Unmet Needs in Hemophilia B Patients
Patients with Hemophilia B often have substantial functional limitations and a reduced health-related quality of life, indicating significant unmet needs in this patient population
- Prophylaxis is the standard of care for people with severe hemophilia B1
- Despite prophylactic treatment, challenges persist in managing hemophilia B2,3
- Real-world evidence has shown that patients with hemophilia B often face challenges with treatment efficacy (ie, bleed protection, joint health), physical and treatment-related burden, along with a reduced quality of life due to their disease
Study design
Data displayed on this webpage is sourced from peer-reviewed literature and real-world sources. These real-world sources include:
Adelphi Hemophilia Disease Specific Programme III is a cross-sectional survey with retrospective data collection and analysis, involving both patient-led and physician-led data collection methods. Includes medical record data for 374 HA and 75 HB patients (US). Objectives include describing real-world hemophilia patient demographics, clinical unmet needs, humanistic burden, and healthcare resource utilization.
PicnicHealth – US captures and structures information from the entire medical record, enriched with direct to patient PRO surveys. Includes electronic medical record data for 354 HA and 118 HB patient Objectives include assessing cross-sectional treatment burden using Hemo-TEM, physical and mental health using PROMIS-29, describing ABRs, bleeds and corresponding treatment patterns using survey and medical record data by patient subgroups, disease severity, joint health and medication usage.
Adelphi Bespoke (2024) is a non-interventional, cross-sectional, multi-national patient survey, capturing PROs and patient experience, and basic clinical data from the patient perspective. Includes online survey data for 79 HA and 21 HB patients (US only). Objectives include understanding the patient and caregiver perspective on the disease and its treatment burden, characterization and exploration of the remaining clinical, humanistic and economic unmet needs, describing the patient and caregiver perspective on physical, emotional and social burden.
Unmet Needs
Many patients with hemophilia B treated with prophylactic FIX remain insufficiently protected from bleeds, leading to clinical complications and a lack of patient satisfaction
Efficacy:
Almost half of patients with hemophilia B (n=59) reported experiencing one or more bleed annually, despite prophylactic treatment, with the number of bleeds increasing with disease severity4
According to a real-world survey of physicians, more than two-thirds of physicians were not completely satisfied with their patients' current prophylactic treatment (EHL or SHL), with efficacy being the primary driver for lower satisfaction.5
Physicians reported:
were unsatisfied with their patients' EHL treatment (n=19)
were not satisfied because of efficacy (n=6)
were unsatisfied with their patients' SHL treatment (n=20)
were not satisfied because of efficacy (n=8)
Bleed protection may vary due to FIX peaks and troughs with the more time spent below 1% being associated with a higher risk of bleeding6,7,8
Joint health
According to real-world data from the CHESS and CHESS II studies, over half of adults with hemophilia B on prophylactic treatment (n=287) reported having one or more target joint9, and about one-third of patients reported having a history of joint surgery10
Many people with hemophilia B without inhibitors experience an unresolved burden from the disease and its treatment
Treatment burden:
In a real-world observational study of 105 hemophilia B without inhibitor patients, some reported being inconvenienced by medication storage, preparation, and administration3
2 in 10 patients on SHL treatment (n=9)
>3 in 10 patients on EHL treatment (n=36)
Patients are required to complete 30 steps to prepare and self-administer factor treatment, according to Hemophilia of Georgia11
Patients with hemophilia B may have difficulty complying with their medication, with physicians (n=81) reporting that only
patients with hemophilia B
patients with hemophilia B
are completely adherent to their prophylactic factor therapy, based on a real world observational study including 81 patients with hemophilia B12
Physical burden:
Patients with hemophilia B can experience discomfort related to their treatment including13
brusing
physical discomfort
pain
soreness
(n=65)
