AIM:To assess the clinicopathologic features and its relationship with prognosis of pseudomyxoma peritonei(PMP) in Chinese patients.METHODS:The clinicopathologic features and followup data of 92 patients with PMP were...AIM:To assess the clinicopathologic features and its relationship with prognosis of pseudomyxoma peritonei(PMP) in Chinese patients.METHODS:The clinicopathologic features and followup data of 92 patients with PMP were reviewed and retrospectively analyzed.The cases were categorized into three groups:disseminated peritoneal adenomucinosis(DPAM),peritoneal mucinous carcinomatosis(PMCA),and peritoneal mucinous carcinomatosis with intermediate or discordant features(PMCA-I/D).The log-rank test was used to analyze survival for each group and various clinicopathological parameters.Multivariate Cox proportional-hazard models were constructed to determine the important factors associated with survival.RESULTS:The median age at diagnosis was 51.9 years(range:22-76 years).The median follow up was 124 mo.The 3-,5-and 10-year survival rates were 74.0%,67.4% and 49.1%,respectively.There were 49(53.2%)patients with DPAM,26(28.3%) with PMCA-I and 17(18.5%) with PMCA.Patients with DPAM,PMCA-I/D and PMCA exhibited statistically significant difference in survival(P = 0.001).The 3 year survival for DPAM,PMCAI/D and PMCA was 97.0%,80.0% and 67.0%,respectively;the 5 year survival was 80.0%,67.0% and 50.0%,respectively;and the 10 year survival was 65.0%,28.0% and 14.0%,respectively.Survival rate was significantly lowest in patients < 40 age years of age(P = 0.011).Appendiceal tumor and extra-ovarian parenchymal organ involvement were significantly related to overall survival.Patients with appendiceal mucinous adenocarcinoma(MACA) showed the significantly poorer prognosis(P = 0.011).Multivariate analysis showed that pathological classification,age,appendiceal tumor were significant related to overall survival.CONCLUSION:The clinical process "PMP" should be pathologically classified into DPAM,PMCA and PMCA-I/D.Pathological classification,age,appendiceal MACA are survival independent predictors in Chinese patients with PMP.展开更多
AIM:To present statistical tools to model and optimize the cost of a randomized clinical trial as a function of the stringency of patient inclusion criteria.METHODS: We consider a two treatment, dichotomous outcome tr...AIM:To present statistical tools to model and optimize the cost of a randomized clinical trial as a function of the stringency of patient inclusion criteria.METHODS: We consider a two treatment, dichotomous outcome trial that includes a proportion of patients who are strong responders to the tested intervention. Patients are screened for inclusion using an arbitrary number of test results that are combined into an aggregate suitability score. The screening score is regarded as a diagnostic test for the responsive phenotype, having a specific cutoff value for inclusion and a particular sensitivity and specificity. The cutoff is a measure of stringency of inclusion criteria. Total cost is modeled as a function of the cutoff value, number of patients screened, the number of patients included, the case occurrence rate, response probabilities for control and experimental treatments, and the trial duration required to produce a statistically significant result with a specified power. Regression methods are developed to estimate relevant model parameters from pilot data in an adaptive trial design. RESULTS: The patient numbers and total cost are strongly related to the choice of the cutoff for inclusion. Clear cost minimums exist between 5.6 and 6.1 on arepresentative 10-point scale of exclusiveness. Potential cost savings for typical trial scenarios range in millions of dollars. As the response rate for controls approaches 50%, the proper choice of inclusion criteria can mean the difference between a successful trial and a failed trial. CONCLUSION: Early formal estimation of optimal inclusion criteria allows planning of clinical trials to avoid high costs, excessive delays, and moral hazards of Type II errors.展开更多
文摘AIM:To assess the clinicopathologic features and its relationship with prognosis of pseudomyxoma peritonei(PMP) in Chinese patients.METHODS:The clinicopathologic features and followup data of 92 patients with PMP were reviewed and retrospectively analyzed.The cases were categorized into three groups:disseminated peritoneal adenomucinosis(DPAM),peritoneal mucinous carcinomatosis(PMCA),and peritoneal mucinous carcinomatosis with intermediate or discordant features(PMCA-I/D).The log-rank test was used to analyze survival for each group and various clinicopathological parameters.Multivariate Cox proportional-hazard models were constructed to determine the important factors associated with survival.RESULTS:The median age at diagnosis was 51.9 years(range:22-76 years).The median follow up was 124 mo.The 3-,5-and 10-year survival rates were 74.0%,67.4% and 49.1%,respectively.There were 49(53.2%)patients with DPAM,26(28.3%) with PMCA-I and 17(18.5%) with PMCA.Patients with DPAM,PMCA-I/D and PMCA exhibited statistically significant difference in survival(P = 0.001).The 3 year survival for DPAM,PMCAI/D and PMCA was 97.0%,80.0% and 67.0%,respectively;the 5 year survival was 80.0%,67.0% and 50.0%,respectively;and the 10 year survival was 65.0%,28.0% and 14.0%,respectively.Survival rate was significantly lowest in patients < 40 age years of age(P = 0.011).Appendiceal tumor and extra-ovarian parenchymal organ involvement were significantly related to overall survival.Patients with appendiceal mucinous adenocarcinoma(MACA) showed the significantly poorer prognosis(P = 0.011).Multivariate analysis showed that pathological classification,age,appendiceal tumor were significant related to overall survival.CONCLUSION:The clinical process "PMP" should be pathologically classified into DPAM,PMCA and PMCA-I/D.Pathological classification,age,appendiceal MACA are survival independent predictors in Chinese patients with PMP.
文摘AIM:To present statistical tools to model and optimize the cost of a randomized clinical trial as a function of the stringency of patient inclusion criteria.METHODS: We consider a two treatment, dichotomous outcome trial that includes a proportion of patients who are strong responders to the tested intervention. Patients are screened for inclusion using an arbitrary number of test results that are combined into an aggregate suitability score. The screening score is regarded as a diagnostic test for the responsive phenotype, having a specific cutoff value for inclusion and a particular sensitivity and specificity. The cutoff is a measure of stringency of inclusion criteria. Total cost is modeled as a function of the cutoff value, number of patients screened, the number of patients included, the case occurrence rate, response probabilities for control and experimental treatments, and the trial duration required to produce a statistically significant result with a specified power. Regression methods are developed to estimate relevant model parameters from pilot data in an adaptive trial design. RESULTS: The patient numbers and total cost are strongly related to the choice of the cutoff for inclusion. Clear cost minimums exist between 5.6 and 6.1 on arepresentative 10-point scale of exclusiveness. Potential cost savings for typical trial scenarios range in millions of dollars. As the response rate for controls approaches 50%, the proper choice of inclusion criteria can mean the difference between a successful trial and a failed trial. CONCLUSION: Early formal estimation of optimal inclusion criteria allows planning of clinical trials to avoid high costs, excessive delays, and moral hazards of Type II errors.
