A varied group of rare diseases including juvenile forms of arthritis, lupus, fibromyalgia, and other conditions are under the umbrella of pediatric rheumatic diseases. As it stands, there is an inconsistency in how patients with these diseases are treated in clinical practice. This makes it challenging to understand which therapies are the most effective. Recent efforts to address this challenge have been published in a new review article in Arthritis & Rheumatology.
For most diseases, randomized placebo-controlled clinical trials are conducted to help find the best treatment methods, but these trials are hard to perform in rare diseases. Alternative solutions are consensus treatment plans (CTPs), which are standardized methods developed by a consensus among experts, designed to help reduce treatment variability, and allow for better comparisons of different therapies. The largest pediatric rheumatology research collaborative in North America, The Childhood Arthritis and Rheumatology Research Alliance (CARRA), is pioneering the use of CRPs to standardize the common treatments for childhood rheumatic diseases.
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Sarah Ringold, MD, MS, of Seattle Children’s Hospital and her colleagues present their relationship for the CARRA CTP approach in their review and observe its advantages and disadvantages.
“Once a diagnosis is made, providers and patients and their families can choose together the CTP strategy that they believe will work the best for that disease,” Dr. Ringold explained. “Information on how the patient is doing on that treatment is then collected at regular clinic visits through a registry. At the end of the proposed study duration, researchers can then compare how the patients do between the different CTPs.”
She added that ongoing large-scale studies are currently examining this approach in juvenile idiopathic arthritis. The results from these studies will shed light on how effective this approach is, and may provide more insights on which therapeutic studies are most successful for certain patients. “The CTP approach will be even more powerful when coupled with biospecimen collection to facilitate translational research aimed at identifying biomarkers of response and non-response, paving the way towards personalized medicine,” she said.
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