Patient-reported outcome measures in adults with hemophilia: A scoping review

Abstract

Background:Hemophilia is a generic bleeding disorder that significantly affects patient quality of life (QoL). Patient-reported outcome measures (PROMs) can capture health outcomes from a patient’s perspective. The identification of PROMs used in adult patients with hemophilia (PwHs) can improve our understanding of patient care needs.
Objective:To examine the current use of PROMs in adult PwHs, evaluate the frequency of PROMs, and summarize the main evaluation dimensions and descriptive data of the included publications.
Methods:In this scoping review, PubMed, EMBASE, The Cochrane Library, Web of Science, Scopus, and EBSCO were searched from January 2000 to June 2023. This review focused on descriptions of the use of PROMs in adult PwHs. The search strategy followed the PRISMA-ScR guidelines.
Results:Of 32 934 sources identified, 394 articles including 56028 patients were included. A wide range of measures was available, and many were used only once. One hundred and fifty-four different PROMs were identified. The most commonly used generic PROMs were the EuroQoL Five-Dimensional Questionnaire (EQ-5D) (n=105, 26.6 %) and the 36-item Short Form General Health Survey (SF-36) (n=95, 24.1 %). The most commonly used hemophilia-specific PROM was the Hemophilia-specific health-related QoL questionnaire (A36 Hemofilia-QoL) (n=15, 3.8 %). However, psychological health has rarely been separately assessed.
Conclusion:This study provides a valuable summary of PROMs to facilitate research on hemophilia. Disease-specific instruments should be used more frequently to capture changes in patients, and psychological health should be considered in future studies.

Ethics approval

Ethics approval was not required for this scoping review as it involved only the analysis of publicly available data and did not involve any direct contact with human subjects or experimental procedures. All data used in this review were obtained from peer-reviewed journals and established databases, ensuring the integrity and reliability of the information and compliance with ethical standards of data use.

Funding

This work was supported by the National Natural Science Foundation of China (No. 72174110).

CRedit authorship contirbution statement

Yingrong Bai: Conceptualization, Formal analysis, Investigation, Methodology, Validation, Visualization, Writing-original draft. Lulu Liu: Conceptualization, Formal analysis, Investigation, Methodology, Visualization, Writing-original draft. Haode Wang: Data curation, Formal analysis, Writing-original draft, Writing-review and editing. Jianglin Wang: Data curation, Formal analysis, Writing-review and editing. Zhao Shi: Supervision, Writing-review and editing. Junchao Feng: Writing-review and editing. Shunping Li: Conceptualization, Funding acquisition, Project adminstration, Resources, Writing-review and editing. All the authors have read and approved the final version of this manuscript.

Declaration of Competing Interest

The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Acknowledgments

There was no external funding source for this project. We thank the editage group for its linguistic assistance during the preparation of this manuscript.

Appendix A. Supplementary material

Supplementary data associated with this article can be found in the online version at doi:10.1016/j.hcr.2024.100007.

Data Availability

Data are available in the article and appendix.

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