Background: Currently, it is unknown which patient-reported outcomes are important for patients with autosomal inherited bleeding disorders.  

Aims: The purpose of this study is to systematically review the available literature assessing patient-reported outcomes and their measurement methods in autosomal inherited bleeding disorders.

Methods: The Embase, Medline ALL, Web of Science Core Collection, Cochrane Central Register of Controlled Trails and Google Scholar databases were searched from inception until August 1^st  2020 using a combination of registered and non-registered terms. Studies on patient-reported outcomes in von Willebrand disease, inherited platelet function disorders and rare factor deficiencies were included.

Results: The systematic literature search yielded a total of 1959 non-duplicate references, of which 21 articles met the inclusion criteria. Figure 1 shows the risk of bias assessment of the included studies. Three studies were assessed as having poor quality and therefore a high risk of bias. The remaining articles had a fair quality rating. The majority of included studies focused on patients with von Willebrand disease. Patients with von Willebrand disease were reported to have lower health-related quality of life compared to the general population. Overall, this trend was especially visible in the following domains: vitality, physical and social functioning and pain. Women with inherited bleeding disorders generally scored lower on health-related quality of life compared to men, especially those with heavy menstrual bleeding. Health-related quality of life was associated with bleeding assessment tool scores in most studies. Patients with joint bleeds or heavy menstrual bleeding experienced an increased level of pain.

Risk of bias assessment using the quality assessment tool for observational cohort and cross-sectional studies from the National Heart, Lung, and Blood Institute 

Conclusions: Our systematic review highlights the urgent need for studies using established, standardised measurement methods to analyse patient-reported outcomes in patients with autosomal inherited bleeding disorders, in order to effectively capture all aspects of disease. Future studies should especially focus on women and the association between bleeding phenotype and patient-reported outcomes.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/patient-reported-outcomes-in-autosomal-inherited-bleeding-disorders-a-systematic-literature-review/