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)
Summary of Results
“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.”
- 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
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
Current Treatments (Patient Survey)
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)
On what type of schedule are you currently receiving treatments?
Quality of Life (QoL) and Well-being (Patient Survey)
12 Item Short-Form
Health Survey (SF-12)
Hemophilia Well-Being Index (HWBI)
Impact of Treatment on Bleed Control, Satisfaction, and QoL/Burden
How would you best describe your experience with your treatments for hereditary factor X deficiency? (Patient Survey)
How would you best describe your experience with your person with a bleeding disorder’s treatments for hereditary factor X deficiency? (Caregiver Survey)
Bleeding Episodes (Patient Survey)
Menorrhagia Impact Questionnaire (MIQ) (Patient Survey)
Disease Impact (Caregiver Survey)
|Raw Mean Score (SD)
|Highest possible score = 35; higher scores relate to higher (worse) impact.
|Positive Emotion Summary
|Mean Score (SD)
|Highest possible score = 5; higher scores relate to more positive aspects of caregiving.
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.