Treating Hemophilia Depends on Available Types of Clotting Factors

Hemophilia is a rare disorder that causes an individual’s blood to not clot properly, because of a lack of clotting proteins, causing excessive bleeding.

In patients with an extremely low level of clotting factor proteins, hemophilia can cause more serious issues and even be fatal.¹

The disease has 2 types: hemophilia A and B. Hemophilia A, which is more common, is a disease where there is a clotting factor VIII deficiency, whereas hemophilia B is deficient in factor IX. A rarer type, hemophilia C, is caused by low levels of factor XI. ²

Hemophilia A affects about 1 in 5000 male births or approximately 400 babies per year. An estimate of 33,000 males in the United States live with hemophilia, mostly type A, according to recent data from 2012 to 2018.³

The first article published on the disorder dates to 1803, when John Conrad Otto, MD, a physician from the Philadelphia, Pennsylvania, area, wrote about a rare, hemorrhagic bleeding disease that affected mostly men. Following this, in 1828, he and Friedrich Hopff developed the term “haemorrhaphilia,” which was shortened to “haemophilia.” In 1947, Alfredo Pavlovsky, a physician from Buenos Aires, discovered that hemophilia can be categorized into A and B. It was not until the 1920s that other factors of the disorder were discovered.⁴

Treating hemophilia properly depends on the kind of clotting factors that available. An ideal way to replace these clotting factors is through continuous replacement therapies, which use infusions to treat patients. Clotting factors can be made from donated blood or in the laboratory. These are also known as recombinant clotting factors.¹

Plasma-derived and recombinant clotting factors remain the most popular for patients. Some factor VIII recombinant products include Advate, Eloctate, Esperoct, NovoEight, and Xyntha. As for humanized factors, Alphanate, Hemlibra, and Hemofil M are just a few examples.⁵

Amicar, Desmopressin Acetate (DDAVP), and Stimate, are other medications that are similar to hormones produced in the body. Amicar can be administered intravenously or orally, while DDAVP and Stimate can be given intravenously or via a nasal spray.⁴

One of the most recent FDA-approved factors for hemophilia A and B is Sevenfact, or coagulation factor VIIA (recombinant), for use in individuals aged 12 years and older. Sevenfact used at 75 mcg/kg dosing, repeated every 3 hours until bleeding stops, has an active ingredient expressed in genetically engineered rabbits.

The demand for hemophilia drug treatment in Europe and North America has increased in recent years, amid increased awareness of the disease. Growth is seen continuing this year.⁶

References

• Hemophilia. Mayo Clinic. August 7, 2020. Accessed June 17, 2021. https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333
• Types of hemophilia. Indiana Hemophilia & Thrombosis Center. 2021. Accessed June 17, 2021. https://www.ihtc.org/types-of-hemophilia
• Hemophilia B. National Organization of Rare Disorders. Accessed June 17, 2021. https://rarediseases.org/rare-diseases/hemophilia-b/
• History. National Hemophilia Foundation. 2021. Accessed June 17, 2021. https://www.hemophilia.org/bleeding-disorders-a-z/overview/history
• Products licensed in the US. National Hemophilia Foundation. 2021. Accessed June 17, 2021. https://www.hemophilia.org/healthcare-professionals/guidelines-on-care/products-licensed-in-the-us
• Hemophilia drugs market size 2021 is expected to progress at a CAGR of 6% and reach USD 15,830 million by the end of 2025. Intrado GlobeNewswire. May 17, 2021. Accessed June 17, 2021. https://www.globenewswire.com/en/news-release/2021/05/17/2230352/0/en/Hemophilia-Drugs-Market-Size-2021-Is-Expected-to-Progress-at-a-CAGR-of-6-and-Reach-USD-15-830-Million-by-the-end-of-2025.html