Research and BSPAR

BSPAR members are very active in lots of different areas of research in paediatric rheumatology. Research activities cover clinical research, basic science and education and clinical trials. Much of this work involves collaboration with between centres in the UK and increasingly internationally with Europe and North America.

There has been great progress in the management for children with rheumatic disease and much of this success comes from strong research teams and collaboration between units. However there are lots of questions that still need to be answered, such as how to choose the very best treatment path for each child, how to decide when it is safe to stop treatment when a child improves, and what are the long term effects of both the medicines and other treatments as well as the diseases themselves.

The Research subcommittee of BSPAR meets regularly, at BSPAR meetings and at the annual BSPAR Research day to share information on research progress and update on results.   

Many of these studies fit into the priority areas that have been identified by the MCRN/ARUK Paediatric Rheumatology Clinical Studies Group: You can find information about the CSG here.

 

Other Information / published articles:

 

Research Subcommittee: List of current active studies

Juvenile Idiopathic Arthritis

Registries and Cohorts and associated clinical and Laboratory Research

BSPAR Etanercept Cohort Study ( Formerly BSPAR Etanercept Registry)

The BSPAR Etanercept registry, established in 2004, collects detailed information on the effectiveness and safety of Etanercept in children with JIA. A parallel group of  children with JIA treated with Methotrexate is also being recruited in order to compare side effects and long term outcomes between the two treatment groups. The study, previously known as the Biologics and New Drugs Register (BNRD), is currently undergoing a transition and is moving to The University of Manchester to sit among the other national rheumatic disease biologic registers, including the Biologics for Children with Rheumatic Diseases study (See below). Recruitment and follow-up continue as normal although changes to the website and data collection are underway.

The lead investigators are: Professor Tauny Southwood (University of Birmingham) and Dr. Kimme Hyrich (University of Manchester)

Website:  https://www.aruk.manchester.ac.uk/bspar/

The study is overseen by an independent steering committee which is chaired by Professor DGI Scott and includes a consumer representative.

NEWS!  The study has now been adopted onto the MCRN Portfolio - Now known as the  BSPAR Etanercept Cohort Study.  See: BSPAR study update dec 2012.pdf BSPAR study update dec 2012.pdf  

 

Biologics for Children with Rheumatic Diseases – The Extended Biologics Study

The BCRD is a second biologics register in the UK which has a focus on biologics other than etanercept and is currently recruiting and following children with JIA starting any non-etanercept biologic. A parallel group of  children with JIA treated with Methotrexate is also being recruited in order to compare side effects and long term outcomes between the two treatment groups. Adopted onto the UK CRN and MCRN Portfolios, recruitment began in June 2010.

Lead investigator Dr KimmeHyrich, University of Manchester.  

Find out more at:

http://www.bcrdstudy.org/

The study is overseen by an independent steering committee which is chaired by Professor R Moots and also includes a consumer representative.

 

Childhood Arthritis Prospective Study (CAPS)

CAPS is a prospective observational study of children presenting with new onset inflammatory arthritis.

The key aims of CAPS are to find out what happens over time to children with all types of arthritis and to identify the relative contributions of socio-demographic, clinical psychological, laboratory and genetic factors and treatment in explaining outcomes.

The lead investigators of this study are Professor Wendy Thompson and Dr Kimme Hyrich, Manchester.

CAPS is undertaken in collaboration with researchers in Manchester, Liverpool, Glasgow, Newcastle and London.  CAPS is funded by ARUK. The study is overseen by an Independent Steering Committee, chaired by Dr Eileen Baildam.

Find out more at:

http://www.medicine.manchester.ac.uk/epidemiology/research/arc/inflammatorymusculoskeletal/ outcomestudies/caps

 

SPARKS CHARMS: The Childhood Arthritis Response to Medication Study

This is a large cohort study of the mechanisms of response to methotrexate and ant TNF medicines in JIA, at genetic, biological, immunological and psychological levels. The study is based in London with collaborating partners in Birmingham and Utrecht.  The Chief investigator is Prof Lucy Wedderburn.

Target recruitment is 800 currently over 600 children have been recruited in London. The study is overseen by an independent steering committee chaired by Dr R Leech including a consumer representative.

Phase 1 of the study was funded by a Lottery (Community fund research) grant in partnership with SPARKS UK, and phase 2 funded by SPARKS UK. This Study is adopted into the UK MCRN portfolio.  

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=3227

 

Validation of prognostic biomarkers predictive of disease outcome in (JIA)

This prospective observational study wishes to validate whether a fingerprint of proteins identified (using Mass Spectrometry techniques) at diagnosis can predict outcome at 2 years.This study has been undertaken in two sites Belfast and St Louis USA. The cohorts have now been followed for 2 years and the results are currently being evaluated.

