Hereditary Factor X Deficiency (HFXD) Survey shows unmet need in patients with rare bleeding disorder

Data presented at the 2022 Thrombosis & Hemostasis Summit of North America

DURHAM, N.C., Aug. 16, 2022 /PRNewswire/ -- Bio Products Laboratory USA (BPL), a leading manufacturer of plasma-derived protein therapies, today presented initial data from the Hereditary Factor X Deficiency (HFXD) in America Survey at the Thrombosis and Hemostasis Summit of North America (THSNA) in Chicago, IL. 

The HFXD in America Survey is the first survey of its kind to directly assess the long and difficult journey from diagnosis to treatment for patients and caregivers suffering from Hereditary Factor X Deficiency, as well as seeks to better understand the significant disease burden, quality of life issues and other factors that impact overall care and well-being.  HFXD is a rare bleeding disorder affecting 1 in 1 million individuals and this innovative survey provides valuable insight into this rare disease. 

According to the survey's lead expert, Brian Branchford, MD, Associate Medical Director of the Versiti Medical Sciences Institute, Associate Investigator at the Versiti Blood Research Institute, and Associate Professor of Pediatrics, Hematology/Oncology at the Medical College of Wisconsin, the survey fills a critical gap in knowledge and understanding about patients and caregivers impacted by this rare orphan disease.  "The impact of rare coagulation disorders on patients and their caregivers is often underrepresented.  In addition to clinical data, it is critically important that we better understand how these disorders impact patients' health and well-being.  The HFXD in America Survey is an important step along that path for clinicians who provide care for patients with HFXD."  

The survey included a national, cross-sectional sample of patients with Hereditary Factor X Deficiency and their associated caregivers.  Participants were recruited through Hemophilia Treatment Centers (HTCs), rare bleeding disorder patient advocacy groups, and social media.  Overall, 30 patients and 38 caregivers completed the survey, which represents robust input from approximately 10% of the estimated patients with HFXD in the United States.

While the majority of patients in the survey reported receiving treatment after their diagnosis with HFXD, their current treatments varied significantly.  Despite expert consensus recommendations advising clinicians to use single-factor replacement therapy for hereditary bleeding disorders^1,2, only about half of patients in this survey reported treatment with single factor replacement therapy either as monotherapy or in combination with other treatments. Furthermore, approximately 1 in 4 patients in the survey reported receiving fresh frozen plasma (FFP).  Perhaps of concern, many patients reported still having bleeding episodes in the four weeks prior to taking the survey, suggesting a need to better optimize their treatment plan. 

Overall quality of life (QoL) and well-being were lower for patients with HFXD versus the general population, and caregivers also reported the negative impact of HFXD on QoL, although caregivers did also report high positive aspects of caregiving, including self-worth and inner strength.

"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."

BPL looks forward to continuing the analysis of this important data and publishing further insights that may contribute to helping improve the overall care and quality of life for patients with HFXD and their caregivers.  Additional data from the HFXD Survey in America will be presented at the National Hemophilia Foundation (NHF) 74th Annual Bleeding Disorders Conference in Houston, Texas later this month.  

1. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 272. MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Other Bleeding Disorders. National Hemophilia Foundation.  https://www.hemophilia.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-272-masac-recommendations-concerning-products-licensed-for-the-treatment-of-hemophilia-and-other-bleeding-disorders    Accessed August 12, 2022.

 2.  Giangrande P, Seitz R, Behr-Gross ME, Berger K, Hilger A, Klein H, Schramm W, Mannucci PM. Kreuth III: European consensus proposals for treatment of haemophilia with coagulation factor concentrates. Haemophilia. 2014 May;20(3):322-5. doi: 10.1111/hae.12440. PMID: 24731129. 

Recognizing the importance of plasma and with many years of experience in the industry, BPL supplies high-quality plasma derived medicines to meet the needs of clinicians, patients, and customers globally. Headquartered in the United Kingdom, with US offices in Durham, NC, and plasma collection centers across the United States, we are dedicated to producing medicines for the treatment of immune deficiencies, bleeding disorders and infectious diseases as well for critical care. BPL invests in the latest R&D, technology and manufacturing methods, and continuously adapts to ensure that we continue to serve all our stakeholders effectively. For more information visit https://www.bplgroup.com or https://www.bpl-us.com.

BPL consists of two operating divisions — BPL Plasma and BPL Therapeutics. BPL Plasma, headquartered in Austin, Texas and operating in the USA, collects plasma from donors in around 28 centers across the U.S. BPL Plasma employs over 900 staff to support the needs of donors and to ensure high-quality plasma collection in all their centers. Plasma collection is regulated by both FDA and MHRA, and BPL Plasma follows industry guidelines. BPL Plasma operates plasma facilities, staffed with trained personnel, dedicated to supporting donors through the process that leads to the donation of plasma. Plasma is shipped to the headquarters of BPL Therapeutics in Elstree, United Kingdom. The plasma is fractionated, purified, and filled through the efforts of our over 1,000 employees involved in production, quality, R&D, commercial, customer services, and administrative activities. BPL's plasma-derived medicines are commercially available in the U.K., USA, and 30 plus other countries around the world through our network of local affiliates and distribution partners.

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SOURCE Bio Products Laboratory USA