Shannon van Hoorn

27 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 other databases’ syntax (Supplement 1). Key terms include congenital blood clotting disorders, blood clotting deficiency, patient-reported outcome and quality of life. The literature search was performed in Embase, Medline ALL, Web of Science Core Collection, Cochrane Central Register of Controlled Trails and Google Scholar. All databases were searched from inception until 14 August 2020. Study selection Two reviewers (ESvH and MEH) independently screened the articles on potential eligibility. Disagreements between the reviewers were discussed until uniform consensus was reached. English articles reporting on all different types of patient-reported outcomes in both adult and pediatric patients with autosomal inherited bleeding disorders including von Willebrand disease, inherited platelet function disorders and coagulation factor deficiencies, were included. Articles solemnly focusing on bleeding symptoms or bleeding assessment tools were not included in this study, since bleeding symptoms are not necessary patient-reported; they can also be reported by someone other than the patient himself/herself, for example in case of grading of bleeding after surgery. Case studies, commentaries, editorials, conference abstracts, economic evaluations and articles about acquired bleeding disorders were excluded. According to the COSMIN methodology, articles were excluded if less than 80 percent of the research population consisted of patients with autosomal inherited bleeding disorders. The reference lists of included studies identified by the search were checked for further relevant studies. Data assessment For each included study, the following information was collected: study design, characteristics of the patient population, mean or median age at study inclusion, used PROMs and measured PRO’s. In case the psychometric properties of a PROM were assessed, including its acceptability, internal consistency, reliability, validity and responsiveness this was also reported. Two reviewers (ESvH and MEH) read and abstracted each article; a third reviewer (WA) checked table entries for accuracy with regard to the original articles. Data were reviewed descriptively. Risk of bias assessment A single reviewer from the team (ESvH) assessed risk of bias of included publications using the National Institutes of Health (NIH) quality assessment tool for observation and cross-sectional studies. 21 Two other reviewers (MEH

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