Research and publications from the Rheumatology department

Research by the Rheumatology department at Great Ormond Street Hospital (GOSH) focuses on childhood arthritis, dermatomyositis and vasculitis.

Many of these complex diseases are still hard to treat and we have few ways to tell parents how severe or mild their child’s illness will be.


Our clinical and research teams collaborate closely with one another and are constantly improving the treatment that we provide as well as the patients' experiences. All our work, from basic science right through to patient-centred work, is informed by patient need and the need for better ways to understand, diagnose and treat these conditions.

Examples of our work that have direct benefit to patients include our leading role in drug trials of new medicines, for example for arthritis and the rare fever syndromes of childhood. These are medications such as Anakinra and Toculizumab. Some of the key basic science work that led up to the use of Toculizumab in childhood arthritis was carried out by our team at GOSH/University College London.

A major effort has been our study to understand the mechanisms of response (or failure) to first line drugs used to treat arthritis, like Methotrexate (MTX) and anti-TNF medicines. Our CHARMS study (Childhood Arthritis Response to Medication Study) has collected the experiences of more than 250 families and built a family website, now under testing, to help parents manage their life with a child with arthritis.

In parallel we have undertaken the largest study of genetics of the response to MTX to help us to understand what controls children’s response to these drugs. We are the host centre for the UK-wide Cohort study of Juvenile Dermatomyositis (JDM) which is an invaluable resource both for researchers and also families with children with JDM.

The research expertise in the Rheumatology department is wide, with active research programmes on arthritis, myositis, vasculitis, juvenile systemic lupus erythematosus (JSLE) and hypermobility.

Our current work ranges from:

  • Basic science laboratory work (which includes immunology, genetics and vascular biology).

  • Translational work - such as finding out ways to measure the activity of these rare diseases, including developing biomarkers, or predict how a child’s disease will develop.

  • Our family-centred work such as our juvenile idiopathic arthritis (JIA) family website project.

Current active research programmes:

Immunology of childhood arthritis

We are working to understand what has changed in a child’s immune system when arthritis develops and how we can help the immune system to restore its balance to make arthritis go into remission

Understanding response to medication in childhood arthritis

The large CHARMS includes more than 800 children and aims to understand immunological, genetic and psychological factors that control response to drugs in JIA.

Vascular biology of vasculitis

The key aims of the vasculitis research are to further understand the pathogenesis of vasculitis in the young, develop novel diagnostic and treatments and systematic documentation of different types of childhood vasculitis.

Muscle biology and myositis programme

We are custodians of the UK-wide JDM Cohort study, now the largest of its kind in the world, which supports many clinical/research studies and now has its own Juvenile Dermatomyositis Research Group website.

Clinical trials

We are involved in many clinical trials including POPS (Prevention and treatment of steroid induced OsteoPenia Study) and SYCAMORE, a new trial of Adalimumab in uveitis. Many of our studies are carried out in the GOSH Clinical Research Facility and are supported by the Medicines for Children Research Network. We play an active role in the UK Arthritis Research Clinical Studies Group for Paediatric Rheumatology.


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Moncrieffe H, Hinks A, Ursu S, Kassoumeri L, Etheridge A, Hubank M, Martin P, Weiler T, Glass DN, Thompson SD, Thomson W, Wedderburn LR (2010) Generation of novel pharmacogenomic candidates in response to methotrexate in juvenile idiopathic arthritis: correlation between gene expression and genotype. Pharmacogenet Genomics 20 (11): 665-76

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Ruperto N, Pistorio A, Ravelli A, Rider LG, Pilkington C, Oliveira S, Wulffraat N, Espada G, Garay S, Cuttica R, Hofer M, Quartier P, Melo-Gomes J, Reed AM, Wierzbowska M, Feldman BM, Harjacek M, Huppertz HI, Nielsen S, Flato B, Lahdenne P, Michels H, Murray KJ, Punaro L, Rennebohm R, Russo R, Balogh Z, Rooney M, Pachman LM, Wallace C, Hashkes P, Lovell DJ, Giannini EH, Gare BA, Martini A, Paediatric Rheumatology International Trials Organisation (PRINTO), Pediatric Rheumatology Collaborative Study Group (PRCSG) (2010) The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis. Arthritis Care Res (Hoboken) 62 (11): 1533-41

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Reference 30
Stringer E, Bohnsack J, Bowyer SL, Griffin TA, Huber AM, Lang B, Lindsley CB, Ota S, Pilkington C, Reed AM, Scuccimarri R, Feldman BM (2010) Treatment approaches to juvenile dermatomyositis (JDM) across North America: The Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM Treatment Survey. J Rheumatol 37 (9): 1953-61

Reference 31
Visvanathan S, Wagner C, Marini JC, Lovell DJ, Martini A, Petty R, Cuttica R, Woo P, Espada G, Gattorno M, Apaz MT, Baildam E, Fasth A, Gerloni V, Lahdenne P, Quartier P, Saurenmann R, Travers S, Mendelsohn A, Xu S, Giannini EH, Ruperto N, the Paediatric Rheumatology INternational Trials Organization (PRINTO), the Pediatric Rheumatology Collaborative Study Group (PRCSG) (2010) The effect of infliximab plus methotrexate on the modulation of inflammatory disease markers in juvenile idiopathic arthritis: analyses from a randomized, placebo-controlled trial. Pediatr Rheumatol Online J 8 (1): 24

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Reference 34
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Reference 36
Abinun M, Flood TJ, Cant AJ, Veys P, Gennery AR, Foster HE, Friswell M, Baildam E, Davidson J, Southwood TR, Livermore P, Wedderburn LR (2009) Autologous T cell depleted haematopoietic stem cell transplantation in children with severe juvenile idiopathic arthritis in the UK (2000-2007). Mol Immunol 47 (1): 46-51

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