Biologics for Children with Rheumatic Diseases

1. Biologics for Children with Rheumatic Diseases An Introduction

2. Objectives • To document the short and long term outcomes (risk and benefit) in children with JIA exposed to biologic treatments other than etanercept • To collect a DNA/serum sample from each child to use in pharmacogenetic studies. Only one sample per participant will be taken, and this can be collected at any point in the study.

3. Inclusion criteria: Biologics Cohort • Satisfying the revised ILAR classification criteria for Juvenile Idiopathic Arthritis (JIA) at the time of registration or diagnosed with JIA by the consultant rheumatologist • About to start a biologic drug or started within the preceding 6 months • Willingness to give informed consent.

4. Methotrexate Cohort • Purpose: to compare the rates of adverse events in an exposed cohort to those receiving methotrexate (standard first line DMARD) Inclusion criteria: • 1. Children satisfying revised ILAR classification for JIA with active JIA • 2. Starting therapy with methotrexate and have never been prescribed a biologic agent.

5. Study Design  Long term prospective observational cohort study • Participants are followed for at least five years via entry of baseline and follow-up questionnaires onto online database. • Participants are also flagged with the NHS Information Centre for malignancies and death Baseline registration Follow up at six months Follow up at one year Then annual follow up for a further four years Consultant follow-up – 5 years NHS IC flagging - lifelong

6. Registration Process • When decision to treat with biologic/MTX is made, family provided with study information (or posted to home if missed in clinic) • At the next scheduled visit, the family approached again and further info provided. If agree, asked to provide written consent. • Once consented, information will be obtained/extracted from the child’s case notes by the research nurse using a standardised form • All data will be submitted using a secure web-based system.

7. Information collected at baseline includes:  Demographic details  Arthritis details  Disease activity  Current therapy details (including new biologic therapy)  Previous DMARD therapy  Co-morbidities  Details of consent

8. Follow-up • Follow-up completed at 6 months, 12 months and then annually • Data again extracted from the case notes • No additional visits are required and follow-ups will capture all available data up until the follow-up date.

9. Information collected at follow up includes: •  Changes to biologic therapy and dates of infusions •  Changes to DMARD therapy •  Disease activity •  Adverse event and surgery information • Disease status

10. Events of Special Interest Form • If a serious adverse event is reported, further specific information will be obtained via a ESI form. • Serious = death, hospitalisation, significant disability or specified medically significant events

11. MCRN Adoption/Research Support • Adopted by the NIHR National Portfolio and the Medicines for Children Research Network • As part of funding, up to £500/child is available to support data collection, either via the MCRN/LRN or to the rheumatology department directly • Paid as £71.50/completed follow-up, including serious event follow-up data

12. Contact Details • If you have any questions about the study please contact: Dr Kimme Hyrich BCRD Chief Investigator Kimme.hyrich@manchester.ac.uk Katy Evans Biologic Studies Group Assistant Studies Co-ordinator Katy.evans@manchester.ac.uk