because dosing is variable due to age and size — something that pediatricians are used to dealing with.”
Dosing in pediatric patients is largely based on weight, according to Sule. “It is imperative to calculate the child’s weight at each visit because they are growing,” she said. “Also, children metabolize medications differently than adults, which is something many adult rheumatologists don’t consider.”
Findings from Van Mater and colleagues published in Arthritis Care & Research indicated that while 23% of adult rheumatologists reported treating children, most dealt primarily with older adolescent patients. Additionally, although 94% of adult rheumatologists reported being comfortable in diagnosing the most common pediatric rheumatologic disease, juvenile idiopathic arthritis (JIA), only 76% felt comfortable treating this condition.
This discomfort can carry consequences, according to Van Mater. “The effects of arthritis or inflammatory diseases on growth are important to consider when determining treatments and disease activity targets,” she said. “In pediatrics, given the potential for permanent limb length discrepancies, joint damage and metabolic changes from chronic inflammation, the goal is no disease activity while minimizing — or hopefully avoiding — systemic steroids.”
For Van Mater, the goal of no disease activity comes down to understanding which therapies to use, and aggressiveness in prescribing them. “We try to avoid prolonged courses of steroids due to growth and metabolic impacts,” she said. “When dosing medications in children, it is essential to not assume they will need lower doses than adults. Many adult rheumatologists might be afraid to be too aggressive, but you have to understand that we have an important — but often small — window of opportunity to make a significant impact on these patients during formative times.”
If a clinician fails to properly deal with a mechanical issue in a child’s joint, they may have that issue for the rest of their life, Van Mater added. “We need to make sure they maintain healthy joints for the long haul,” she said.
“In many ways, the physical exam and dosing issues are minor compared to the challenges in talking with these patients and their parents,” Onel said. “Children and adolescents have specific vulnerabilities and emotional needs due to age in addition to their chronic illness. If you treat someone with these issues inappropriately, it can cause long-term, lifelong problems.”
Onel said that pediatric rheumatologists are trained to talk to young patients on their terms, to explain decision-making processes to parents and guardians, and to understand ancillary services pertaining to schools and other activities. “We know to ask about vaping and contraception, and we understand how the mood disorders specific to young people can impact disease activity,” she said.
For Van Mater, the way pain is perceived and processed is yet another critical component to consider. “Some children are very sensitive to pain,” she said. “For most children, differentiating active disease from mechanical joint pain and secondary pain syndromes can be challenging. Other children are very guarded and will not complain of pain due to fear of shots or additional treatments. A thorough exam is therefore needed, even of joints they are not complaining of issues with, to evaluate for signs of arthritis, as the history alone can often be misleading in children.”
In addition, Sule noted that pain itself can often prompt a referral to a rheumatologist, but usually after other diagnoses have been ruled out, along with the included expenses. “These kids are probably going to emergency rooms because they are in pain,” she said. “This increases costs and the burden to the system overall.”
Looking at the Numbers
As with adult rheumatology, pediatric patients can always benefit from a more robust armamentarium of therapies. While the concerns for individual patients are real, the numbers may not add up to warrant the type of attention that pharma and the FDA are willing to give to yield more options and approvals.
JIA impacts upwards of 300,000 children in the U.S., according to the American College of Rheumatology; other rheumatic diseases are even more rare, with an estimated 30,000 lupus cases and 3,000 dermatomyositis cases, and cases of systemic vasculitis and chronic recurrent multifocal osteomyelitis numbering in the hundreds. Apart from JIA, all of them would be considered rare or orphan diseases, according to the FDA criteria.
“Because these are rare diseases, we do not have FDA-approved doses for medications,” Onel said. “No matter how much we would want pharmaceutical companies to test a medication in children, the companies will only manage them if it gives them something. Although there are rules within the FDA for patent exclusivity that will grant companies additional years if they test in children, there is not a real fiscal advantage to conducting these pediatric trials.”
But Onel persists in spreading awareness of these diseases and tries to remain focused on the big picture. “We are a very funny society: We treat diseases when they happen but are less interested in pre-emptively treating children so they don’t become unhealthy adults,” she said.
This particular group of unhealthy adults could become a significant burden on the health care system, according to Sule. “Joint damage often requires surgery and other expensive treatments,” she said. “The same is true for permanent kidney disease or neurologic diseases. If they go untreated in children, it becomes a burden on adult rheumatologists as well.”
A closer look at the rheumatology workforce reveals an even starker picture. Roughly one-quarter of children are receiving appropriate care from a pediatric rheumatologist, according to statistics from the Arthritis Foundation. In eight U.S. states, there are no certified and practicing pediatric rheumatologists, and five states have just one such qualified practitioner. Moreover, as in other rheumatology practices, the average age of pediatric rheumatologists is older than 50 years, and the retirement of older rheumatologists is likely to worsen access in even densely populated areas.
“With so few certified practitioners, this represents a serious problem of access,” Sule said. However, despite these many obstacles, she remains hopeful. “There are great resources available through the ACR, and through the individual disease foundations,” Sule said. “There are many ways to educate providers and families. ACR has a whole packet to help adult rheumatologists deal with pediatric patients who are transitioning into adults.”
“The reality is that because our diseases are rare, and because there are so few of us pediatric rheumatologists, we have become a feisty bunch,” Onel added. “We have banded together, and it has become part of what we do to raise awareness. This has helped us move forward. This has helped us stand together.”
For Sule, it’s not only about standing together, but also about reaching out. “Pediatric rheumatologists are all open to help,” she said. “Don’t be afraid to ask for help with specific diseases.” – by Rob Volansky
For more information:
Karen Brandt Onel, MD, can be reached at 535 E 70th St 5th floor, New York, NY 10021; email: randolphs@HSS.EDU.
Sangeeta Sule, MD, PhD, can be reached at 111 Michigan Ave. N.W., Washington, D.C. 20010; email: email@example.com.
Heather Ann Van Mater, MD, can be reached at Box 3212, Durham, NC 27710; email: Heather.firstname.lastname@example.org.
Onel KB. CARRA Registry and Current Pediatric Practice. Presented at: Rheumatology Nurses Society Annual Conference; Aug. 8-11, 2018; Fort Worth, Texas.
van Mater H, et al. Arthritis Care Res (Hoboken). 2018;doi:10.1002/acr.23273.
Disclosures: Onel reports _________. Sule and Van Mater report no relevant financial disclosures.