46 Chapter 2 Author, (year), country Study design and characteristics of population (n; gender; disease type; age) PROMs Measured PROs PROs and conclusions* Rae, C., et al. (2013). Canada 39 Prospective, single center 417 patients with different inherited bleeding disorders (75.5% female) • VWD: 359 • Other bleeding disorders (all female): 56 Age (years) • range: 12-84 HUI3 • HRQoL • Daily activities • Patients with different type or severity of disease have similar HRQoL (p=0.914). • Women with VWD have lower HRQoL compared to women with other inherited bleeding disorders (p=0.017) and men with VWD (p=0.039). • Women aged from 20 to 84 years have lower HRQoL compared to women with other inherited bleeding disorder and normative data (p<0.05). • Women with VWD and HMB have similar HRQoL compared to women with other inherited bleeding disorder and HMB (p=0.100). • Women with HMB have lower HRQoL compared to women with no HMB (p<0.001), especially on the domains of cognition (p<0.001) and pain (p<0.002). • Women with HMB have more interference of HMB with daily activities compared to women with no HMB (p<0.001). • Women with iron deficiency have similar HRQoL compared to women with no iron deficiency (p=0.459). Sumner, M. and J. Williams (2004). USA 37 Prospective, multicenter 79 patients with VWD (58% female) • Type 3: 79 Age (years) • mean: 24.2 • range: 1-60 Ad hoc questionnaire • Participation • Approximately 40% of patients lost days from work or school, the majority caused by bleeding or other VWD related complications.
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