INTRODUCTION: Treatment of severe atopic dermatitis in children with systemic cyclosporine costs €7.000/y. Improvement is seen in about 50% of patients. Objective diagnostic tools for effective use of cyclosporine are lacking, resulting in over- and undertreatment.
RESEARCH QUESTION: Does stratification of children with eczema/ atopic dermatitis (AD) on the diagnostic NMF biomarker tool improve efficient use of cyclosporine?
HYPOTHESIS: This project hypothesizes that cyclosporine will be more effective in children with moderate AD and low NMF biomarker and less effective in children with severe AD and normal NMF biomarker.
STUDY DESIGN: The study is a partially blinded RCT with two equally sized parallel groups.
STUDY POPULATION: Children 2-18 y, with moderate to severe eczema.
INTERVENTION: Cyclosporine for 3 months according to stratification on NMF. Control group: cyclosporine treatment for 3 months according to usual care.
OUTCOME MEASURES: Disease severity over 6 months period, cost of cyclosporine use, QoL
SAMPLE SIZE: In each arm: 86 patients. Sampling ratio of 1:3 results in 85% power to detect a difference of 0.5 standard deviations. Sample size is feasible.
DATA-ANALYSIS: Linear and logistic regression adjusted for patient characteristics. Effect estimates presented as beta’s and odds ratios with 95% confidence intervals.
ECONOMIC EVALUATION: from societal perspective, timeframe of 6 months. Health care costs measured in accordance with economic guidelines. Primary effect measure for economic evaluation is quality of life by 3 and 6 month EuroQol-5D. The cost-effectiveness assessed by calculating incremental cost-effectiveness.