Shannon van Hoorn

241 Addendum A Willebrand disease, inherited platelet function disorders and rare bleeding disorders, and how they can be measured in routine care. Chapter 2 gives an overview of which health outcomes might be impacted in this patient population. This review showed that patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders experience a decreased general health, vitality, physical and social functioning, and an increased level of pain compared to the general population. Moreover, this review demonstrated that women with bleeding disorders who experience heavy menstrual bleeding report a lower health-related quality of life compared to those without heavy menstrual bleeding. In Chapter 3, I provide a consensus-based set of health outcomes which are important and should be measured in routine care for patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders as seen from the perspective of the patient, caregivers, and healthcare professionals. Dutch patients, caregivers and healthcare professionals all agreed on the importance of the following six health outcomes: 1) number of bleeding episodes per year that require treatment, 2) total number of life-threatening bleeding episodes, 3) severity of bleeding episode, 4) lifethreatening complications, 5) intensity of menstrual bleeding, and 6) impact of menstrual bleeding on daily life. In addition to these health outcomes, the separate groups (e.g., patients and caregivers, and healthcare professionals) identified several additional health outcomes which they deemed important for this patient population. Patients and caregivers identified health outcomes related to menstruation and the impact of the bleeding disorder on their daily life to be of importance, while healthcare professionals prioritized outcomes related to treatment side effects, joint health, and pain. Recommended patient-reported outcome measures Subsequently to identifying which health outcomes are important to be routinely collected in the care for patients with inherited bleeding disorders, consensus needs to be reached on how to measure these relevant health outcomes. In Chapter 2, I found that fifteen different patient-reported outcome measures (PROMs) have been utilized to measure patient-reported outcomes in patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders. The most frequently used PROMs were the generic Short Form-36 (SF-36), followed by the Health Utilities Index, and disease-specific Hemophilia Activities List.

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