Heather F. Burnett

The use of biologic response modifiers in polyarticular-course juvenile idiopathic arthritis: A systematic review

OBJECTIVE To systematically review the clinical efficacy and safety evidence of biologic drugs used to treat the polyarticular category of juvenile idiopathic arthritis (JIA). METHODS The literature was searched between 2000 and September 2012 for randomized controlled trials (RCTs), non-randomized comparative studies, and non-comparative observational cohort studies. The drugs evaluated included etanercept, infliximab, adalimumab, abatacept, anakinra, and ritixumab. Eligible studies included 20 or more patients with JIA, the majority of whom had polyarticular course disease. Outcomes of interest were disease improvement defined by the American College of Rheumatology criteria for Pediatrics, disease flares, rates of inactive disease, remissions, discontinuations, and adverse events (severe and non-severe). RESULTS Thirty-seven studies were included, the majority focused on etanercept. Seven RCTs were identified, including one each for etanercept, infliximab, adalimumab, abatacept, and anakinra, and one each looking at etanercept or infliximab as first-line therapies. There was strong evidence to support the efficacy and safety of biologics over the short-term, but a lack of long-term data for treatments other than etanercept. Several high-quality patient registries confirmed the efficacy and safety of etanercept over the long-term. CONCLUSIONS Current evidence shows that a short-term improvement in treatment response is achieved when patients with polyarticular JIA with an inadequate response to conventional treatment are treated with biologics. Long-term effectiveness data, however, are sparse leaving many questions regarding switches between biologics, handling patients that achieve disease remission, and long-term safety. Study designs other than RCTs may be important in understanding the role of biologics in JIA over the long-term.

Cost-effectiveness analysis of clinic-based chloral hydrate sedation versus general anaesthesia for paediatric ophthalmological procedures.

Background/aims The inability of some children to tolerate detailed eye examinations often necessitates general anaesthesia (GA). The objective was to assess the incremental cost effectiveness of paediatric eye examinations carried out in an outpatient sedation unit compared with GA. Methods An episode of care cost-effectiveness analysis was conducted from a societal perspective. Model inputs were based on a retrospective cross-over cohort of Canadian children aged <7 years who had both an examination under sedation (EUS) and examination under anaesthesia (EUA) within an 8-month period. Costs (

Values and evidence colliding: health technology assessment in child health

CAN), adverse events and number of successful procedures were modelled in a decision analysis with one-way and probabilistic sensitivity analysis. Results The mean cost per patient was

Cost-Effectiveness Analysis of First-Line Treatment With Biologic Agents in Polyarticular Juvenile Idiopathic Arthritis.

406 (95% CI

Parents' Preferences for Drug Treatments in Juvenile Idiopathic Arthritis: A Discrete Choice Experiment

401 to

Task complexity and response certainty in discrete choice experiments: An application to drug treatments for juvenile idiopathic arthritis

411) for EUS and

Parents' willingness to pay for biologic treatments in juvenile idiopathic arthritis.

1135 (95% CI

Using A Crossover Study Design For Early Hospital-Based Health Technology Assessment: The Cost-Effectiveness Of Clinic-Based Chloral Hydrate Sedation Versus General Anaesthesia For Paediatric Ophthalmological Procedures

1125 to

what happens when personalized medicine guidelines don’t agree? A systematic review of clinical guidelines for thiopurine methyltransferase testing for dose selection of thiopurines

1145) for EUA. The mean number of successful procedures per patient was 1.39 (95% CI 1.34 to 1.42) for EUS and 2.06 (95% CI 2.02 to 2.11) for EUA. EUA was

Thiopurine Dosing using Thiopurine Methyltransferase Status: A Systematic Review of Clinical Guidance

729 more costly on average than EUS (95% CI