A global community of physicians cooperated to share experiences with systemic drugs for pediatric psoriasis, and to get experience with a joint registry. A uniform dataset could be tapped to learn more about disease manifestations and treatment outcomes
Emerging research from an international dataset of pediatric psoriasis patients is revealing much needed information about how children fare with commonly used systemic treatments, says dermatologist Amy S. Paller, M.D., M.S.
And she says the collaborative effort is powered by pediatric dermatologists - not industry.
Dr. Paller, professor and chair of dermatology at Northwestern University Feinberg School of Medicine and pediatric dermatologist at Ann and Robert H. Lurie Children’s Hospital of Chicago, presented findings from the PeDRA-EPPWG Study of Systemic Therapy in Pediatric Psoriasis at the July World Congress of Pediatric Dermatology in Chicago. She not only talked about the soon-to-be-published study’s findings, but also how a retrospective analysis could inform a prospective registry.
Dr. Paller and colleagues launched the Pediatric Dermatology Research Alliance, or PeDRA, in 2012, recognizing that pediatric dermatologists needed to work as a group to research dermatologic conditions in children because many of the conditions are rare and lack pediatric-specific data.
“That’s exactly what has happened in this work with pediatric psoriasis,” Dr. Paller says.
Colleagues in the European Pediatric Psoriasis Working Group, or EPPWG were willing to buy in. The groups shared goals to better understand dermatologists’ experiences with systemic drugs for pediatric psoriasis, and to get experience with a joint registry, which hopefully would pave the way for a prospective pediatric psoriasis registry, according to Dr. Paller.
Ten centers from PeDRA in North America and 10 centers in Europe came together to perform the study.
“It was a tremendous learning experience about some of the challenges of retrospective data collection and the benefit to prospective research using common data collection,” Dr. Paller says.
The researchers extracted 54 different items from charts of patients treated with systemic therapy or phototherapy, but only allowed patients to be included who had at least a minimum dataset that could provide important information on demographics, clinical characteristics and severity, systemic agents used, treatment duration and efficacy, side effects and reasons for discontinuation of medications.
A review of thousands of patient records revealed 446 which met criteria for the minimal dataset; of those, 390 involved systemic therapy for pediatric psoriasis. In this joint PeDRA-EPPWG study, which was funded by the International Psoriasis Council, data was collected using the Research Electronic Data Capture, or REDCap, web-based data capture tool.
NEXT: Lessons learned
One of the lessons in that research is the importance of question specificity.
“If you just walk into a patient’s room, who is on systemic therapy, and ask, ‘How are you feeling? Any problems?’ you may well miss having the person report the nausea that he is experiencing. In contrast, you likely would capture that data if you said, ‘I know you’re on methotrexate. Tell me a little about whether you’ve had any abdominal discomfort or nausea or anything else that has to do with your stomach.’ Then follow that with questions about how soon it occurs after taking the drug and if persistent, allowing the doctor to make a decision about the possible relationship to the nausea to methotrexate,” she says. “The more we can be consistent in our data collection, the more value there is for pooling datasets to understand disease better or to understand the outcome of treatment.”
Without uniformity in questioning and a fairly comprehensive collection of data, it’s difficult to get good data from a retrospective review, she says.
“That’s one of the reasons we think prospective registries would be great,” Dr. Paller says.
While establishing prospective registries can be challenging and costly, Dr. Paller and her colleagues in PeDRA and the International Eczema Council are jointly planning to create uniform minimal datasets for use in clinical practice and incorporation into electronic medical records.
These would include the same questions about history, findings on examination, and planned intervention for children with atopic dermatitis that are important to ask in the caring for the patient. This uniform dataset could be tapped to learn more about disease manifestations and treatment outcomes. If successful, a similar process for documentation could be applied to pediatric psoriasis and other diseases.
Comparing apples to apples offers important insight. For example, in the PeDRA-EPPWG study researchers found big differences in gastrointestinal side effects from methotrexate, depending on whether the patients were in Europe versus North America. European children experienced significantly more gastrointestinal side effects from the medication, despite receiving the same amount of folic acid weekly. The difference was that most European kids were given the folic acid to take once a week on the day after the methotrexate; in contrast, the North American children were largely taking a smaller dose of folic acid daily or on the six days when methotrexate was not being given.
“I can tell you that many of our colleagues in Europe have switched to folic acid on six to seven days a week as a result of this study’s data,” Dr. Paller says.
A key study finding: Worldwide, methotrexate is the most commonly used systemic therapy for pediatric psoriasis. Overall, approximately 70% of the children used methotrexate and 27% used tumor necrosis factor (TNF) inhibitors.
Compared to methotrexate, cyclosporin, acitretin and fumaric acid esters (not used in the U.S.) had more medication-related adverse events, overall, and per patient years. TNF inhibitors had fewer overall medication-related adverse events than methotrexate.
Dr. Paller and colleagues are now analyzing this data to consider differences in efficacy, as well as the differences between Europe versus North America beyond the utilization of folic acid, she says.
Dermatologists who are interested in pediatric skin research are encouraged to become members of PeDRA (http://pedraresearch.org) and get involved in international studies, Dr. Paller says.
“This study was a great example of the power of a research collaborative. And, in this particular case, the power of getting investigators together internationally to ask important questions,” Dr. Paller says.
Disclosures: Dr. Paller reports no relevant disclosures.