What is ADAMTS13 (Recombinant): Uses, Dosage, Side Effects and More

ADAMTS13 is a specialized enzyme replacement therapy used to treat a rare blood clotting disorder called thrombotic thrombocytopenic purpura (TTP). This medication works by replacing a missing or deficient enzyme in your blood that helps prevent dangerous blood clots from forming throughout your body.

If you or someone you know has been prescribed this medication, you're likely dealing with a serious but treatable condition. Understanding how this therapy works can help you feel more confident about your treatment plan.

What is ADAMTS13?

ADAMTS13 is a laboratory-made version of an enzyme that naturally exists in your body. This enzyme's job is to break down large proteins in your blood called von Willebrand factor multimers, which can cause dangerous clotting when they become too large.

When your body doesn't make enough of this enzyme, or when it doesn't work properly, you can develop TTP. This condition causes tiny blood clots to form throughout your body, which can block blood flow to vital organs like your brain, heart, and kidneys.

The recombinant version of ADAMTS13 is created in a laboratory using advanced biotechnology. It's designed to work exactly like the natural enzyme your body should be producing, giving you back the protection you need against abnormal blood clotting.

What is ADAMTS13 Used For?

ADAMTS13 is primarily used to treat hereditary thrombotic thrombocytopenic purpura (hTTP), a rare genetic condition that affects fewer than 1 in 100,000 people. This condition occurs when you're born with a deficiency in the ADAMTS13 enzyme due to genetic mutations.

People with hTTP experience episodes where their blood forms dangerous clots throughout their body. These episodes can cause severe symptoms including confusion, seizures, kidney problems, and dangerously low platelet counts. Without treatment, these episodes can be life-threatening.

Your doctor might also consider this medication if you have acquired TTP that hasn't responded well to other treatments. However, the hereditary form is the primary indication for this specific enzyme replacement therapy.

How Does ADAMTS13 Work?

ADAMTS13 works by replacing the enzyme your body is missing or not producing enough of. Think of it as giving your blood the right tool to keep clotting proteins at a safe size.

In your bloodstream, proteins called von Willebrand factor naturally form chains of different sizes. When these chains become too large, they can trigger excessive clotting. The ADAMTS13 enzyme acts like molecular scissors, cutting these oversized protein chains down to a safer size.

This medication is considered a highly targeted therapy because it addresses the specific enzyme deficiency that causes hTTP. By restoring normal enzyme levels, it helps prevent the formation of dangerous blood clots while maintaining your body's ability to clot normally when needed for healing.

How Should I Take ADAMTS13?

ADAMTS13 is given as an intravenous (IV) infusion, which means it's delivered directly into your bloodstream through a vein. This medication cannot be taken by mouth because your digestive system would break down the enzyme before it could work.

Your healthcare team will typically administer this medication in a hospital or specialized infusion center. The infusion usually takes about 30 minutes to complete, and you'll be monitored during and after the treatment to ensure you're tolerating it well.

Before your infusion, you don't need to follow any special dietary restrictions. You can eat normally and take your other medications as prescribed. However, make sure to tell your healthcare provider about all medications and supplements you're taking, as some may interact with the treatment.

The timing of your infusions will depend on your individual needs and how your body responds to treatment. Some people need regular preventive infusions, while others may only receive treatment during acute episodes.

How Long Should I Take ADAMTS13 For?

The duration of ADAMTS13 treatment varies significantly from person to person, depending on whether you have hereditary TTP or acquired TTP. For hereditary TTP, this may be a lifelong treatment since the underlying genetic condition doesn't go away.

Your doctor will work with you to develop a personalized treatment schedule. Some people with hTTP need regular prophylactic (preventive) infusions to maintain adequate enzyme levels, while others may only need treatment during acute episodes or periods of increased risk.

Regular monitoring through blood tests will help your healthcare team determine how well the treatment is working and whether any adjustments to your schedule are needed. These tests measure your ADAMTS13 activity levels and monitor for signs of TTP episodes.

