Shannon van Hoorn

39 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* Blaauwgeers, M.W, et al. (2020). The Netherlands 23 Prospective, multicenter 156 confirmed or suspected patients with CPD (81% female) B-IPQ SF-36 • HRQoL • Physical and social functioning • Pain • General health • Vitality • Patients have lower HRQoL compared to normative data, especially in the domains physical functioning, pain, general health, vitality and social functioning (p≤ 0.01). • Patients with confirmed CPD have similar HRQoL compared to patients with suspected CPD. • Women have more pain compared to men. • Bleeding phenotype is not correlated with HRQoL. Chi, C., et al. (2010). UK 29 Retrospective, single center 42 patients with different inherited bleeding disorders and HMB (all female) • VWD: 20 • IPFD: 13 • HA carrier: 3 • FXI def.: 6 Age (years): • mean: 16 • range: 13-19 Ad hoc questionnaire • HRQoL • General health • Daily activities • Dysmenorrhea • Before treatment, around half of patients had low HRQoL, especially on the domain of general health and daily activity and after treatment, almost no patient had low HRQoL. • Before treatment, 88% of patients had dysmenorrhea and after treatment, 17% of patients had dysmenorrhea. • Before treatment, almost half of patients reported missing school because of their menstruation, and after treatment, none of the patients reported missing school.

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