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ASH 2021 | DEFIFrance: Outcomes of patients with post-HCT VOD/SOS receiving defibrotide

Mohamad Mohty, MD, PhD, Saint Antoine Hospital, Paris, France, presents long-term results from the DEFIFrance study which analyzed real world data on patients receiving defibrotide for the treatment of severe or very severe veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), a potentially life-threatening complication of hematopoietic stem cell transplantation (HCT). In total, 798 patients treated with defibrotide from more than 50 centers across France were investigated, with 251 treated for severe/very severe VOD/SOS post-HCT. Overall, the results highlighted the feasibility and efficacy of defibrotide treatment for severe/very severe VOD/SOS. The estimated day 100 post-HCT survival rate was 61% in these patients and the estimated complete response rate was 74%. Survival rates were significantly higher in patients with severe VOD/SOS compared to very severe VOD/SOS, emphasizing the need for early detection and treatment. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.

Transcript (edited for clarity)

I’m Mohamad Mohty, from the Sorbonne University in Saint-Antoine Hospital in Paris, France. It is my pleasure to be with you today and give you an overview about a study we presented during this last ASH 2021 Annual Meeting. Mainly the final results of the so-called DEFIFrance Study. Just as a reminder, this is a real-world data, a real-life registry about patients treated with defibrotide in different centers in France...

I’m Mohamad Mohty, from the Sorbonne University in Saint-Antoine Hospital in Paris, France. It is my pleasure to be with you today and give you an overview about a study we presented during this last ASH 2021 Annual Meeting. Mainly the final results of the so-called DEFIFrance Study. Just as a reminder, this is a real-world data, a real-life registry about patients treated with defibrotide in different centers in France.

The study included more than 700 patients. Among these patients, we have 251 patients who received defibrotide for the treatment of severe or very severe veno-occlusive disease, according to the EBMT criteria. Here we had some very interesting results, highlighting first, the feasibility and efficacy of defibrotide for the treatment of severe and very severe V.O.D. This is in line with the label of the drug. The other key information we have from this large registry study is that the results, whether it comes to the response rate, especially complete response, or when it comes to the percentage of being alive at day 100, which is a traditional endpoint in these kind of studies. Actually the results are significantly better when it comes to the severe V.O.D group compared to the very severe V.O.D group.

I think this is an important message, which is in line actually with our current understanding and belief of this very complex disease, V.O.D, because it means that early intervention is crucial to improve the outcome of these patients. Although the drug can be effective at a later stage, like very severe, actually, the best results are achieved when we have an early intervention. That brings us actually, to the issue of trying to identify, detect and diagnose V.O.D very early in order to establish the appropriate monitoring and introduce defibrotide whenever needed.

Last but not least, the other important message from this large study was about the safety of the drug. The good news is that now with such a large number of patients receiving it, actually, the safety profile is definitely in line with what has been previously published and known about the drug, so no major or no new safety signal. This is always good news.

In summary, we have here the largest, I would say, one of the largest registry-based studies, when it comes to the use of defibrotide and clearly establishing the value of the drug, but most importantly, the importance of early intervention. Because as we all know, veno-occlusive disease is a very serious life-threatening complication after stem cell transplantation. Although by the way, it can exist outside stem cell transplantation after high dose chemotherapy, for instance, or after using antibody-drug conjugates like gemtuzumab or inotuzumab based chemotherapy. Nevertheless, the key message: early intervention is crucial if we want to improve the outcome of these patients.

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