226 Chapter 8 (Table 1). This set consists of the following clinical and patient-reported outcomes: 1) frequency of bleeding episodes in a set time period, 2) number of severe and life-threatening bleeding episodes, 3) complications, 4) pain, 5) joint health, 6) physical functioning, 7) social functioning, 8) mental health, 9) intensity of menstrual bleeding, and 10) impact of menstruation/heavy menstrual bleeding on activities of daily life. With the addition of disease or symptom-specific health outcomes, this set is comparable to the generic outcome set as defined by the Dutch OutcomeBased Healthcare program for implementation in medical specialist care. This outcome set namely consists of the health outcomes: 1) quality of life, 2) perceived health, 3) participation in social roles, 4) physical functioning, 5) mental functioning, 6) fatigue, and 7) pain 8. To facilitate continuous learning and quality improvement across medical conditions, I suggest implementing the generic outcome set as defined by the Outcome-Based Healthcare program, supplemented with the health outcomes related to bleeding frequency, complications, joint health, and menstruation in routine clinical care for patients with inherited bleeding disorders. Recommended patient-reported outcome measures Many different PROMs have been used in literature to measure patientreported outcomes in patients with inherited bleeding disorders. In our systematic review, I identified fifteen different PROMs (Chapter 2), of which the SF-36 has been utilized the most frequently. In patients with hemophilia, the most frequently used PROMs are the generic SF-36 and EuroQoL Five Dimensions (EQ-5D), followed by the hemophilia-specific Haemo-A-QoL 9-11. Since the SF-36 is the most frequently used PROM, it has been used as a reference in studies aiming to evaluate the feasibility, psychometric properties, and/or relevance of the PROMIS instruments for patients with hemophilia 12,13. When combining the results of these previous studies in hemophilia with the findings of our own validation study (Chapter 5), I can conclude that the PROMIS CATs Physical Function, Pain Interference, Fatigue, and Satisfaction with Social Roles and Activities are feasible, valid and reliable alternatives to the SF-36 for all patients with inherited bleeding disorders 12. In addition, the PROMIS CATs Anxiety, Depression, and Ability to Participate in Social Roles and Activities are a feasible, valid, and reliable alternatives to the SF-36 for patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders.
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