Maxime Verhoeven
13 General introduction strategies in (early) RA patients , and the second part to explore whether monitoring of disease activity, a prerequisite for implementation of treat-to-target treatment strategy, can be performed using the HandScan without the need for a healthcare professional . Outline PART I: Evaluation of (cost-)effectiveness of DMARD therapy approaches in treat-to-target strategies in (early) RA patients. In chapter 2 , an overview of the literature is given of the effectiveness of intensive treatment strategies aiming at remission compared to that of standard of care of MTX therapy with or without GC bridging in early RA. Chapter 3 reports the effectiveness and safety of initiating TCZ, with or without MTX, versus those of initiating MTX with a GC (long-term use of low-moderate dose, i.e., prednisone 10mg/day) within a treat-to- target strategy in early RA, analysing individual patient data of two clinical trials. Since it is known that some patients respond insufficiently to first-line treatment with MTX or are intolerant to MTX, and because specifically in this subgroup, a more intensive initial DMARD strategy could be of value, we performed an external validation of a prediction model for MTX non-response, described in chapter 4 . Due to the in general more effective treatment nowadays, joint damage in RA is typically limited, making it more difficult to show differences on this outcome between therapies within a single trial. By using individual patient data of several randomized controlled trials, chapter 5 reports the difference in effect of preventing radiographic progression of TCZ-monotherapy compared to TCZ+MTX, and identifies modifiers (i.e., baseline joint damage, disease activity, disease duration) of this effect. The ultimate impact of treatment of a chronic disease like RA only becomes apparent over the longer-term. Therefore, the longer-term effectiveness and safety outcomes of patients initially treated with TCZ, MTX or their combination is reported in chapter 6, and the cost effectiveness over this period is reported in chapter 7. An editorial about different methodologies and interpretations of costing analyses of bDMARD strategies, in response to a paper on the budget impact of introducing an etanercept biosimilar, is provided in chapter 8. PART II: Monitoring disease activity in treat-to-target strategies: exploring usefulness of the HandScan. In chapter 9 , the utility of the HandScan in monitoring disease activity and prediction of clinical response in RA patients treated within a treat-to-target strategy was assessed in an exploratory study. In extension of chapter 9, chapter 10 describes the development 1
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