229 General discussion 8 Table 1: Relevant outcomes for all patients with an inherited bleeding disorders. Outcomes Definition Patient or clinician reported Recommended measuring instrument 1. Frequency of bleeding episodes in a set time period The total number of bleeding episodes that required treatment during a set time period Patient and clinician reported No recommendation 2. The number of severe and life-threatening bleeding episodes The total number of severe and lifethreatening bleeding episodes across the patient lifetime Patient and clinician reported No recommendation 3. Complications Any health complication caused by the bleeding disorder or by the administration of treatment Clinician reported No recommendation 4. Pain The presence of pain and the interference of pain with activities of daily living Patient reported PROMIS Pain Interference 5. Joint health The presence and degree of joint damage due to joint bleeding Clinician reported Hemophilia Joint Health Score 6 Physical functioning The ability and degree to which a person is able to perform actions that are essential for maintaining independence and carry out activities of daily living Patient reported PROMIS Physical Function 7 Social functioning The degree to which a person is able to maintain and manage interactions with other people and contribute to society Patient reported PROMIS Ability to Participate in Social Roles and Activities, and PROMIS Satisfaction with Social Roles and Activities 8 Mental health Degree of overall well-being, anxiety, depression and satisfaction with life Patient reported PROMIS Anxiety and PROMIS Depression 9 Intensity of menstrual bleeding Number of times a person needs to change their menstrual products per day Patient reported No recommendations 10 Impact of menstruation/ heavy menstrual bleeding on activities of daily living The impact of menstrual bleeding on a person’s ability to engage in activities of daily living (including social relations, work/ school, sleep) Patient reported No recommendations Definitions were adapted based on the definition provided by Skinner et al. (2018), Dover et al. (2020), van Balen et al. (2021), van Hoorn et al. (2023).
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