Dataset Information

Bayesian design of superiority clinical trials for recurrent events data with applications to bleeding and transfusion events in myelodyplastic syndrome.

ABSTRACT: In many biomedical studies, patients may experience the same type of recurrent event repeatedly over time, such as bleeding, multiple infections and disease. In this article, we propose a Bayesian design to a pivotal clinical trial in which lower risk myelodysplastic syndromes (MDS) patients are treated with MDS disease modifying therapies. One of the key study objectives is to demonstrate the investigational product (treatment) effect on reduction of platelet transfusion and bleeding events while receiving MDS therapies. In this context, we propose a new Bayesian approach for the design of superiority clinical trials using recurrent events frailty regression models. Historical recurrent events data from an already completed phase 2 trial are incorporated into the Bayesian design via the partial borrowing power prior of Ibrahim et al. (2012, Biometrics 68, 578-586). An efficient Gibbs sampling algorithm, a predictive data generation algorithm, and a simulation-based algorithm are developed for sampling from the fitting posterior distribution, generating the predictive recurrent events data, and computing various design quantities such as the type I error rate and power, respectively. An extensive simulation study is conducted to compare the proposed method to the existing frequentist methods and to investigate various operating characteristics of the proposed design.

SUBMITTER: Chen MH

PROVIDER: S-EPMC4276515 | biostudies-literature | 2014 Dec

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Bayesian design of superiority clinical trials for recurrent events data with applications to bleeding and transfusion events in myelodyplastic syndrome.

Chen Ming-Hui MH Ibrahim Joseph G JG Zeng Donglin D Hu Kuolung K Jia Catherine C

Biometrics 20140716 4

In many biomedical studies, patients may experience the same type of recurrent event repeatedly over time, such as bleeding, multiple infections and disease. In this article, we propose a Bayesian design to a pivotal clinical trial in which lower risk myelodysplastic syndromes (MDS) patients are treated with MDS disease modifying therapies. One of the key study objectives is to demonstrate the investigational product (treatment) effect on reduction of platelet transfusion and bleeding events whi ...[more]

PMID: 25041037

Similar Datasets

Project description:Background Surgery is the main treatment for recurrent retroperitoneal liposarcoma (RPLS). The aim of the present study was to explore the factors associated with blood loss during surgery for recurrent RPLS. Methods This retrospective study included patients with first recurrence of RPLS who were treated at our hospital between January 2015 and December 2019. Factors associated with intraoperative blood loss were identified by univariate and multivariate logistic regression analyses. Receiver-operating characteristic (ROC) curve analyses were conducted to evaluate whether tumor size and number of tumor-containing abdominal/pelvic zones were predictive of the need for blood transfusion. Results The study included 67 cases. The number of zones containing tumors was 1 in 4 cases (6%), 2 in 36 cases (53.7%), 3 in 14 cases (20.9%), and 4 in 13 cases (19.4%). Tumor size was associated with blood loss >500 mL [odds ratio (OR): 1.153, 95% confidence interval (CI): 1.051–1.266, P=0.003]. The number of tumor-containing zones was associated with blood loss >1,000 mL (OR: 3.161, 95% CI: 1.248–8.003, P=0.015) and >1,500 mL (OR: 2.674, 95% CI: 1.061–6.739, P=0.037). Multiple tumors were associated with blood loss >2,000 mL (OR: 3.161, 95% CI: 1.092–13.133, P=0.036) and >2,500 mL (OR: 2.674, 95% CI: 1.243–16.299, P=0.022). Tumor dedifferentiation was associated with blood loss >1,000 mL (OR: 4.802, 95% CI: 1.287–17.916, P=0.019) and >1,500 mL (OR: 9.249, 95% CI: 1.927–44.39, P=0.005). ROC curve analysis showed that tumor size >15.25 cm [area under the ROC curve (AUC): 0.772, P<0.001] and the number of tumor-containing zones >2.5 (AUC: 0.670; P=0.023) were predictive of the need for blood transfusion. Conclusions The main finding of the present study was that a larger tumor size, a larger number of tumor-containing zones, multiple tumors, and dedifferentiation were independently associated with a larger volume of intraoperative blood loss in patients with recurrent RPLS. The tumor size >15.25 cm and the tumor area >2.5 areas predicted the need for blood transfusion. Formulating the intraoperative blood transfusion plan for recurrent RPLS, it is necessary to pay attention to two spatial factors, tumor size and affected area, rather than one of them.

Dataset Information

Bayesian design of superiority clinical trials for recurrent events data with applications to bleeding and transfusion events in myelodyplastic syndrome.

Publications

Bayesian design of superiority clinical trials for recurrent events data with applications to bleeding and transfusion events in myelodyplastic syndrome.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets