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General Updates | Future research directions in ITP: improving disease understanding & aiming for disease modification

In this interview, Hanny Al-Samkari, MD, Massachusetts General Hospital, Boston, MA, comments on the research directions that he believes should be prioritized in the field of immune thrombocytopenia (ITP), highlighting the importance of aiming for disease modification to potentially free patients from the need for long-term therapy. Dr Al-Samkari also suggests that there is a need to better understand the disease biology and multiple intrinsic subtypes of ITP, allowing for personalized therapeutic strategies. This interview took place virtually.

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Transcript

You know, the areas that I think should be prioritized really are disease modification, because we’re treating ITP, you know, the model has been chronic controller medications and that served our patients well to a point, right? But I think that the possibility of intervening early on in the course of disease with a drug that is able to modify the course of disease and maybe free the patient from the need for long-term therapy is a very, very attractive option...

You know, the areas that I think should be prioritized really are disease modification, because we’re treating ITP, you know, the model has been chronic controller medications and that served our patients well to a point, right? But I think that the possibility of intervening early on in the course of disease with a drug that is able to modify the course of disease and maybe free the patient from the need for long-term therapy is a very, very attractive option. And this initially was, there was some hope that rituximab as an off-label drug would help do this, and we know that it doesn’t, but the question is now, do drugs like ianalumab or possibly rilzabrutinib or some of these other drugs that are very potent and act on multiple aspects of autoimmunity, can they lead us to that potential promised land, so to speak, of disease modification? That’s an important area. 

And then, of course, we need to understand more about this disease. There’s no question that there are multiple true intrinsic subtypes of ITP that we do not see clinically, we just see a low platelet count clinically. And understanding more about, you know, the fact that this disease is probably multiple different diseases, really, that we kind of lump together as one, and that’s why we see such varied presentations, varied symptomatologies, and varied responses to therapies. And ultimately, right, I think the fourth era of ITP management will be the one characterized by personalized therapies, where we really are able to understand the disease better and therefore treat it better.

 

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Disclosures

Consultancy: Amgen, Alnylam, Alpine, Agios, Takeda, Pharmacosmos, Sanofi, Sobi, Novartis; Research funding to institution: Amgen, Agios, Novartis, Sobi, Vaderis.