Hereditary Factor X Deficiency in America Survey¹

The “Hereditary Factor X Deficiency (HFXD) in America Survey” is the first known study of its kind to directly assess patient and caregiver HFXD burden and impact on quality of life (QoL)

View Dr. Brian Branchford of Versiti Blood Research Institute reviewing Survey results

Summary of Results

Graphic showing that based on the number of patient-reported bleeds over four weeks, nearly 75% of hereditary factor X deficiency patients surveyed have an annualized bleed rate of 12 to >120 bleeds.

“Based on the number of patient-reported bleeds over four weeks, nearly 75% of hereditary factor X deficiency patients surveyed have an annualized bleed rate of 12 to >120 bleeds. It’s important to note that rates this high would be considered absolutely unacceptable for other more common bleeding disorders, such as hemophilia. Clinicians can better help patients with factor X deficiency by frequently assessing bleed control and quality of life factors—and using that information to adjust and optimize treatment.”
Dr. Brian Branchford
Lead Expert for the Survey and physician scientist at the world-renowned
Versiti Blood Research Institute in Milwaukee, WI

HFXD was shown to be associated with considerable patient burden and lower QoL
Approximately half of patients reported using single-factor replacement as monotherapy or in combination with other treatments
The study showed that HFXD impacts caregivers’ QoL negatively; however, they also reported high positive aspects of caregiving, including self-worth and inner strength

Improvements are needed to ensure prompt diagnosis and appropriate management to ultimately improve QoL and decrease the burden faced by patients with HFXD and their caregivers

Study Description

This prospective cross-sectional study analyzed survey data from 30 patients diagnosed with HFXD and 38 caregivers of patients with HFXD. Eligible patients were identified by Hemophilia Treatment Centers (HTCs), physicians, specialty pharmacies, social media, and patient advocacy groups. Study subjects completed a Web-based survey developed from validated general and disease-specific patient-reported outcomes measures adapted for HFXD. Expert input was obtained from physicians, caregivers, and health outcomes researchers. Survey items addressed patient journey, QoL, humanistic, and unmet needs.

Results – Survey Items

Ease of Obtaining an Accurate Diagnosis

Findings: Only about half of patients and caregivers found that HFXD had been easily/accurately diagnosed

Insight: To support earlier, accurate diagnosis, increase familiarity with diagnostic indicators and clinical presentations of HFXD

Graphic showing responses from patients and caregivers describing the process of getting diagnosed with hereditary factor X deficiency. 46.7% of patients and 57.9% of caregivers selected Very easy/somewhat easy; 20.0% of patients and 10.5% of caregivers selected Neither easy nor difficult; 23.3% of patients and 26.3% of caregivers selected Very difficult/somewhat difficult; 10.0% of patients selected Do not recall; 5.3% of caregivers selected Not involved.

Patient Survey: How would you describe the process of getting accurately diagnosed with hereditary factor X deficiency originally (such as going to doctor appointments, receiving tests or labs, etc.)?

Caregiver Survey: How would you describe the process of getting your person with a bleeding disorder accurately diagnosed originally (such as going to doctor appointments, receiving tests or labs, etc.)?

Current Treatments (Patient Survey)

Findings: Although NHF’s MASAC guidelines and expert consensus recommend single factor X concentrate for HFXD, only about half of patients were treated with single factor replacement therapy

Insight: Assess bleeding control, satisfaction, and QoL on current therapy. Consider single-factor replacement therapy per NHF’s MASAC guidelines

Graphic showing responses from patients reporting the types of treatment they receive for hereditary factor X deficiency (HFXD): 53.3% receive single-factor replacement (SFR); 26.7% receive fresh frozen plasma (FFP); 6.7% receive blood transfusions; 6.7% receive Prothrombin Complex Concentrate (PCC); 10.0% receive tranexamic acid; 10.0% receive aminocaproic acid; 13.3% receive oral contraceptives; and 6.7% report receiving no treatment for HFXD.

Approximately half of all patients reported using SFR as monotherapy or in combination with other treatments while 1 in 4 reported the use of FFP.

SFR: Single-factor replacement
FFP: Fresh frozen plasma
PCC: Prothrombin Complex Concentrate

Treatment Schedule (Patient Survey)

Findings: Regular prophylaxis was used most often as a treatment schedule

Insight: Reassess treatment type and frequency and consider regular prophylaxis, taking into account the severity of disease and bleed control

On what type of schedule are you currently receiving treatments?

Please select all that apply.

Graphic showing responses from patients reporting the types of treatment schedules they are currently receiving: 25.0% receive episodic or on-demand treatment; 7.1% receive intermittent prophylaxis; 71.4% receive regular prophylaxis; 21.4% receive treatment before surgery; and 0.0% report receiving Other schedules.

Of those on regular prophylaxis, 30% receive prophylaxis monthly, 15% biweekly, 25% weekly, 20% twice weekly, and 10% other.

Quality of Life (QoL) and Well-being (Patient Survey)

Findings: Patients with HFXD scored lower on QoL based on their SF-12 mean physical score and mean mental score versus the US average of 50 for either one. Patients also perceived overall lower well-being (HWBI)

Insight: To support earlier, accurate diagnosis, increase familiarity with diagnostic indicators and clinical presentations of HFXD

12 Item Short-Form
Health Survey (SF-12)

Graphic showing responses from patients assessing their overall quality of life (Physical and Mental) and well being with scores from 0 to 100 (higher is better): the physical component was assessed at 45.13%; the mental component received a score of 48.79%; and the Hemophilia Well-Being Index (HWBI) received a score of 20.04%.

