The list of treatments mentioned in various sources for Hemophilia includes the following list. Always seek professional medical advice about any treatment or change in treatment plans.
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The introduction of factor VIII concentrate products in the 1970s allowed treatment at home and largely replaced the need for transfusion of whole blood or plasma in the hospital. An unfortunate result of increased freedom from bleeding as a cause of disability and death was infection with hepatitis from products made from pooled plasma. This culminated in the early 1980s with the unexpected and devastating infection of about 70 % of severe hemophiliac patients with HIV, the cause of AIDS. Since then, the development and improvement of methods for inactivating viruses with lipid envelopes in plasma, including factor VIII and IX concentrates, has improved the safety of transfused products to the point where the threat of infection with HIV, hepatitis B(HBV) or hepatitis C (HCV) viruses has been essentially eliminated. (Source: excerpt from Hemophilia Update 1997: NHLBI)
Although most individuals with hemophilia can use replacement products repeatedly without problems, about 20% develop neutralizing antibodies that make the product less effective. Antibody inhibitors are more likely to occur in individuals with severe hemophilia. At this time, it is not possible to predict who will develop the antibody inhibitors, but there is some evidence for genetic predisposition for an immune response. The treatment for people with inhibitors can be complex and expensive. Often more than one approach is tried before the bleeding is arrested. The decreased ability to control bleeding in the joints can lead to earlier development of arthritis. In some cases, immune tolerance can be induced which allows standard treatment to again be effective. Studies are being conducted to avoid or modify the immune response and to prepare recombinant factor VIII proteins with reduced antigenicity. (Source: excerpt from Hemophilia Update 1997: NHLBI)
Approximately 15 percent of severe hemophilia A patients and 2.5 percent of hemophilia B patients develop such antibodies after exposure to transfused factors. When inhibitors are present in large amounts, the patient may require very high and expensive quantities of transfused clotting factors to stem bleeding, and, in some instances, even that may not be effective. The factor VIII products produced through biotechnology have been found to cause inhibitors in only about 5 percent of patients and are, thus, safer in this respect. Nevertheless, these inhibiting antibodies will remain a concern for hemophilia patients unless our ability to understand and control the immune system is improved. A number of NHLBI-supported scientists are directing research at this problem. (Source: excerpt from Hemophilia: NHLBI)
Several European countries are treating hemophiliacs by periodic infusion (prophylaxis) regardless of bleeding status. This approach maintains the factor level high enough that bleeding, joint destruction, and life-threatening hemorrhage are almost entirely avoided. There are, nevertheless, serious disadvantages such as the need for frequent infusions, the requirement for almost continuous access to veins by catheters, and the considerable cost of factor. In the United States, it is estimated that most patients on prophylaxis which is begun in the first few years of life will easily exceed the common life-time insurance cap of $1,000,000 by the second decade of life. The treatment decisions are not easy ones. (Source: excerpt from Hemophilia: NHLBI)
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Hemophilia isn't curable, but treatment can prevent crippling deformities and prolong life expectancy. Correct treatment quickly stops bleeding by increasing plasma levels of deficient clotting factors to help prevent disabling deformities that result from repeated bleeding into muscles and joints. (See Factor replacement products.)
Desmopressin (DDAVP — 1-deamino-8-d-arginine vasopressin) administered I.V. or intranasally is usually sufficient to manage bleeding episodes of children and adolescents with mild hemophilia A. Persons with moderate to severe hemophilia A require commercially prepared factor VIII concentrates to treat bleeding episodes. Concentrates derived from human plasma are virally attenuated by one or more available methods significantly minimizing the risk for human immunodeficiency virus (HIV)-1, HIV-2, hepatitis B, and hepatitis C contamination. However, no currently available method has been successful in eradicating parvovirus B 19 from blood products. Factor VIII concentrate derived from recombinant technology (rFVIII) has been shown in multiple clinical trials to be as effective as virally attenuated plasma-derived concentrate. Risk for viral contamination is essentially nonexistent in preparations of rFVIII that avoid human serum albumin as a stabilizer.
In hemophilia B, administration of factor IX concentrate during bleeding episodes increases factor IX levels.
The U.S. National Hemophilia Foundation first recommended prophylaxis with factor concentrates in 1994 after investigators in Sweden demonstrated repeated success with this approach. The ultimate goal is to prevent irreversible destructive arthritis that results from repeated hemarthrosis and synovial hypertrophy. Prophylaxis of persons with hemophilia A or B may begin as early as age 1 or 2.
A person with hemophilia who undergoes surgery needs careful management by a hematologist with expertise in hemophilia care. The patient will require replacement of the deficient factor before and after surgery, possibly even for minor surgery such as a dental extraction. (DDAVP may be given before dental extractions and surgery to prevent bleeding.) In addition, epsilon-aminocaproic acid is commonly used for oral bleeding to inhibit the active fibrinolytic system present in the oral mucosa.
Development of factor VIH or factor XI inhibitors occurs in up to 3.5% of children with severe hemophilia A and up to 3% of those with hemophilia B. Studies indicate that certain gene mutations predispose to an increased risk for inhibitor development. Patients with hemophilia who can't achieve hemostasis after use of previously effective factor concentrate doses should be evaluated for factor inhibitors.
Preventive measures include teaching the patient how to avoid trauma, manage minor bleeding, and recognize bleeding that requires immediate medical intervention. Genetic counseling helps carriers understand how this disease is transmitted. (See Managing hemophilia, page 28.)
Source: Professional Guide to Diseases (Eighth Edition), 2005
Hemophilia isn’t curable, but treatment can prevent crippling deformities and prolong life expectancy. Correct treatment quickly stops bleeding by increasing plasma levels of deficient clotting factors to help prevent disabling deformities that result from repeated bleeding into muscles and joints. Treatment varies according to the type of hemophilia. Gene therapy has shown promise for both types.
❑ In hemophilia A, cryoprecipitated antihemophilic factor (AHF), lyophilized AHF, or both, given in doses large enough to raise clotting factor levels above 25% of normal can permit normal hemostasis. Before surgery, AHF is administered to raise clotting factors to hemostatic levels. Levels are then kept within a normal range until the wound has completely healed.
❑ In hemophilia B, administration of factor IX concentrate during bleeding episodes increases factor IX levels.
A person with hemophilia who undergoes surgery needs careful management by a hematologist with expertise in hemophilia care. The patient requires replacement of the deficient factor before and after surgery. Such replacement may be necessary even for minor surgery such as a dental extraction.
In addition, aminocaproic acid is commonly used for oral bleeding to inhibit the active fibrinolytic system present in the oral mucosa. Preventive treatment teaches the patient how to avoid trauma, manage minor bleeding, and recognize bleeding that requires immediate medical intervention. (See Managing hemophilia.)
Source: Handbook of Diseases, 2003
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