129 PROMIS in VWD, IPFDs and RBDs 5 Additionally, the domain scores can be aggregated in two summary scores: a physical component summary and a mental component summary. The component scores were standardized using normative data from the United States general population with a mean score of 50 and a standard deviation of 10 40. Patient characteristics Patient characteristics were collected from the WiN-Pro, TiN and RBiN studies and included age, sex, type of bleeding disorder, and disease severity. Patients with VWD were categorized as having a severe bleeding disorder if they had von Willebrand factor (VWF) levels (VWF:antigen and/or VWF:collagen binding and/or VWF:activity) ≤10 U/dL and/or factor (F)VIII:C ≤20 U/dL. Patients with VWD and VWF levels between 10-30 U/dL and/or FVIII:C between 20-40 U/dL were classified as having a non-severe bleeding disorder. Patients with the IPFDs Glanzmann trombasthenia and Bernard Soulier Syndrome, and patients with RBDs with a severe deficiency, or with a grade III bleeding were classified as having a severe bleeding disorder 41,42. Grade III bleeding was defined as spontaneous major bleeding such as intramuscular hematomas requiring hospitalization, hemarthrosis, central nervous system, gastrointestinal, and umbilical cord bleeding 41. The classification of VWD, IPFDs and RBDs is consistent with the disease severity classification used in the WiN, TiN, and RBiN study 3,10. In addition, the use of prophylactic treatment was collected at the moment of PROMs completion. Information on the patients’ race or ethnicity was not collected due to Dutch privacy regulations. Statistical analysis All statistical analyses were conducted in R version 2022.07.2+576. Descriptive statistics were used to analyze demographics and clinical characteristics of the participants. All completed questionnaires were included in the analyses. Feasibility Feasibility of the PROMIS measures and the SF-36v2 for use in clinical practice was assessed based on the number of completed items (mean [SD], range) and the presence of floor and ceiling effects. For all measures, floor and ceiling effects were defined to be present if more than 15% of the participants reported the lowest or highest possible score 43. We determined the PROMIS measures to be feasible if the mean number of items and/or the presence of floor/ceiling effects was lower on the PROMIS measures than the SF-36v2.
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