THE
TRUTH IS...
Despite advancements in Hemophilia A therapies, bleeds are still happening1-3
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
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 A2
~50%
(N=70)
ON FVIII MIMETIC EXPERIENCED ≥1 SPONTANEOUS BLEED during 18 months of follow up2,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 patients ABR data from medical records among a subset of patients who had bleed data for ≥ 1 year. The study is limited to patients seeking hemophilia care, with potential bias toward those with severe illness or higher healthcare interaction. Additionally, the cross-sectional design may limit changes over time.1
aA real-world study of 70 patients treated at the Israeli National Hemophilia Center with severe hemophilia A receiving FVIII mimetic therapy for at least 18 months. Patients were evaluated for bleeding episodes.
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 (eg, FISH, HJHS).
ARE PATIENTS REPORTING ALL BLEEDS?
In a real-world, 2-year study of 472 patients with hemophilia1
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 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
Bentley 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 three or more spontaneous bleeds have occurred within a consecutive 6-month period.3
NEARLY
60%
of patients had
target joints5
According to real-world data from the CHESS study in 949 adults with hemophilia A. Patient data were collected retrospectively between January and April 20155
Compromised joint health
calls for intervention
Joint bleeds Still cause3,6,7:
REDUCED MOBILITY
CHRONIC PAIN
TARGET JOINTS
JOINT COMORBIDITIES
DO THEY FEEL
PROTECTED?
In a real-world observational study of 472 patients with hemophilia1
33%
(N=75)
On FVIII
MIMETIC
47%
(N=83)
ON FVIII
PROPHYLAXIS
FELT ANXIOUS ABOUT BLEED PROTECTION AT LEAST SOMETIMES1
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.
The objective of this real-world, observational survey was to assess the nature of treatment burden and annualized bleed rates (ABR) for people with hemophilia A and B in the United States. From June 6th 2020 to June 15th 2022, 472 patients with hemophilia responded to a survey that captured concerns around protection from bleeding episodes. Additional cross-sectional data on treatment burden were collected via a validated questionnaire (Hemophilia Treatment Experience Measure [Hemo-TEM]) by PicnicHealth. PicnicHealth collects and extracts medical records on behalf of patients and invites them to participate in recurring outcome surveys. Patients were recruited to the PicnicHealth research platform using digital marketing, direct referrals, and community and affiliate partnerships.
Nikola lives with Hemophilia A
WHAT ARE PATIENTS
GIVING UP?
In a real-world study of 348 patients with hemophilia1
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 is limited to patients seeking hemophilia care, with potential bias toward those with severe illness or higher healthcare interaction. Additionally, the cross-sectional design limits tracking 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.
- 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.
- WFH Shared Decision Making Tool: The Role of the Healthcare Team. World Federation of Hemophilia. Last reviewed: August 2024. Accessed September 8, 2024. https://www1.wfh.org/publications/files/pdf-2371.pdf
- 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, et al. 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.
ABR=annualized bleeding rate; CHESS=Cost of Haemophilia in Europe: a Socioeconomic Survey; EHL=extended half-life; FVIII=Factor VIII; SHL=standard half-life.
