管理出血性疾病：Wilate 如何帮助治疗血管性血友病 (VWD) 和血友病 A

Bleeding disorders like 血管性血友病 (VWD) and 血友病A can make your life difficult. In these conditions, your blood doesn’t clot properly. As a result, you may experience heavy bleeding, even from minor cuts and injuries. These diseases are not curable. Fortunately, modern medicine has given us several treatment options for patients to manage these conditions and lead active lives. One of them is Wilate. In this article, we’ll break down everything you need to know about Wilate, including its side effects, dosing, benefits, and much more.

What Is Wilate?

Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) is a medication used to control major and minor bleeding episodes in von Willebrand disease (VWD) patients. It is also used in patients with hemophilia A for routine prophylaxis (prevent bleeding episodes) and on-demand treatment of bleeding episodes [1]. 

Octapharma, a Swiss pharmaceutical company, developed Wilate, and the FDA first approved Wilate on December 8, 2009 [2]. Patients suffering from von Willebrand disease (VWD) have a lack or abnormality of a protein called von Willebrand factor (VWF). On the other hand, people suffering from hemophilia A have a lack or abnormality of a protein called factor VIII (FVIII). Wilate is a plasma-derived concentrate that contains both von Willebrand factor (VWF) and factor VIII (FVIII). So, by replacing these missing factors, Wilate helps with blood clotting in patients with von Willebrand disease (VWD) and hemophilia A.

Unlike some other treatments, Wilate is free of albumin (which can cause side effects like allergic reactions). All plasma-derived products carry the risk of virus transmission. To reduce this risk, Wilate undergoes two separate viral inactivation processes [4].

What Is Wilate Used To Treat?

Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) is FDA-approved to treat the following conditions [3]:

Von Willebrand Disease (VWD): This is a lifelong bleeding disease in which your blood doesn’t clot properly. A protein in our blood called von Willebrand factor helps platelets stick together and form blood clots. People with von Willebrand disease (VWD) lack enough von Willebrand factor to clot properly. Wilate is used in children and adults with all types of VWD for on-demand treatment and controlling bleeding episodes. It is also used before any surgery to prevent bleeding.

Hemophilia A: This is a condition in which your blood doesn’t clot properly because you lack a protein called clotting factor VIII (FVIII). Wilate is used in children and adults with hemophilia A for routine prophylaxis (preventing bleeding episodes) and on-demand treatment of bleeding.

How Does Wilate Work?

Wilate is a human plasma-derived product that contains a balanced 1:1 ratio of von Willebrand factor (VWF) and factor VIII (FVIII). These are proteins that are critical for blood clotting. VWF helps the platelets to stick to the site of a blood vessel injury and to each other. This forms the initial blood clot. FVIII works with other clotting proteins to form a stable fibrin clot.

People with von Willebrand disease have a lack of VWF, and people with hemophilia A have a lack of FVIII. Wilate works by replacing both. It helps blood to clot normally and stops or prevents bleeding episodes.

Benefits of Wilate

Wilate is specially designed to help people with von Willebrand disease (VWD) and hemophilia A. Here are some key advantages of using Wilate [4]:

Low Recommended Dosing: Wilate works effectively with a low recommended dose.

Safe for Repeated Use: If you take repeated doses of a VWF/FVIII product, such as before and after surgery, your FVIII levels can rise temporarily. This is known as FVIII accumulation, which might increase your risk of blood clots. However, clinical trials showed no FVIII accumulation after repeat dosing with Wilate [5], so it is safe for repeated use.

Controls Bleeding Effectively: Wilate can effectively control both major and minor bleeding episodes [5].

Purified for Safety: An advanced purification process is used to make Wilate, which helps remove unwanted proteins and impurities.

Double Viral Protection: To reduce the risk of viral transmission and ensure your safety, Wilate undergoes two separate viral inactivation processes.

