IDENTIFY BARRIERS
for more effective shared decision-making in hemophilia A1,2
Nikola lives with hemophilia A
THE TRUTH IS...
Not all physicians are completely satisfied with current prophylaxis1
In real-world study with 62 physicians,
40%
AND
53%
Reported not being completely satisfied with FVIII Mimetic and EHL Prophylaxis, respectively, due to1,a,b
Are trade-offs being made with current prophylaxis?
aPhysicians responded on a 5-point scale: Completely dissatisfied, Dissatisfied, Neither satisfied nor dissatisfied, Satisfied, or Completely satisfied. The number of physicians who reported being "satisfied" with current treatment is unknown.
bPercentages reflect the top reason given for not being completely satisfied with treatment.
Questions asked: "Please indicate your overall satisfaction with the current treatment in terms of how well it manages the patient’s hemophilia" and "Why are you not completely satisfied with the patient's current prophylactic treatment?"
The study is limited to patients seeking hemophilia care, with potential bias toward those with severe illness or higher health care interaction. Additionally, the cross-sectional design limits tracking changes over time1
The objective of this study was to use the Adelphi Real World (ARW) hemophilia Disease Specific Programme (DSP)™ to gain insights into the real-world characteristics and unmet needs of patients with hemophilia A and B within the current treatment landscape in the United States. From July 2023 to February 2024, 62 physicians who treat and manage patients with hemophilia were identified by ARW through local fieldwork partners. Both the recruited physicians and their patients subsequently completed the survey, which included data from 423 patients (348 with hemophilia A and 75 with hemophilia B). The comprehensive observational survey utilized online physician surveys and self-completion questionnaires for patients and caregivers, employing validated instruments such as EQ-5D-5L, WPAI, Haemo-QoL, and joint health assessment tools (e.g., FISH, HJHS).
Breakthrough bleeds and administration challenges have consequences
Giving up on any activity or interference with activities is a profound compromise patients could be making with current prophylaxis.
DO PATIENTS FEEL LIMITED IN THEIR DAILY LIVES?
In a secondary analysis of data from a real-world study of 431 adults with hemophilia A
Patients on FVIII mimetic (N=43) reported a
29%
Patients on FVIII prophylaxis (n=210) reported a
30%
loss of ability to do daily activities
Response to survey question: "During the past seven days, how much did your hemophilia affect your ability to do your regular activities, other than your paid job?" Patients responded on a scale of 0 to 10, where 0 is 'hemophilia had no effect on my daily activities' and 10 is 'hemophilia completely prevented me from doing my daily activities.
Secondary analysis of data collected as part of a repeated cross-sectional, retrospective observational study of 431 adult patients with hemophilia A in the US. Data collected as part of the CHESS US data platform were used from 2019, 2023, and 2024. Information was collected using two questionnaire forms: a web-based 'case record form' (CRF), completed by physicians, and a 'patient and public involvement element' (PPIE) completed by patients. Limitations included the retrospective, non-interventional design and voluntary participation, which may introduce selection and recall bias.
Patients with hemophilia
may experience emotional and psychosocial burden3
According to a systematic literature review and meta-analysis of 28 studies, people with hemophilia A and B had an increased risk of3,c:
2.64x
Depression
1.74x
Anxiety
2.60x
Depression &
anxiety
cMeta-analysis was used to determine the prevalence of anxiety and depression in people with hemophilia (PWH). Pooled odds ratios for depression diagnosis, anxiety, and anxiety/depression were determined. Comparisons were between PWH and controls. Meta-analysis included 9 studies (depression), 13 studies (anxiety), and 6 studies (anxiety and depression combined) to calculate the risk of depression, anxiety, or anxiety/depression in PWH.3
Challenges with current treatment regimens can lead to feelings of distress, worry, frustration, and anger4-6
Luke lives with hemophilia A.
Key questions to ask your patients
- Do you feel confident that your regimen can fit into your lifestyle? Why or why not?
- Are you concerned or are your patients concerned with high-volume doses with an every-4-week regimen or vein health due to frequent infusions?
- Is bleed protection a concern for you with the current dosing schedule?
- What information will you need to feel more confident about a less frequent dosing schedule?
Lack of patient confidence in treatment outcomes may act as a barrier when it comes to shared decision-making.
CAN DEEPER CONVERSATIONS HELP UNCOVER TRADE-OFFS?
Fostering in-depth discussions with your patients may help uncover physical or emotional burdens.3
Some QUESTIONS TO REVEAL UNSPOKEN COMPROMISES
FVIII MIMETIC
How would you describe your level of confidence around bleed protection with less frequent dosing?
What administration challenges have you experienced, if any?
What factors do you consider when thinking about your preferred dosing interval (ie: every 4-week dosing)?
How does the time it takes to prepare and inject treatment impact you?
FVIII PROPHYLAXIS
What is your level of confidence around bleed protection with SHL or EHL prophylaxis?
How concerned are you with venous access due to frequent infusions?
What role does infusion frequency play in your decisions to start, continue, or stop treatment?
How does the time it takes to prepare and inject treatment impact you?
BRING THEIR TRUTH
TO LIGHT
Consider patient trade-offs when addressing hemophilia A management
IDENTIFY UNEXPRESSED PATIENT GOALS7
Explore what is working and not working with their current management approach to inform future decisions7
Luke lives with hemophilia A
EHL=Extended half-life; FVIII=Factor VIII; SHL=Standard half-life.
References
- Data on file. Novo Nordisk Inc; Plainsboro, NJ.
- Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition [published correction appears in Haemophilia. 2021;27(4):699]. Haemophilia. 2020;26(suppl 6):1-158.
- Al-Huniti A, Reyes Hernandez M, Ten Eyck P, Staber JM. Mental health disorders in haemophilia: systematic literature review and meta-analysis. Haemophilia. 2020;26(3):431-442.
- Thornburg CD, Duncan NA. Treatment adherence in hemophilia. Patient Prefer Adherence. 2017;11:1677-1686.
- Brod M, Bushnell DM, Neergaard JS, et al. Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM). J Patient Rep Outcomes. 2023;7(1):17.
- duTreil S. Physical and psychosocial challenges in adult hemophilia patients with inhibitors. J Blood Med. 2014;5:115-122.
- World Federation of Hemophilia. WFH Shared Decision Making Tool: World Federation of Hemophilia. Last reviewed: August 2024. Accessed September 8, 2024. https://www1.wfh.org/publications/files/pdf-2371.pdf
