VJHemOnc is committed to improving our service to you

EHA 2019 | Updates on defibrotide therapy

VJHemOnc is committed to improving our service to you

Paul Richardson

Paul Richardson, MD, Dana-Farber Cancer Institute, Boston, MA, discusses the use of defibrotide for the treatment of veno-occlusive disease and sinusoidal obstruction syndrome at the 24th Congress of the European Hematology Association (EHA) 2019, held in Amsterdam, Netherlands.

Transcript (edited for clarity):

Yes. In terms of, you know, we’re where defibrotide sits at the moment, it’s in a very good place in terms of its global use and uptake in the context of treating veno-occlusive disease, sinusoidal obstruction syndrome.

In terms of clinical trials, ongoing. The ongoing prevention study has been very successful in its initial phase...

Yes. In terms of, you know, we’re where defibrotide sits at the moment, it’s in a very good place in terms of its global use and uptake in the context of treating veno-occlusive disease, sinusoidal obstruction syndrome.

In terms of clinical trials, ongoing. The ongoing prevention study has been very successful in its initial phase. And at the moment it’s undergoing an interim look to determine whether the study gets expanded and continues or whether it’s actually meeting some of its primary endpoints.

My expectation is that we’ll need to continue to expand that trial in the prevention setting in order to get a very clear signal, simply because I think the nature of the study is such that to me anyway, it makes sense that the sample size will have to be substantial.

The good news is that in terms of defibrotide overall, we continue to see a very strong signal in terms of real-world experience with very similar response rates and outcomes, as demonstrated in the treatment studies, being reflected in real world experiences. And it’s nice to see case series and publications reflecting that.

In terms of future directions, there are a number of very exciting new developments. The graft versus host disease study is up and going. There are also specific studies addressing the microangiopathies of transplant that are getting going as well.

And finally there is exploratory work into evaluating the role of defibrotide in the abrogation of a what’s called cytokine release syndrome, which is a very important complication of cellular therapies from CAR T through to bispecific T-cell engagers.

The protocols are specifically looking at CRS and related toxicities in CAR T, and these studies are getting going and I think that’s very exciting. So it’s showing the spectrum of applications of the drug as an endothelial stabilizer, growing, reflecting, on the one hand it’s promising activity, but on the other hand also it’s excellent tolerability profile.

Read more...

Share this video  
Similar topics

Sign-up for our Newsletter!

Keep up to date with all the latest news with our monthly newsletter