Sample size calculations for randomized controlled trials and for prediction models

Fiorentino, Francesca; Treasure, Tom

doi:10.1111/codi.15489

Cited by 10 publications

(7 citation statements)

References 18 publications

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“…Considering the HR for overall survival found in this pilot trial of 0.52, the needed samples size for a future trial, based on alfa significance level of 0.05 and 80% of power 17 , should be of 145 patients with approximately 73 events/deaths.…”

Section: Resultsmentioning

confidence: 97%

Comparison of VATS Pleurectomy/Decortication Surgery plus Hyperthermic Intrathoracic Chemotherapy with VATS talc pleurodesis for the treatment of Malignant Pleural Mesothelioma: a randomized pilot study

Fiore

Tumino²,

Filippini³

et al. 2021

Preprint

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IntroductionNo previous study provides compelling evidence to convince surgeons to opt for one procedure over another for the treatment of Malignant Pleural Mesothelioma (MPM). Hyperthermic intrathoracic chemotherapy (HITHOC) adjunct to surgery for MPM has no definite role. The primary objective of this randomized pilot trial was to evaluate the feasibility for future large studies.MethodThe study design was a prospective randomized three-centric pilot trial (ISRCTN12709516). We recruited patients diagnosed with MPM and prospectively assigned them to two groups: Group A Video Assisted Thoracic Surgery (VATS) talc pleurodesis or Group B VATS P/D plus HITHOC. The main outcome measures were description of study feasibility. We collected socio-demographic and clinical patient information. Data of Kaplan-Meier survival analysis and Cox regression analysis are presented.ResultsFrom November-2011 to July-2017 24 males and 3 females, with a median age of 68-years were enrolled (recruitment rate 5 patients/year). Preoperative stage was I-II, and 18 had epithelioid type. 14 patients were in the Group A. Operative mortality was 0. Follow-up ranged from 6 to 80 months. The median overall survival time started to diverge at 20 months. In Group A, it was 19 months (95% CI:12-25) and in Group B, it was 28 months (95% CI:0-56). Survival rate for the epithelioid type was 15 months (0-34) in groups A. and 45 months (0-107) in the Group B with an HR 0.77 (95% CI:0.28-2.2).ConclusionThese findings suggest that VATS P/D plus HITHOC may improve survival time in MPM patients undergoing surgical treatment and support the need for a larger multicenter randomized clinical trial.Strengths and limitations of this studythis pilot study represents an important step forward of the treatment of malignant pleural mesotheliomaThe study demonstrates the feasibility for a multicenter randomized trial to compare VATS P/D plus HITHOC with VATS talc pleurodesis in the management of MPMAlthough survival for the epitheliod type is 45 months, the inclusion of small number of patients is a limitation.Although neoadjuvant, adjuvant chemo-radio or immunotherapy have been administered to the patients, we have no specific information about doses for every single patient.A potential source of bias could be that the house staff of the Centre A has not been formally educated about the study while in the Centre B house staff was informed about the pilot trial.

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Section: Resultsmentioning

confidence: 97%

Comparison of VATS Pleurectomy/Decortication Surgery plus Hyperthermic Intrathoracic Chemotherapy with VATS talc pleurodesis for the treatment of Malignant Pleural Mesothelioma: a randomized pilot study

Fiore

Tumino²,

Filippini³

et al. 2021

Preprint

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“…Sufficiently large, collaborative, and independently monitored controlled trials are needed. PulMiCC illustrates the difficulties encountered but also shows an approach to planning such trials 16 .…”

Section: Letter To the Editormentioning

confidence: 99%

The Pulmonary Metastasectomy in Colorectal Cancer study calls for reconsideration of the clinical effectiveness of this widespread practice

Treasure

Macbeth

Younes

2022

Rev. Assoc. Med. Bras.

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“…3 The number randomized (N = 93) precluded proof of noninferiority, but the generally believed large benefit can be confidently refuted. 8…”

Section: To the Editormentioning

confidence: 99%

“…3 The number randomized (N = 93) precluded proof of noninferiority, but the generally believed large benefit can be confidently refuted. 8 Moran cited median survival of 42 months in PRODIGE as evidence for effectiveness of peritoneal resection, but it was hyperthermic intraperitoneal chemotherapy that was under test. There were no unoperated patients.…”

mentioning

confidence: 99%

Comment on Moran’s “Light at the End of the Tunnel”: The Pulmonary Metastasectomy in Colorectal Cancer Study Sheds Some Light on the Question

Treasure

Milosevic

Macbeth

2022

Diseases of the Colon &Amp; Rectum

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To the Editor-The editorial about resection of colorectal cancer peritoneal metastases referred to the pulmonary metastasectomy in colorectal cancer (PulMiCC) randomized controlled trial (RCT) 1 only to dismiss it. 2 We write to update the citation 3 and summarize some published data from the full PulMiCC study. 4,5 Recruitment reached 512 patients, but the Society of Thoracic Surgeons published consensus, that without metastasectomy survival would be zero 6 , led to the majority of patients (N = 263) being selected for metastasectomy. 4 The 22% 5-year survival among 128 unoperated patients refutes the zero assumption (p < 0.001). 4 All prognostic factors favored the operated patients: fewer (31% vs 65%) had solitary metastases, elevated carcinoembryonic antigen, liver involvement, and impaired performance status. They were younger with better lung function. Fiveyear survival (~60%) was comparable with the best of "real world" outcomes. 7 For the patients who were randomly assigned, prognostic factors were excellently balanced in the 2 arms of the nested RCT. There was no survival difference at any time point. 3 The number randomized (N = 93) precluded proof of noninferiority, but the generally believed large benefit can be confidently refuted. 8 Moran cited median survival of 42 months in PRODIGE as evidence for effectiveness of peritoneal resection, but it was hyperthermic intraperitoneal chemotherapy that was under test. There were no unoperated patients. 9 Without well-balanced controls, there is no proof of benefit. Similar median survival values were seen in the PulMiCC RCT, that is, 42 months for treated group and 45.6 months for control. 9 Selection for inclusion and guarantee time bias play a major part in the much better than expected survival rates. 5,10

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Sample size calculations for randomized controlled trials and for prediction models

Cited by 10 publications

References 18 publications

Comparison of VATS Pleurectomy/Decortication Surgery plus Hyperthermic Intrathoracic Chemotherapy with VATS talc pleurodesis for the treatment of Malignant Pleural Mesothelioma: a randomized pilot study

Comparison of VATS Pleurectomy/Decortication Surgery plus Hyperthermic Intrathoracic Chemotherapy with VATS talc pleurodesis for the treatment of Malignant Pleural Mesothelioma: a randomized pilot study

The Pulmonary Metastasectomy in Colorectal Cancer study calls for reconsideration of the clinical effectiveness of this widespread practice

Comment on Moran’s “Light at the End of the Tunnel”: The Pulmonary Metastasectomy in Colorectal Cancer Study Sheds Some Light on the Question

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