Other Treatment Products
Desmopressin Acetate (DDAVP®)
Desmopressin acetate is a drug that causes VWF and some other coagulation proteins that are stored in the lining of blood vessels to be released into the bloodstream. This can help some people with von Willebrand Disease, mild Hemophilia, some platelet disorders and in some people who have undefined cause of bleeding. The rise in VWF in the blood can help raise the amount of factor VIII, it may also help some people with mild Hemophilia. Desmopressin is not made from blood and carries no risk of viruses. In the United States, it is given in two ways. One way is to inject the drug into the body through an IV. The brand name of this type of desmopressin is DDAVP®.
The other way desmopressin is given is by a nasal spray (spraying it in the nose). The brand name is Stimate® Nasal Spray. Stimate® is easy to use. The nurse in the Hemophilia Treatment Center (HTC) teaches people how to use Stimate® correctly. People can carry the nasal spray wherever they go and use it whenever they need it. However, there are some side effects to desmopressin. These include headaches, changes in blood pressure, and feeling flushed in the face. The drug also makes you retain water. If you drink too much liquid after taking desmopressin, the amount of sodium (salt) in your body can go too low. This can cause a seizure or even lead to a coma. For this reason, people taking desmopressin are told to limit the amount of liquids they drink.
Before a person with a bleeding disorder begins using desmopressin, a trial dose is given and the levels of VWF and factor VIII are measured. If the desmopressin is effective, there is usually a two-to-five-fold increase in the amount of factor VIII and VWF in the blood. The level begins to drop again after about six-eight hours. The medication does not work if used more than 3 days in a row. Desmopressin does not work for everyone with VWD. Please discuss with your healthcare team if this is the right medication for you.
Hemlibra® (emicizumab-kxwh)
Hemlibra is a prescription medication that works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It is used for prophylaxis to either prevent or reduce the frequency of bleeding episodes in people with Hemophilia A with and without inhibitors. This treatment product is given by injection under the skin. People who use Hemlibra for prophylaxis need to use factor to treat breakthrough bleeds. Specialized testing method called the chromogenic Bethesda assay needs to be used when testing for inhibitors in people on Hemlibra.
HEMGENIX® (etranacogene dezaparvovec-drlb)
In 2022, HEMEGENIX, a factor IX gene therapy, was approved for treatment of adults (age 18 years and older) with Hemophilia B. HEMGENIX is the first and currently the only gene therapy for Hemophilia B. Gene therapy uses advanced science to address the gene causing a condition. HEMGENIX is a one-time infusion administered through a vein. If you are a person with Hemophilia B who is 18 years or older you may be eligible for gene therapy. You will need to speak with your hematologist to assist in determining your eligibility to receive HEMGENIX.
Amicar® (epsilon aminocaproic acid) and Cyklokapron® (tranexamic acid)
Amicar and Cyklokapron are drugs that prevent a fibrin clot from breaking down. They do not help the body form a clot. These drugs stop one of the body’s enzymes, plasmin, from dissolving the clot. They keep the clot in place after it has already been made.
Amicar can be taken by mouth as a pill or as a flavored syrup. It is also available as a solution to be injected into a vein. Cyklokapron is available as a pill or solution to be injected.
These drugs are useful when you have bleeding in your mouth. Your saliva (spit) will break down a clot. Amicar and Cyklokapron can stop this from happening. Take these drugs exactly as your doctor orders. If you skip a dose or stop taking them too soon, you can start bleeding again.
Do not use Amicar or Cyklokapron if you have blood in your urine. It can make you have clots in your kidneys which will damage them.