Shannon van Hoorn

115 Harmonizing patient-reported outcome measurements in inherited bleeding disorders with PROMIS 4 Table 1: Differences across outcome sets for persons with hemophilia. Auteurs Target population Purpose Outcomes Recommended measure (Disease specific) Recommended measure (Generic) Iorio et al. (2018) Persons with hemophilia Gene therapy trails 1. Frequency of bleeds 2. Factor activity level 3. Duration of factor VII or factor IX expression 4. Chronic pain 5. Utilization of healthcare system (direct costs) 6. Mental health Not mentioned Not mentioned Skinner et al. (2018) Persons with hemophilia and persons without a bleeding disorder Research 1. General health problems 2. Use of mobility devices 3. Use of pain medication 4. Acute pain 5. Chronic pain 6. Difficulties with activities of daily living 7. Work/school life 8. Surgical history 9. Concurrent medical problems 10. Mobility 11. Self-care 12. Usual activities 13. Pain/discomfort 14. Anxiety/depression 15. Global health Not mentioned 1-9. PROBE questionnaire 10-14. EQ-5D-5L 15. EQ-VAS Dover et al. (2020)* Persons with hemophilia Research and clinical care 1. Total bleeding events 2. Mobility 3. Self-care 4. Usual activities 5. Pain/discomfort 6. Anxiety/depression 7. Treatment adherence 8. Joint health 9. Number and location of bleeds per unit time 8. HJHS 2-6. EQ-5D

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