THE
TRUTH IS...
Despite advancements in Hemophilia A therapies, bleeds are still happening1-4
aBased on a real-world study with 354 people with hemophilia A. Analysis is limited to ABRs in medical records and PROs from the survey and may have been limited by medical record discrepancies, recall bias, and potential reporting bias for bleeds.1
ABR=annual bleed rate; PROs= patient-reported outcomes.
Luke lives with Hemophilia A
aBased on a real-world study with 472 people with Hemophilia. Analysis is limited to ABRs in medical records and PROs from the survey and may be limited by medical record discrepancies, recall bias, and potential reporting bias for bleeds.1
DESPITE ADVANCEMENTS
IN THERAPY
In a real-world study in Israel of 70 patients with severe hemophilia A3
~50%
(N=70)
ON FVIII MIMETIC EXPERIENCED ≥1 SPONTANEOUS BLEED after 18 months of follow-up3,a
In a real-world study with 62 physician responses1
42%
(N=40)
AND
50%
(N=28)
ON SHL AND EHL PROPHYLAXIS EXPERIENCED ≥1 BLEED IN A YEAR, RESPECTIVELY1,b
According to physicians reporting ABR data from medical records of a subset of patients who had bleed data for ≥1 year. The study was limited to patients seeking hemophilia care, with potential bias toward those with severe illness or higher health care interaction. Additionally, the cross-sectional design may limit changes over time.1
aData from a real-world observational study including patients with severe HA or HAwI, treated at the Israeli National Hemophilia Center, who had received FVIII mimetic therapy for at least 18 months, and experienced at least 1 spontaneous bleed.
bThe 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).1
ARE PATIENTS REPORTING ALL BLEEDS?
In a real-world, 2-year study of 354 patients with hemophilia A1
Patients reported more bleeds in a survey than were recorded in their medical records1
This discrepancy may mean patients are not reporting all bleeds, warranting the need for deeper discussions1,4
Analysis was limited to ABRs in medical records and PROs from the survey and may be limited by medical record discrepancies, recall bias, and potential reporting bias for bleeds.1
Bentlee lives with hemophilia A (and mom Teilei)
JOINT BLEEDS ARE STILL PREVALENT5
A target joint in this study was defined as a joint with known chronic synovitis, with study investigators given discretion to apply additional criteria.5 The World Federation of Hemophilia defines a target joint as a single joint where 3 or more spontaneous bleeds have occurred within a consecutive 6-month period.4
NEARLY
60%
of patients had
target joints5
According to real-world data from the CHESS study of 949 adults with hemophilia A. Patient data were collected retrospectively between January and April 2015.5
Compromised joint health
calls for intervention
Joint bleeds Still cause4,6,7:
REDUCED MOBILITY
CHRONIC PAIN
TARGET JOINTS
JOINT COMORBIDITIES
PATIENTS CAN BECOME TRAPPED IN A VICIOUS CYCLE OF PHYSICAL INACTIVITY8
Reduced physical activity can lead to weight gain, which may then lead to an increased load on joints. That load can then be followed by further bleeds and mobility problems.8
DO THEY FEEL
PROTECTED?
In a real-world observational study of 354 patients with hemophilia A1,2
33%
(N=75)
On FVIII
MIMETIC
47%
(N=83)
ON FVIII
PROPHYLAXIS
Of Patients WITH SEVERE HEMOPHILIA A FELT ANXIOUS ABOUT BLEED PROTECTION AT LEAST SOMETIMES1,2
Response to survey question: “Because of taking your current treatment, how often do you feel anxious that your current treatment might not be adequately protecting you from bleeding?” Response options were “Never,” “Rarely,” “Sometimes,” “Often,” and “Always".1
Survey responses were collected in a cross-sectional study design. Responses in individuals over time may vary and may not account for continuous patient participation.
Data from an observational study including 354 patients with hemophilia A, conducted from 2022 to 2023, were collected through online surveys as part of the PicnicHealth research platform. Patients responded to supplemental questions assessing anxiety about bleed protection using a 5-point scale: never, rarely, sometimes, often, or always. Limitations may include selection bias due to the cross-sectional study design, variability in individual responses over time and does not account for continuous patient participation.
Nikola lives with hemophilia A
WHAT ARE PATIENTS
GIVING UP?
In a real-world study of 348 patients with hemophilia A1
29%
(N=77)
On FVIII
MIMETIC
18%
(N=103)
ON EHL
PROPHYLAXIS
Admitted limiting activities
to reduce bleeds1
Response to survey question: “What actions do you take to reduce the overall rate of bleeding episodes.” The study was limited to patients seeking hemophilia care, with potential bias toward those with severe illness or higher health care interaction. Additionally, the cross-sectional design limited the ability to track changes over time.1
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 (eg, FISH, HJHS).
Luke lives with hemophilia A
References
- Data on file. Novo Nordisk Inc; Plainsboro, NJ.
- Wheeler AP, Cibelli E, Hanson G, et al. Treatment and disease burden in a cohort of people with haemophilia without inhibitors in the United States. Haemophilia. 2025:e70078.
- Levy-Mendelovich S, Brutman-Barazani T, Budnik I, et al. Real-world data on bleeding patterns of hemophilia A patients treated with emicizumab. J Clin Med. 2021;10(19):4303.
- 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.
- Booth J, Oladapo A, Walsh S, et al. Real-world comparative analysis of bleeding complications and health-related quality of life in patients with haemophilia A and haemophilia B. Haemophilia. 2018;24(5):e322-e327.
- Olasupo OO, Lowe MS, Krishan A, Collins P, Iorio A, Matino D. Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B. Cochrane Database Syst Rev. 2021;8(8):CD014201.
- Knobe K, Berntorp E. Haemophilia and joint disease: pathophysiology, evaluation, and management. J Comorb. 2011;1:51-59.
- Matlary RED, Grinda N, Sayers F, et al; EAHAD Physiotherapists Committee. Promoting physical activity for people with haemophilia in the age of new treatments. Haemophilia. 2022;28(6):885-890.
ABR=annualized bleeding rate; CHESS=Cost of Haemophilia in Europe: a Socioeconomic Survey; EHL=extended half-life; FVIII=Factor VIII; SHL=standard half-life.