Patients with hemophilia B experience moderate to severe impacts on mobility and activity due to their disease14
report problems with mobility
report problems with usual activities
report problems with pain and discomfort
(n=299)
Venous health:
Due to frequent IV infusions, people with hemophilia B may experience major complications to venous health15
experience
venous scarring
experience blown
or ruptured veins
(n=112)
Major long-term treatment complications can arise, including:16
- Blood loss
- Blood clot
- Bruising
- Swelling
- Permanent artery or vein weakness
- Increased risk of injury
Patients requiring CVADs are at risk of potentially life-threatening infections
of implanted catheters may lead to potentially life-threatening conditions, including bacterial endocarditis17,18
of patients experienced CVAD-related deep vein thrombosis with the risk of loss of potential access sites19,20
Despite improvements in treatment, many patients with hemophilia B and their caregivers continue to experience a reduction in quality of life with substantial impacts on their emotional, psychological, and social well-being
Daily activities:
Patients and caregivers of patients with hemophilia B report that the disease, and the treatment of, negatively impacts their work21
of patients report an impact on their work (n=299)
of caregivers report an impact on their work (n=150)
Some patients with hemophilia B treated with EHL also report that their disease negatively impacts the progression of their education22
of patients with moderate hemophilia B (n=3)
of patients with severe hemophilia B (n=10)
Emotional well-being:
Patients and caregivers of patients with hemophilia B report having anxiety and/or depression due to the disease14
of patients report having anxiety/depression (n=299)
of caregivers report having depression (n=150)
of caregivers report having anxiety (n=150)
Protection from breakthrough bleeding remains a concern among ~20% of patients with hemophilia B without inhibitors despite prophylactic treatment17
Understand
The burden of treatment in patients with hemophilia B
View the infographic
Discover
Coming soon
An adult hematologist’s perspective on the evolving needs in hemophilia B management
Explore
Coming soon
A pediatric nurse’s views on the care challenges faced by people with hemophilia B
ABR, Annualized Bleeding Rate; CHESS / CHESS II, Cost of Haemophilia in Europe: A Socioeconomic Survey; CVAD, Central Venous Access Device; EHL, Extended Half-Life factor; FIX, Factor IX; HA, Hemophilia A; HB, Hemophilia B; Hemo-TEM, Hemophilia Treatment Experience Measurement; IV, Intravenous; PRO, Patient-Reported Outcome; PROMIS, Patient-Reported Outcomes Measurement Information System; SHL, Standard Half-Life factor; UPMC, University of Pittsburgh Medical Center
References:
- Srivastava A, et al. Haemophilia. 2020;26(suppl 6):1-158.
- Windyga J, et al. J Haematol. 2024;113(5):631-640.
- Jiménez-Yuste V, et al. Presented at: American Society of Hematology Annual Meeting; December 7–10, 2024; San Diego, CA, USA. Abstract 5077.
- Data on file. Novo Nordisk Inc.; Plainsboro, NJ.
- Data on file. Novo Nordisk Inc.; Plainsboro, NJ.
- Role of new prolonged half-life clotting factors in hemophilia. National Hemophilia Foundation. Accessed September 20, 2025. https://www.bleeding.org/sites/ default/files/document/files /phlbrochure.pdf
- Soucie JM, et al. Blood Adv. 2018;2(16):2136-2144.
- Miesbach W, et al. Eur J Haematol. 2024;112:339-349.
- Booth J, et al. Haemophilia. 2018;24(5):e322-e327
- Burke T, et al. Orphanet J Rare Dis. 2021;16(1):521.
- Hemophilia of Georgia [online]. Steps to prepare and infuse factor. Hemophilia of Georgia. Accessed September 20, 2025. https://www.hog.org/handbook/article/3/35/steps-to-prepare-and-infuse-factor
- Thakkar S, et al. Presented at: American Society of Hematology Annual Meeting; December 9-12, 2023; San Diego, CA, USA. Abstract P2616.
- Data on file. Novo Nordisk Inc.; Plainsboro, NJ.
- Buckner TW, et al. Eur J Haematol. 2018;100(6):592-602.
- Data on file. Novo Nordisk Inc.; Plainsboro, NJ.
- Vascular trauma. UPMC. Accessed September 20, 2025. https://www.upmc.com/services/heart-vascular/conditions/vascular-trauma
- Castaman G, Linari S. Expert Rev Hematol. 2018;11:7:567-576.
- Hothi D, et al. Haemophilia. 2001;7:5:507-510.
- Journeycake J, et al. Blood. 2001;98:6:1727-1731.
- Ettingshausen C, et al. Blood. 2002;99(4):1499-1500.
- Cutter S, et al. Eur J Haematol. 2017;98(suppl 86):18-24.
- Data on file. Novo Nordisk Inc.; Plainsboro, NJ.
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