Transcript www.bcrdstudy.org

Biologics for Children with
Rheumatic Diseases
An Introduction
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.
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.
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.
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
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.
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
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 followup date.
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
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
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
Contact Details
• If you have any questions about the study please contact:
Dr Kimme Hyrich
Katy Evans
BCRD Chief Investigator
Biologic Studies Group
[email protected]
Assistant Studies Co-ordinator
[email protected]