Issue: August 2017
August 18, 2017
14 min read

Advancing Standards of Care in Pediatric Rheumatology

Issue: August 2017
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The most important challenge facing pediatric rheumatology is a lack of pediatric rheumatologists, according to many experts in the field. The shortage of clinicians has a ripple effect. It impacts the amount and nature of research conducted in the field. Communication gaps exist between young patients, their parents and clinicians, largely because many people simply do not realize rheumatic diseases can occur in children. Diagnoses are delayed. While treatments can be more or less effective in children compared to adults, primary pediatricians or general practitioners may not be familiar with these uses. All of this points to suboptimal patient outcomes.

“A problem is the current rheumatology workforce shortage,” Cathy Patty-Resk, MSN, RN, CPNP, pediatric rheumatology nurse practitioner at Children’s Hospital of Michigan and Specialty Center Detroit, told Healio Rheumatology. “It will continue to worsen unless we all take an active role in educating and inspiring our future rheumatology providers while they are in medical school or pursuing a nurse practitioner education.”

Patty-Resk personalized the far-ranging effects of these knowledge and awareness gaps.

“I have had parents of children with aggressive [rheumatoid-factor positive juvenile idiopathic arthritis] RF+JIA who have delayed treatment with methotrexate (MTX) and biologics out of fear of harming their child’s internal organs despite all the time we spend educating them,” she said. “We have given them great literature, encouraged them to network with other JIA families and the Arthritis Foundation and conducted frank discussions about this potentially crippling disease,” she said.

Cathy Patty-Resk, MSN, RN, CPNP
Cathy Patty-Resk

Despite these efforts, the result is often permanent joint damage, according to Patty-Resk. “I wish we had a crystal ball we could show these parents so they could see the organs and joints prevail and be healthy,” she said. “I spend a great deal of time with these parents at each visit, not only providing care for their child, but emotional support to empower the parents so they can let their child be a kid and say, ‘Go play.’ We need to have a better appreciation of the mind-body connection.”

Charles H. Spencer, MD, professor of clinical pediatrics at Nationwide Children’s Hospital/Ohio State University, said awareness may be improving, albeit slowly.

“It is better than it was in the 20th century, but still could be improved upon a great deal,” he said. “Primary physicians in the community still do not seem to have good musculoskeletal exam skills to detect arthritis or know how to order the right laboratory tests. Changing the ability of these more senior physicians to diagnose arthritis diseases is hard, and we get many inappropriate or delayed referrals due to these issues.”


Workforce Shortage

Bullock and colleagues suggested telemedicine may be an important way of compensating for the shortage of pediatric rheumatologists in the United States. They surveyed 221 parents or guardians of patients during a recent 6-week period to determine barriers to care and interest in exploring alternative models of care for children with these diseases. Results indicated more than 25% of respondents had traveled more than 3 hours to visit the pediatric rheumatology clinic and nearly half reported travel as an inconvenience. Most respondents (95%) reported that an in-person visit would be preferential to a telemedicine visit. This trend held for respondents who claimed travel was inconvenient (92%) or convenient (97%), even when those in the inconvenient group reported travel as a barrier to care. However, among respondents who were familiar with telemedicine, the preference for telemedicine visits (27%) was greater than the preference for in-person visits (3%). Sixty-three percent of the group was amenable to an outreach clinic. However, an adult rheumatology center was acceptable to 22% of the cohort, while a shared-care clinic was acceptable to 34%. “Ultimately, the parent perspective can shape acceptable ways to address barriers and provide accessible care,” the researchers concluded.

Brian Feldman, MD, MSc, Ho Family Chair in Childhood Autoimmune Diseases and head of the division of rheumatology at The Hospital for Sick Children said the health care system issues in his native Canada are different from those in the United States.

“More pediatric rheumatologists are being trained in the [United] States, and hopefully there will soon be no states that don’t have one,” he said. “In the meantime, it is important that awareness is broadened among primary care specialists and that timely referrals to experienced rheumatologists, neurologists or dermatologists experienced in the care of dermatomyositis in children are encouraged.”

