Hemophilia medications & treatments
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Hemophilia is a rare genetic disorder that occurs equally among all ethnic groups worldwide. It is estimated that the number of people living with hemophilia is about 400,000 worldwide. Hemophilia A is the more common type, affecting around 1 in 5,000 live male births. Hemophilia B is found in 1 in 30,000 live male births. Hemophilia C only occurs in about 1 in 100,000 people. It is usually diagnosed relatively soon after birth. The average age of diagnosis is 36 months for cases of mild hemophilia, 8 months for cases of moderate hemophilia, and 1 month for cases of severe hemophilia.
What is hemophilia?
Hemophilia is typically an inherited bleeding disorder that is caused by a deficiency of blood clotting factors. This can cause spontaneous bleeding as well as prolonged, excessive bleeding after an injury or surgery.
There are several types of hemophilia. The 2 most common types of inherited hemophilia are:
- Type A. Hemophilia A is the most common type and is caused by low levels of factor VIII, a protein that helps blood form clots. It is also known as classic hemophilia.
- Type B. Hemophilia B is caused by low levels of factor IX. It is also known as Christmas disease.
Rarely, a person develops a form of hemophilia with no family or personal history of this disease. This acquired hemophilia is an autoimmune disorder where your body’s immune system attacks clotting factors in your blood.
Hemophilia is almost always an inherited condition that is due to a clotting factor defect or gene mutation. It is inherited in an X-linked recessive pattern. You are born with 2 sex chromosomes, one from each parent. Males inherit an X chromosome from their mother and a Y chromosome from their father. Females inherit an X chromosome from each parent. Because the faulty gene is on the X chromosome, males have a 50% chance of developing it if their mother carries this defect. Females rarely inherit hemophilia because the faulty gene would have to be on both X chromosomes, or the faulty gene would have to be on one X chromosome, and inactive or missing in the other. If a female has one faulty gene, they are carriers that can pass it on to children they may have.
How is hemophilia diagnosed?
To confirm a diagnosis of hemophilia, your doctor or healthcare professional will look at your symptoms, review your medical history, and perform blood tests, or other diagnostic tests.
If you have a family history of hemophilia or if you are a known carrier of the disease, you should have your baby tested soon after birth to see if they have hemophilia. This can be done by taking a blood sample from the umbilical cord or a vein from your newborn immediately after birth and checking the clotting factor levels. Hemophilia A (clotting factor VIII deficiency) can be confirmed if its level is low. However, hemophilia B (clotting factor IX deficiency) can be difficult to diagnose in a newborn because their levels can be naturally low after childbirth. It takes about six months for them to reach their normal levels.
Even if there is no family history, those with severe hemophilia will have symptoms right away and are typically diagnosed within the first year of life. Milder cases might not be diagnosed until years later.
Some common signs and symptoms of hemophilia include:
- Bleeding after a circumcision
- Bleeding into the muscle or soft tissue (hematoma)
- Bleeding from the mouth and gums
- Blood in the stool or urine
- Bleeding after vaccinations or other shots
- Frequent nosebleeds that are hard to stop
- Bleeding into the joints
Screening tests, including a complete blood count, an activated partial thromboplastin time test (APTT), or a prothrombin time test (PT) may be ordered to show if the blood is clotting properly.
What are some hemophilia treatment options?
Your hemophilia treatment will depend on the type and severity of your condition. The main goal of treatment is to replace the clotting factor that is low or missing so that you can form clots and prevent excessive bleeding.
Your doctor may prescribe the following to treat your condition:
- Blood factor concentrates. These treatments can be made from human blood plasma or genetically engineered in a lab. Plasma-derived products such as Monoclate-P (factor VIII) and AlphaNine SD (factor IX) are collected from many donors and tested and treated for any viruses that may be present. Recombinant products such as Adynovate (factor VIII) and BeneFIX (factor IX) are safer options as they avoid the transmission of any blood-borne viral infections such as hepatitis. Severe cases of hemophilia usually require regular, ongoing therapy to prevent bleeding episodes, known as prophylaxis. Milder cases might only need demand therapy to stop bleeding once it starts.
- Hemlibra (emicizumab). This medication is a monoclonal antibody that does not contain clotting factors. It replaces their function to prevent or reduce the number of bleeding episodes in people with hemophilia A.
- DDAVP (desmopressin). Desmopressin is a synthetic hormone that stimulates the release of factor VIII from where it’s stored in your body to treat hemophilia A. DDAVP is injected into a vein. Desmopressin also comes as a nasal spray under the brand name Stimate.
- Amicar (aminocaproic acid). This medication can be given via injection or by mouth to prevent the breakdown of clots. It is typically given for bleeding in the mouth or after dental procedures.
In severe cases of hemophilia, you may need rehabilitation if internal bleeding has caused joint damage.
What is the best medication for hemophilia?
The best medication for the treatment of hemophilia will depend on the individual’s specific medical condition, medical history, medications that the individual is already taking that may potentially interact with hemophilia medications, and the individual’s potential response to the treatment. It is advisable to always speak with your healthcare provider about the best medication for you. The table below includes a list of the most prescribed or over-the-counter hemophilia medications approved by the Food and Drug Administration (FDA).
