29 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 were found in items related to sample size justification, exposure assessment prior to outcome measurements and statistical analyses. Diseases studied The majority of included articles focused solely on von Willebrand disease (VWD) (n=13) 22,24-28,34,35,37-41. Other studies included patients with inherited platelet function disorders (n=2) 23,33, coagulation factor deficiencies (n=2) 32,42, or a combination of different autosomal inherited bleeding disorders (n=4) 29-31,39. Measurement methods Studies used different measurement methods including predefined, routinely assessed PROMs (n=11, 54%), self-developed PROMs (n=5, 23%), or a combination of both (n=2, 9%). Three studies used semi-structured questionnaires assessing PROs in an interview format (n=3, 14%). The most frequently used predefined PROMs were the Short Form-36 (SF-36) and the Health Utilities Index (HUI). Of the 21 included studies, two studies validated the applied PROMs specifically in patients with an inherited bleeding disorder 26,28. Assessment of the development and psychometric properties of each PROM are summarized in Table 1. Figure 2: Publication year of included studies.
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