Patient-Reported Outcomes in Pleural Effusions: A Systematic Review.

Area of Science:

• Pulmonary Medicine
• Clinical Trials Methodology
• Patient-Reported Outcomes

Background:

• Pleural effusions significantly impact patient quality of life, causing breathlessness and reduced activity.
• Patient-reported outcome measures (PROMs) are crucial for evaluating interventions in clinical trials for pleural effusions.
• Assessing the validity and responsiveness of PROMs is essential for accurate outcome measurement.

Purpose of the Study:

• To systematically review PROMs used in pleural effusion drainage clinical trials.
• To identify assessed variables, responsiveness to interventions, and validation status of PROMs.
• To determine if a minimal clinically important difference (MCID) has been established for PROMs in this patient group.

Main Methods:

• Systematic review of clinical trials identified through Medline, EMBASE, Emcare, and CINAHL databases.
• Data extraction from 29 identified clinical trials that utilized PROMs for pleural effusion drainage.
• Analysis of 16 different PROMs for their usage, assessed variables, responsiveness, and validation.

Main Results:

• Sixteen distinct PROMs were identified, with unidimensional breathlessness measures being most common.
• The visual analogue scale for dyspnoea (VASD) demonstrated responsiveness and is the only PROM validated with a defined MCID.
• Multidimensional PROMs showed inconsistent responsiveness, and no single PROM captured all key outcomes.

Conclusions:

• A variety of PROMs are employed in pleural effusion clinical trials, but none comprehensively address all outcomes.
• Unidimensional breathlessness measures, particularly VASD, are responsive to pleural fluid drainage.
• There is a critical need for validated, responsive PROMs that capture key outcomes for patients with pleural effusions.