The goal of growth hormone (GH) treatment in a short child is to attain a fast catch-up growth toward the target height (TH) standard deviation score (SDS), followed by a maintenance phase, a proper pubertal height gain, and an adult height close to TH. The short-term response variable of GH treatment, first-year height velocity (HV) (cm/year or change in height SDS), can either be compared with GH response charts for diagnosis, age and gender, or with predicted HV based on prediction models. Three types of prediction models have been described: the Kabi International Growth Hormone Study models, the Gothenburg models and the Cologne model. With these models, 50-80% of the variance could be explained. When used prospectively, individualized dosing reduces the variation in growth response in comparison with a fixed dose per body weight. Insulin-like growth factor-I-based dose titration also led to a decrease in the variation. It is uncertain whether adding biochemical, genetic or proteomic markers may improve the accuracy of the prediction. Prediction models may lead to a more evidence-based approach to determine the GH dose regimen and may reduce the drug costs for GH treatment. There is a need for user-friendly software programs to make prediction models easily available in the clinic.

BackgroundWarfarin, an inexpensive drug that has been available for over half a century, has been the mainstay of anticoagulant therapy for stroke prevention in patients with atrial fibrillation (AF). Recently, rivaroxaban, a novel oral anticoagulant (NOAC) which offers some distinct advantages over warfarin, the standard of care in a world without NOACs, has been introduced and is now recommended by international guidelines.ObjectiveThe aim of this study was to evaluate, from a Belgian healthcare payer perspective, the cost-effectiveness of rivaroxaban versus use of warfarin for the treatment of patients with non-valvular AF at moderate to high risk.MethodsA Markov model was designed and populated with local cost estimates, safety-on-treatment clinical results from the pivotal phase III ROCKET AF trial and utility values obtained from the literature.ResultsRivaroxaban treatment was associated with fewer ischemic strokes and systemic embolisms (0.308 vs. 0.321 events), intracranial bleeds (0.048 vs. 0.063), and myocardial infarctions (0.082 vs. 0.095) per patient compared with warfarin. Over a lifetime time horizon, rivaroxaban led to a reduction of 0.042 life-threatening events per patient, and increases of 0.111 life-years and 0.094 quality-adjusted life-years (QALYs) versus warfarin treatment. This resulted in an incremental cost-effectiveness ratio of €8,809 per QALY or €7,493 per life-year gained. These results are based on valuated data from 2010. Sensitivity analysis indicated that these results were robust and that rivaroxaban is cost-effective compared with warfarin in 87 % of cases should a willingness-to-pay threshold of €35,000/QALY gained be considered.ConclusionsThe present analysis suggests that rivaroxaban is a cost-effective alternative to warfarin therapy for the prevention of stroke in patients with AF in the Belgian healthcare setting.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-013-0087-9) contains supplementary material, which is available to authorized users.

No abstract

Costs and effects of micafungin compare to those of caspofungin in the treatment of systemic Candida infections in the UK. The results indicate that micafungin is cost-effective compared to caspofungin, although the difference was not found to be significant.

BackgroundResponse to growth hormone (GH) therapy may vary between individual patients. Therefore the use of GH in children should be closely monitored to avoid over, under, or ineffective treatment regimens. The treatment response can be evaluated using growth prediction models. In an effort to improve the accuracy of these prediction models, Ranke et al. (J Clin Endocrinol Metab 95(3):1229–37) proposed a novel ‘data-driven’ approach based on a quantitative analysis of a large cohort of patients from the Pfizer International Growth Database (KIGS) treated with Genotropin (human growth hormone). This model allows physicians to predict and evaluate the level of growth response and responsiveness for their patients so they can adapt treatment accordingly. By comparing the actually observed and the predicted growth response the ability of an individual to respond to GH (responsiveness) can be estimated and further treatment can be adapted accordinglyObjectiveTo determine the potential population level reduction in the amount of GH used and impact on height outcome of using this data-driven approach to guide treatment decisions, compared to conventional, ‘experience-based’ GH treatment in prepubertal patients with growth hormone deficiency (GHD) or Turner syndrome (TS).MethodsA model was developed to study the height outcome and the total amount of GH used in the presence or absence of data-driven treatment decisions. The proportion of patients for whom height outcome could be improved or GH use could be reduced (i.e. for low compliance, high or low responder) was estimated using the KIGS cohort. The analysis assumed that this segmentation allows physicians to tailor dosage to the individual patient’s needs or even to discontinue therapy when it is not effective. The analysis used a 4-year time horizon, with Germany as an example country, but results are extendable to other countries. Only the total amount of GH used was included, and effects were defined as the height outcome after 4 years.ResultsThe analysis estimated that an evidence-driven approach may reduce the total amount of GH utilized by 7.0 % over 4 years for the treatment of short stature in prepubertal patients with GHD and TS in Germany. Despite the reduction in drug use the average growth outcomes remained unaffected with the new treatment approach. Univariate and probabilistic sensitivity analyses showed that the results are robust.ConclusionsOur analysis showed that using a data-driven approach to guide treatment decisions for children with GHD or TS is estimated to result in efficiencies in the amount of GH used, without reducing the average growth in the population.