TPS4685 Background: Since the identification of Von Hippel Lindau (VHL) gene mutations as a critical step in the development of most Clear Cell Renal Cell Carcinomas (CCRCC), neoangionesis inhibition has become a cornerstone in their treatment. Despite the proven efficacy of antiangiogenic drugs, most patients will not achieve partial response by RECIST criteria. Thus, to accurately determine tumor progression is a challenging question incompletely answered by traditional radiological assessment. In recent years CECs counts have been proposed as surrogate biomarkers of angiogenesis that could be used for monitoring tumor evolution while on targeted therapies. We aim to figure out if CECs elevations could anticipate radiological progression in CCRCC. Methods: An observational prospective study is being performed in 10 institutions members of the Spanish Oncology Genitourinary group (SOGUG). Patients older than 18 years with histologically proven CCRCC on first line treatment with any targeted drug who have not progressed after 3 months of therapy are considered elegible. Recruitment begun on August the 1st 2011 and 15 of the 75 scheduled patients have been included so far. CECs are periodically collected in peripheral blood every 6 weeks for 15 months or radiological tumor progression, whatever occurs first. Median CEC values will be calculated and stated by descriptive statistics (Cellsearch, VERIDEX). To explore the evolution of CECs counts along treatment a linear model will be constructed. An association among CECs counts changes and time to progression will be analyzed with Cox model.
significant benefits of prophylaxis treatment with FIX versus OD treatment. However, these benefits are associated with an increase in FIX consumption and considerable costs, and most of Chinese HB pediatric patients are on OD treatment. The objective of this study was to assess the cost effectiveness of prophylaxis treatment compared with OD treatment in HB children without inhibitors from the payer perspectives. Methods: We developed a Markov model to perform a cost-utility analysis that comparing prophylaxis versus OD treatment for HB. The model ran over a 16-year time horizon using 1 yearly cycle. Transition probabilities and utility weights were estimated using published studies, and cost were gathered from HB patients with medical insurance in Beijing Children hospital, including drug cost, utilization of both in-and outpatient. Both one-way and probabilistic sensitivity analysis were performed to assess the robustness of results. Results: The model projected prophylaxis had an incremental gain of 1.62 quality-adjusted life years of with an incremental cost of $13,943.The incremental cost per quality-adjusted life years gained for individuals with haemophilia B receiving prophylaxis were $8611.26 compared with OD arm, which is under the willingness-to-pay threshold(3 times of GDP per capital) in Beijing of $ 29,306.33. Moreover, one-way sensitivity analyses showed that the results were sensitive to the transition probability of normal to joint bleeding and PSA results showed that it had 97.8% probability to prove the conclusion robust. Conclusions: Although prophylaxis is a costly treatment, the results show that it's cost-effective compared with on demand treatment in HB children in China.
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