A patient sitting with a doctor, receiving IV treatment for aTTP.

How to Treat aTTP

6 Treatment Options for Acquired Thrombotic Thrombocytopenic Purpura

Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare and potentially life-threatening blood disorder. Fortunately, effective treatment options are available to manage the condition, including the breakthrough therapy Cablivi. Let's take a look at common treatment options.

1. Cablivi (Caplacizumab-yhdp)

Cablivi is one of the most effective medications for treating aTTP. It works by targeting the interaction between von Willebrand factor (vWF) and platelets, which are key contributors to the formation of clots in people with aTTP. This targeted approach prevents the small clots that cause the disease's complications, allowing blood to flow normally.

Cablivi is often used in combination with plasma exchange and immunosuppressive therapy to address both the immediate symptoms and the underlying cause of aTTP. It is given as an injection, starting with an intravenous dose followed by subcutaneous injections.

For many patients, the cost of Cablivi can be a concern. However, financial assistance programs are available to help. The drug’s manufacturer offers savings programs, such as co-pay assistance for insured patients and financial support for those without insurance. Additionally, non-profit organizations like the HealthWell Foundation or Good Days provide funding for people with high out-of-pocket expenses. Talking to a healthcare provider about these options can make Cablivi more accessible.

2. Plasma Exchange Therapy

Plasma exchange therapy, also known as plasmapheresis, is a first-line treatment for aTTP. It involves removing the patient’s plasma, which contains harmful autoantibodies, and replacing it with healthy donor plasma. This process helps restore a balance of essential proteins in the blood, including the ADAMTS13 enzyme, which is deficient in people with aTTP.

Plasma exchange is typically performed daily until a patient’s platelet count and other clinical markers return to normal. While it’s a highly effective treatment, it is often paired with medications like Cablivi and corticosteroids to ensure long-term disease management.

3. Corticosteroids

Corticosteroids, such as prednisone, are commonly prescribed to reduce the immune system’s production of the autoantibodies that attack the ADAMTS13 enzyme. These medications work by suppressing the immune system, which helps to control the underlying cause of aTTP.

Corticosteroids are usually taken in pill form or given intravenously, depending on the severity of the condition. While effective, they can have side effects, including increased susceptibility to infections, so close monitoring by a healthcare provider is essential during treatment.

4. Rituximab

Rituximab is a monoclonal antibody that targets and reduces the number of B cells, which are responsible for producing the autoantibodies seen in aTTP. This treatment is often used when patients do not respond to initial therapies or when there is a high risk of relapse.

Rituximab is administered through an intravenous infusion, typically over several weeks. It is effective in preventing relapses and may help reduce the need for plasma exchange or corticosteroids. Patients undergoing rituximab therapy are closely monitored for side effects, such as allergic reactions during the infusion.

5. Immunosuppressive Drugs

In cases where aTTP is particularly severe or recurrent, additional immunosuppressive medications like cyclosporine or mycophenolate mofetil may be used. These drugs further suppress the immune system, reducing the production of harmful autoantibodies. They are often reserved for patients who don't respond to other treatments.

Immunosuppressive drugs can be very effective in controlling aTTP but require careful monitoring due to their potential to increase the risk of infections and other complications. Regular follow-ups with a healthcare provider ensure that the treatment remains effective while minimizing side effects.

6. Lifestyle and Supportive Care

In addition to medical treatments, certain lifestyle adjustments can support recovery from aTTP. Eating a balanced diet, staying hydrated and reducing stress can help improve overall health and support the body’s ability to heal. Regular check-ups and lab tests are also critical for detecting potential relapses early and ensuring the effectiveness of ongoing treatments.

Signs of Acquired Thrombotic Thrombocytopenic Purpura

Recognizing the signs of aTTP is crucial for early diagnosis and treatment. Common symptoms include severe fatigue, unexplained bruising or red spots on the skin, confusion, shortness of breath and dark-colored urine. Because these symptoms can develop suddenly and worsen quickly, it's important to seek medical attention immediately if they appear.

Final Notes

Managing aTTP requires a comprehensive approach, combining effective treatments like Cablivi, plasma exchange and immunosuppressive therapies with supportive care and regular monitoring. Recognizing the signs of aTTP and seeking timely treatment can make a significant difference, improve outcomes and enhance quality of life.