Empower Families

Parsons and colleagues wrote that including young patients in deciding on directions for research may be useful in guiding and prioritizing clinical studies. They conducted 13 focus groups among patients aged 11 years to 24 years with rheumatic conditions in the United Kingdom to gain a better understanding of their research-related priorities. The study included 63 participants. Results showed basic science was a key priority, along with psychosocial research. As many respondents were satisfied with the care they were receiving, health services research was of a lower priority. Similarly, clinical medicine was not a priority because respondents reported satisfaction with their therapies and discomfort with trying new treatments. Public health also was a lower priority. Differences in response were reported among age groups and across geographic locations. “The results highlight the importance of considering the whole age range of adolescence and young adulthood, as well as geographical diversity,” the researchers concluded.


Feldman addressed the challenges in understanding what pediatric patients and their parents need and want from care and research. “I’m not sure there are any special challenges, per se, but because dermatomyositis in children is such a rare disease, everything needs to be explained in detail. As physicians, we can’t assume that patients and families have any baseline knowledge of the disease,” he said.

For Jay Mehta, MD, MS, clinical director of the division of rheumatology at Children’s Hospital of Philadelphia, simple awareness of the most commonly reported diseases can go a long way in helping clinicians and parents understand what they are up against.

“The most common autoimmune diseases we see are JIA, lupus, juvenile dermatomyositis and vasculitis,” he said. “The major signs and symptoms that raise the possibility of rheumatic disease for a pediatrician are joint and back pain or stiffness that is worse in the morning and gets better as the day goes on, persistent joint swelling that isn’t just associated with activity and lasts more than a day at a time, persistent daily or recurrent — for example, monthly — fevers without a clear infectious source, a facial rash that comes out after spending time in the sun and isn’t a typical sunburn, a rash that looks like broken blood vessels known as petechiae or purpura, or muscle weakness, especially with going up stairs, brushing hair or teeth and getting up from a chair. This is not an exhaustive list by any means, but they’re what I think the pediatrician should be most on the lookout for.”

Spencer encouraged ongoing education in the medical community.

“It is best to focus on medical students and residents,” he said. “Improving musculoskeletal exam skills of medical students and pediatric and family medicine residents is crucial. It is also important for rheumatologists to give at least one pediatric talk to each of these young doctors or doctors-in-training. It will take time, but these young professionals will be better trained for early diagnosis of rheumatic diseases than their seniors.”

Managing expectations may also be useful, particularly at the treatment stage, according to Spencer. “Parents may focus on length of treatment and inability to obtain full remission as a major frustration,” he said. “We get 50% improvement or 70% improvement, but not the 100% parents and patients want.”

Juvenile Dermatomyositis

Cantez and colleagues investigated cardiac outcomes in a cohort of 105 children with juvenile dermatomyositis (JDM) treated at a single center between 1991 and 2007. Abnormal electrocardiographic findings were reported in 6% of the cohort, while 25% demonstrated abnormal echocardiographic findings. The researchers noted, however, that many of these cases were mild or possibly not related to JDM. “Our findings suggest that cardiac abnormalities at JDM disease onset are frequently seen, but are rarely significant findings due to disease; however, JDM patients should be considered for screening for cardiac disease as late cardiac complications are well recognized,” they concluded.


“While still unclear and awaiting more extensive research, it may be that earlier and better control of the inflammatory disease and more restricted use of corticosteroids will help prevent cardiac and other damaging complications as children with JDM age,” Feldman said.

Spencer and colleagues investigated the use of biologics in inflammatory juvenile dermatomyositis using data from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry, which has information on these drugs used between 2011 and 2016. In the study, they conducted an online survey of clinicians who used these drugs in this patient population in 2012. They report on data for 105 pediatric rheumatologists who responded. At that time, 35 of 90 had never used a biologic therapy for juvenile dermatomyositis. Among those who did use a biologic, 32% had used rituximab, 5% had used etanercept, and 4% had used infliximab. Seventeen percent used more than one of those three therapies. Biologic therapy was the strategy for 10%, while 19% used a biologic with MTX; 52% used a biologic with MTX and corticosteroids; 42% used a biologic with MTX, corticosteroids, and immunosuppressant; and 43% used a biologic with IVIG and MTX. A second survey was conducted in 2013 and included substantially more drugs and drug combinations. While the 2013 survey revealed no recognizable patterns in usage of these drugs and combinations, rituximab emerged as the preferential treatment, followed by adalimumab, infliximab, abatacept, tocilizumab, and even etanercept. A final survey in 2016 confirmed this result.

