Standardising Outcomes in Malignant Pleural Effusion Trials: The STANDOUT MPE Study

Background There are multiple treatment options available for MPE with a high-quality evidence base, so a patient-focused management strategy has been emphasised [1] & [2]. Improving MPE management strategies in the NHS is dependent on the synthesis of validated research trial findings. Without a consensus in MPE clinical trial design, there is significant heterogeneity in the selection of outcomes.

There is also a high risk of outcome reporting bias, with potential for outcomes to be prioritised based on their statistical power rather than clinical relevance [3].

This variation prevents direct comparability and meta-analysis of findings, resulting in research waste and delays to clinical implementation of findings.

A Cochrane review of MPE management [4] concluded that the standardisation of outcomes measures is essential for future trials in this area.

Research Aims This project aims to support the standardisation of outcome reporting by establishing international consensus on outcomes to be reported as a minimum in future interventional MPE clinical trials: The STANDOUT-MPE Core Outcome Set (COS).

This study is aligned with the COS development standards set out by the COMET initiative [5] and consists of three work packages (WPs): WP1: Creating an exhaustive list of MPE outcomes 'What' aspects of MPE care are important to patients and healthcare professionals? Qualitative interviews will be conducted with patients with MPE, their family members and pleural clinicians (n=20).

The transcripts will be analysed using the framework method.

Existing published outcomes from MPE clinical trials will be collated with outcomes generated in interviews to form an exhaustive list. WP2: Developing an MPE Core Outcome Set 'Which' outcomes should be collected in all future MPE trials? WP2a) Outcomes generated in WP1 will be used to develop a draft Delphi survey, co-designed with PPI collaborators.

The survey will be evaluated for usability and clarity through cognitive 'think aloud' interviews (n = 16) to reduce potential participation response burden.

Content analysis will be conducted on interview transcripts and survey responses with findings used to finalise the survey. WP2b) International stakeholders (n=200) will then be surveyed. Participants will rate the importance of each MPE outcome over two rounds.

This will be followed by a consensus meeting, in which a representative sample of the survey group (n=30) will review and finalise outcomes to be included in the COS. WP3: Reviewing MPE Outcome Measurement Instruments (OMIs) 'How' should information be measured in future MPE trials? WP3 will support effective implementation of the STANDOUT MPE COS.

To achieve this, the construct of each outcome to be measured will be defined. The quality of available OMIs in the literature will then be reviewed. Two focus groups will also be conducted with patients (n=14) to determine patient acceptability of these OMIs.

Outputs Three peer-reviewed papers will be published from this study and findings will be presented at three conferences: Qualitative Health Research Network Conference, British Thoracic Society meeting and European Respiratory Society international congress. I will also develop a STANDOUT-MPE website to host key study information.