Investigating the safety and effectiveness of biologic treatments in children with rheumatic disease

Disease - Juvenile idiopathic arthritis

Lead applicant - Professor Kimme Hyrich

Organisation - University of Manchester

Type of grant - Clinical Studies

Status of grant - Active

Amount of the original award - £583,277.60

Start date - 1 October 2015

Reference - 20747

Public Summary

What are the aims of this research?

Juvenile idiopathic arthritis (JIA) is a serious condition of children and young people. Until recently, treatment was limited to methotrexate and steroids which were not always effective and caused side effects in many children. The development of new biologic drugs has significantly improved treatment for many children with arthritis. However, there are concerns that biologics may increase the risk of infections or cancers because they suppress the body's immune response. This study aims to investigate the current use, effectiveness and risk of this group of drugs in children and young people with JIA.

Why is this research important?

Although there have been clinical trials of biologic drugs in JIA, they have been small in size, with limited numbers of patients tracked long-term. Therefore, information on the long-term risk of serious side effects is still lacking. Using the large quantities of information stored in medical records, questions about the safety of these drugs in adults have been answered, and it is important to do the same work in children and young people treated with these drugs.

Information over five years will be collected from the medical records of children and young people with JIA who agree to be involved in this study. Researchers will look for patterns between the treatment types and their effects. Blood and saliva samples collected as part of routine monitoring will also be used to help the investigators investigate whether the treatment benefits or side effects seen in patients are related to their genes.

How will the findings benefit patients?

This research will give families and doctors important information when choosing biologic medications for this patients with JIA so that the safest and most effective treatment(s) can be offered to each child at each stage of their arthritis.