Brian Feldman, MD, MSc
Brian Feldman

The 2016 survey also included more drugs and drug combinations. While this survey revealed no recognizable patterns in usage of these drugs and combinations, rituximab emerged as the preferential treatment, followed by adalimumab, infliximab, abatacept, tocilizumab and etanercept.

“These survey results and our consensus meetings determined our choice of four biologic therapies (rituximab, abatacept, tocilizumab and anti-TNF-[alpha] drugs) to consider for refractory JDM treatment when indicated and to evaluate for comparative effectiveness and safety in the future,” the researchers concluded.

“Most children are treated with corticosteroids and a steroid-sparing agent, which is most often MTX,” Feldman said. “While biological therapies have a huge impact in other areas of rheumatology, there has not been a biological therapy that has shown to be of great impact in dermatomyositis.”

Spencer spoke more broadly about therapeutic options, and whether current regimens are effective enough.

“Effective enough is a tricky phrase,” he said. “Are there some children who do not do well enough on current treatment? Yes, but there many fewer kids who do poorly with the current treatment. Kids in wheelchairs are rare.”

Charles H. Spencer, MD
Charles H. Spencer

Most clinicians, including Spencer, recognize that biologics are more effective than MTX, NSAIDs and steroid treatments, even though these are more expensive.

“Remission is a real possibility with every child, and some children are spared any sign of serious disease,” Spencer said. “Quality improvement projects allow us to make sure our patients get the best care, obtain the needed labs, the eye checkups, and are taking their medications.”

Physical Activity

Another debate in the profession is the extent to which children with rheumatic diseases should be exercising and the nature of that activity.

Coda and colleagues suggested that although low-intensity exercise regimens have been shown to be safe in patients with JIA and lead to improved physical function in those patients, use of these approaches is not consistent across populations. The researchers added that adherence to MTX and other therapies is problematic in this patient group. With this in mind, it was proposed that advances in interactive mobile and wearable technologies may solve some of these problems.

“Modern interactive technologies can be customized and accessed directly in the hands or wrists of children with JIA,” they wrote. “These secured networks could be accessible ‘live’ at anytime and anywhere by the child, parents and clinicians.” Such technology may yield improvements in biological parameters, symptom progression, adherence to medications, quality of life and participation in physical activities. These outcomes may improve clinical decision-making for physicians, parental awareness and self-management for patients. The benefits may be useful in both the clinical and research arenas, according to the researchers.

“We have been encouraging physical activity and participation in normal activities of childhood for decades now,” Patty-Resk said. “We see first-hand the detrimental effects of social isolation when children are kept out of school. Granted, sometimes it is warranted, as in the case of a newly diagnosed severe JDM in an adolescent who has to trek through a large high school campus with several flights of stairs. However, there are still times when kids are missing school for reasons we, as providers, do not agree with and will not provide school excuses.”

Gualano and colleagues noted that several levels of evidence have supported a departure from the traditional paradigm of prescribing bed rest to children with rheumatic diseases. They wrote that physical activity may benefit a range of conditions, from juvenile systemic lupus erythematosus to JIA, juvenile fibromyalgia and JDM. Increased activity may improve aerobic fitness, pain, fatigue, muscle weakness and poor health-related quality of life, according to their findings. They suggested that psychosocial factors, such as social self-isolation, overprotection and fear and/or ignorance on the part of parents, teachers and health practitioners, may also have a detrimental impact on these patients.


“In support of this rationale, a growing number of studies have demonstrated that the systemic benefits of exercise training clearly outweigh the risks in these diseases,” they wrote. “In this sense, health professionals are advised to assess, track and fight against physical inactivity and sedentary behavior on a routine basis, as they are invaluable health risk parameters in rheumatology.”

“Physical therapy and kids with JIA is different than it used to be,” Mehta said. “Physical therapists used to be an integral part of every pediatric rheumatology clinic, and divisions would highlight what a close relationship they had with physical therapists. Since the biologic era and the recognition that early aggressive treatment of kids with JIA leads to better outcomes, there has been a rapidly diminishing need for physical therapy in kids with JIA. The only times I’ve tended to recommend physical therapy is if there was some significant delay in diagnosis or treatment, or in the occasional child who is refractory to treatment.”

Some parents fear the activity of a typical day in school is too much for a child with arthritis, according to Patty-Resk. “In fact, it is too much for the parent to send their ‘sick’ child to school, where they cannot be near or protect them,” she said. “Parents who have a trusted relationship with the school staff are much better able to send their newly diagnosed JIA child to school.”

