Clinical trials of pneumonia management assess heterogeneous outcomes and measurement instruments

Objectives: To inform clinical practice guidelines, randomized controlled trials (RCTs) of the management of pneumonia need to address the outcomes that are most important to patients and health professionals using consistent instruments, to enable results to be
compared, contrasted, and combined as appropriate. This systematic review describes the outcomes reported in clinical trials of pneumonia management and the instruments used to measure these outcomes.
Study Design and Setting: Based on a prospective protocol, we searched MEDLINE/PubMed, Cochrane CENTRAL and clinical trial registries for ongoing or completed clinical trials evaluating pneumonia management in adults in any clinical setting. We grouped reported outcomes thematically and classified them following the COMET Initiative’s taxonomy. We describe instruments used for assessing each outcome.
Results: We found 280 eligible RCTs of which 115 (41.1%) enrolled critically ill patients and 165 (58.9%) predominantly noncritically ill patients. We identified 43 distinct outcomes and 108 measurement instruments, excluding non-validated scores and questionnaires.
Almost all trials reported clinical/physiological outcomes (97.5%). Safety (63.2%), mortality (56.4%), resource use (48.6%) and life impact (11.8%) outcomes were less frequently addressed. The most frequently reported outcomes were treatment success (60.7%), mortality (56.4%) and adverse events (41.1%). There was significant variation in the selection of measurement instruments, with approximately two-thirds used in less than 10 of the 280 RCTs. None of the patient-reported outcomes were used in 10 or more RCTs.
Conclusion: This review reveals significant variation in outcomes and measurement instruments reported in clinical trials of pneumonia management. Outcomes that are important to patients and health professionals are often omitted. Our findings support the need for a rigorous core outcome set, such as that being developed by the European Respiratory Society.

Contributors

Alexander G. Mathioudakisa, Markus Fally, Jan Hansela, Rebecca C. Robeya,
Faiuna Haseeba, Thomas Williamse, Ahmed Koutaa, Tobias Weltef, Dan G. Woottong,
Mike Clarkeh, Grant Waterer, Paul Darka, Paula R. Williamson, Jørgen Vestbo,
Timothy W. Felton, Pneumonia Outcomes Group

Publication

Journal: Journal of Clinical Epidemiology
Volume: 164
Issue:
Pages: 88 - 95
Year: 2023
DOI: 10.1016/j.jclinepi.2023.10.011

Further Study Information

Current Stage: Completed
Date:
Funding source(s): AGM, JH, RCR, PD, JV, and TWF were supported by the NIHR Manchester Biomedical Research Centre (NIHR203308). AGM was supported by an NIHR Clinical Lectureship in Respiratory Medicine. JH was supported by an NIHR Academic Clinical Fellowship in Intensive Care Medicine. PD was supported by an NIHR Senior Investigator’s award (NIHR203745). DGW is supported by an NIHR Advanced Fellowship (NIHR300669).


Health Area

Disease Category: Infectious disease, Lungs & airways

Disease Name: Pneumonia (community-acquired), Pneumonia (hospital-acquired), Pneumonia (ventilator-associated)

Target Population

Age Range: 18

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

Study Type

- Systematic review of outcomes measured in trials

Method(s)

- Systematic review

We identified potentially relevant RCTs using a structured search strategy consisting of controlled vocabulary and free search terms describing pneumonia, along with filters that
included the Cochrane sensitivity and precision maximising filter for RCTs (available in the online appendix). We searched MEDLINE/PubMed, the Cochrane Register of Controlled Trials (CENTRAL), the U.S. National Library of Medicine Clinical Trials Register (clinicaltrials.gov) and the World Health Organisation (WHO) International Clinical Trials Registry Platform for articles published from 1st January 2010 to 13th December 2021.