First data show tocilizumab (RoActemra®) can significantly improve symptoms in children with systemic juvenile idiopathic arthritis
More than a third of children given tocilizumab (RoActemra®), the only treatment to target the interleukin-6 (IL-6) pathway, saw the symptoms of their systemic juvenile idiopathic arthritis (systemic JIA), also known as Still’s disease, improve by 90% at 12 weeks; just 5% of those given placebo saw the same improvement1 (JIA ACR90)*. The most serious type of childhood arthritis, systemic JIA – which is often resistant to standard JIA treatments2 – affects up to 2,500 children in the UK. 3,4,5 Tocilizumab is currently used to treat adult rheumatoid arthritis, but data presented today at the European League Against Rheumatism (EULAR) annual meeting in Rome, suggest that it may also have an important part to play in tackling childhood arthritis.
Prof Patricia Woo, Professor of Paediatric Rheumatology at Great Ormond Street Hospital for Children NHS Trust (GOSH) commented: “This is a major advance for these young people. Children with systemic JIA remain a group with a high unmet medical need, and those at the more severe end of the disease spectrum have significant morbidity throughout their lives as well as a higher rate of mortality than other types of JIA. As a notoriously difficult disease to treat, we need new treatments like tocilizumab to help combat systemic JIA.”
“This condition can seriously disrupt the child's development and the day to day life of the family. Without effective treatment up to half of the affected children develop chronic and persistent arthritis and a majority of these children can be left with significant disability.”
As one type of JIA, ‘systemic’ juvenile arthritis means that along with joint inflammation the disease typically begins with symptoms of illness throughout the body, such as high fever accompanied by a salmon coloured rash, gland swelling, and the a risk of affecting a child’s internal organs such as the tissue that covers the heart, liver or spleen.
The cause of systemic JIA is unknown but studies have shown that patients have high levels of IL-6 in their blood and joints6 and that IL-6 contributes to all the major features of systemic JIA including joint inflammation, joint damage, fever, anaemia, growth impairment and osteoporosis.6 By blocking the IL-6 pathway, tocilizumab is shown through these study results to have an important impact on systemic JIA.
In the TENDER trial, in addition to the 90% improvement seen in over a third of children, significantly more patients in the tocilizumab arm achieved JIA ACR30 response plus absence of fever at week 12 as compared to placebo (85% vs 24%, p<0.0001).1 These results were just 12 weeks into treatment and the trial – involving 112 children – will continue over a five year period to evaluate the long term effects of treatment with tocilizumab.1
No patients given placebo and only three patients treated with tocilizumab experienced serious adverse events. These were angioedema and hives (urticaria), varicella (chicken pox) and bacterial arthritis, all of which resolved without lasting or recurring effects.1
Tocilizumab is not yet licensed for the treatment of systemic JIA however a licence application will be submitted toward the end of 2010.
*About JIA ACR Scores:
JIA ACR90 improvement is defined as three of any six core outcome variables improved by at least 90% from the baseline assessments, with no more than one of the remaining variables worsened by more than 30%.
The JIA Core Outcome Variables consist of:
- Physician Global Assessment of Disease Activity (100 mm VAS)
- Parent/patient global assessment of overall well-being (100 mm VAS)
- Number of Joints with Active Arthritis
- Number of Joints with Limitation of Movement
- Functional Ability (Childhood Health Assessment Questionnaire)s
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 De Benedetti F et al. Efficacy and safety of tocilizumab in patients with systemic Juvenile Idiopathic Arthritis (sJIA): 12-week data from the phase III TENDER trial. Abstract and oral presentation at EULAR 2010
2 M W Beresford, E M Baildam. New advances in the management of juvenile idiopathic arthritis—2: The era of biologicals. Arch Dis Child Educ Pract Ed 2009 94: 151-156
3 National statistics – The health of children and young people. Office for National Statistics, March 2004. http://www.statistics.gov.uk/children/downloads/child_pop.pdf
. Accessed June 2010
. Accessed June 2010
5 Woo P. Systemic juvenile rheumatoid arthritis: diagnosis, management, and outcome. Nature Clinical Practice: Rheumatology. 2006. 2:1
6 De Benedetti F. Inflammatory cytokines in the pathogenesis and treatment of systemic juvenile idiopathic arthritis – Basic science for the clinician. Pediatric Rheumatology Online Journal 2005. Vol 3:2
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