What Are the Side Effects of ADAMTS13?

Most people tolerate ADAMTS13 well, but like all medications, it can cause side effects. The most common side effects are generally mild and manageable with proper monitoring and care.

Here are the side effects you might experience, starting with the most common ones:

• Headache and mild fatigue
• Nausea or stomach discomfort
• Infusion site reactions like redness, swelling, or mild pain
• Dizziness or lightheadedness
• Muscle aches or joint pain

These common side effects usually improve on their own and don't require stopping treatment. Your healthcare team can help you manage any discomfort you experience.

More serious but less common side effects include allergic reactions, which can range from mild skin rashes to severe anaphylaxis. Signs of an allergic reaction include difficulty breathing, swelling of your face or throat, severe rash, or rapid heartbeat.

Very rarely, some people may develop antibodies against the medication, which can make it less effective over time. Your doctor will monitor for this through regular blood tests and can adjust your treatment plan if needed.

Who Should Not Take ADAMTS13?

ADAMTS13 isn't suitable for everyone, and your doctor will carefully evaluate whether it's right for you. People with known severe allergies to any components of the medication should not receive this treatment.

If you have a history of severe allergic reactions to other protein-based medications or enzyme replacement therapies, your doctor will need to weigh the risks and benefits very carefully. They may recommend additional precautions or alternative treatments.

Pregnant and breastfeeding women require special consideration, as there's limited safety data available for these situations. Your doctor will discuss the potential risks and benefits with you if you're pregnant, planning to become pregnant, or breastfeeding.

People with certain immune system disorders or those taking immunosuppressive medications may need modified treatment plans, as these conditions can affect how your body responds to the medication.

ADAMTS13 Brand Names

The recombinant ADAMTS13 medication is available under the brand name Adzynma. This is currently the only FDA-approved recombinant ADAMTS13 product available in the United States.

Adzynma was specifically developed for treating hereditary thrombotic thrombocytopenic purpura and represents a significant advancement in TTP treatment. Before this medication became available, treatment options were much more limited.

Your insurance coverage and pharmacy may refer to this medication by either its brand name (Adzynma) or its generic name (recombinant ADAMTS13). Both names refer to the same medication.

ADAMTS13 Alternatives

Before ADAMTS13 became available, the primary treatment for TTP was plasma exchange (plasmapheresis), which involves removing your blood, separating out the plasma, and replacing it with donor plasma that contains the missing enzyme.

Plasma exchange is still used in many cases, particularly for acquired TTP or when ADAMTS13 isn't available. However, it's a more invasive procedure that requires specialized equipment and carries its own risks, including exposure to donor blood products.

Fresh frozen plasma infusions can also provide temporary enzyme replacement, but they're less concentrated than the recombinant medication and may require larger volumes of fluid. This approach is sometimes used in emergency situations or when other treatments aren't available.

For some people with acquired TTP, immunosuppressive medications like corticosteroids or rituximab may be used to reduce the immune system's attack on the ADAMTS13 enzyme. However, these don't directly replace the missing enzyme.

Is ADAMTS13 Better Than Plasma Exchange?

ADAMTS13 offers several advantages over plasma exchange, particularly for people with hereditary TTP. The recombinant enzyme is more convenient, safer, and provides more consistent results than traditional plasma-based treatments.

Unlike plasma exchange, which requires several hours in a specialized medical facility with complex equipment, ADAMTS13 can be given as a relatively quick infusion. This makes it much more practical for regular, preventive treatment.

From a safety standpoint, ADAMTS13 eliminates the risks associated with donor blood products, including potential infections and allergic reactions to plasma proteins. The recombinant medication is also more standardized, so you receive the same consistent dose every time.

However, plasma exchange may still be the preferred initial treatment for some people with acquired TTP, especially when the cause is related to immune system dysfunction. Your doctor will determine which approach is best based on your specific type of TTP and individual circumstances.