Side Effects of Wilate

Like any other medication, Wilate can also have some side effects. These side effects range from mild to severe. Here are some common to severe side effects of Wilate [1][3]:

常见副作用

Some common side effects of Wilate include:

• 发烧
• 头晕
• 头痛
• 麻疹
• 胸部不适
• Injection site reactions (redness, swelling, pain)

严重的副作用

In some cases, Wilate can cause severe side effects. Here are some of the severe side effects of Wilate:

Hypersensitivity Reaction: In rare cases, you may encounter serious allergic reactions. Symptoms include rash, itching, shortness of breath, chest tightness, and swelling of the face or throat. Inform your doctor immediately if you experience these symptoms.

血栓： In rare cases, continued treatment with Wilate in patients with VWD can increase the risk of blood clots.

Inhibitor Development: Patients with VWD and hemophilia A may develop antibodies (inhibitors) that make Wilate less effective or ineffective over time. In such cases, you need to consider other treatment options.

This is not a list of all possible side effects. Contact your doctor immediately if you experience any severe side effects of Wilate.

Dosing of Wilate

The dosage of Wilate depends on several factors, including your age, weight, disease type, and the severity of your condition. Your doctor will determine the right dosage for you based on your situation. Here are some typical dosing regimens of Wilate [3]:

Dosing for Von Willebrand Disease (VWD)

The dosing varies based on your situation and treatment purpose. The recommended dosage is given below:

For On-Demand Treatment and Control of Bleeding: For minor bleeding, the recommended starting dose is 20 to 40 IU per kg of body weight. After that, a 20 to 30 IU/kg maintenance dose is usually given every 12 to 24 hours. For major bleeding, the recommended starting dose is 40 to 60 IU/kg. Then, to maintain proper clotting levels, doctors usually give 20 to 40 IU/kg every 12 to 24 hours.

For Surgery: The recommended starting dose for minor surgeries (including tooth extractions) is 30 to 60 IU/kg body weight. After that, a 15 to 30 IU/kg maintenance dose is given every 12 to 24 hours for up to 3 days. For major surgeries, the starting dose is 40 to 60 IU/kg, and the maintenance dose is 20 to 40 IU/kg every 12 to 24 hours for up to 6 days or more.

Dosing for Hemophilia A

The recommended dosage of Wilate for hemophilia A is given below:

For Routine Prophylaxis: To prevent bleeding episodes in adolescents and adults, the usual dose is 20 to 40 IU/kg every 2 to 3 days. 

For On-Demand Treatment of Bleeding Episodes: If you start bleeding, you can use Wilate to stop it. The dosage depends on how severe your bleeding is. 

• For minor bleeding, the recommended dose is 30 to 40 IU/kg, repeated every 12 to 24 hours until the bleeding stops. 
• For moderate bleeding, the recommended dose is 30 to 40 IU/kg. You have to repeat it every 12 to 24 hours for 3 to 4 days or more until the bleeding stops. 
• For major bleeding, the dose is 35 to 50 IU/kg body weight, repeated every 12 to 24 hours. You have to continue this dose for 3 to 4 days or more, depending on the severity.

常见问题解答

Here are some frequently asked questions about Wilate:

1. How is Wilate different from other clotting factor products?

Wilate is the first VWF/FVIII treatment developed especially for von Willebrand disease (VWD). It is made from human plasma and contains both von Willebrand factor (VWF) and factor VIII (FVIII) in a balanced 1:1 ratio. It is double virus-inactivated and does not contain albumin, making it a safe and effective option [3][6].

2. Can I use Wilate at home?

Yes. Home administration of Wilate is possible, which is convenient and time-saving. But first, you or your caregiver has to undergo training by a healthcare provider.

3. Can I use Wilate during pregnancy or breastfeeding?

There is still no data available to recommend or limit the use of Wilate during pregnancy and breastfeeding [3]. Your doctor will weigh the benefits and risks before prescribing it during pregnancy or breastfeeding.

4. What if I experience side effects after using Wilate?

If you have mild side effects like a headache or nausea, they may go away on their own. But if you experience allergic reactions (rash, swelling, trouble breathing) or signs of a serious problem like blood clots, contact your doctor or seek emergency care immediately.