VIDEO: Unmet needs in myelodysplastic syndrome

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription.

We have a ton of unmet needs. You know, maybe finally, you could argue that transfusion dependent lower risk anemia were making major, you know, thresholds and is not the most critical unmet need, but patients with other cytopenias, neutropenia, thrombocytopenia, big unmet needs changing the natural history of the disease big unmet needs, having targeted therapy, we have nothing. And the targeted therapy, again, outside of IDH1 and IDH2, those mutations don't really exist in MDS. So could we target splicing mutations which may have some excitement with this IRAC14 class? You know, can we finally improve outcomes in P53 mutant patient populations? We talked about HMA failure disease and then sort of, you know, just the concept of overall improving chances of cure for this disease. You know, allogeneic stem cell transplant is the only curative therapy, and I think there's still a lot of sort of misinformation about who can go to transplant. You know, we know that very clearly and fit patients up to the age of 75. And for example I have an 80 year old going to transplant this month. So age is really not the factor.And I think the problem in the challenge really even in the entire US community is it's thought about a little bit as an afterthought, I think sometimes. So yes, the young under 50 patient which is rare, yes, everybody, okay send them to academic center, think about transplant. But this needs to be thought about in any higher risk MDS patient that is on the fit side. Because once you fail standard therapy like HMA our options are extremely limited. And getting patient to transplant becomes less and less and less. But if you think about really optimizing therapy beforehand ideally getting deeper remissions, transplanting them early meaning within the first six months of treatment taking them to transplant and then even in some settings thinking about, you know, maintenance trials or other considerations, sort of this concept of total therapy, I think we can really continue to improve long-term survival. But it's really a multi, you know, team collaboration a community academic collaboration and is really a huge area that I think even right now we can significantly improve the outcomes of our patients.