This study was funded by Arthritis Research UK.

 

Treatment and Management

Prevention and treatment of Steroid-induced Osteopaenia Study in children and adolescents with rheumatic diseases (POPS)

This is a randomised controlled study into the prevention and treatment of bone thinning (osteopaenia) in children and adolescents with rheumatic diseases either commencing or receiving steroids as part of their treatment.

The study compares treatment with a potent form of Vitamin D –One alpha or the bisphosphonate Risedronate (widely used in the treatment of adults) to a placebo group. All three groups receive calcium and vitamin D supplementation.

Eleven sites in the UK have been involved in this multicentre trial. Recruitment is now complete.

Follow-up will be completed in Jan 2013. Results will be available in mid 2013.

The Chief investigator is Dr Madeleine Rooney, Belfast. The study is overseen by an independent steering committee including a consumer representative. Funded by ARUK. The study is adopted onto the UKMCRN portfolio.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=2312

 
 
 

A Prospective Comparative Study of Clinical Examination, Ultrasound and MRI of the Knee in JIA

This study seeks to investigate the correlation between clinical examination, ultrasound examination and the current gold standard MRI in evaluation knee joint disease in Juvenile idiopathic arthritis.

This study was enabled by the R&D office NI.  Lead investigator Dr Madeleine Rooney, Belfast.  The initial findings of this study are currently being reviewed for publication.

 

Tocilizumab (Anti-Il-6R) in active sJIA phase III trial

A 12-week randomized, double blind, placebo-controlled, parallel group, 2-arm study to evaluate the efficacy and safety of tocilizumab in patients with active systemic juvenile idiopathic arthritis (sJIA); with a 92-week single arm open-label extension to examine the long term use of tocilizumab.

The study is now completed and results are awaited

This study involved two centres in the UK. Lead investigator in the UK is Professor Pat Woo. The study was funded by Roche and was adopted onto the UK MCRN portfolio.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=4497

 

Tocilizumab (Anti-Il-6R) in polyarticular  JIA trial

A multi-center international study to evaluate the efficacy and safety of tocilizumab in subjects with active polyarticular-course juvenile idiopathic arthritis; followed by an open-label extension to examine the long term use of Tocilizumab. The lead investigator in the UK is Dr A Ramanan, Bristol.

The study is now closed to recruitment and is in follow up.  The study was adopted into the UKMCRN portfolio see.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=7050

 

Canakinumab in Systemic JIA trial

A randomized, double-blind, placebo controlled, single-dose study to assess the initial efficacy of Canakinumab (ACZ885) with respect to the adapted ACR Paediatric 30 criteria in patients with Systemic Juvenile Idiopathic Arthritis (SJIA) and active systemic manifestations. Lead investigator in the UK is Professor Pat Woo. This trial is now closed and results are currently being analysed.

 

SYCAMORE trial

A randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis.

This multicentre trial is currently recruiting in centres throughout the UK.  The Lead investigators are Dr A Ramanan Bristol and Professor M Beresford Liverpool.  The study has an independent steering committee, including a consumer representative. It has been adopted onto the UKMCRN portfolio.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=10320

CSG Letter to BSPAR re SYCAMORE Nov_12.pdf CSG Letter to BSPAR re SYCAMORE Nov_12.pdf

 

Development of a new method to measure methotrexate levels in children with JIA  & JDM

This is a pilot study to develop a method to measure methotrexate (MTX) product levels in blood in children with arthritis using simple finger prick blood samples known as dried blood spots (DBS). We will determine if this method can be used by children and their parents or guardians to participate effectively in DBS sampling in their own home. The study was undertaken in Belfast, Liverpool and London. Lead investigator is Professor James Mc Elnay, Belfast. This study was funded by Arthritis Research UK.

 

Educational research in paediatric rheumatology

Recent projects include:

A programme of research to improve access to care through education and training. The  focus is Juvenile Idiopathic Arthritis (JIA) albeit much of our work is transferable to other conditions within paediatric rheumatology. This work is based at Newcastle University, lead by Professor Helen Foster and involves collaboration with the Institute of Health and Society (Dr Tim Rapley), Medical Education (Dr Jane Stewart) and clinical colleagues based at Great North Children’s Hospital (Dr Sharmila Jandial). Many of our projects involve colleagues in BSPAR to explore educational strategies to promote access to specialist care. Much of our work is funded by Arthritis Research UK.

Improving musculoskeletal skills in medical students.