Pediatric Rheumatololgy visits

Patty-Resk acknowledged it may be difficult for some parents to surrender “protector mode,” but she reminds parents it is a necessary step in bringing these patients back to normal activity levels.

“I have patients who are competitive figure skaters, travel soccer and baseball players, track and cross-country runners, swim team, competitive dancers and musicians,” she said. “It is more than rewarding seeing them go from sick at diagnosis to back to being kids and enjoying their childhood and special friendships.”

Delays to Care

Rubinstein and colleagues wrote that delayed presentation to a pediatric rheumatologist may be a barrier to care in pediatric lupus. They explored differences in demographic and clinical characteristics of lupus patients and delays to care in a cohort of 598 children from the within the CARRA Legacy Registry. A typical time to presentation was defined as between 1 month and 3 months of symptom onset. Results showed 44% of the cohort presented early (within less than 1 month of symptom onset), while moderate delays (3 months to 12 months) occurred in 23% and severe delays (more than or equal to 1 year) occurred in 9%. Factors associated with early presentation included a family history of lupus, absence of discoid rash and location in a state with a higher density of pediatric rheumatologists, according to the findings. Conversely, younger patients, household income of less than $25,0000 per year and a family history of lupus were factors associated with severe delay. “In this large and diverse sample of patients, access to care and family resources played an important role in predicting time to presentation to a pediatric rheumatologist,” the researchers concluded.


“We still see delays in diagnosis,” Spencer said.

Patty-Resk also sees delays and she offered further perspective.

“I have only seen a few delays in lupus diagnosis, and these were from large outside institutions of the young child without a family history of lupus,” she said. “It stemmed from physicians who did not look for lupus immediately as the underlying cause of pleural or pericardial effusions or proteinuria in the young child.”

Patty-Resk acknowledged the shortage of rheumatologists could easily impact these outcomes, as well. “It seems that poverty, young age and a family history of lupus had the most severe delay in diagnosis, presumably from a lack of education about signs or symptoms of lupus,” she said and added that the disease may occur in young school-age children. “Just from my personal experience with newly diagnosed children with lupus, it seems that parents are still surprised their child has lupus when other family members were diagnosed with lupus later in life. The parents who are less surprised and more suspect of lupus tend to be the ones who were diagnosed in early adulthood themselves or have a close relative that was.”

Spencer put the issue into practical terms. “We believe in the theory of window of opportunity, which means that you only have a few months to get the arthritis disease under good control or the disease will be tougher to control,” he said. “Early diagnosis means better outcome.”

Education, perhaps in the form of a public service announcement blitz about childhood rheumatic diseases, could be effective, according to Patty-Resk. “We see so many ‘rare’ diseases that they hardly seem rare to us,” she said.

Moving Forward

Patty-Resk once again highlighted the shortage of pediatric rheumatologists as a critical need in the field. “They need to be challenged at developing their differential diagnosis in pediatric rheumatology to feel our excitement and love for this career path,” she said. “Too often they think it is all about kids with an old person’s disease and that couldn’t be farther from the truth. As a former emergency department nurse, I can tell you that pediatric rheumatology has the same level of excitement and challenge, but can be much more rewarding from the sustained relationships with the children and their parents.”

For Mehta, a cornerstone treatment of these patients is physical activity.

“We encourage all our patients to stay as active as possible,” he said. “We encourage them to fully participate in gym class and generally recommend no restrictions at all. This is especially true of the pain amplification syndromes, like juvenile fibromyalgia or complex regional pain syndrome. Inactivity in those patients can lead to worsening symptoms, and daily vigorous physical activity is part of the treatment plan.”


Spencer spoke to the role that professional organizations like the American College of Rheumatology and EULAR may play in advancing the cause.

“It would be useful to see more emphasis on kids with arthritis,” he said. These organizations are run by adult rheumatologists and, truthfully, pediatric rheumatology receives some focus but not enough.”

Education efforts will go a long way in improving outcomes, as will increases in the workforce of rheumatologists who specifically target pediatric patients, he said. There is a final component to the effort for Spencer, as well. “We need to support our research organizations worldwide,” he said. — by Rob Volansky

Disclosures: Feldman, Mehta, Patty-Resk and Spencer report no relevant financial disclosures.