Unmet Needs and Future Perspectives in Treatment of MDS

EP: 1.Risk Stratification of Myelodysplastic Syndromes [MDS]

EP: 2.Importance of Early Diagnosis of MDS for Lower and Higher-risk Patients

EP: 3.Frontline Treatment Options for Lower-Risk MDS

EP: 4.Clinical Use of Luspatercept on Patients with Lower-risk MDS

EP: 5.Safety and Efficacy of Luspatercept in Frontline Setting in Patients with MDS

EP: 6.Emerging Therapies for Treatment of MDS

EP: 7.Clinical Management of Patients with Lower-risk MDS

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EP: 8.Unmet Needs and Future Perspectives in Treatment of MDS

Transcript:

Ralph Boccia, MD, FACP: This has been a real eye-opener since we got to see the presentation yesterday. I think that it holds a lot of promise. I’m very excited that we potentially will have this option. I would be surprised if the FDA didn’t think this was a worthwhile therapy, but only time will tell. If that’s the case, to think that we can have almost 60% of patients who are no longer transfusion-dependent, almost 75% of patients that’ll have at least a benefit, if not becoming transfusion-independent, with a relatively safe safety profile there. There’s nothing better than that I think for this population of patients.

Hetty Carraway, MD: I think it’s so promising. I do agree. I worry that there’s more patients on the study that are ring sideroblasts SF3B1-positive, so that data are fantastic and provocative. The remaining question is: Is that representative in general of our lower-risk MDS [myelodysplastic syndromes] population as a whole? The way that you’re framing it is 75% of people are going to have a response. I hope that’s the case, and hopefully we’ll get there to have that type of impact on our patients.

Ralph Boccia, MD, FACP: Looking at the different subsets has been looking at patients who were SF3B1-positive or -negative, those who had the higher or lower transfusion burden, the higher or lower under 500, of course, EPO [erythropoietin]levels, it seems like the luspatercept [Reblozyl] data was always better. Response rates always higher than the ESA [erythropoiesis-stimulating agent] data.

Hetty Carraway, MD: It’s true for the ring sideroblasts-positive and the SF3B1 mutated. But for the patients that were ring sideroblasts-negative or SF3B1 unmutated, they had similar responses and some similar durability. We’re hopeful that all patients will benefit from this, and we’re hopeful that the FDA will really want to see all of the data when it’s fully mature, particularly the safety data as you talked about. We’re really excited about having a potential new option for patients with MDS.

Ralph Boccia, MD, FACP: I don’t think the FDA’s going to approve ring sideroblasts-negative patients, but hopefully they will see there were enough patients that were ring sideroblasts-positive to feel that this is a reasonable therapy to give us approval for.

Hetty Carraway, MD: Although we use ESA-based therapies in the upfront management of patients with MDS, I am not sure that we really have an FDA-approved agent in that space. I think [it] is also really an exciting time.

Ralph Boccia, MD, FACP: I agree. No doubt about it. Being a hematologist oncologist today is an exciting field.

Hetty Carraway, MD: I completely agree. It truly is humbling. And an honor to be in this field

Ralph Boccia, MD, FACP: Very different than 15, 20 years ago.

Hetty Carraway, MD: I want to thank you, Dr Boccia, for this discussion. Before we conclude, I would like to get your closing thoughts on unmet needs and future perspectives in the field.

Ralph Boccia, MD, FACP: The unmet need is not still met. I think we would all agree that even with this very nice, statistically significant data that we still have further to go. I really am excited that we have the other options of we’ve got the HIF [hypoxia-inducible factor]inhibitors, we’ve got luspatercept [Reblozyl] and some other things coming along. And as we begin to further evaluate them and combine them, I think we have a bright future that certainly will push the envelope and bring us to a better and better state every year or so.

Hetty Carraway, MD: Well, I think this data that we’ve talked about with the COMMANDS study [NCT03682536] is really exciting and promising. I’m looking forward to the role of imetelstat and looking at the data that have been presented here and the promising hematologic improvements that we see in patients with that agent, particularly in patients that have a high transfusion burden and nice responses. And so, two really exciting agents for lower-risk MDS patients. A high transfusion burden is really challenging, and it challenges our patients in terms of symptoms. Even in the high-risk MDS, which we didn’t even get to talk about today, there are ongoing phase 3 studies that we’re eagerly awaiting data for their final outcomes in the coming months to year. I’m hopeful that we’re inching forward to better outcomes for our patients and a better understanding of the pathogenesis of disease, particularly because of the molecular annotation that we’ve been able to do for our patients. I want to thank you and thank our viewing audience. We hope you found our OncLive® Insights discussion to be useful and valuable in the treatment of your patients with lower-risk MDS.

Transcript edited for clarity.