Hemophilia Well-Being Index (HWBI)

The 12 Item Short-Form Health Survey (SF-12) is used to assess overall quality of life (Physical and Mental) and can be compared to the general population. Higher scores (0-100) indicate greater functioning. The Hemophilia Well-Being Index (HWBI) assesses patient well-being. Patients are asked about how their life has been negatively affected by hemophilia. Higher scores (0-32) indicate lower well being^3,4

Impact of Treatment on Bleed Control, Satisfaction, and QoL/Burden

Findings: Over 35% of patients and more than 40% of caregivers were not completely satisfied with the patient’s treatment in controlling bleeding

Insight: Assess efficacy and optimize treatment regimen in patients with HFXD to improve bleed control, satisfaction, and QoL

How would you best describe your experience with your treatments for hereditary factor X deficiency? (Patient Survey)

Graphic showing survey responses from patients about their experience with treatments for hereditary factor X deficiency: 78.6% strongly agree that treatment helps control bleeding; 64.3% strongly agree they are satisfied that treatment is controlling bleeding; and 71.4% strongly agree that treatment helps improve their quality of life.

How would you best describe your experience with your person with a bleeding disorder’s treatments for hereditary factor X deficiency? (Caregiver Survey)

Graphic showing survey responses from caregivers for patients with hereditary factor X deficiency: 55.3% say treatment helps control bleeding all of the time, 29.0% that treatment helps control bleeding most of the time, and 15.8% that treatment helps control bleeding some of the time; 57.9% are satisfied that treatment is controlling bleeding all of the time, 23.7% most of the time, 15.8% some of the time, and 2.6% none of the time; 47.4% agree that treatment helps improve the overall caregiver burden all of the time, 36.8% most of the time, 7.9% some of the time, 2.6% a little of the time, and 5.3% prefer not to answer.

Bleeding Episodes (Patient Survey)

Findings: Over the course of 4 weeks, only about 1 in 4 patient’s bleeding was fully controlled

Insight: Reassess treatment type and frequency and consider regular prophylaxis, taking into account the severity of disease and bleed control

Most patients (63.3%, N=19) reported having

1 to 3

bleeding episodes in the 4 weeks prior to taking the survey

while 26.7% (N=8) patients reported no bleeding episodes

Menorrhagia Impact Questionnaire (MIQ) (Patient Survey)

Findings: Nine patients completed the MIQ. Over 20% (2/9) of patients reported very heavy blood loss with their most recent period. Two-thirds (6/9) of patients reported that menstrual bleeding limited their physical activities moderately or worse. Over half (5/9) reported it also limited their social/leisure activities moderately or worse

Insight: In women with heavy menstrual bleeding, consider ruling out a bleeding disorder as a potential cause

The MIQ measures the impact of heavy menstrual bleeding on QoL, including menstrual blood loss, limitations in social/leisure activities, physical activities, and ability to work²

Disease Impact (Caregiver Survey)

Findings: HFXD negatively impacts caregiver burden based on the Hemophilia Caregiver Impact (HCI) mean Burden Summary score. However, caregivers also reported experiencing positive aspects of caregiving (self worth and inner strength)

Insight: Consider caregiver burden and QoL when assessing how to optimize each patient’s treatment

Burden Summary
Total Responses	37
Raw Mean Score (SD)	15.88 (4.63)
Highest possible score = 35; higher scores relate to higher (worse) impact.

Positive Emotion Summary
Total Responses	37
Mean Score (SD)	4.20 (0.99)
Highest possible score = 5; higher scores relate to more positive aspects of caregiving.

The HCI, which was adapted to HFXD, is a 36-item questionnaire that assesses the negative and positive aspects of caring for people with HFXD.⁵

NHF, National Hemophilia Foundation; MASAC, Medical and Scientific Advisory Council.

References: 1. Huang SP, et al. Hereditary factor X deficiency (HFXD) burden of disease and impact on quality of life in patients and caregivers: Findings from the HFXD in America Survey. Poster presented at 2022 Thrombosis and Hemostasis Summit of North America (THSNA). August 16–18, 2022. Chicago, Illinois. 2. Bushnell BBM, et al. Menorrhagia impact questionnaire: Assessing the influence of heavy menstrual bleeding on quality of life. Curr Med Res Opin. 2010;26(12):2745-2755. 3. Ware J, et al. A 12-item short-form health survey. Construction of scales and preliminary tests of reliability and validity. Med Care. 1996;34(3):220–233. 4. Remor E. Development and psychometric testing of the Hemophilia Well-being Index. Int J Behav Med. 2013;20(4):609-617. 5. Schwartz CE, et al. Measuring hemophilia caregiver burden: validation of the Hemophilia Caregiver Impact measure. Qual Life Res. 2017;26:2551-2562.

Indications and Usage for COAGADEX

COAGADEX, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children with hereditary factor X deficiency for:

Routine prophylaxis to reduce the frequency of bleeding episodes
On-demand treatment and control of bleeding episodes
Perioperative management of bleeding in patients with mild, moderate and severe hereditary factor X deficiency

Contraindication for COAGADEX

COAGADEX is contraindicated in patients who have had life-threatening hypersensitivity reactions to COAGADEX.

Important Safety Information for COAGADEX

Allergic type hypersensitivity reactions, including anaphylaxis, are possible with COAGADEX. If symptoms occur, patients should discontinue use of the product immediately, contact their physician, and administer appropriate treatment.

The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking COAGADEX for the development of inhibitors by appropriate clinical observations and laboratory tests.

COAGADEX is made from human plasma and may contain infectious agents, e.g. viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of COAGADEX.

In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with COAGADEX were infusion site erythema, infusion site pain, fatigue and back pain.

Please see complete Prescribing Information for COAGADEX.

Hereditary Factor X Deficiency in America Survey1