Patient-reported outcomes in autosomal inherited bleeding disorders: A systematic literature review

Erasmus MC, University Medical Centre Rotterdam, Department of Public Health, Rotterdam, The Netherlands. Erasmus MC - Sophia Children's Hospital, University Medical Centre Rotterdam, Department of Paediatric Haematology, Rotterdam, The Netherlands. Erasmus MC, Erasmus University Medical Centre Rotterdam, Department of Haematology, Rotterdam, The Netherlands. Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Department of Paediatric Haematology, Amsterdam, The Netherlands. Van Creveldkliniek, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands. Radboud University Medical Centre, Department of Haematology, Nijmegen, The Netherlands.

Haemophilia : the official journal of the World Federation of Hemophilia. 2022
AIM: Currently, it is unknown which patient-reported outcomes are important for patients with autosomal inherited bleeding disorders. Therefore, 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 14 August 2020. Studies on patient-reported outcomes in patients with von Willebrand disease, inherited platelet function disorders and coagulation factor deficiencies were included. RESULTS Twenty-one articles met the inclusion criteria. Three studies were assessed as having poor quality, and therefore a high risk of bias. Nineteen studies had fair quality rating. Different measurements methods were used, ranging from predefined to self-developed questionnaires. The majority of included studies focused on von Willebrand disease. Patients with von Willebrand disease reported 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 scored lower on health-related quality of life compared to men, especially women with heavy menstrual bleeding. Patients with joint bleeds or heavy menstrual bleeding reported an increased level of pain. CONCLUSION Patients with autosomal inherited bleeding disorders report lower health related quality of life, especially those with joint bleeds or heavy menstrual bleeding. Numerous measurement methods are used in patients with autosomal inherited bleeding disorders, highlighting the need for studies using established, standardized measurement methods.
Study details
Study Design : Systematic Review
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine