Location of Repository

A national registry for juvenile dermatomyositis and other paediatric idiopathic inflammatory myopathies: 10 years' experience; the Juvenile Dermatomyositis National (UK and Ireland) Cohort Biomarker Study and Repository for Idiopathic Inflammatory Myopathies

By N Martin, P. Krol, S. Smith, K. Murray, C.A. Pilkington, J.E. Davidson, L.R. Wedderburn, J. [JD Research Group] Gardner-Medwin and Juvenile Dermatomyositis Research Group

Abstract

Objectives: The paediatric idiopathic inflammatory myopathies (IIMs) are a group of rare chronic inflammatory disorders of childhood, affecting muscle, skin and other organs. There is a severe lack of evidence base for current treatment protocols in juvenile myositis. The rarity of these conditions means that multicentre collaboration is vital to facilitate studies of pathogenesis, treatment and disease outcomes. We have established a national registry and repository for childhood IIM, which aims to improve knowledge, facilitate research and clinical trials, and ultimately to improve outcomes for these patients. \ud Methods: A UK-wide network of centres and research group was established to contribute to the study. Standardized patient assessment, data collection forms and sample protocols were agreed. The Biobank includes collection of peripheral blood mononuclear cells, serum, genomic DNA and biopsy material. An independent steering committee was established to oversee the use of data/samples. Centre training was provided for patient assessment, data collection and entry. \ud Results: Ten years after inception, the study has recruited 285 children, of which 258 have JDM or juvenile PM; 86% of the cases have contributed the biological samples. Serial sampling linked directly to the clinical database makes this a highly valuable resource. The study has been a platform for 20 sub-studies and attracted considerable funding support. Assessment of children with myositis in contributing centres has changed through participation in this study. \ud Conclusions: This establishment of a multicentre registry and Biobank has facilitated research and contributed to progress in the management of a complex group of rare muscloskeletal conditions

Topics: RJ
Publisher: Oxford University Press
Year: 2011
OAI identifier: oai:eprints.gla.ac.uk:48913
Provided by: Enlighten

Suggested articles

Preview

Citations

  1. Age-dependent inhibition of ectopic calcification: a possible role for fetuin-A and osteopontin in patients with juvenile dermatomyositis with calcinosis. doi
  2. Age-dependent inhibition of ectopic calcification: a possible role for fetuin-A and osteopontin in patients with juvenile dermatomyositis with calcinosis. Rheumatology 2008;47: doi
  3. (2006). An international consensus survey of the diagnostic criteria for juvenile dermatomyositis (JDM). Rheumatology doi
  4. (1998). Chung A et al. Juvenile dermatomyositis at diagnosis: clinical characteristics of 79 children.
  5. (2009). Complete and sustained remission of juvenile dermatomyositis resulting from aggressive treatment. Arthritis Rheum doi
  6. (2008). Effectiveness of infliximab in the treatment of refractory juvenile dermatomyositis with calcinosis. Rheumatology doi
  7. (2007). Failure to over express MHC-CLASS-1 on muscle biopsy in a case of amyopathic juvenile dermatomyositis. Clin Exp Rheumatol
  8. (2007). HLA class II haplotype and autoantibody associations in children with juvenile dermatomyositis and juvenile dermatomyositis-scleroderma overlap. Rheumatology doi
  9. (2009). HLA-DPB1 associations differ between DRB1*03 positive anti-Jo-1 and anti-PMScl antibody positive idiopathic inflammatory myopathy. Rheumatology doi
  10. (2008). Hsp60 in inflamed muscle tissue is the target of regulatory autoreactive T cells in patients with juvenile dermatomyositis. Arthritis Rheum doi
  11. International consensus on a proposed score system for muscle biopsy evaluation in patients with juvenile dermatomyositis: a tool for potential use in clinical trials. doi
  12. (2004). International consensus outcome measures for patients with idiopathic inflammatory myopathies. Development and initial validation of myositis activity and damage indices in patients with adult onset disease. Rheumatology doi
  13. (2004). Intravenous cyclophosphamide pulse therapy in juvenile dermatomyositis. A review of efficacy and safety. Rheumatology doi
  14. Juvenile dermatomyositis: new developments in pathogenesis, assessment and treatment. doi
  15. Long-term outcome and prognostic factors of juvenile dermatomyositis: a multinational, multicenter study of 490 patients. doi
  16. Medium- and long-term functional outcomes in a multicenter cohort of children with juvenile dermatomyositis. Arthritis Rheum 2000;43:541–9. 22 Pachman doi
  17. MHC Class I overexpression on muscles in early juvenile dermatomyositis.
  18. National registry of patients with juvenile idiopathic inflammatory myopathies in Hungary—clinical characteristics and disease course of doi
  19. National registry of patients with juvenile idiopathic inflammatory myopathies in Hungary—clinical characteristics and disease course of 44 patients with juvenile dermatomyositis. doi
  20. (2007). Oropharyngeal dysphagia in juvenile dermatomyositis (JDM): an evaluation of videofluoroscopy swallow study (VFSS) changes in relation to clinical symptoms and objective muscle scores. Rheumatology doi
  21. Over expression of MHC class l heavy chain protein in young skeletal muscle leads to severe myositis: implications for juvenile myositis. doi
  22. patients with juvenile dermatomyositis. doi
  23. (2008). Persistent association of nailfold capillaroscopy changes and skin involvement over thirty-six months with duration of untreated disease in patients with juvenile dermatomyositis. Arthritis Rheum doi
  24. (1975). Polymyositis and dermatomyositis (first of two parts).
  25. (1975). Polymyositis and dermatomyositis (second of two parts).
  26. Predicting the course of juvenile dermatomyositis: significance of early clinical and laboratory features. doi
  27. (2011). Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. doi
  28. Protocols for the initial treatment of moderately severe juvenile dermatomyositis: results of a Children’s Arthritis and Rheumatology Research Alliance Consensus Conference. doi
  29. Protocols for the initial treatment of moderately severe juvenile dermatomyositis: results of a Children’s Arthritis and Rheumatology Research Alliance Consensus Conference. Arthritis Care Res 2010;62:219–25. doi
  30. (2008). Quantification of normal range of inflammatory changes in morphologically normal pediatric muscle. Muscle Nerve doi
  31. Quantitative assessment of MRI T2 relaxation time of thigh muscles in juvenile dermatomyositis. doi
  32. Quantitative assessments of the effects of a single exercise session on muscles in juvenile dermatomyositis. doi
  33. Rheumatol 2009;21:575–80. 9 Rider LG. The heterogeneity of juvenile myositis.
  34. (1995). The incidence of juvenile dermatomyositis: results from a nation-wide study. doi
  35. The Juvenile Dermatomyositis National Registry and Repository (UK and Ireland)—clinical characteristics of children recruited within the first 5 yr. doi
  36. Upregulation of MHC class I in transgenic mice results in reduced force-generating capacity in slow-twitch muscle. doi
  37. Validation and clinical significance of the Childhood Myositis Assessment Scale for assessment of muscle function in the juvenile idiopathic inflammatory myopathies. Arthritis Rheum 2004;50:1595–603. doi
  38. Validation of manual muscle testing and a subset of eight muscles for adult and juvenile idiopathic inflammatory myopathies. Arthritis Care Res 2010;62:465–72. doi
  39. Validation of the Childhood Health Assessment Questionnaire in the juvenile idiopathic myopathies. Juvenile Dermatomyositis Disease Activity Collaborative Study Group. doi

To submit an update or takedown request for this paper, please submit an Update/Correction/Removal Request.