Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.

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 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.

Patient-centred care in haemophilia: Patient perspectives on visualization and participation in decision-making.

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 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.

Clinical practice of personalized prophylaxis in hemophilia: Illustrations of experiences and benefits from two continents.

These cases of people with hemophilia (PWH) illustrate the importance of understanding the patient's expectations and desires and adapting treatment to meet these needs, in addition to traditional clinical targets. Population PK modeling and FVIII products with improved PK profiles provide the opportunity to individualize care and improve long-term outcomes.

Men, Masculinities, and Hemophilia.

Hemophilia is a congenital bleeding disorder that predominantly affects men. Home intravenous replacement of missing clotting factor is the most effective treatment; however, the uptake of preventative treatment (also known as prophylaxis) varies among men with hemophilia. The purpose of the current qualitative study was to describe the connections between masculinities and men's (n = 11) experiences of hemophilia across varying age groups. The inductively derived findings revealed bleed-related joint pain as the primary prompt for men to treat or seek medical help. Many men reported experiencing a high number of bleed-related injuries in adolescence, particularly in high school, oftentimes as a result of engaging in idealized masculine physical activities. Though the limitations imposed by hemophilia were contested by most men early on in their lives, as men grow older more conservative approaches were employed both in terms of treatment and activity to reduce the potential for residual bleed-related disabilities. Overall, the results indicate that men with hemophilia may benefit from peer and professional education about recognition, prevention, and optimal treatment of bleeds. Furthermore, masculine ideals act as important context in which men navigate hemophilia management practices and may facilitate contesting or conceding behaviors. Masculine ideals of strength and control may be garnered to facilitate optimal hemophilia management practices.

Joining the patient on the path to customized prophylaxis: one hemophilia team explores the tools of engagement.

BACKGROUND: The relationship between hemophilia team interventions and achievement of optimal clinical outcomes remains to be elucidated. The British Columbia Hemophilia Adult Team has previously reported results of a comprehensive approach to individualize prophylaxis that has resulted in substantially reduced bleeding rates. In order to facilitate knowledge exchange and potential replication, it was important to gain a thorough understanding of the team's approach. METHODS: A focus group of the British Columbia Hemophilia Adult Team was conducted to identify specific roles and processes that might be contributing to the prophylaxis regimen outcomes in this clinic. The focus group consisted of two workshops; one to describe the individual and collective roles of the clinic team in providing clinical care and guiding patients toward individualized prophylaxis; and the other to describe the patient journey from initial contact through reaching a successful engagement with the clinic. RESULTS: Analysis of the results revealed team roles and processes that underpinned a shared decision-making relationship with the patient with a particular focus on supporting the patient's autonomy. Within this relationship, team focus shifts away from "adherence" toward the process whereby patients design and implement prophylaxis regimens resulting in reduction or elimination of bleeding episodes. LIMITATIONS: Using the current methodology, it is not possible to demonstrate a causal link between specific team processes and improved bleeding rates in patients. CONCLUSION: Through the active support of patient autonomy in all aspects of decisions related to hemophilia management, the British Columbia Hemophilia Adult Team approach de-emphasizes "adherence" as the primary goal, and focuses on a prophylaxis plan that is customized by the patient and aligned with his priorities. Adoption of this comprehensive team approach facilitates shared goals between the patient and the team that may optimize treatment adherence, but more importantly, reduce bleeding rates.

Assessing the reliability and validity of the revised WCCNR stomatitis staging system for cancer therapy-induced stomatitis.

Before developing interventions for stomatitis, nurses require a simple, valid and reliable approach to staging severity. The eight-item WCCNR(R) was previously validated for chemotherapy-induced stomatitis. In this study, the validity and reliability of the WCCNR(R), a shorter three-item tool for staging stomatitis caused by chemotherapy, radiotherapy, or both, was assessed. Pairs of data collectors evaluated 207 patients from 10 Canadian cancer centres. The WCCNR(R) correlated well with the MacDibbs Mouth Assessment (r = 0.44, p = 0.0002 to r = 0.54, p < 0.0001), a standardized tool for staging radiotherapy-induced stomatitis. Agreement between data collectors at five sites was acceptable (kappa = 0.75); three additional sites were close to this target. Findings indicate that the WCCNR is a valid and reasonably reliable tool for staging stomatitis due to cancer therapy.