UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Diseases
Childhood arthritis describes a group of diseases that commonly present as pain, stiffness and swelling of the joints and other supporting structures of the body. Paediatric patients who are diagnosed with arthritis are often faced with debilitating symptoms that can dramatically decrease quality of life. This can also lead to lifelong chronic rheumatic disease. As such, providing timely and effective interventions is crucial for improving short and long term health outcomes for children living with this disease.
Below are some crucial facts about childhood arthritis and biologic therapies:
- Juvenile idiopathic arthritis (JIA) is the most common immune mediated inflammatory musculoskeletal disease affecting more than 20,000 children in Canada and the Netherlands
- Children that are at high-risk of permanent joint damage due to their arthritis often require advanced therapeutic agents called biologics
- One in three paediatric and adult patients currently receive biologic therapies for arthritis without any treatment end-date
- Biologic therapies can dramatically improve disease outcomes, but also have adverse side effects associated with this course of treatment
- The use of biologic therapies imposes a substantial economic burden on the patients, their families and society
How can we improve treatment approaches and outcomes for children with arthritis?
One of the challenges when it comes to treating childhood arthritis is providing the correct treatment at the opportune time. While advanced biologic therapies are frequently used and can be highly effective, we are currently unable to accurately predict which children should start biologic therapies and which can discontinue treatment without having disease flares. The overarching goal of the UCAN CAN-DU study is to address this gap in treatment approaches and support translational research for all children with juvenile arthritis.
- Recent work by our team has established that patients and treatment response can be classified by integrating biologic and clinical data. UCAN CAN-DU will have a diverse approach with a focus on the development of new concepts for standardized evaluation of:
- Disease activity outcomes
- Novel eHealth tools for clinicians and patients
- Real-time integration of individual biological profiles
The commitment of all paediatric rheumatology care providers across Canada and the Netherlands to the UCAN CAN-DU mission of integrating innovative precision medicine strategies into practice will forever transform the care of children with arthritis in our countries and beyond.
Our network research program will have a multi-pronged approach with the following interacting research themes:
- Early identification of children at high-risk of poor outcome
- Identify the best treatments to improve outcomes and quality of life in affected children
- Define the optimal ways to manage affected children
- Develop a sustainable transdisciplinary network
UCAN CAN-DU has three main activities:
- Biomarker program
- The biomarker program will have two complementary arms. A retrospective discovery phase and a prospective validation phase, each with three cohorts
- There are three aims for the biomarker program:
- To develop a disease classification system that could categorize children with arthritis based on their biology and clinical data
- To develop a clinical tool that could be used to predict response to therapy
- To identify risk of relapse after discontinuation of therapy through the use of our predictive tool
- eHealth
- The eHealth arm of the study will focus on electronic, web based health tools for clinical and patient reported data capture. These have been developed with our partners at The Centre of Global eHealth Innovations at the University Health Network in Toronto
- The web based tools include platforms for both clinicians and patients
- Health Economics
- This arm of the study will focus on the complexity and dynamic nature of treatment pathways for patients with JIA, including patient and clinician preferences for biomarker-based testing information
- The health economic study will look at defining integrated multi-dimensional disease trajectories and their associated health care resource use and costs
Cohort 1 – Biologic Basis of JIA: The objectives for this group are to help researchers look at childhood arthritis and determine which management approach is best for each individual child.
- To be eligible for this group:
- Participant must be ≤ 18 years of age at time of study enrollment
- Participant is suspected to have JIA
- Participant has not received any treatment other than non-steroidal anti-inflammatory drugs, such as acetaminophen
Cohort 2 – START Biologics: The objectives for this group are to help researchers develop a tool to predict response to therapy.
- To be eligible for this group:
- Participant ≤ 18 years of age at time of study enrollment
- Participant has been diagnosed with JIA and your arthritis is active
- Participant will be starting, re-starting or switching to a new biologic therapy
Cohort 3 – STOP Biologics: The objectives for this group are to help researchers develop a tool to predict who will remain in remission after discontinuing therapy.
- To be eligible for this group:
- Participant ≤ 18 years of age at time of study enrollment
- Participant has been diagnosed with JIA and your arthritis is inactive
- Participant will be stopping or tapering biologic therapy
Cohort 4 – Extreme Phenotypes: The objective for this group is new gene discovery and drug target identification.
- To be eligible for this group:
- There is high suspicion of genetic contribution
- Participants are severely affected with difficult to control arthritis or systemic disease
- There is unexplained systemic inflammation with arthritis/arthralgia as a part of manifestations