Talk to Your Doctor About Treatments
Effective treatment is crucial to manage acquired thrombotic thrombocytopenic purpura (aTTP), prevent complications and improve outcomes for patients. This article reviews the most common treatment options for aTTP, starting with the FDA-approved medication Cablivi.
1. Cablivi (Caplacizumab-yhdp)
Cablivi is a targeted therapy approved for the treatment of aTTP. It works by inhibiting the interaction of von Willebrand factor (vWF) with platelets, which is a key mechanism in the formation of the small blood clots characteristic of aTTP. Cablivi is used in combination with plasma exchange and immunosuppressive therapy to address the underlying causes of the disorder.
The benefits of Cablivi include:
- Reduced formation of blood clots in the small vessels.
- Lowers the risk of relapses during treatment.
- Improves platelet counts more quickly than other therapies.
Cablivi is administered as a subcutaneous injection. The treatment typically begins with a single intravenous dose during the first plasma exchange session, followed by daily subcutaneous injections.
How to Save on Cablivi
The cost of Cablivi can be significant, but several options may help reduce the financial burden:
- Manufacturer savings programs. The manufacturer offers assistance programs for eligible patients, including co-pay cards and financial aid for uninsured or underinsured individuals.
- Patient assistance foundations. Organizations such as the HealthWell Foundation or Good Days may provide financial support for high-cost treatments.
- Insurance optimization. Work with your healthcare provider to ensure insurance coverage and explore prior authorization options to secure reimbursement.
2. Plasma Exchange (Plasmapheresis)
Plasma exchange is the cornerstone of aTTP treatment and is often initiated immediately upon diagnosis. The procedure involves removing the patient’s plasma, which contains the autoantibodies responsible for the disorder, and replacing it with donor plasma.
Benefits of plasma exchange include:
- Rapidly reduces the levels of harmful autoantibodies.
- Restores a healthy balance of von Willebrand factor and ADAMTS13 enzyme activity.
- Improves platelet counts and reduces symptoms quickly.
Treatment typically occurs daily until clinical and laboratory indicators of aTTP normalize. The procedure is often combined with other therapies, such as corticosteroids and Cablivi.
3. Corticosteroids
Corticosteroids, such as prednisone or methylprednisolone, are commonly used in aTTP treatment to suppress the immune system and reduce the production of autoantibodies targeting ADAMTS13, the enzyme responsible for preventing clot formation.
Benefits of corticosteroids include:
- Helps reduce inflammation and immune system overactivity.
- Complements other therapies such as plasma exchange.
Corticosteroids are typically administered orally or intravenously and may be used throughout the treatment process.
4. Rituximab
Rituximab is a monoclonal antibody used to target and suppress B cells, which produce the autoantibodies responsible for aTTP. It is often prescribed for patients who do not respond well to initial treatments or those at high risk of relapse.
Benefits of rituximab include:
- Long-term prevention of relapses.
- Reduces the need for plasma exchange and corticosteroids.
Rituximab is given as an intravenous infusion, usually on a weekly basis over four to eight weeks.
5. Immunosuppressive Therapies
Additional immunosuppressive drugs, such as cyclosporine or mycophenolate mofetil, may be prescribed in cases of refractory or relapsing aTTP. These medications help regulate the immune system and decrease autoantibody production.
Benefits of using immunosuppressive drugs include:
- An alternative for patients unresponsive to standard treatments.
- May provide longer-term disease control.
Potential side effects of these drugs including suppressing the immune system, increasing the risk of infections and other complications. Close monitoring is required.
Signs of Acquired Thrombotic Thrombocytopenic Purpura
ATTP can present with a range of symptoms, often developing suddenly. Common signs include:
- Fatigue. Caused by reduced oxygen delivery due to anemia.
- Bruising or petechiae. Small red or purple spots on the skin due to low platelet counts.
- Neurological symptoms. Confusion, headache or difficulty concentrating, often caused by blood clots in the brain.
- Shortness of breath. A result of reduced oxygen delivery to tissues.
- Dark urine. Caused by the breakdown of red blood cells.
If you experience any of these symptoms, especially in combination, seek immediate medical attention. ATTP is a medical emergency that requires urgent treatment.