Rare Factor X Deficiency Survey Shows Disparity in Care
Initial data from the survey were presented at the recent Thrombosis and Hemostasis Summit of North America and NHF’s Bleeding Disorders Conference last week in Houston.
A newly released survey of individuals with Hereditary Factor X Deficiency (HFXD) and caregivers of those with HFXD yielded noteworthy insights related to treatment and quality of life (QoL).
The initial data gleaned from HFXD American Survey were presented at the recent Thrombosis and Hemostasis Summit of North America in Chicago, IL. The survey was funded by Bio Products Laboratory (BPL), which manufactures the coagulation factor X (Human) product Coagadex®.
Coagadex® is a plasma derived concentrate that was first approved by the U.S. Food and Drug Administration in 2015. It is indicated for adults and children with HFXD for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment, and control of bleeding episodes, and for the perioperative management of bleeding in patients with mild and moderate HFXD.
The survey recruitment effort, which encompassed hemophilia treatment centers, patient advocacy groups, and social media, ultimately yielded 68 survey participants (30 with HFXD, 38 were caregivers). The feedback provided by participants, particularly that related to treatment, is eye opening and suggests often disparate and suboptimal utilization of available therapies.
While the majority of patient participants reported being treated after their HFXD diagnosis, the treatment approach amongst participants was inconsistent. A new BPL press release reported that despite expert consensus recommendations advising clinicians to use single-factor replacement therapy for hereditary bleeding disorders, only about half of patient participants indicated treatment with single factor replacement therapy either as monotherapy or in combination with other treatments. In addition, approximately one in four patients reported receiving fresh frozen plasma. Further, “many patients” reported still having bleeding episodes in the four weeks prior to taking the survey.
“We have definitely come a long way in treating patients with HFXD,” said Eric Wolford, PharmD, Vice President of Global Medical for BPL, “but the survey demonstrates significant gaps and opportunities to improve care and treatment.”
Survey responses also indicated that overall QoL and well-being were lower for HFXD patients when compared to the general population. While caregivers did report that HFXD contributed to a negative impact on QoL they also conveyed “high positive aspects of caregiving, including self-worth and inner strength.”
Additional data from the HFXD Survey were presented at NHF’s 74th Bleeding Disorders Conference last week in Houston.
Learn more about Factor X Deficiency.
Source: BPL press release/ PRNewswire (Cision) dated August 16, 2022