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EBMT 2026 | The current approach to response assessment in TA-TMA

Eleni Gavriilaki, MD, PhD, Aristotle University of Thessaloniki, Thessaloniki, Greece, shares insights into the current approach to response assessment in patients with transplant-associated thrombotic microangiopathy (TA-TMA), highlighting that the current desired treatment outcome is achieving a clinically meaningful partial response. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.

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Transcript

Response assessment is controversial in this field. Up to now we didn’t have any specific target and specific treatment, so it was hard to assess response. So that is why probably clinical trials had some big problem in getting a drug approved because the actual response assessment was overall survival. Overall survival was better with the drug, so the drug was eventually approved...

Response assessment is controversial in this field. Up to now we didn’t have any specific target and specific treatment, so it was hard to assess response. So that is why probably clinical trials had some big problem in getting a drug approved because the actual response assessment was overall survival. Overall survival was better with the drug, so the drug was eventually approved. But in the meantime, we tried to identify some response assessment criteria that are based on the diagnostic criteria. This was with the help of all the community in the national community and this process was very hard because we don’t have enough data to justify all decisions in this process, for example we don’t know exactly when to assess response, but as we said we will learn more from data that are coming up. Up to now, we have this response assessment definition criteria, and we’re trying to use them in the clinical trial setting, but also in the real-world setting. And our goal for our patients is for them to achieve a clinically meaningful partial response. We think that we don’t have a target to achieve CR, complete response, but a clinically meaningful partial response would benefit our patients.

 

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