In clinical research, study outcomes usually consist of various patients’ information corresponding to the treatment. To have a better understanding of teh effects of different treatments, one often needs to analyze multiple clinical outcomes simultaneously, while the data are usually mixed with both continuous and discrete variables. We propose the multivariate mixed response model to implement statistical inference based on the conditional grouped continuous model through a pairwise composite-likelihood approach. It can simplify the multivariate model by dealing with three types of bivariate models and incorporating the asymptotical properties of the composite likelihood via the Godambe information. We demonstrate the validity and the statistic power of the multivariate mixed response model through simulation studies and clinical applications. This composite-likelihood method is advantageous for statistical inference on correlated multivariate mixed outcomes.
Background The objective of this study was to assess the effect of parathyroidectomy (PTX) treatment on prolonging overall survival (OS) as well as decreasing levels of intact parathyroid hormone (iPTH), calcium (Ca), and phosphorus (P) in elderly hemodialysis patients with severe secondary hyperparathyroidism (SHPT). Methods A total of 304 elderly hemodialysis patients with severe SHPT were consecutively enrolled in this cohort study. According to whether PTX operations were applied, patients were classified into PTX group (N = 112) and Control group (N = 192) and were followed up for 3 years. Mortality rate and OS were evaluated, and iPTH, Ca, and P levels were recorded. Results Compared to control group, increased iPTH (P < 0.001), higher Ca (P = 0.003), elevated AST (P = 0.022), and lower Hb (P = 0.049) concentrations were observed in the PTX group at baseline. The 1‐year mortality (P < 0.001), 2‐year mortality (P < 0.001), and 3‐year mortality (P < 0.001) was reduced in PTX group compared to Control group, and PTX was correlated with prolonged OS (P < 0.001). Multivariate Cox's regression analysis further revealed that PTX treatment (P < 0.001, HR = 0.177) was an independent factor for better OS. Moreover, patients in PTX group had decreased iPTH (P < 0.05) and Ca (P < 0.05) levels compared to Control group at M1‐M36, while no difference was found in serum P level between the two groups at M1‐M36. Conclusion Parathyroidectomy decreases iPTH and Ca levels, and it associates with favorable survival in elderly hemodialysis patients with severe SHPT.
This study aimed to compare the long-term survival of patients with hepatocellular carcinoma (HCC) within the Milan criteria who underwent hepatic resection (HR) or transarterial chemoembolization (TACE).Medical records were retrospectively analyzed for HCC patients within the Milan criteria treated at Affiliated Tumor Hospital of Guangxi Medical University between March 2003 and March 2008, 159 of whom underwent HR and 42 of whom underwent TACE. Long-term overall survival (OS) was evaluated using the Kaplan–Meier method before and after propensity score matching. Cox proportional hazard modeling was used to identify possible predictors of OS.Propensity score matching was used to generate 32 pairs of patients, for which OS was significantly higher after HR than TACE at 1 year, 96.6% versus 84.4%; 3 years, 75.4% versus 53.1%; 5 years, 48.8% versus 29.7%, respectively (P = .038). Among all patients with multinodular HCC (2–3 tumors ≤3 cm), HR was also associated with significantly higher OS than TACE at 1 year, 95.2% versus 72.7%; 3 years, 71.4% versus 9.1%; 5 years, 35.1% versus 0%, respectively (P < .001). By contrast, among all patients with a single HCC tumor ≤5 cm, HR and TACE were associated with similar OS at 1 year, 85.9% versus 90.3%; 3 years, 62.0% versus 61.3%; 5 years, 42.1% versus 33.2%, respectively (P = .332).HR provides survival benefit over TACE in HCC patients within the Milan criteria, especially patients with multinodular HCC involving 2 to 3 tumors ≤3 cm. However, HR and TACE appear to be similarly effective for patients with single-tumor HCC ≤5 cm.
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