Consensus treatment plans can help reduce variation in treatment approaches, identify effective treatment strategies and standardize outcomes for pediatric patients with rheumatic diseases, according to a recent review published in Arthritis & Rheumatology.
Developed by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) in 2009, consensus treatment plans (CTPs) are intended to supply health care providers with a set of standardized treatments that match the common clinical presentations of various rheumatic diseases, allowing meaningful direct comparisons among treatment strategies.
“Randomized controlled trials are considered the gold standard for evidence in clinical research, but intrinsic limitations — including cost and relatively restrictive eligibility criteria — hinder their feasibility and generalizability for answering many of the pressing questions facing providers caring for children with rheumatic diseases,” Sarah Ringold, MD, MS, from Seattle Children’s Hospital, and colleagues wrote.
In a recent review, Sarah Ringold, MD, and colleagues highlighted the clinical potential of consensus treatment plans to treat pediatric rheumatic diseases and other rare diseases.
The researchers noted that “in contrast to treatment that is provided in the setting of a [randomized controlled trials], treatment following the published CARRA CTPs represents clinical standard of care, given current evidence, with providers choosing the CTP that best fits their usual practice, along with input from the patient and family.”
Researcher review of completed pilot studies on the use of CTPs for systemic onset juvenile idiopathic arthritis, lupus nephritis, moderate juvenile dermatomyositis, polyarticular juvenile idiopathic arthritis, and juvenile localized scleroderma confirmed that CTPs successfully reflected current standards of care among health care providers.
Ringold and colleagues noted that two large-scale comparative effectiveness trials — Start Time Optimization in Polyarticular JIA (STOP-JIA) and First Line Options for Systemic JIA Treatment (FROST) — are also underway, using the CARRA Registry, a nationwide registry of children and young adults with pediatric-onset rheumatic conditions, as a data collection platform.
“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,” Ringold said in a press release. “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.”
Despite the advantages offered through CARRA CTPs, the researchers noted that there were challenges commonly associated with observational research — including selection bias and confounding by indication — as well as limitations found in any prospective study, such as missing data and visits beyond the time frames for follow-up. However, more importantly, Ringold and colleagues found that the lack of validated response measures for many of the rheumatic diseases limited the researchers’ ability to assess the efficacy of different treatment arms.
“The are a unique approach to identifying best treatment strategies for pediatric rheumatologic diseases, employing an iterative observational design that allows for the addition of new treatments and the removal of unused or ineffective strategies, as well as the addition and validation of new outcome and response measures including biomarkers, as available,” Ringold and colleagues wrote.
The researchers added, “The recently established standardized guidelines for CTP development within CARRA will ensure consistent quality and widespread consensus on the use of the resultant plans. It is anticipated that they will provide an efficient and effective framework to compare treatments for pediatric rheumatic diseases, and if successful, the CARRA CTP concept could be applied to other rare diseases even beyond pediatric rheumatology.” – by Bob Stott