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EBMT 2022 | Updates from the DEFIFrance study

Mohamad Mohty, MD, PhD, Saint-Antoine Hospital, Paris, France, discusses some updates and results from the DEFIFrance study (NCT02851407) investigating the safety and efficacy of defibrotide for the treatment of hepatic veno-occlusive disease (VOD), a complication of hematopoietic stem cell transplantation (HSCT). Prof. Mohty highlights some important findings from this study, including complete response (CR) and survival of patients, as well as the safety profile of this agent. Prof. Mohty also emphasizes the importance of early detection and treatment of VOD, as this results in improved patient outcomes. This interview took place at the 48th Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) 2022, which was held virtually.

Transcript (edited for clarity)

The DEFIFrance study is a multicenter national trial from 53 transplant centers in France. It’s a real-life study, post-marketing and it collected mainly prospective data, but also some retrospective data. And the idea was about collecting real-life data from patients who received defibrotide for VOD treatment, but also for other reasons, in case it happened. And actually it is the largest real-life dataset, almost 800 patients among them, 250+ received defibrotide for severe or very severe VOD...

The DEFIFrance study is a multicenter national trial from 53 transplant centers in France. It’s a real-life study, post-marketing and it collected mainly prospective data, but also some retrospective data. And the idea was about collecting real-life data from patients who received defibrotide for VOD treatment, but also for other reasons, in case it happened. And actually it is the largest real-life dataset, almost 800 patients among them, 250+ received defibrotide for severe or very severe VOD. And what is very reassuring from the results of this DEFIFrance study, is that when you look to the efficacy in terms of CR rate, after defibrotide therapy, or even looking into the day 100 survival in these patients with severe or very severe VOD the results are in line with what we already know. And there is an important piece of information, which is about the importance of early treatment, because the severe VOD patients are doing better than the very severe VOD. So this highlights the fact that we need to be always very vigilant and try to track VOD very quickly in order to initiate the right treatment. And, of course, the other important information from the DEFIFrance study is about the safety profile. And in this real-world, real-life setting, actually, there are no additional or unexpected safety signals compared to what we already know about the drugs, which has been used now for more than 15 years ago.

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