Treatment Overview
Living with hemophilia A means your blood does not clot as well as it should because of missing or defective factor VIII, a clotting protein. Medicines can help replace the missing factor, prevent bleeding and lower the risk of complications. The right treatment depends on your health, lifestyle, medical history and how your body responds.
Your care plan is personal, and your doctor will work with you to understand your options, track progress and adjust treatment as needed.
Types of medicines
There are several kinds of medicines used to treat hemophilia A:
- Clotting factor replacement therapy: These medicines replace the missing clotting factor VIII. They may be given when bleeding happens or on a regular schedule to help prevent bleeding. Examples include antihemophilic factor (Advate), octocog alfa (Kogenate FS) and moroctocog alfa (Xyntha).
- Extended half-life factor products: These work like regular factor VIII but last longer in the body, so fewer infusions may be needed. Examples include efmoroctocog alfa (Eloctate) and rurioctocog alfa pegol (Adynovate).
- Non-factor therapy: These medicines do not replace factor VIII directly but help the blood clot in other ways. An example is emicizumab (Hemlibra), which can be given under the skin instead of into a vein.
- Antifibrinolytics: These medicines help clots stay in place once they form. They are often used along with other therapies for dental work or surgery. An example is tranexamic acid (Lysteda).
How doctors choose a medicine
Your doctor will help decide which medicine to use based on how severe your hemophilia A is, your age, lifestyle and past reactions to treatment. Some people may do well with standard factor VIII infusions, while others may need extended half-life products or non-factor therapies for easier dosing. If inhibitors (antibodies that block factor VIII) develop, special medicines may be needed.
Monitoring and follow-up
Regular checkups are important. Blood tests help show whether you are getting the right amount of medicine and if your body is responding well. Doctors also look for side effects, check for inhibitors and ask about any bleeding episodes. Follow-up visits help make sure your treatment is safe and effective.
Adjusting therapy over time
Treatment plans can change. A child may begin with infusions only during bleeds and later move to regular preventive therapy. Adults may switch to newer medicines that need fewer doses. If bleeding happens too often, or if inhibitors develop, your care team may adjust or “escalate” therapy.
Working together with lifestyle changes
Medicines work best when combined with healthy habits. Protecting joints, staying active with safe exercises like swimming and avoiding high-risk activities that may cause injuries are all important. Sometimes surgery, physical therapy or dental procedures may be needed, and medicines are adjusted around those times.
Precautions and safety
Different medicines have different risks, and it is important to know what to watch for:
- Risk of inhibitors (antibodies): The most common and serious complication of hemophilia A treatment is the development of inhibitors, which are antibodies that block factor VIII. These make treatment less effective and can make bleeding harder to control.
- Pregnancy and organ health: During pregnancy, both dosing and medicine choice may need careful adjustment under specialist care. Antifibrinolytics such as tranexamic acid (Lysteda) may not be safe in people with kidney disease, and clotting factor use in people with liver disease often requires close monitoring.
- Drug interactions: Some antifibrinolytics can interact with other medicines that affect blood clotting, which may raise the risk of complications. Emicizumab (Hemlibra) is generally safe but may increase the risk of serious clotting problems if combined with high doses of certain bypassing agents, so doctors monitor these situations closely.
- Side effects: Most side effects are mild and may include infusion-site pain, headache, joint discomfort or low fever. Allergic reactions can occur with factor VIII products, and in rare cases medicines may cause serious clotting problems that may require emergency treatment. Regular follow-up helps detect these issues early so treatment can remain safe and effective.
Always tell your care team about allergies, health conditions and all the medicines or supplements you take. It’s also important to take medicines exactly as prescribed. Skipping doses or stopping suddenly can lead to bleeding episodes. If cost, side effects or remembering doses is hard, talk to your doctor—there are often solutions.
Medications for Hemophilia A
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Hemophilia A.