223 General discussion 8 In Chapter 3, I provide a consensus-based set of health outcomes which are important and should be measured in routine clinical 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 episodes, 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. 6 Recommended patient-reported outcomes 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 identified which patient-reported outcome measures (PROMs) have previously been used to measure patient-reported outcomes in patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders. I found that fifteen different PROMs have been utilized, including generic, disease-specific, and self-developed PROMs. The most frequently encountered PROMs were the generic Short Form-36 (SF-36), followed by the Health Utilities Index, and disease-specific Hemophilia Activities List 5. In Chapter 4, I introduced and described the possible advantages of using patient-reported outcomes measurement information system (PROMIS) instruments to assess the identified relevant patient-reported outcomes. PROMIS has several advantages compared to more traditional PROMs including the application of Computer Adaptive Tests (CATs). Using CAT, the next item presented to the person completing the PROM depends on the responses on earlier items. In theory, the use of CAT lowers the burden of outcome assessment by administrating a limited number of more relevant questions with a higher reliability. Moreover, PROMIS instruments are
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