132 Chapter 5 RESULTS Patient characteristics During the study period, 174 patients with VWD, 84 patients with IPFD and 80 patients with an RBD were approached to participate in this study. In total, 111 patients completed one or multiple PROMs resulting in an overall response rate of 33% (response rate: VWD 39%, IPFDs 21%, and RBDs 33%). Of these 111 patients, 105 completed all PROMs. The median age of the patients was 57 (IQR 44-67) years, and 81 (74%) were female (Table 1). The majority of the patients were diagnosed with VWD (60%), had a non-severe bleeding disorder (86%), and did not use prophylactic treatment at the moment of inclusion (100%). Table 1: Patient characteristics. Overall N=111 von Willebrand disease N= 67 Inherited platelet function disorders N=18 Rare bleeding disorders N=26 Gender Female 81 (73%) 49 (73%) 15 (83%) 17 (65%) Male 30 (27%) 18 (27%) 3 (17%) 9 (35%) Age Median (IQR) 57 (44-67) 59 (46-69) 54 (38-60) 58 (44-66) Severity* Non-severe 96 (86%) 61 (91%) 15 (83%) 20 (77%) Severe 15 (14%) 6 (9%) 3 (17%) 6 (23%) Prophylactic treatment Yes 0 0 0 0 No 108 (100%) 67 (100%) 15 (100%) 26 (100%) Missing 3 0 3 0 * Patients with von Willebrand factor (VWF) levels (VWF:Ag and/or VWF:CB and/or VWF:Act) ≤10 U/ dL and/or factor VIII:C ≤20 U/dL, Glanzmann thrombasthenia, Bernard Soulier Syndrome, and rare bleeding disorders with a grade III bleeding and/or undetectable factor activity were categorized as severe bleeding disorders. Abbreviations: VWF:Ag, von Willebrand factor antigen; VWF:Act, von Willebrand factor activity; VWF:CB , von Willebrand factor collagen binding; VWF, von Willebrand factor. Due to the low response rate and heterogeneity within the patients with IPFDs and RBDs, we present the results of patients with VWD. The results for patients with an IPFD or RBD can be found in the supplementary information (Supplementary Tables S5–S19).
RkJQdWJsaXNoZXIy MTk4NDMw