most frequently reported TEAE (vomiting, upper respiratory tract infection, cough, and constipation) none were considered AVXS-101 related, with most events related to patients' underlying illness, not to study drug. CONCLUSIONS: Magnitude of the observed relative treatment effects indicates that AVXS-101 offers promising efficacy compared to nusinersen regarding EFS, reduced requirement for ventilation, and increased achievement of motor milestones.
analyses (DSA and PSA) were conducted. Results: Over a lifetime horizon, treating moderate-to-severe active UC with tofacitinib resulted in additional quality-adjusted life-years (QALYs) and lower total costs compared to vedolizumab (0.018; V6,408) and infliximab (biosimilar) (0.009; V3,031), hence, tofacitinib was estimated to dominate over both comparators. DSA showed model results were mostly influenced by differences in treatment efficacy from the NMA and target patient population. PSA confirmed robustness of findings below a threshold of V34,000 in 100% and 98% of the simulations comparing tofacitinib to vedolizumab and infliximab respectively. Conclusions: The results of the analysis suggest that in the Greek setting, tofacitinib could be considered a cost-effective (dominant) treatment option for the treatment of patients with moderate-to-severe active UC.
IntroductionColorectal cancer (CRC) is the third most common malignant neoplasm among men and the second most common among women. According to the World Cancer Report, the number of people suffering from this disease is growing steadily. In 2012, there were more than 1.36 million new cases of CRC, and approximately 694,000 people died from this disease worldwide.MethodsA sensitive literature search identified 12 relevant publications, including: a CORRECT phase III study assessing the effect of regorafenib in patients with metastatic CRC that continued to progress despite using all standard treatment methods; a CONCUR Phase III study evaluating the clinical effect of regorafenib in Asian patients with metastatic CRC; a CONSIGN study conducted after the CORRECT and CONCUR studies that assessed the safety profile of regorafenib prior to market entry; and various systematic reviews evaluating the safety of regorafenib.ResultsThe efficacy and safety of regorafenib for treating patients with metastatic CRC was evaluated in two major clinical studies: CORRECT and CONCUR. Although the studies were randomized, double-blind, and placebo-controlled, they were conducted in different patient populations. Before treatment with regorafenib, patients received, depending on the country, fluoropyrimidines, oxaliplatin, irinotecan, or bevacizumab, and patients with the wild-type KRAS gene also received cetuximab and panitumumab. Results from both studies indicated that regorafenib had a clinically significant positive effect on rates of progression-free survival and overall survival in patients with treatment-resistant metastatic CRC.ConclusionsRegorafenib can be recommended as a monotherapy for resistant metastatic CRC when there are no contraindications to use. Considering the safety profile of regorafenib, further research is needed to determine the best dosage of regorafenib and the most appropriate clinical and molecular biomarkers for determining which patients would benefit most from this treatment.
Islamic Republic of) Objectives: Free access to information as a public right has become a critical issue in healthcare. Some health systems disclose information about hospitals performance to influence patients' choice. The main purpose of this study is to explore barriers to transparency improvement of hospitals performance in Iran. Methods: This qualitative study was conducted during January to August 2017 in Iran through face-toface interviews (18 various interviewees in macro, meso and micro levels of health system with different academic orientation and expertise) and through a comprehensive and purposive document analysis. The data was analyzed using a thematic analysis method. Results: Five main themes were identified, including: lack of regular hospitals' performance data available to the government; lack of transparency culture among health system authorities; concerns about transparency of hospitals' poor performance; absence of rule of law and Lack of adequate maturity in the country's health system governance which weaken democracy. Therefore the decision making system mostly supports special groups rather than public. Conclusions: There are serious barriers against improving transparency of hospitals in Iran. The most important challenge found was "authorities unwillingness to promote transparency". This challenge has become more prominent due to the existence of conflict of interests among authorities and power of the pressure groups to influence the public sector. Therefore, interviewees suggested that the necessary infrastructures be created prior to promoting transparency. Among the most important preliminaries is managing conflict of interest in the health system to have decisions in favor of public interests.
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