Dataset Information

Detailed statistical analysis plan for the Danish Palliative Care Trial (DanPaCT).

ABSTRACT: Advanced cancer patients experience considerable symptoms, problems, and needs. Early referral of these patients to specialized palliative care (SPC) could offer improvements. The Danish Palliative Care Trial (DanPaCT) investigates whether patients with metastatic cancer will benefit from being referred to 'early SPC'. DanPaCT is a multicenter, parallel-group, superiority clinical trial with 1:1 randomization. The planned sample size was 300 patients. The primary data collection for DanPaCT is finished. To prevent outcome reporting bias, selective reporting, and data-driven results, we present a detailed statistical analysis plan (SAP) for DanPaCT here.This SAP provides detailed descriptions of the statistical analyses of the primary and secondary outcomes in DanPaCT. The primary outcome is the change in the patient's 'primary need'. The 'primary need' is a patient-individualised outcome representing the score of the symptom or problem that had the highest intensity out of seven at baseline assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30). Secondary outcomes are the seven scales that are represented in the primary outcome, but each scale evaluated individually for all patients, and survival. The detailed description includes chosen significance levels, models for multiple imputations, sensitivity analyses and blinding. In addition, we discuss the patient-individualized primary outcome, blinding, missing data, multiplicity and the risk of bias.Only few trials have investigated the effects of SPC. To our knowledge DanPaCT is the first trial to investigate screening based 'early SPC' for patients with metastatic cancer from a broad spectrum of cancer diagnosis.Clinicaltrials.gov identifier: NCT01348048 (May 2011).

SUBMITTER: Johnsen AT

PROVIDER: S-EPMC4190470 | biostudies-literature | 2014 Sep

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Detailed statistical analysis plan for the Danish Palliative Care Trial (DanPaCT).

Johnsen Anna Thit AT Petersen Morten Aagaard MA Gluud Christian C Lindschou Jane J Fayers Peter P Sjøgren Per P Pedersen Lise L Neergaard Mette Asbjoern MA Vejlgaard Tove Bahn TB Damkier Anette A Nielsen Jan Bjoern JB Strömgren Annette S AS Higginson Irene J IJ Groenvold Mogens M

Trials 20140926

<h4>Background</h4>Advanced cancer patients experience considerable symptoms, problems, and needs. Early referral of these patients to specialized palliative care (SPC) could offer improvements. The Danish Palliative Care Trial (DanPaCT) investigates whether patients with metastatic cancer will benefit from being referred to 'early SPC'. DanPaCT is a multicenter, parallel-group, superiority clinical trial with 1:1 randomization. The planned sample size was 300 patients. The primary data collecti ...[more]

PMID: 25257804

Similar Datasets

Project description:IntroductionCoronary artery bypass grafting can be conducted using the radial artery as a bypass graft. However, it remains unclear which harvesting method is superior, i.e. endoscopic or open radial artery, and which site for proximal anastomosis of the radial artery has the greatest benefits?MethodsThe NEO Trial is a single site randomised clinical trial with a 2 × 2 factorial design. The first comparison assesses endoscopic versus open radial artery harvest with a primary outcome of hand function and secondary outcomes of neurological deficits through clinical exams and neurophysiological studies. The primary outcome is postoperatively hand function at three months. We anticipate a mean difference of 3 points with a standard deviation of 8 points, a power of 90%, and a type I error of 5%, resulting in a required sample size of 300 participants randomised 1:1. Secondary outcomes are neurological deficits (based on nerve conduction measurements, algometry test and von Frey hair test), clinical neurological examination of cutaneous sensibility, and registration of complications in the donor arm (haematoma formation, wound dehiscence, and/or infection). The second comparison assesses two different proximal anastomotic sites, i.e. aorto-radial anastomosis versus mammario-radial anastomosis. The primary outcome is a composite of cerebrovascular events and the secondary outcome is graft patency evaluation by multi-slice computer tomography-scan. These outcomes will be assessed at 1 year postoperatively, and the results of this comparison will be exploratory only. Both comparisons will be analysed using intention-to-treat and intervention groups will be compared using linear regression, logistic regression, or Mann-Whitney U test depending on data type. Two independent statisticians will follow the present plan and conduct the analyses which will hereafter be fused into a final analysis based on consensus.ConclusionThis detailed analysis plan will increase the validity of the NEO trial results by predefining the statistical analysis in detail.Trial registrationClinicalTrials.gov identifier: NCT01848886 . Registered 25 February 2013. Danish Ethics committee number: H-3-2012-116. Danish Data Protection Agency: 2007-58-0015/jr. n:30-0838.

