Cost-effectiveness analysis of top-down versus step-up strategies in patients with newly diagnosed active luminal Crohn's disease.

BACKGROUND: Top-down (TD) strategy with frontline infliximab proved to be more effective than the traditional step-up (SU) approach in newly diagnosed luminal moderate-to-severe CD patients. However, the considerable cost of infliximab calls its universal use as frontline treatment into question. The aim of this study is to evaluate the cost-effectiveness of the TD approach using a Markov decision model. METHODS: Four states were modelled, namely step 1, step 2, step 3 and death. The first three steps were in TD infliximab induction plus azathioprine, infliximab rechallenge plus azathioprine and steroids plus azathioprine, and in SU steroid induction, azathioprine plus steroid rechallenge and infliximab plus azathioprine. Each health state lasted 1 month. The time horizon of the model was 5 years. Transition probabilities and quality of life were estimated from a randomised trial. First- and second-order sensitivity analyses were done to test the robustness of the results. RESULTS: At baseline analysis, TD improved quality-adjusted life expectancy from 3.76 to 3.90 quality-adjusted life years (QALYs), that is, 0.14 QALYs, while allowing a saving of euro 773, proving dominant when compared to SU. TD was cost-saving in 66% of the Monte Carlo simulations and cost

Response criteria for myelofibrosis with myeloid metaplasia: results of an initiative of the European Myelofibrosis Network (EUMNET).

The European Myelofibrosis Network (EUMNET), a European research network on myelofibrosis with myeloid metaplasia (MMM), has developed a definition of response for the disease by using clinicohematologic, histologic, and cytogenetic criteria. A core set of 5 clinicohematologic criteria was selected out of 9 candidates on the basis of their sensitivity to change measured in 196 patients treated either during clinical trials or routine clinical practice. A consensus panel of 16 international experts was convened and asked to score the level of response in 104 patient profiles as major, moderate, minor, or no response according to changes of the clinicohematologic criteria. Using the experts' consensus as the gold standard, the performance of 100 possible definitions of response was evaluated. Criteria for major or moderate clinicohematologic response were determined to be changes in hemoglobin (Hb) and spleen size and the presence of constitutional symptoms, while changes in platelet count and white blood cell (WBC) count served as complementary criteria and were of value for defining minor responses. A histologic response was defined by changes in bone marrow fibrosis and cellularity grades. The combined use of these response definitions should help standardize the design and reporting of future clinical studies in MMM.