Targeting Antithrombin to Treat Hemophilia  

Oleh:

Ragni, Margaret V.

Jenis: Article from Journal - ilmiah internasional
Dalam koleksi: The New England Journal of Medicine (keterangan: ada di Proquest) vol. 373 no. 04 (Jul. 2015), page 389-391.

Ketersediaan

Perpustakaan FK Nomor Panggil: N08.K Non-tandon: 1 (dapat dipinjam: 0) Tandon: tidak ada

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Isi artikel

Hemophilia is an X-linked bleeding disorder resulting from a deficiency of coagulation factor VIII or IX (causing hemophilia A or B, respectively) and is characterized by spontaneous and traumatic bleeding into the muscles, joints, and body cavities. Treatment is expensive, with an annual cost of several hundred thousand dollars for an average adult, and the complications of bleeding often result in greater severity of disease, poor quality of life, a requirement for orthopedic surgery, and a shortened life span. To prevent bleeding and its complications, the missing coagulation factor is replaced several times weekly by means of an intravenous infusion, termed “prophylaxis,” which begins in infancy. Despite advances in the manufacture of clotting factor, including recombinant and extended-half-life technologies that have led to safer and simpler treatment, many problems remain. As many as 50% of adults abandon prophylaxis because it is invasive and inconvenient, and 25% have development of inhibitory antibodies, which render the infused factor ineffective and lead to high morbidity and early death.1 The development of simpler, less invasive, and less costly treatment is clearly a goal.

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