Hemlibra: un medicamento prometedor para pacientes con hemofilia A

Si tienes hemofilia A, your doctor might suggest Hemlibra as a treatment option. Approved by the FDA on November 16, 2017, this medicine is used to prevent or reduce bleeding episodes in people with hemophilia A. Hemlibra is seen as a promising alternative to the usual treatments for this condition. In this article, we will discuss Hemlibra, its side effects, dosing information, and much more.

What Is Hemlibra?

Hemlibra (emicizumab-kxwh) is a medication made by Genentech, a subsidiary of the Roche corporation. This medication is used for preventing or reducing bleeding episodes in people with hemophilia A, whether they have factor VIII inhibitors or not. It was first approved by the FDA on November 16, 2017, for hemophilia A patients with factor VIII inhibitors [1]. Later, on October 4, 2018, the approval was extended for patients with hemophilia A without factor VIII inhibitors [2].

People with hemophilia A don’t have enough of a protein called factor VIII in their bodies.

This protein plays a very crucial role in the blood clotting process. Therefore, when someone with hemophilia A gets a cut or injury, their blood doesn’t clot properly. This can lead to longer bleeding, internal bleeding, and more problems.

Before the FDA approved Hemlibra, factor VIII replacement therapy was the primary treatment for hemophilia A. This therapy involved injecting external factor VIII proteins to compensate for the deficiency in hemophilia A patients. However, a common problem with this therapy is that, over time, some people develop inhibitors. Inhibitors are antibodies that attack factor VIII, making the treatment ineffective. Hemlibra offers a solution to this issue. It can prevent bleeding in people with hemophilia A, whether they have inhibitors or not. 

What Is Hemlibra Used To Treat?

Hemlibra is a breakthrough medication that is primarily used to treat hemophilia A. Hemophilia A is a rare genetic disorder that affects blood clotting due to a deficiency in clotting factor VIII. 

Hemlibra is prescribed to be taken regularly. It prevents or reduces bleeding episodes in patients of all ages, including newborns, with hemophilia A [3]. This medication is effective for both adults and children with this condition, whether they have factor VIII inhibitors or not. 

How Does Hemlibra Work?

Hemophilia A is a bleeding disorder caused by a missing clotting factor called factor VIII, which is a protein that helps in blood clotting. When you have an insufficient amount of factor VIII, your blood struggles to form clots when you have an injury. 

Hemlibra is an antibody that mimics factor VIII, the missing protein in hemophilia A.

Normally, factor VIII links factor IX and factor X to aid in blood clotting. However, in hemophilia A, this link is missing. Hemlibra fills this gap by bringing factor IX and factor X together, helping clot formation. This action reduces the danger of bleeding episodes, providing relief to people who have hemophilia A.

The usual treatment for hemophilia A is replacing factor VIII. However, some people may develop inhibitors over time, which can make this treatment ineffective. However, Hemlibra works uniquely. Rather than directly replacing factor VIII, Hemlibra performs the same function as factor VIII by joining other blood proteins. This means it remains effective, even if inhibitors are present. 

Efectos secundarios

Like all medications, Hemlibra can also cause side effects. In rare cases, it can cause severe side effects. 

Efectos secundarios comunes

The following are some common side effects:

• Dolor de cabeza
• Joint Pain
• Reactions at the injection site, including redness, warmth, itching, or tenderness

These side effects usually go away on their own after a short time. But, if they worsen or do not go away, consult your doctor or pharmacist.

Efectos secundarios graves

Severe side effects from Hemlibra are rare, but they can occur. Contact your doctor right away if you encounter any severe side effects, which include:

• Blood clots and injury to small blood vessels that cause harm to the kidney, brain, and other organs
• Blood clots that may form in blood vessels in your arms, legs, lungs, or head
• Stomach, chest, and back pain
• Confusion and weakness
• Swelling, pain, or redness
• Eye pain or trouble seeing
• Shortness of breath, coughing up blood

The above list of Hemlibra side effects is not exhaustive. If you encounter any other side effects that are not mentioned above, please consult your doctor. 

Información de dosificación

Hemlibra is administered subcutaneously through injections. The dosing depends on various factors like your weight and treatment history. Hemlibra treatment usually starts with loading doses, followed by maintenance doses. Loading doses rapidly increase the drug’s concentration in your body. They are higher than maintenance doses.

The first four doses are loading doses. They are given at 3 mg/kg of body weight once weekly. After that, each subsequent dose is known as a maintenance dose. They are given as:

• 1.5 mg/kg of body weight once weekly, or
• 3 mg/kg of body weight once every 2 weeks, or
• 6 mg/kg of body weight once every 4 weeks

You have to discontinue the use of bypassing agents the day before starting Hemlibra treatment. If you miss a dose, take it as soon as you remember and then continue with your regular dosing schedule. 

Hemlibra Cost

Hemlibra can be expensive, priced at around $122 per mg [5]. The total cost of treatment varies based on your weight. For example, if you weigh 80 kg, the cost of treatment for the first year would be $411,136. For the following years, it would be $381,841. Please remember that this is a rough estimation. The real cost of treatment varies depending on your insurance plan, region, and pharmacy. 

Preguntas frecuentes

Here are some answers to frequently asked questions about Hemlibra:

Is Hemlibra Safe for Use During Pregnancy or Breastfeeding?

Hemlibra was mainly tested in males. There is no information about whether Hemlibra can cause harm to an unborn child or if it is present in breast milk. Hemlibra should only be used during pregnancy or breastfeeding if the benefits outweigh the risks.

Can Hemlibra Be Used in Patients With Liver or Kidney Disease?

If you have mild liver or kidney disease, you can still use Hemlibra without needing any changes to the dosage. Data for use in patients with severe liver or kidney disease is insufficient to provide specific recommendations [6]. Please consult your doctor.

Does Hemlibra Cure Hemophilia A?

Unfortunately, there is currently no cure for hemophilia A. Hemlibra can only prevent or reduce the occurrences of bleeding episodes. It cannot cure the disease.