Volume 6 Supplement 1

15thPaediatric Rheumatology European Society (PreS) Congress

Open Access

UK paediatric rheumatology and its clinical trials network

  • MW Beresford1,
  • EM Baildam1,
  • PA Brogan2,
  • HE Foster3,
  • AV Ramanan4,
  • ME Rooney5,
  • UU Shah1,
  • W Thomson7,
  • LR Wedderburn2 and
  • P Woo6
Pediatric Rheumatology20086(Suppl 1):P150

DOI: 10.1186/1546-0096-6-S1-P150

Published: 15 September 2008

Background

There is striking paucity of a rigorous evidence-base for the treatment of paediatric musculoskeletal diseases. The Medicines for Children Research Network (MCRN) improves coordination and quality of clinical trials and related studies across the UK. It has joined in partnership with the Arthritis Research Campaign (arc), the major UK charitable funder of musculoskeletal research, to form the MCRN/arc Paediatric Rheumatology CSG.

Methods

The MCRN has extensive expertise in supporting non-commercial, pharmaceutical-sponsored and investigator-led studies. The CSG is a multi-disciplinary group comprising clinicians, academics, basic scientist, pharmacy, and clinical trials unit support. Through consultation with stakeholders, its remit includes: developing a comprehensive portfolio for clinical trials/related studies covering the spectrum of paediatric rheumatology; ensuring consumer involvement; upholding Good Clinical Practice.

Results

The CSG has developed topic-specific groups (TSGs) outlined in Table 1. Key "themes" across TSGs include: acceptability of intervention; quality of life; health economics; cardiovascular risk; skeletal health; rare diseases; orphan drugs; international collaboration. Key "tools" include: characterised phenotype cohort studies; biomarkers; genotype/phenotype/proteomics; pharmacogenomics; pharmacokinetic studies; mechanism of disease; Phase II and III studies. Strategic development of protocols is underway. Regionally based networks assist with feasibility, site selection, financial, ethical and governance arrangements, staff recruitment, training, participant recruitment and trial monitoring. Table 2 illustrates studies already MCRN adopted and supported in this way.
Table 1

Topic-specifc Groups

Topic Specific Group

Auto Inflammatory Diseases

Bone Health

Juvenile Dermatomyositis

Juvenile idiopathic arthritis (JIA)

Juvenile-onset SLE

Rare disorders (Initial focus on Scleroderma)

Uveitis in JIA

Vasculitis

Formulations & Pharmacy

Table 2

Studies recently adopted by MCRN

Acronym

Study Title

POPS Study

Prevention and treatment of steriod-induced osteopaenia in children and adolescents with rheumatic diseases

UK JSLE Cohort Study and Repository

UK Juvenile-onset Systemic Lupus Erythematosis Cohort Study epository: Clinical Characteristics and Immunopathology of Juvenile-onset SLE

MYCYC

A randomised clinical trial of mycophenolate mofetil versus cyclophosphamide for remission induction in ANCA associated vasculitis

SPARKS CHARMS

A study of the immunological and genetic mechanisms of response, and psychological response to, standard disease management in juvenile idiopathic arthritis (JIA)

Conclusion

An integrated national research network provides an excellent infrastructure to collaborate closely with international trial networks (e.g. CARRA, PRINTO) while fostering a unique contribution to the transformation of clinical care of children.

Authors’ Affiliations

(1)
Royal Liverpool Children's Trust
(2)
Institute of Child Health
(3)
Newcastle Hospitals NHS Trust
(4)
Bristol Royal Hospital for Children & Royal National Hospital for Rheumatic Diseases
(5)
Queen's University Belfast
(6)
University College London
(7)
University of Manchester

Copyright

© Beresford et al; licensee BioMed Central Ltd. 2008

This article is published under license to BioMed Central Ltd.

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