116 Chapter 4 Auteurs Target population Purpose Outcomes Recommended measure (Disease specific) Recommended measure (Generic) van Balen et al. (2021)† Persons with hemophilia Clinical care 1. Cure 2. Impact of disease on life expectancy 3. Ability to engage in normal daily activities 4. Severe bleeding episodes 5. Number of days lost (work or school) 6. Chronic pain 7. Complications 8. Sustainability of physical functioning 9. Social functioning 10. Mental health 3. HAL Use of transportation, Self-care, Household tasks 6. PROBE Chronic pain 8. HAL Lying down/ sitting/kneeling/standing, functions of the legs, function of the arms 8. HJHS 9. Haemo-QoL-A: Role functioning 10. Haemo-QoL-A: Emotional impact 3. PROMIS Self-efficacy for managing chronic conditions – managing daily activities 6. PROMIS Pain intensity, Pain interference 8. PROMIS Physical functioning, Physical functioning for samples with mobility aid users 9. PROMIS Ability to participate in social roles and activities, Self-efficacy for managing social interactions 10. PROMIS Anxiety, Depression, General life satisfaction, Positive affect The health outcomes in bold represent patient-reported outcomes that can only be reported by the patients themselves. * Dover et al. propose a core outcome set for children and adults with hemophilia. The table only shows the proposed set for adults with hemophilia. † van Balen et al. provide recommendations on measurement instruments for both adults and children and for high- and low-income societies. The table only shows the recommended measurement instruments for adults in high income societies. Abbreviations: QoL, quality of life; PROBE, Patient Reported Outcomes; Burdens and Experiences; EQ-5D-5L, EuroQol 5-dimensions 5-level; EQ-VAS, EuroQol visual analog scale; HJHS, Hemophilia Joint Health Score; EQ-5D, EuroQol 5-dimensions; HAL, Hemophilia Activities List; PROMIS, Patient-Reported Outcomes Measurement Information System; Haemo-QoL-A, Hemophilia Quality of Life Questionnaire for Adults.
RkJQdWJsaXNoZXIy MTk4NDMw