Factor VIII (medication)

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Factor VIII
Fviii 2R7E.png
A depiction of factor VIII
Clinical data
Trade names Aafact, Kovaltry, others [1]
Other namesoctocog alfa
AHFS/Drugs.com Monograph
License data
Routes of
administration 		Intravenous (IV)
ATC code
Legal status
Legal status
Identifiers
ChemSpider
  • none

Factor VIII is a medication used to treat and prevent bleeding in people with hemophilia A and other causes of low factor VIII. [6] [7] Certain preparations may also be used in those with von Willebrand's disease. [7] It is given by slow injection into a vein. [6]

Contents

Side effects include skin flushing, shortness of breath, fever, and red blood cell breakdown. [6] [7] Allergic reactions including anaphylaxis may occur. [7] It is unclear if use during pregnancy is safe for the fetus. [8] A purified factor VIII concentrate is made from human blood plasma. [7] A recombinant version is also available. [6] People may develop antibodies to factor VIII such that this medication becomes less effective. [8]

Factor VIII was first identified in the 1940s and became available as a medication in the 1960s. [9] [10] Recombinant factor VIII was first made in 1984 and approved for medical use in the United States in 1992. [11] [12] It is on the World Health Organization's List of Essential Medicines. [13]

Available forms

Factor VIII is delivered by intravenous infusion. [14]

History

This transfer of a plasma byproduct into the blood stream of a hemophiliac often led to the transmission of diseases such as HIV and hepatitis before purification methods were improved. In the early 1990s, pharmaceutical companies began to produce recombinant synthesized factor products, which now prevent nearly all forms of disease transmission during replacement therapy.[ citation needed ]

Society and culture

Economics

The cost of Factor VIII and similar clotting factors has been described as "highly expensive". [14] The cost of the clotting factors is 80% of all medical costs for people with hemophilia. [15] They are so expensive that gene therapy for haemophilia might be less expensive, especially for people with severe hemophilia. [15]

See also

Related Research Articles

<span class="mw-page-title-main">Haemophilia</span> Genetic disease involving blood clotting

Haemophilia, or hemophilia, is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or a decreased level of consciousness.

<span class="mw-page-title-main">Haemophilia A</span> Medical condition

Haemophilia A is a blood clotting disorder caused by a genetic deficiency in clotting factor VIII, thereby resulting in significant susceptibility to bleeding, both internally and externally. This condition occurs almost exclusively in males born to carrier mothers due to X-linked recessive inheritance. Nevertheless, rare isolated cases do emerge from de novo (spontaneous) mutations.

<span class="mw-page-title-main">Haemophilia B</span> Genetic X-linked recessive bleeding disorder

Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency.

<span class="mw-page-title-main">Coagulation factor VII</span> Mammalian protein found in humans

Coagulation factor VII is one of the proteins that causes blood to clot in the coagulation cascade, and in humans is coded for by the gene F7. It is an enzyme of the serine protease class. Once bound to tissue factor released from damaged tissues, it is converted to factor VIIa, which in turn activates factor IX and factor X.

<span class="mw-page-title-main">Factor IX</span> Protein involved in blood clotting in humans

Factor IX is one of the serine proteases of the coagulation system; it belongs to peptidase family S1. Deficiency of this protein causes haemophilia B. It was discovered in 1952 after a young boy named Stephen Christmas was found to be lacking this exact factor, leading to haemophilia.

<span class="mw-page-title-main">Fresh frozen plasma</span> Liquid portion of whole blood

Fresh frozen plasma (FFP) is a blood product made from the liquid portion of whole blood. It is used to treat conditions in which there are low blood clotting factors or low levels of other blood proteins. It may also be used as the replacement fluid in plasma exchange. Using ABO compatible plasma, while not required, may be recommended. Use as a volume expander is not recommended. It is administered by slow injection into a vein.

<span class="mw-page-title-main">Pegaspargase</span> Pharmaceutical drug

Pegaspargase, sold under the brand name Oncaspar, is a medication used in the treatment of acute lymphoblastic leukemia (ALL). Often it is used together with anthracycline, vincristine, and corticosteroids. Pegaspargase can be administered either via an intravenous infusion or a intramuscular injection.

Prothrombin complex concentrate (PCC), also known as factor IX complex, sold under the brand name Kcentra among others, is a combination medication made up of blood clotting factors II, IX, and X. Some versions also contain factor VII. It is used to treat and prevent bleeding in hemophilia B if pure factor IX is not available. It may also be used for reversal of warfarin therapy. It is given by slow injection into a vein.

<span class="mw-page-title-main">Contaminated blood scandal in the United Kingdom</span> The historical contamination of blood products in the UK with HIV and hepatitis C virus

In the 1970s and 1980s, a large number of people – most of whom had haemophilia – were infected with hepatitis C and HIV, the virus that leads to acquired immune deficiency syndrome (AIDS), as a result of receiving contaminated clotting factor products. In England, these were supplied by NHS England. Many of the products were imported from the US.

Moroctocog alfa is a recombinant antihemophilic factor genetically engineered from Chinese hamster ovary (CHO) cell line. Chemically it is a glycoprotein. It is manufactured by Genetics Institute, Inc. and used to control and prevent hemorrhagic bleeding and prophylaxis associated with surgery or to reduce the number of spontaneous bleeding episodes in patients with hemophilia A. It is partially a recombinant coagulation factor VIII since it has an amino acid sequence which compares to the 90 + 80 kDa form of factor VIII (BDDrFVIII). It also has posttranslational modifications which are similar to those of the plasma-derived molecule. It can not prevent hemorrhagic bleeding associated with von Willebrand's disease since it is not a von Willebrand factor.

