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Identifying Trajectories of Disease Activity States in Juvenile Idiopathic Arthritis (JIA) Early After Treatment: Shortening Time to Decision to Change Treatment








Identifying Trajectories of Disease Activity States in Juvenile Idiopathic Arthritis (JIA) Early After Treatment: Shortening Time to Decision to Change Treatment


Lily Lim


Rady Faculty of Health Sciences, University of Manitoba Children's Hospital Research Institute of Manitoba, University of Manitoba


An application has been submitted to the Childhood Arthritis & Rheumatology Research Alliance (CARRA) group (USA) for funding the analysis. This is currently under review, results are to be expected in February/ March 2017.


Lily Lim- none
Sarah Ringold
Salary support from CARRA
Eleanor Pullenayegum
None


01 May 2018


Juvenile arthritis (JA) is the most common childhood rheumatic disease and affects 1 in 1000 children in the USA. Untreated arthritis destroys joints, resulting in pain, discomfort, physical limitations. In recent decades, many new and improved medications- which targets proteins of inflammation specifically- are now available to treat children with arthritis. However, not every child responds the same way to these powerful medicines. A child may respond eventually, or sometimes not at all. Currently, physicians have no means of knowing who will respond aside from waiting. Trials commonly wait at least 4 months for patients to respond. However, waiting exposes children with JA to increased risks of joint damage.

We believe that perhaps the different patterns of treatment response can be found earlier than 4 months. The aims of this study are: 1) To test if we can identify non-responsive or poorly responsive patients using their disease activity patterns earlier than 16 weeks; 2) To forecast different response patterns using basic information such as individual factors and treatment factors. We will do this by using information from four trials in JA. We will calculate the possibilities of reaching different meaningful treatment response states as well as the duration of time spent in each of these states. We will do this using special test methods that allow us to study disease patterns over time; this has not been done before.

By finding out who will likely not do well earlier, doctors can change JA patients' medications earlier, so helping these children improve sooner. By controlling the JA faster, we can then protect these children from joint damage. This also means that trials of new medications can be shorter, saving money for the companies and allowing new medications to get to more patients who need them sooner than now.

We intend to reach doctors looking after JA patients and drug companies that test new JA medications about our study results at annual scientific meetings such as the CARRA scientific meeting, ACR and EULAR. As our team includes several leading opinion leaders and partners (for drug companies) in trials, we will have unique opportunities to introduce drug companies to the use of more efficient study design that our study will identify. We will reach out to communicate our results to the lay public through PARTNERS, a network that links patients, families, the CARRA network, the Arthritis Foundation and a quality improvement network to conduct research that matters to patients and families. We will do this in the form of short briefs for the lay public and encourage them to discuss the results further with their physicians as to the implications for themselves or their children.



[{ "PostingID": 1358, "Title": "ROCHE-WA19977", "Description": "A 24 week randomized, double-blind, placebo-controlled withdrawal trial with a 16 week open-label lead-in phase, and 64 week open-label follow-up, to evaluate the effect on clinical response and the safety of tocilizumab in patients with active polyarticular-course juvenile idiopathic arthritis" }]

Statistical Analysis Plan


The publication citation will be added after the research is published.