Authors' objectives:

"The objectives were as follows: to review outcome measures in children with JIA and low BMD and/or fragility fractures to review evidence for effectiveness and safety of bisphosphonates and calcium and/or vitamin D in these children to assess long-term bone health in adults with JIA to review costs of treating children with JIA and low BMD and/or fragility fractures to evaluate the cost of treating JIA." (from executive summary)

Authors' recommendations: Assessment of outcome measures relating to bone health in children with JIA BMD, adjusted for size, should be assessed as the primary outcome in studies of bone health in children with JIA. QCT could be used where equipment is available as it offers the advantage of measuring volumetric density. Other outcome measures may also be useful but further data are needed to establish their role. Systematic review of effectiveness of bisphosphonate and calcium and/or vitamin D Bisphosphonates are a promising treatment for osteoporosis in children with JIA, but the quality of the current evidence is poor. Better studies are needed to assess more clearly their role and permit licensing of these agents for treatment of children. In particular, longer-term studies are needed to evaluate the effectiveness and safety of this treatment into adulthood. The accurate assessment of outcome is crucial. There are still uncertainties about the use of bisphosphonates in children, including whether the positive effects of treatment continue over time, the length of treatment and the maximal bone mass gain that can be achieved. In particular, longer-term studies are needed to evaluate the effectiveness and safety of this treatment into adulthood. Long-term bone health in JIA Adults with JIA may have persistent low BMD compared with an otherwise healthy population together with an increased risk of fracture. Systematic review of costs for managing children with JIA and low BMD or fragility fractures There are no studies evaluating the costs of treating children with JIA and low BMD and/or fragility fractures. There are few data evaluating the costs of treating JIA in general. Assessment of cost of treatment for JIA In the first 12 months after diagnosis, children with all JIA disease subtypes consume large, but highly variable, quantities of health service resources. The largest component of health provider costs was consultant rheumatology appointments. The right-skewed distribution of costs suggests that a few high cost outliers increased the mean costs for the group overall, and within individual disease subgroups. Data from a larger cohort, over a longer period, are required to substantiate these results further. Implications for healthcare All methods of assessing outcome have limitations; DXA is the current most practical measure but results in children must be interpreted with care. Fractures would be the ideal outcome measure but a study with this end-point would require large numbers of patients and long-term follow up. However, fracture data could be routinely collected for local and other registers. Bisphosphonates seem to be effective in the management of children with JIA but the evidence is limited. Few children with JIA have been treated with bisphosphonates; the studies include case series and case reports and there are no true controlled studies, the studies are heterogeneic in design (different subtypes of JIA are included in different studies, children with other connective tissue disease also included, different bisphosphonates and varied doses and routes of administration are used and durations of treatment and follow-up times vary), together with poor assessment of outcome measures and varied methods of reporting results. There are still many unanswered questions about bisphosphonates' use, including the optimum dose and frequency of administration and length of treatment. The maximal BMD gain that can be achieved is not known. It is not clear whether the positive effects of treatment continue over time. There is limited evidence on the use of calcium and/or vitamin D. Assessment of outcome was poor in all studies. The problems of poor bone health persist into adulthood; adults with JIA have an increased numbers of fractures compared with expected values in otherwise healthy adults. Recommendations for research Areas for further research are as follows: The arc has initiated an RCT of bisphosphonates and 1-a-hydroxycholecalciferol (hydroxylated derivative of vitamin D) in children with JIA. This study should address some of the research issues raised in this report. Longer-term follow-up of studies with bisphosphonates and calcium and/or vitamin D is needed to determine the longer-term effect of treatment on both bone mass and fracture risk, and also safety. A cohort study of children with newly diagnosed JIA should examine the effects of disease and current management approaches on bone health in these children. Large prospective studies are needed to determine the predictors of bone mass and fractures in adults with JIA. Longitudinal studies of DXA should be conducted to consider whether bone mass measured by DXA predicts bone mass and fracture risk in adults. Most current evidence relates to the use of DXA for assessing bone health in children. Further evaluation of other quantitative imaging techniques is required. More studies are needed looking at the performance of biochemical markers in children with JIA. The effect of treatment on markers in children with JIA should be assessed. An HRQoL measure should be validated specifically for use in children with low trauma fractures. Future studies should examine costs of management of bone health in JIA in both the short and medium term. A cost-effectiveness or cost;utility evaluation could be incorporated. Future studies examining bone health in children should have an economic component

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.hta.ac.uk/1403

Year Published: 2008

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Health Technology Assessment programme

Contact Address: NIHR Journals Library, National Institute for Health and Care Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK

Contact Name: journals.library@nihr.ac.uk

Contact Email: journals.library@nihr.ac.uk

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