Patient-Reported Outcome Measures in Liver and Gastrointestinal Cancer Randomized Controlled Trials

Carolin Winkelmann; Anna Mezentseva; Bodo Vogt; Thomas Neumann

doi:10.3390/ijerph20136293

Patient-Reported Outcome Measures in Liver and Gastrointestinal Cancer Randomized Controlled Trials

Int J Environ Res Public Health. 2023 Jul 4;20(13):6293. doi: 10.3390/ijerph20136293.

Authors

Carolin Winkelmann^{1

2}, Anna Mezentseva¹, Bodo Vogt^{1

2

3}, Thomas Neumann^{1

2

4

5}

Affiliations

¹ Chair in Empirical Economics, Otto-von-Guericke-University Magdeburg, Universitätsplatz 2, 39106 Magdeburg, Germany.
² Research Campus STIMULATE, Otto-von-Guericke-University Magdeburg, Otto-Hahn-Straße 2, 39106 Magdeburg, Germany.
³ Chair in Health Economics, Institute of Social Medicine and Health Systems Research, Otto-von-Guericke-University Magdeburg, Leipziger Str. 44, 39120 Magdeburg, Germany.
⁴ Chair in Health Services Research, Department of Digital Health Sciences and Biomedicine, School of Life Sciences, University of Siegen, Am Eichenhang 50, 57076 Siegen, Germany.
⁵ University Department of Neurology, Otto-von-Guericke-University Magdeburg, Leipziger Str. 44, 39120 Magdeburg, Germany.

Abstract

Objective: For many years, outcomes such as mortality and morbidity were the standard for evaluating oncological treatment effectiveness. With the introduction of patient-reported outcome measures (PROMs), the focus shifted from a mere extension of a patient's life or release from disease to the improvement of a multilayered concept of health, decisively affecting life satisfaction. In this study, we deal with the topic of PROMs in liver and gastrointestinal randomized controlled trials.

Results: The final database included 43 papers reporting results of randomized controlled trials (RCTs) for liver or gastrointestinal cancer interventions where one of the primary or secondary outcomes was a health-related quality of life measure. The most often used PROM was the European Organization for Research and Treatment of Cancer Quality of Life questionnaire (EORTC QLQ-C30) for both liver cancer and gastrointestinal cancer (in 62% of gastrointestinal cancer studies and 57% of liver cancer studies). For the gastrointestinal cancer group, the QLQ-STO22, a cancer-specific extension of the QLQ-C30, was the second most commonly used PROM. In liver cancer, the generic PROM Short Form 36 and the EORTC QLQ-HCC18, a cancer-specific extension of the QLQ-C30, were the second most commonly used PROMs.

Conclusion: We found that RCTs often do not include comprehensive quality-of-life measures. When quality of life is part of an RCT, it is often only a secondary outcome. For a holistic view of the patient, a stronger integration and weighting of patient-reported outcomes in RCTs would be desirable.

Keywords: RCT; gastrointestinal cancer; health-related quality of life (HRQoL); liver cancer; patient-reported outcome (PRO); patient-reported outcome measures (PROMs); quality of life (QoL); randomized controlled trial.

Publication types

Review
Research Support, Non-U.S. Gov't

MeSH terms

Gastrointestinal Neoplasms* / therapy
Humans
Liver Neoplasms* / therapy
Patient Reported Outcome Measures
Quality of Life
Randomized Controlled Trials as Topic
Surveys and Questionnaires

Grants and funding

The work of this paper is partly funded by the European Regional Development Fund under the operation number ‘ZS/2016/04/78123’ as part of the initiative “Sachsen-Anhalt WISSENSCHAFT Schwerpunkte” and by the Federal Ministry of Education and Research within the Research Campus STIMULATE under grant number ‘13GW0095C’.