43 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 Author, (year), country Study design and characteristics of population (n; gender; disease type; age) PROMs Measured PROs PROs and conclusions* Khair, K., et al. (2013). UK 31 Prospective, single center 45 patients with different inherited bleeding disorders (all female) • VWD: 23 • HA carrier: 9 • FXIII def.: 3 • IPFD: 4 • FXI def. : 2 • FX def.: 1 • FVII def.: 1 • FV def.: 1 • GT: 2 • Unknown: 1 Age (years, SD) • mean: 19.8 (5.21) • range: 9-34 Ad hoc questionnaire Focus group • HRQoL • Sport participation • Almost all patients (90%) are not adversely affected by their disease. • 80% of patients is limited in sport participation due to their bleeding disorder. • Around half of patients have concerns about fertility. • A subgroup of patients experience stigmatization, isolation and bullying. Khair K. et al. (2018). UK 33 Prospective, single center 16 patients with CPD (50% female) Age (years) • mean: 11 • range: 6-17 Kids’ ITP Tool Focus group • General health • Psychosocial functioning • Most patients have good general health. • More than half of children are more often than not tired. • Most patients have good psychosocial functioning.
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