Cablivi (caplacizumab) is a medication that has been recently approved by the FDA for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura. This disease is a rare and life-threatening condition which is characterized by the formation of blood clots in small blood vessels. These clots can lead to low levels of blood platelets, hence the name thrombocytopenic purpura.
Cablivi is a type of monoclonal antibody that is designed to bind to a protein called von Willebrand factor (vWF), which plays a critical role in blood clotting. In patients with aTTP, the interaction between vWF and blood platelets is excessively active, which leads to the formation of clots. Cablivi works by preventing the interaction between vWF and platelets, which reduces the formation of blood clots and improves outcomes in these patients.
Cablivi is available in both powder form and solution for injection. It is administered in combination with standard medical therapy, which involves plasma exchange and immunosuppressive therapy. Plasma exchange is a process that removes the patient’s plasma and replaces it with a donor plasma, which helps to remove the antibodies that are causing the disease. Immunosuppressive therapy is a type of treatment that suppresses the immune system to prevent the body from making more antibodies.
Clinical trials have demonstrated the efficacy and safety of Cablivi in the treatment of aTTP. In one study, patients who received Cablivi in addition to standard therapy experienced a faster resolution of their symptoms and a lower rate of recurrence compared to those who received standard therapy alone. The most common side effects of Cablivi include bleeding, which can be severe or life-threatening in some cases.
Cablivi is an important breakthrough in the treatment of aTTP, a rare and potentially deadly disease. By targeting vWF, it addresses the root cause of the disease and provides patients with a new treatment option that can improve their outcomes. Patients who are diagnosed with aTTP should work closely with their healthcare providers to determine the best course of treatment, which may include Cablivi in addition to standard therapy.
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