Best medications for hemophilia
|Drug name||Drug class||Administration route||Standard dosage||Common side effects
|Hemlibra (emicizumab)||Monoclonal antibody||Injection||3mg/kg of body weight under the skin every week for 4 weeks, then 3mg/kg every 2 weeks thereafter.||Injection site reactions, injection site pain, headache, joint pain, fever, diarrhea, altered taste
|BeneFIX (factor IX, recombinant)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Headache, dizziness, injection site reaction, injection site pain, nausea, rash, hives, altered taste
|AlphaNine SD (factor IX, human)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Headache, dizziness, injection site reaction, injection site pain, nausea, rash, hives
|Idelvion (factor IX, recombinant/albumin)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Headache, dizziness, hypersensitivity, rash, eczema
|Bebulin (factor IX complex)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Headache, rash, injection site reaction, injection site pain, cough
|Adynovate (factor VIII, recombinant)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Factor VIII inhibitor disorder, fever, nausea, dizziness, headache, altered taste
|Monoclate-P (factor VIII, human)||Coagulation factor||Injection||The dose is based on your body weight and desired factor IX increase.||Injection site reactions, headache, fever, nausea, vomiting,
|Feiba NF (anti-inhibitor coagulant complex)||Coagulation factor||Injection||The dose is based on your body weight and where the hemorrhage occurs.||Headache, nausea, rash, injection site pain, chills, hives
|Humate P (antihemophilic factor/von Willebrand factor complex)||Coagulation factor||Injection||The dose is based on your body weight and desired factor VIII increase.||Pain, itching, rash, hives, fever, joint pain, headache, fatigue
|DDAVP (desmopressin)||Synthetic hormone||Injection||0.3mcg/kg of body weight via 15-30 minute infusion 30 minutes before the procedure.||Headache, nausea, upset stomach, facial swelling, injection site reactions
|Stimate (desmopressin)||Synthetic hormone||Nasal spray||150mcg to 300mcg 2 hours before the procedure.||Runny nose, stomach pain, chills, nausea, pink eye, headache
|Amicar (aminocaproic acid)||Antifibrinolytic agent||Injection||4g to 5g via IV during the 1st hour, the 1g/hr for up to 8 hours. Max of 30g/day.||Headache, stomach pain, loss of appetite, nausea, vomiting, diarrhea, fatigue, dizziness
Your healthcare provider will determine the right dosage based on your response to the treatment, medical condition, weight, and age. Other possible side effects may exist; this is not a complete list.
What are the most common side effects of hemophilia medications?
As with all medicines, those used for hemophilia will have some side effects, depending on the class you are taking:
- Blood factor concentrates can commonly cause headache, dizziness, injection site reaction, injection site pain, nausea, rash, hives, and altered taste.
- Hemlibra (emicizumab) is a monoclonal antibody that can cause injection site reactions, injection site pain, headache, joint pain, fever, and diarrhea.
- DDAVP (desmopressin) can cause headaches, nausea, upset stomach, facial swelling, and injection site reactions. The nasal spray version Stimate causes runny nose, stomach pain, chills, nausea, pink eye, and headache.
- Amicar (aminocaproic acid) common side effects include headache, stomach pain, loss of appetite, nausea, vomiting, diarrhea, fatigue, sleepiness, and dizziness.
What are some home remedies for hemophilia?
While there is no way to reduce your chance of developing hemophilia, there are some self-care measures you can do to reduce your chance of excessive bleeding and protect your joints.
- Regular low-impact exercise such as walking or swimming to strengthen your muscles and protect your joints. Be sure to avoid any contact sports.
- Avoid medications that can increase your risk of excessive bleeding such as aspirin, Motrin (ibuprofen), Coumadin (warfarin), heparin, Plavix (clopidogrel), and Eliquis (apixaban).
- Prevent gum disease and tooth decay by practicing good dental hygiene.
- The Centers for Disease Control and Prevention (CDC) recommends getting vaccinated for hepatitis A and B.
- Find support through a Hemophilia Treatment Center (HTC). A CDC study showed that people who used an HTC were 40% less likely to be hospitalized or die from hemophilia-related complications.
Frequently asked questions about hemophilia
Is there a cure for hemophilia?
There are treatments to help manage this condition, but there is currently no cure for hemophilia.
What are some complications of hemophilia?
Some complications from hemophilia include joint damage due to internal bleeding and developing inhibitors in your immune system that can make it very difficult to treat or prevent bleeding episodes.
Do females develop hemophilia?
Hemophilia A and B typically affect males, but it can affect females as well. A study by the CDC looked at the number of people treated at HTCs. They found that almost 1700 of the patients were women while over 23,000 were males.
Related resources for hemophilia
The content on this website is intended for information purposes only. It does not constitute medical advice. The information on this website should not be relied upon and is not a substitute for professional medical advice. You should always speak to your doctor regarding the risks and benefits of any treatment.