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ASH 22 Presentation Preview: Most/All Patients With Acquired (Immune) Thrombotic Thrombocytopenic Purpura Receive Caplacizumab

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Dr. Masias will be speaking on Monday, December 12, 2022, 10:30 A.M. - 11:45 A.M at Debate: Most/All Patients With Acquired (Immune) Thrombotic Thrombocytopenic Purpura Receive Caplacizumab. She elaborates on what to look forward to below.

What are the highlights that attendees should take away from your presentation?

I will be presenting an educational Spotlight Session on the use of caplacizumab for immune-mediated TTP. This session will be in the form of a debate, in which we will discuss the pros and cons of the medication. The format will leave ample room for questions as the use of caplacizumab has become a controversial topic in the United States due to the cost of the drug. What I want attendees to get from my presentation, is that the efficacy of the drug is not what is in question. This is a highly effective drug with a high cost, unfortunately a common scenario in rare diseases, not unique to iTTP or caplacizumab.

How do these findings and/or conclusions potentially impact clinical practice?

I will be reviewing the data on the efficacy of caplacizumab in patients with iTTP. What both HERCULES and TITAN studies have shown, as well as multiple real life data studies, is that caplacizumab provides benefits beyond the primary endpoint, but equally or more important, reducing risk of exacerbations and life threatening complications, including deaths and refractoriness. Therefore, every patient with a diagnosis of iTTP should receive caplacizumab upfront. This is the same recommendation made by experts in the field after seeing the effect of caplacizumab in our patients with TTP.

What are 3–5 questions you would ask attendees about the topic of your presentation to spark an engaging conversation?

I would ask attendees to pay special attention about the risk of bleeding associated with the medication; while statistically significant, you will see that most of the bleeding is self-resolved epistaxis and gingival bleeding. Taking into consideration the benefits of the drug, the risks of manageable bleeding are minor.

Another important factor to consider is the reduction exacerbations when patients receive caplacizumab upfront. Patients with iTTP have a 30-40% risk of exacerbations, that is another iTTP episode in the next 30 days after stopping plasma exchange. For our patients, this is extremely important, it means not having to be readmitted to the hospital, not having to get another central line. Every iTTP episode carries significant associated morbidity and mortality, not to mention the tremendous impact in quality of life by having to be away from family and work due to readmissions.  

Finally, I would ask attendees to  what is our role as physicians, deny our patients a medication with proven clinical benefit or advocate to address issues related to the cost of medications.

What sessions are you personally looking forward to listening to and sitting in on?

Besides this education spotlight session, there are very exciting sessions on classical hematology. On Friday, there will be a Scientific Workshop on Thrombotic Microangiopathies in which leaders in the field will discuss new insights in the pathogenesis, treatment and long term outcomes of TTP. In the plenary Scientific session, we will hear the results of the ADVANCE IV trial, on the efficacy and Safety of Efgartigimod for ITP. Finally, on the health services and quality session for non-malignant conditions, we will hear about new developments on the diagnosis of ITP and TTP.

Dr. Masias has received consulting fees or honorarium from Sanofi, Rigel, Takeda. She has received payment for lectures from Sanofi.

Image by Denis Novikov / GettyImages

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