'Potentially curative therapies' for hepatocellular carcinoma: how many patients can actually be cured?

BACKGROUND: Treatment of hepatocellular carcinoma (HCC) is predicated on early diagnosis such that 'curative therapies' can be successfully applied. The term 'curative' is, however, poorly quantitated. We aimed to complement our previous work by developing a statistical model to predict cure after ablation and to use this analysis to compare the true curative potential of the various 'curative' therapies. METHODS: We accessed data from 1571 HCC patients treated in 5 centres receiving radiofrequency (RFA) or microwave (MWA) ablation and used flexible parametric modelling to determine the curative fraction. The results of this analysis were then combined with our previous estimations to provide a simple calculator applicable to all patients undergoing potentially curative therapies. RESULTS: The cure fraction was 18.3% rising to about 40% in patients with good liver function and very small tumours. CONCLUSION: Cure for HCC treated with ablation occurs in the order of 20% to 30%, similar to that achievable by resection but much inferior to transplantation where the analogous figure is >70%. We provide a 'calculator' that permits clinicians to estimate the chance of cure for any individual patient, based on readily available clinical features.

Developing a topic-based repository of clinical trial individual patient data: experiences and lessons learned from a pilot project.

BACKGROUND: Building a dataset of individual participant data (IPD) for meta-analysis represents considerable research investment as well as collaboration across multiple institutions and researchers. Making arrangements to curate and share the dataset beyond the IPD meta-analysis project for which it was established, for reuse in future research projects, would maximise the value of this investment. METHODS: Our aim was to establish the Cochrane repository for individual patient data from clinical trials in pregnancy and childbirth (CRIB) as an example of how an IPD repository could become part of Cochrane infrastructure. We believed that establishing CRIB under Cochrane auspices would engender trust and encourage trial investigators to share data, and at the same time position Cochrane to take steps towards expanding the number of reviews with IPD synthesis. RESULTS: CRIB was designed as a web-based platform to receive, host and facilitate onward sharing of de-identified data. Development was not straightforward and we did not fully achieve our aim as intended. We describe the challenges encountered and suggest ways that future repositories might overcome these. In particular, securing the legal agreements required to facilitate data sharing proved to be the main barrier, being time-consuming and more complex than anticipated. CONCLUSIONS: We would recommend that researchers conducting IPD meta-analysis should consider discussing the option to transfer the curated IPD datasets to a repository at the end of the initial meta-analysis and this should be recognised within the data sharing agreements made with the original data contributors.

Consensus Building in OMERACT: Recommendations for Use of the Delphi for Core Outcome Set Development.

OBJECTIVE: Developing international consensus on outcome measures for clinical trials is challenging. The following paper will review consensus building in Outcome Measures in Rheumatology (OMERACT), with a focus on the Delphi. METHODS: Based on the literature and feedback from delegates at OMERACT 2018, a set of recommendations is provided in the form of the OMERACT Delphi Consensus Checklist. RESULTS: The OMERACT delegates generally supported the use of the checklist as a guide. The checklist provides guidance for clearly outlining the multiple aspects of the Delphi process. CONCLUSION: OMERACT is deeply committed to consensus building and these recommendations should be considered a work in progress.

Higher number of items associated with significantly lower response rates in COS Delphi surveys.

OBJECTIVES: The Delphi method is commonly used to achieve consensus in core outcome set (COS) development. It is important to try to maximize response rates to Delphi studies and minimize attrition rates and potential for bias. The factors that impact response rates in a Delphi study used for COS development are unknown. The objective of this study was to explore the impact of design characteristics on response rates in Delphi surveys within COS development. METHODS: Published and ongoing studies that included Delphi to develop a COS were eligible. Second round voting response rates were analyzed, and multilevel linear regression was conducted to investigate whether design characteristics were associated with the response rate. RESULTS: Thirty-one studies were included. Two characteristics were significantly associated with a lower response rate: larger panels and studies with more items included. CONCLUSION: COS developers should pay attention to methods when designing a COS development study; in particular, the size of the panels and the size of the list of outcomes. We identified other potential design characteristics that might influence response rates but were unable to explore them in this analysis. These should be reported in future reports to allow for further investigation.