The aim is to produce a core list of skills and knowledge relating to musculoskeletal (MSK) problems in children and young people to be acquired by medical students at the time of graduation.  The study is now complete and papers in preparation.

Musculoskeletal problems in children from a primary care perspective. What does the General Practice registrar need to know and how should this be taught?

The aim is to produce a core list of skills and knowledge relating to MSK problems in children and young people to be acquired by trainees in primary care. The study is now complete and papers in preparation.  

Exploring the barriers to care for children with JIA

This project aims to identify, define and understand factors that promote or inhibit referral and understand how these are mediated through different health care and social networks. This work will lead to strategies to facilitate access to care for children with JIA. This work is completed and papers are in progress.

Development of evidence based clinical skills

pGALS (paediatric Gait, Arms, Legs and Spine) and pREMS (paediatric Regional Examination of the Musculoskeletal System) are now published. Free educational resources to facilitate teaching and learning of pGALS are available on the Arthritis Research website and resources for pREMS are in the pipeline.

Developing an educational framework to facilitate optimal care delivery for management of Juvenile Idiopathic Arthritis within clinical networks

In the UK clinical networks in paediatric rheumatology are developing, as a means of providing a solution to the widely prevalent inequalities in optimal care that are well recognised for children and young people with arthritis. This project starts Sept 2012 and aims to provide an educational framework for doctors working within a clinical network.

The effectiveness of spreading a health message using a shareable online video targeted at the social networks of families with a recently diagnosed child with JIA

Online videos and the spread of social networks present a new opportunity to spread health messages, and will be explored as part of this research. This project starts Sept 2012.

 

Dermatomyositis

Registries and Cohorts and associated clinical and Laboratory Research

The Juvenile Dermatomyositis Cohort Biomarker Study and Repository (UK and Ireland)

This UK and Ireland wide study and research network, aims to understand the causes and types of juvenile dermatomyositis (skin and muscle inflammation), as well as improving treatment and outcomes.

The lead investigator is Prof Lucy Wedderburn, London. The study is overseen by an independent steering committee chaired by Dr M Friswell and includes a consumer representative.

The work is funded by grants from the WellcomeTrust, Action Medical UK , Myositis Support group UK.

Find out more at:

http://www.juveniledermatomyositis.org.uk

 

Treatment and Management

PRINTO JDM trial

The Paediatric Rheumatology International Trials Organisation (PRINTO) trial in juvenile dermatomyositis (JDM) is currently taking place in 46 countries involving 185 centres. This trial will run over 5 years

This is a phase III single-blind randomised clinical trial in children with newly diagnosed JDM has 3 arms: 1. prednisone (PDN) versus 2. PDN plus methotrexate (MTX) versus 3. PDN plus cyclosporine A. The study is designed as an effectiveness trial aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity.

In the UK the lead investigator is Dr Clarissa Pilkington, London. This study is funded by PRINTO via an EU grant. For more information see the PRINTO website: http://www.printo.it

 

Juvenile Systemic Lupus Erythematous

Registries and Cohorts and associated clinical and Laboratory Research

Juvenile Systemic Lupus Erythematosus (SLE) Cohort Study and Repository

The UKJSLE study group is a multi-centre, multidisciplinary collaborative network comprising most of the tertiary and most of the tertiary nephrology centres in the UK.

This cohort study and repository aims to define the clinical presentation severity extent prognosis and disease characteristics of JSLE. The lead investigator is Prof Michael Beresford Liverpool. The study has an independent Steering Committee, including a consumer representative. It has been funded by grants from Lupus UK and  ARUK.

The study is adopted onto the UKMCRN Protfolio.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=383HYPERLINK "http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=3836"6

 

Pediatric LUpus Trial Of Belumimab (PLUTO)

 

Juvenile Scleroderma

Quality of Life in Childhood Scleroderma and the Principle Investigator in a Prospective Study of JIA

Lead Investigator is Dr Eileen Baildam, Liverpool.

 

Auto-inflammatory syndromes

Treatment and Management

Cryopyrin Associated Periodic Syndromes (CAPS) trial

An international multicentre open-label extension study to assess efficacy, safety and tolerability of canakinumab and the efficacy and safety of childhood vaccinations in patients with Cryopyrin Associated Periodic Syndromes (CAPS).

The lead UK investigator is Dr Paul Brogan, London.

This industry sponsored (Novartis Pharma) study has been adopted onto the UKMCRN portfolio and is currently awaiting NHS permission.

Find out more at:

http://public.ukcrn.org.uk/Search/StudyDetail.aspx?StudyID=11667