Magazine article Drug Topics

Hemophiliac Patients Offered Pathogen-Free Clotting

Magazine article Drug Topics

Hemophiliac Patients Offered Pathogen-Free Clotting

Article excerpt

Queen Victoria and Czar Nicholas II didn't have much in common, but both had sons that could bleed to death from a punch in the nose. Passed across centuries, hemophilia is a clotting-factor deficiency known to cause frequent hemorrhages in muscles and joints. The genetically acquired disease has so far been treated with clotting-factor products derived mainly from whole blood of donors. But blood-borne pathogens such as the HIV virus have left many of today's victims with more to worry about than just nosebleeds.

The recent approval of BeneFix (recombinant coagulation factor IX), an albumin-free, plasma-free clotting factor made through recombinant technology, offers a potentially safer alternative for control of bleeding in hemophilia B (factor IX deficiency) patients.

Genetics Institute Inc., Cambridge, Mass., which makes BeneFix, acknowledges that manufacturers of plasmaderived factor concentrates have used additional precautions through the years by employing donor screening programs as well as improved purification and viral inactivation methods. The company maintains, however, that despite these precautions, the small nonenveloped viruses like hepatitis A and Parvovirus B19 have managed to slip through the system.

Pam Hilderbrandt, R.Ph., assistant director of pharmacy at Robert Wood Johnson University Hospital, a designated hemophilia center, noted that "a lot of blood products have been recalled, once it was found that some donors had a rare and fatal neurological condition called Creutzfeldt-Jacob disease. Although there have been no reports to date of any transmissions, the hemophiliac population does not believe anything is safe."

Because BeneFax does not contain any components of blood or plasma, the manufacturer expects that the risk of viral contamination will be eliminated. In addition, the company hopes to hush concerns about product shortages due to limitations in blood supply.

Although clinical trials have shown BeneFix to be effective in the treatment of hemorrhagic episodes, including prevention of bleeding in surgery, some patients required higher doses of the recombinant factor compared with plasma-derived products. "The need for higher doses had to do with 20%-30% less peak amounts of factor IX assayed from the plasma in comparison with Mononine [monoclonal antibody purified, Centeon]," commented Kathryn J. …

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