What is the treatment for Thrombotic Thrombocytopenic Purpura (TTP)?

From the Guidelines

The treatment of Thrombotic Thrombocytopenic Purpura (TTP) should include plasma exchange, high-dose glucocorticoids, rituximab, and/or caplacizumab, as these interventions have been shown to effectively manage the condition, particularly in patients with low ADAMTS13 activity. The management of TTP involves a multi-faceted approach, with the following key components:

• Plasma exchange: This is a crucial intervention for TTP, as it helps to remove autoantibodies and replace the deficient ADAMTS13 enzyme 1.
• High-dose glucocorticoids: These are often used in conjunction with plasma exchange to help reduce inflammation and suppress the immune system 1.
• Rituximab: This monoclonal antibody may be added to the treatment regimen for refractory or relapsing cases of TTP, as it can help to deplete B cells and reduce autoantibody production 1.
• Caplacizumab: This von Willebrand factor inhibitor has been shown to be effective in managing TTP, particularly when used in conjunction with plasma exchange and glucocorticoids 1. It is essential to note that the treatment of TTP should be individualized and based on the specific needs of each patient, taking into account factors such as disease severity, response to treatment, and presence of any underlying conditions. The goal of treatment is to rapidly restore ADAMTS13 activity, reduce microvascular thrombosis, and prevent further complications, thereby improving morbidity, mortality, and quality of life for patients with TTP.

From the FDA Drug Label

CABLIVI is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. The efficacy of CABLIVI for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy was established in a pivotal multicenter, randomized, double-blind, placebo-controlled trial (HERCULES)

The treatment for Thrombotic Thrombocytopenic Purpura (TTP) is caplacizumab (IV) in combination with:

• plasma exchange
• immunosuppressive therapy 2, 2

From the Research

Treatment Overview

The treatment for Thrombotic Thrombocytopenic Purpura (TTP) typically involves a combination of therapies aimed at managing the condition and preventing further complications.

Initial Treatment

• Daily therapeutic plasma exchange is often the initial treatment of choice for acquired TTP with demonstrable autoantibodies 3.
• Plasma exchange with plasma replacement has been the mainstay for the treatment of TTP for several decades 4.
• Plasma exchange plus steroids can effectively treat TTP 5.

Adjunct Therapies

• Immunomodulatory therapies, including corticosteroids, rituximab, vincristine, cyclophosphamide, and splenectomy, may be considered to eliminate autoantibodies for a sustained remission 3.
• Rituximab has been shown to be beneficial in refractory/relapsing TTP 6.
• Caplacizumab, a humanized anti-von Willebrand factor-directed nanobody, has been approved for the treatment of TTP and can be used safely and effectively without concomitant plasma exchange in patients with anaphylaxis to plasma 4.
• Other repurposed drugs such as bortezomib and N-acetylcysteine are increasingly used off-label 7.

Emerging Therapies

• Recombinant ADAMTS-13 is slowly emerging as a potential treatment option 3, 7.
• Adeno-associated virus (AAV) 8-mediated gene therapy and platelet-delivered ADAMTS-13 are under investigation as potential therapeutic modalities 3.
• Antagonists targeting the interaction between platelet glycoprotein 1b and VWF are also being explored as potential treatments 3.