treatment for inherited bleeding disease
November 22 2015 by Ray Sahelian, M.D.
an inherited bleeding disorder. Blood contains many proteins, called clotting
factors, which work to stop bleeding. People with hemophilia have a low level or
absence of one of these clotting factors in their blood. The lack of clotting
factor causes people with hemophilia to bleed for longer periods of time than
people whose blood factor levels are normal. People with hemophilia do not bleed
faster than other people, and will not bleed to death from a minor cut or
injury. The main problem for people with hemophilia is bleeding internally,
mainly into muscles and joints.
What is the difference between hemophilia A and hemophilia B?
There are two types of hemophilia: hemophilia A (sometimes called classical hemophilia) and hemophilia B (sometimes called Christmas disease). Both are caused by a low level or absence of one of the proteins in the blood (called factors) that control bleeding. Hemophilia A is caused by a deficiency of factor VIII, and hemophilia B is caused by a deficiency of factor IX. There is no difference between the two types of hemophilia, except that hemophilia B is about five times less common than hemophilia A.
Clotting Factor Concentrates
There is insufficient evidence from randomized controlled trials to determine whether prophylactic clotting factor concentrates decrease bleeding and bleeding-related complications in hemophilia A or B, compared to placebo, on-demand treatment, or prophylaxis based on pharmacokinetic data from individuals.
Factor VIII or IX replacement in a prophylactic manner is utilized for many patients with moderate to severe hemophilia A or B. Studies have shown it to be effective in reducing or preventing degenerative joint disease in many but not all patients. However, many unanswered questions still exist and optimization of this expensive treatment regimen is needed.
Advate for Hemophilia A
Advate (Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (rAHF-PFM), Baxter) A new 2000 IU (5 ml) ultra-high dosage is available for the prevention and control of bleeding episodes in people with hemophilia A.
History of the
Thromb Res. 2014. The history of haemophilia - a short review. The history of haemophilia dates back to the 2nd century AD, with the first "modern" descriptions of the condition appearing during the 1800s. At that time, transfusion medicine and haemophilia became closely linked, with blood transfusion being the only possible treatment option. A turning point in the history of haemophilia came in the middle of the 20th century when researchers identified an "antihaemophilic globulin" that could reduce the clotting time in haemophilic blood, thereby paving the way for the introduction of cryoprecipitate and the first clotting factor concentrates for the treatment of haemophilia A, haemophilia B and von Willebrand disease. The availability of pasteurized, and therefore virus-safe, plasma-derived, clotting factor concentrates, such as Haemate P and Beriate P in Germany and other countries, dramatically improved the quality of life and life expectancy of haemophilia patients. These and other treatment advances enabled home treatment, with many centres introducing prophylaxis to younger patients.