Project description:BackgroundTransforaminal epidural injections with steroids (TESI) are used increasingly for patients with sciatica. However, their safety, effectiveness, and cost-effectiveness are still a matter of debate. This a priori statistical analysis plan describes the methodology of the analysis for the STAR trial that assesses the (cost-)effectiveness of TESI during the acute stage of sciatica (< 8 weeks).MethodsThe STAR trial is a multicentre, randomized controlled, prospective trial (RCT) investigating the (cost-)effectiveness of TESI by making a three-group comparison among patients with acute sciatica due to a herniated lumbar disc (< 8 weeks): (1) TESI combined with levobupivacaine added to oral pain medication (intervention group 1) versus oral pain medication alone (control group), (2) intervention group 1 versus transforaminal epidural injection with levobupivacaine and saline solution added to oral pain medication (intervention group 2), and (3) intervention group 2 versus control group. Co-primary outcomes were physical functioning (Roland Morris Disability Questionnaire), pain intensity (10-point numerical rating scale), and global perceived recovery (7-point Likert scale, dichotomized into 'recovered' and 'not recovered'). For all three comparisons, we defined the following minimal clinically relevant between-group differences: two points for pain intensity (range 0-10), four points for physical functioning (range 0-24) and a 20% difference in recovery rate. Secondary outcomes are health-related quality of life (EQ-5D-5L) and patient satisfaction (7-point Likert scale) and surgery rate. We also collected resource use data to perform an economic evaluation. Analyses will be conducted by intention-to-treat with p < 0.05 (two-tailed) for all three comparisons. Effects will be estimated using mixed models by maximum likelihood. For each comparison, mean differences, or difference in proportions, between groups will be tested per time point and an overall mean difference, or difference in proportions, between groups during the complete duration of follow-up (6 months) will be estimated. In the economic evaluation, Multivariate Imputation by Chained Equations will be used to handle missing data. Cost and effect differences will be estimated using seemingly unrelated regression, and uncertainty will be estimated using bootstrapping techniques.DiscussionThis statistical analysis plan provides detailed information on the intended analysis of the STAR trial, which aims to deliver evidence about the (cost-)effectiveness of TESI during the acute phase of sciatica (< 8 weeks).Trial registrationDutch National trial register NTR4457 (6 March 2014).

Project description:Extremely preterm infants have a high mortality and morbidity. Here, we present a statistical analysis plan for secondary Bayesian analyses of the pragmatic, sufficiently powered multinational, trial-SafeBoosC III-evaluating the benefits and harms of cerebral oximetry monitoring plus a treatment guideline versus usual care for such infants. The SafeBoosC-III trial is an investigator-initiated, open-label, randomised, multinational, pragmatic, phase III clinical trial with a parallel-group design. The trial randomised 1601 infants, and the frequentist analyses were published in April 2023. The primary outcome is a dichotomous composite outcome of death or severe brain injury. The exploratory outcomes are major neonatal morbidities associated with neurodevelopmental impairment later in life: (1) bronchopulmonary dysplasia; (2) retinopathy of prematurity; (3) late-onset sepsis; (4) necrotising enterocolitis; and (5) number of major neonatal morbidities (count of bronchopulmonary dysplasia, retinopathy of prematurity, and severe brain injury). The primary Bayesian analyses will use non-informed priors including all plausible effects. The models will use a Hamiltonian Monte Carlo sampler with 1 chain, a sampling of 10,000, and at least 25,000 iterations for the burn-in period. In Bayesian statistics, such analyses are referred to as 'posteriors' and will be presented as point estimates with 95% credibility intervals (CrIs), encompassing the most probable results based on the data, model, and priors selected. The results will be presented as probability of any benefit or any harm, Bayes factor, and the probability of clinical important benefit or harm. Two statisticians will analyse the blinded data independently following this protocol. This statistical analysis plan presents a secondary Bayesian analysis of the SafeBoosC-III trial. The analysis and the final manuscript will be carried out and written after we publicise the primary frequentist trial report. Thus, we can interpret the findings from both the frequentists and Bayesian perspective. This approach should provide a better foundation for interpreting of our findings. ClinicalTrials.org, NCT03770741. Registered on 10 December 2018.

Dataset Information

Detailed statistical analysis plan for the Danish Palliative Care Trial (DanPaCT).

Publications

Detailed statistical analysis plan for the Danish Palliative Care Trial (DanPaCT).

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets