Symptoms of Hemophilia are often diagnosed through blood tests. The tests help to identify which type of Hemophilia the person has and the severity of the condition.
CUPERTINO, CALIFORNIA | NOW THEN DIGITAL — Having Hemophilia can cause a lot of problems for you and your family. Fortunately, there are many therapies available to help you deal with this illness.
- Symptoms of hemophilia can vary from mild to severe. Depending on the extent of bleeding, you may need to receive factor replacement therapy. In addition, you may need prophylaxis, or a treatment that prevents bleeding. You may also need to receive physical therapy and pain management.
- The condition can affect people of all ages. It is most common in older men and women. However, it can also be diagnosed in young boys and girls. The symptoms can vary, but the main symptom is bleeding that does not stop.
- The genetic condition is passed from parent to child. Females may be carriers, meaning that they carry a mutation in the gene that causes haemophilia. These women can pass the X chromosome with the mutation on it to their children.
- Some people with hemophilia also develop inhibitors. These inhibitors stop clotting factors from working properly. This can cause other problems with bleeding.
- Hemgenix is a new drug, approved Tuesday by the US Food and Drug Administration for treating hemophilia. Manufacturer CSL Behring set the price at $3.5 million per treatment, establishing it as the most expensive drug on the market.
Signs and Symptoms
Symptoms of hemophilia or haemophilia vary depending on the severity of the condition. For example, mild hemophilia may not cause any symptoms until adulthood, while severe hemophilia is extremely severe and may lead to permanent disability.
The most common symptom of hemophilia is bleeding. This may occur in the joints, skin (in the form of bruises), muscles, mouth and gums or other areas. Bleeding is caused by the absence of clotting factors, specialized proteins that help blood clot.
If bleeding isn’t controlled, it can lead to permanent disability. A doctor can diagnose hemophilia by asking about family history and blood testing. He or she may recommend further treatments.
Some people with hemophilia may experience heavy bleeding in their urine or stool, frequent and hard-to-stop nosebleeds, headaches and pain. You should learn how to use safe medications.
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You should also avoid situations where injuries are common. If you have an injury, go to the emergency room as soon as possible.
Causes
Approximately one in 5,000 males are born with hemophilia. This genetic disease is caused by a mutation of a gene that provides instructions for making the clotting factor proteins that are essential for blood clotting.
In some people, the disorder develops later in life.
There are two different types of hemophilia. The first is hemophilia A, which is caused by a deficiency of the blood clotting factor VIII. This condition causes moderate to severe bleeding, which can lead to life-threatening complications.
The second is hemophilia B, which is also called Christmas disease. The two types of hemophilia are related, but are less common than hemophilia A and C.
The condition is inherited, and it affects both males and females. The gene that causes the condition is located on the X chromosome. Males inherit one X chromosome from their mothers and one X chromosome from their fathers.
Treatment
Depending on the severity of hemophilia, the patient may need to undergo a variety of treatment options. Severe hemophilia patients often require long-term therapy to stop bleeding and prevent complications.
Treatments may include replacing or giving clotting factors, preventing infection, and managing bleeding episodes.
Blood tests can help determine the severity of haemophilia and provide information about the clotting factors in the blood. Blood clotting tests can also help determine the type of hemophilia, if it is hemophilia A, B, or C.
Currently, the CDC writes in this article that the main treatment for hemophilia is giving or replacing clotting factors. CNN reported that the US Food and Drug Administration on Tuesday approved Hemgenix, a drug to treat hemophilia that costs $3.5 million per treatment, making it the world’s most expensive medication.
These factors work with platelets to stop bleeding. The type of clotting factor replacement therapy a patient will need will depend on the type of hemophilia and the patient’s medical needs.
Hemophilia A patients are usually treated with a personalized regimen. The regimen varies depending on the patient’s age and medical needs. In addition to replacing the clotting factor in the blood, patients may also have to take other medications.
Non-factor replacement therapies
Historically, hemophilia A treatment has been based on factor replacement therapy. In this approach, clotting factor concentrates are injected into a vein. However, this approach has limitations. In addition, venous access is affected.
Another option is non-factor replacement therapies. These therapies work by inhibiting the anticoagulant pathways. These therapies are also referred to as bypassing agents. They can be administered intravenously or subcutaneously. However, each person will respond differently to treatment.
Non-factor therapies are a major advancement in hemophilia A treatment. They have revolutionized the lives of patients. They also help prevent bleeds. However, they are not for all hemophilia A patients.
These therapies work by boosting the clotting factor levels in the body. They also have an extended half-life, which delays its breakdown. The result is a decreased number of infusions.
Complications
Approximately 20,000 people in the US are affected by hemophilia, a blood clotting disorder. Hemophilia is characterized by a deficiency of one of the two clotting factors (factor VIII or IX).
These factors are essential for the generation of a stable fibrin clot. The level of clotting factor determines the frequency and severity of bleeding episodes.
Hemophilia patients can experience a wide range of complications, including joint disease. These complications are usually related to inadequate treatment or skipping the prophylaxis doses.
If hemophilia is left untreated, it can cause severe and painful joint damage. Joint replacement surgery may be needed. Other treatments include physical therapy and strengthening exercises.
People with haemophilia are also at higher risk for transfusion-related infections. Some patients may develop inhibitors, which are antibodies produced by the immune system. They can interfere with the production of clotting factors and may cause life-threatening complications.
Hemophilia A Vs Hemophilia B
Despite their similarity in genetics, hemophilia A and hemophilia B are two different types of inherited bleeding disorders. They differ in the amount of bleeding and in the ways in which they affect the body.
The main difference between hemophilia A and hemophilia B is that hemophilia A is caused by mutations in the factor 8 gene while hemophilia B is caused by mutations in the factor 9 gene.
The factor 8 and 9 clotting factors control bleeding in the body.
When you are diagnosed with haemophilia A, your clotting factor levels are below normal. This causes the blood to clot slower than normal. In the absence of injury, the bleeding may occur continuously.
Usually, the bleeding is not too heavy and does not cause any problems. However, there are cases where the bleeding is too heavy and may result in bleeding into the brain or deep muscles.
People with hemophilia A have an increased risk of joint bleeds. The risk is three times higher in hemophilia A than in the general population. Hemophilia A can also lead to arthritis.
The severity of haemophilia is determined by the amount of clotting factor that is in the blood. Patients with hemophilia A have lower levels of factor 8 than the normal population. They may also suffer from excessive bleeding after trauma.
The treatment of royal disease is based on the replacement of missing clotting factors. These factors can be administered intravenously or topically to wounds.
Hemgenix – Gene Therapy For Hemophilia B
Previously known as Etranadez, Hemgenix is a new gene therapy for hemophilia B that reduces the number of bleeding episodes in patients.
The US Food and Drug Administration approved Hemgenix on Tuesday, a new drug that treats hemophilia, according to CNN. The manufacturer, CSL Behring, set the price at $3.5 million per treatment, making it the world’s most expensive drug.
It uses a non-infectious viral vector to deliver a working gene for Factor IX to the liver. The AAV5 virus triggers the production of the factor, which helps to stop bleeding.
How Does Hemgenix Work?
Using gene therapy, CSL Behring’s Hemgenix is able to replace a defective gene and help the body produce factor IX. This is a clotting factor that helps form blood clots. It is used to treat haemophilia B, a rare blood clotting disorder.
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