Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an... Show moreBackground: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments.Results: Persons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables.Conclusion: A consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes. Show less
Balen, E.C. van; Krawczyk, M.; Gue, D.; Jackson, S.; Gouw, S.C.; Bom, J.G. van der; Sawatzky, R. 2019
Introduction and Aim The British Columbia Adult Haemophilia Team recently adopted a patient-centred care approach. The team presented visual information on an individual's pharmacokinetic profile... Show moreIntroduction and Aim The British Columbia Adult Haemophilia Team recently adopted a patient-centred care approach. The team presented visual information on an individual's pharmacokinetic profile and bleed history and encouraged patients to participate in treatment decisions. This qualitative study explored how this approach changed patients' understanding of haemophilia and how it facilitated them to make treatment decisions. Methods We interviewed 18 males with mild, moderate or severe haemophilia, using a convenience sample from the adult haemophilia clinic at St. Paul's hospital in Vancouver, Canada. Interviews were recorded and transcribed verbatim and analyzed using descriptive content analysis. Results Most participants reported that reviewing visual information with the Clinic Team helped them in their communication with their care providers during their annual review clinic appointment. Despite this improved communication, for some the most important feature of their treatment was that they had switched from on-demand treatment to prophylactic treatment in recent years and were able to prevent bleeds. Almost half of the participants reported that the visual information presented increased their understanding of haemophilia and the pharmacokinetics of coagulation factor. Three patients improved their treatment adherence or had changed their prophylaxis schedules based on this. Most participants felt that they were involved in decision-making about their treatment schedule, which they appreciated. On the other hand, two participants thought the Clinic Team should make these decisions. Conclusion Participants perceived the patient-centred prophylaxis approach helpful because it enhanced communication with the Clinic Team, increased their understanding of haemophilia and pharmacokinetics of coagulation factor and facilitated treatment decisions. Show less
