Hemophilia Patient & Family Handbook

(under the skin, similar to insulin injections), or as a nasal spray. DDAVP can cause release of Factor VIII stored in cells that line the blood vessels. This medicine should not be used to treat severe or life- or limb-threatening bleeds. Often, a blood test called a DDAVP challenge is done to see if high enough levels of Factor VIII are achieved when this medication is used. Your healthcare team will teach you about side effects and how to use DDAVP correctly if it is prescribed for your child. The type of treatment that the doctor prescribes—on-demand versus prophylaxis—depends on several elements, including the severity of hemophilia and the amount of bleeding a person has. Other medical conditions may also be noted. Both on-demand and prophylaxis infusions can be done in different ways. Your treatment team will help you decide which option is best for your child. The National Hemophilia Foundation’s Medical and Scientific Advisory Council makes recommendations for treatment for hemophilia. They named prophylaxis as the treatment of choice for all persons with severe hemophilia A or B. A recent study compared treatment modes in severe hemophilia patients. In this study, patients who had prophylaxis showed significantly improved joint outcome scores (Manco-Johnson et al., 2007). Bleeding in hemophilia also can be treated using other methods. Antifibrinolytics are drugs used to slow the body’s clot-dissolving enzymes. These medications are particularly helpful for mouth and nose bleeding. Hormones can be used to control heavy menstrual bleeding in women with hemophilia and symptomatic carriers. Topical clotting agents may be used to treat nosebleeds, bleeding after some dental procedures, and small cuts or abrasions. Rest, ice, compression (using an ace wrap), and elevation (RICE) may be helpful when treating hemophilia. Your healthcare team will educate you about each treatment strategy when it is needed. One of the most important treatment strategies for individuals with hemophilia is comprehensive care. Comprehensive care is provided by a team of specialists, including a doctor, nurse, social worker, and physical therapist, who treat hemophilia. People with hemophilia are best treated with prevention and a team approach. Studies show that persons treated in comprehensive treatment centers have a lower risk of death. They also spent less time in the hospital and had better work and school attendance and fewer health complications than persons treated with a different source of medical care. New Therapies Researchers are evaluating various ways to introduce the hemophilia gene into hemophilia A and B patients, which will allow the body to produce more factor on its own. New ways to prevent bleeding without replacing Factor VIII and IX are also being studied.

n ARE THERE ANY TREATMENT-RELATED CONCERNS I SHOULD KNOW ABOUT?

Yes. As with all treatments, some side effects should be noted. Infusion of factor concentrates and subcutaneous injection of nonfactor therapy can cause local irritation at the site of infusion. Another concern with using factor concentrates is the development of an inhibitor. An inhibitor is an antibody. Antibodies form in everyone and are used by the body to fight infections and foreign proteins. The body can sometimes mistake infused factor as one of those foreign proteins, which causes inhibitor formation.

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