Factor XIII deficiency is a rare genetic blood disorder that is characterised by the deficiency of clotting factor XIII. Clotting factors are proteins that play an important role in blood clotting. Patients suffering from factor XIII deficiency are able to form clots, however, these clots are highly unstable and dissolve easily, which results in prolonged uncontrolled bleeding. The severity and symptoms of the factor XIII deficiency varies from person to person thus the factor XIII deficiency treatment differs with respective to the severity. In most patients with factor XIII deficiency (80%), bleeding symptoms appear after birth, which include bleeding from the umbilical stump. Other patients are likely to have only a mild expression of the factor XIII deficiency that can become more apparent in the later stages of life when they encounter a traumatic injury or surgery.
Factor XIII deficiency is a genetic disorder that is recessive in nature. Thus, the risk of two carrier parents passing both the altered genes to their offspring is about 25% with each pregnancy. In case the parents are close relatives of each other, the risk is much higher. Thus, in communities where consanguineous marriages are more common, the risk of factor XIII deficiency is higher in the offspring, hence, it is widely seen in such communities.
Factor XIII deficiency treatment involves the usage of factor XIII concentrates. Fresh frozen plasma is one of the most common factor XIII deficiency treatments, especially in regions where the pharmaceutical version of factor XIII concentrates are not available easily. However, cryoprecipitates are no longer recommended for factor XIII deficiency treatment because of the risk of infections from viruses and other similar pathogens. The risk of allergy is also likely with other unknown factors present in the cryoprecipitates. Individuals with factor XIII deficiency are recommended to undergo preventive factor XIII deficiency treatment that includes the use of factor XIII concentrate every 3-4 weeks. The prophylactic factor XIII deficiency treatment is mainly used to prevent bleeding in the brain.
In extremely rare cases, factor XIII deficiency may be caused due to the development of autoantibodies. These autoantibodies are also called inhibitors since they mistakenly attack replacement factor XIII that is used for the factor XIII deficiency treatment. In case the factor XIII deficiency treatment results in the formation of autoantibodies, additional therapy is required along with the factor XIII deficiency treatment. These additional therapies include the usage of immune suppressant drugs.
In 2011, CSL Behring received FDA approval for its Corifact (FXIII Concentrate) for the routine prophylactic factor XIII deficiency treatment. Similarly, in 2014, Novo Nordisk, Inc. received FDA approval for Tretten, a recombinant factor XIII replacement product. Tretten was approved for the prevention of bleeding in adults and children due to factor XIII A-subunit deficiency. Factor XIII deficiency treatment using recombinant technology is a popular topic for research since it is artificially created and does not contain human blood components, thereby resulting in no risk due to blood-borne viruses or similar other pathogens.
Factor XIII Deficiency Treatment Market: Drivers and Restraints
The significant presence of communities that follow consanguineous marriages increases the risk of passing on factor XIII deficiency to the next generations, which is driving the factor XIII deficiency treatment market. Increasing government support for the treatment of rare diseases such as the factor XIII disease treatment is also an important factor driving the factor XIII deficiency treatment market. However, lack of experience of skilled personals for the factor XIII deficiency treatment like other rare diseases along with the difficulties encountered during the R&D of factor XIII deficiency treatment are some of the factors expected to hamper the growth of the factor XIII deficiency treatment market during the forecast period.
Factor XIII Deficiency Treatment Market: Overview
The global factor XIII deficiency treatment market is largely driven by the significant presence of consanguineous marriages. The factor XIII deficiency treatment that was used in the past involved the usage of fresh frozen plasma. However, due to the risk of infections, this form of treatment is not preferred. Recombinant technology-driven research resulted in products, such as Tretten, a recombinant factor XIII A-subunit, which are now more preferred since they are completely lab-synthesized products. The administration of these products for factor XIII deficiency treatment must be carried out at regular intervals for the prevention of bleeding especially in the brain.
Factor XIII Deficiency Treatment Market: Regional Outlook
Based on epidemiology, factor XIII deficiency cases are more frequent in regions where consanguineous marriages are more common. Thus, the occurrence of the disorder is most frequent in the Middle East and Africa region. However, a large number of cases go undiagnosed in these regions. Furthermore, the factor XIII deficiency treatment market has the highest potential in the MEA. Regions such as Canada, Brazil, Mexico and China also among the most lucrative regions for the factor XIII deficiency treatment. Europe and Australia have the lowest epidemiology, owing to which the treatment rate for the factor XIII deficiency treatment is low.
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Factor XIII Deficiency Treatment Market: Key Players
The global market for factor XIII deficiency treatment has two main pharmaceutical players, however, there are a number of blood banks that offer fresh frozen plasma. Companies that offer factor xiii concentrate are CSL Behring and Novo Nordisk, Inc. Examples of some of the blood banks that offer fresh frozen plasma for factor XIII deficiency treatment are Global Blood Fund, World Blood Bank and other local blood banks.