Recombinant factor VIIa (rfVIIa) is a form of blood factor VII that has been manufactured via recombinant technology. It is administered via an injection into a vein. It is used to treat bleeding episodes in people who have acquired haemophilia, among other indications. There are several disimilar forms, and biosimilars for each. All forms are activated.

Andexanet alfa, sold under the brand name Andexxa among others, is an antidote for the medications rivaroxaban and apixaban, when reversal of anticoagulation is needed due to uncontrolled bleeding. It has not been found to be useful for other factor Xa inhibitors. It is given by injection into a vein.

<span class="mw-page-title-main">Jeanne Lusher</span> American physician

Jeanne Marie Lusher, M.D. was an American physician, pediatric hematologist/oncologist, and a researcher in the field of bleeding disorders of childhood, and has served as the director of Hemostasis Program at the Children's Hospital of Michigan until her retirement on June 28, 2013.

Turoctocog alfa is a recombinant antihemophilic factor VIII used for the treatment of and prophylaxis of bleeding patients with haemophilia A. It is marketed by Novo Nordisk. It was approved in the United States, the European Union, and Japan in 2013.

Cenegermin, sold under the brand name Oxervate, also known as recombinant human nerve growth factor (rhNGF), is a recombinant form of human nerve growth factor (NGF). In July 2017, it was approved in the European Union as an eye drop formulation for the treatment of moderate or severe neurotrophic keratitis in adults.

Susoctocog alfa, sold under the brand name Obizur, is a medication used for the treatment of bleeding episodes in adults with acquired haemophilia, a bleeding disorder caused by the spontaneous development of antibodies that inactivate factor VIII.

Vonicog alfa, sold under the brand names Vonvendi and Veyvondi, is a medication used to control bleeding in adults with von Willebrand disease. It is a recombinant von Willebrand factor.

Efmoroctocog alfa, sold under the brand name Elocta among others, is a medication for the treatment and prophylaxis of bleeding in people with hemophilia A. Efmoroctocog alfa is a recombinant human coagulation factor VIII, Fc fusion protein (rFVIIIFc). It is produced by recombinant DNA technology in a human embryonic kidney (HEK) cell line.

Damoctocog alfa pegol, sold under the brand name Jivi is a recombinant DNA-derived, Factor VIII concentrate medication used to treat hemophilia A.

Efanesoctocog alfa, sold under the brand name Altuviiio, is a medication used for the treatment of hemophilia A.

References

  1. ↑ "Coagulation Factor VIII, Human". www.drugs.com. Archived from the original on 9 January 2017. Retrieved 8 January 2017.
  2. ↑ "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.
  3. ↑ "Prescription medicines: registration of new chemical entities in Australia, 2016". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
  4. ↑ "Prescription medicines: registration of new chemical entities in Australia, 2014". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
  5. ↑ "Search Page - Drug and Health Product Register". 23 October 2014.
  6. 1 2 3 4 World Health Organization (2009). Stuart MC, Kouimtzi M, Hill SR (eds.). WHO Model Formulary 2008. World Health Organization. pp. 259–60. hdl: 10665/44053 . ISBN   9789241547659.
  7. 1 2 3 4 5 British National Formulary : BNF 69. British Medical Association. 2015. p. 171. ISBN   978-0857111562.
  8. 1 2 "Alphanate – Summary of Product Characteristics (SPC) – (eMC)". www.medicines.org.uk. Archived from the original on 9 January 2017. Retrieved 8 January 2017.
  9. ↑ Potts DM (2011). "Chapter 5". Queen Victoria's Gene: Haemophilia and the Royal Family. The History Press. ISBN   978-0752471969. Archived from the original on 9 January 2017.
  10. ↑ High KA (2012). "In vivo characteristics of rDNA Factor VIII: The impact for the future in hemophilia care". In Sibinga CS, Das PC, Overby LR (eds.). Biotechnology in blood transfusion: Proceedings of the Twelfth Annual Symposium on Blood Transfusion, Groningen 1987, organized by the Red Cross Blood Bank Groningen-Drenthe. Springer Science & Business Media. p. 224. doi:10.1007/978-1-4613-1761-6_19. ISBN   978-1461317616. Archived from the original on 9 January 2017.
  11. ↑ Brownlee GG, Giangrande PL (2012). "Clotting factors VIII and IX". In Buckel P (ed.). Recombinant Protein Drugs. Birkhäuser. p. 79. ISBN   978-3034883467. Archived from the original on 9 January 2017.
  12. ↑ Zimring JC, Duncan A (2006). "Chapter 25 - Coagulation Factor Preparations". In Hillyer CD (ed.). Blood Banking and Transfusion Medicine: Basic Principles & Practice. Elsevier Health Sciences. p. 353. doi:10.1016/B978-0-443-06981-9.50030-2. ISBN   0443069816. Archived from the original on 9 January 2017.
  13. ↑ World Health Organization (2019). World Health Organization model list of essential medicines: 21st list 2019. Geneva: World Health Organization. hdl: 10665/325771 . WHO/MVP/EMP/IAU/2019.06. License: CC BY-NC-SA 3.0 IGO.
  14. 1 2 Nathwani AC, Davidoff AM, Tuddenham EG (October 2017). "Gene Therapy for Hemophilia". Hematology/Oncology Clinics of North America. 31 (5): 853–868. doi:10.1016/j.hoc.2017.06.011. PMID   28895852. S2CID   3779939.
  15. 1 2 Machin N, Ragni MV, Smith KJ (July 2018). "Gene therapy in hemophilia A: a cost-effectiveness analysis". Blood Advances. 2 (14): 1792–1798. doi:10.1182/bloodadvances.2018021345. PMC   6058236 . PMID   30042145.
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