Trajectory of MDS Treatment Landscape


The panel of experts provide their closing thoughts, highlighting unmet needs and treatments on the horizon for MDS.

Ryan Haumschild, PharmD, MS, MBA: One thing I think about is the unmet need as we continue the management of myelodysplastic syndrome [MDS]. For us, in integrated delivery networks and community oncology practices, 1 unmet need is our ability to review patients and make sure we’re practicing the best practice. What I mean is that it’s not that we’re not doing best for the patient, but are we dose titrating soon enough? Are we transitioning patients between therapies fast enough? If a patient needs a dose escalation, are we doing that in a timely manner and monitoring our data to make sure? Let’s say 71% of patients get a dose escalation during their treatment and we’re seeing only 40%. How do we explain that difference? How do we make sure we’re providing that to our patients? I want to pose this question to both of you. Ms Mahmoudjafari, what are the unmet needs in terms of the management of myelodysplastic syndrome?

Zahra Mahmoudjafari, PharmD, BCOP, DPLA: We have lots of opportunities. We have the opportunity for utilizing novel therapeutics and targeting difficult-to-treat patient populations, not only high-risk patients but also patients with failed supportive care or standard care. For our high-risk patients, the majority of whom are ineligible to safely receive transplant—we’ve already established this is the only potential cure—we have a significant challenge with disease relapse if they’re not able to receive any transplants. The novel therapeutic is my choice in terms of unmet needs.

Ryan Haumschild, PharmD, MS, MBA: Mr Mancini, I’ve got to get your input on this question as well. What are some of the remaining unmet needs in MDS?

Robert Mancini, PharmD, BCOP, FHOPA: Ms Mahmoudjafari beat me to the punch on that. The other thing, going back to what we talked about in the context of the guidelines, is teasing out some of those patient-specific factors in terms of outcomes for some of those more intermediate-risk groups. If we can figure out what might stratify them in 1 group vs another, I think back to when we had the Oncotype DX conversation about that middle group. Do we do treatment? Do we not do treatment for those that wouldn’t fall into high or low risk? There’s always going to be a gray area. We need to put more time, effort, and research into how to tease out some of those patient-specific factors that allow us to further segment our intermediate-risk patients.

Ryan Haumschild, PharmD, MS, MBA: The way we stratify patients has changed dramatically over the last 5 years, as we’re starting to isolate unique patient populations more within the syndromes. Another thing is that as we look at the prediction of treatment moving forward, I see us leveraging more on precision medicine and having unique treatments for these targeted therapies. We’ll also have more real-world evidence, because it’s such a small patient population. How do we leverage large aggregates of data to make sure we have good-quality improvement initiatives and that we’re monitoring these patients appropriately and providing the best plans? Those are my votes, and you can steal my answers. Ms Mahmoudjafari, what are some things that will predict the change in treatment for myelodysplastic syndrome moving forward?

Zahra Mahmoudjafari, PharmD, BCOP, DPLA: Ihave a lot more knowledge in terms of the genetic mutation associated with MDS. My hope in terms of treatment is individualizing and using the precision medicine knowledge. You hit it on the head in terms of future reported outcomes. It’s not a science. A lot of push and pull has to happen. Potentially, we can see more combination therapy, and that has become the norm in hematologic malignancies as well. The future is bright, and there’s a lot happening in this space.

Ryan Haumschild, PharmD, MS, MBA: Mr Mancini, how do you predict the treatment of MDS changing in the future?

Robert Mancini, PharmD, BCOP, FHOPA: I’m going to steal those answers too. Precision medicine is a big thing. We talked about all these genetic things, but the low-hanging fruit for MDS is that SF3B1 gene that Ms Mahmoudjafari mentioned earlier. Looking at these specific genes and mutations is going to be important. Beyond that, as pharmacists we also play a role in understanding pharmacokinetics and pharmacodynamics of these medications. What are their mechanisms of action? How does that play out. A perfect example is EPO [erythropoietin] levels. It makes physiological sense why we would use 1 vs the other. As we gain more understanding about the physiology, risk factors, and components in MDS, they’re going to help us find better, more streamlined treatments for a lot of our patients.

Ryan Haumschild, PharmD, MS, MBA: MDS is a disease that can burden patients, and we’re constantly trying to improve their quality of life and treatment options. But the future is bright, and we’ve hit on a lot of key tenants throughout this discussion. I’m most excited about more pharmacist-driven protocols and pharmacists getting more involved in titrating to the response and leveraging some of those genomic factors to make decisions that can improve the efficacy in these patients. There are a lot of therapies and treatment opportunities ahead that get people excited. Mr Mancini, you’ve talked about prediction and changing the way we treat it. What are you most excited about in the future of MDS?

Robert Mancini, PharmD, BCOP, FHOPA: We’ve hit on iron chelation and how that’s a big deal for impact, not only in those who are transfusion dependent but also for any patient with MDS. It can greatly improve survival. I don’t know about other places, but we manage a lot of those patients on iron chelation therapy as pharmacists. As we get more information about the supportive care for these patients and how we can play a role in those pieces of things, that’s going to be interesting. There are more data and research to be done to learn about this disease. We’re going to be important in terms of how to manage the treatments but also the supportive care medications.

Ryan Haumschild, PharmD, MS, MBA: Excellent. Ms Mahmoudjafari, you talked about the future being bright. What are you most excited about in the treatment of myelodysplastic syndrome?

Zahra Mahmoudjafari, PharmD, BCOP, DPLA: The things you’re both excited about, I’m equally excited about how we’ve established supportive care as a big component of MDS management. Pharmacists are well positioned to help the health care team when it comes to adjustments in monitoring of our patients. Perhaps related to this, we’re talking more about the social determinants of health. We’re incorporating many additional elements that we didn’t talk about as much in the past when I was in training. We’re moving toward treating the entire patient and treating not just the disease but also the other factors that may be contributing to their outcome. As we continue to teach our learners and ourselves and advocate for change, it’s a very exciting time for all patients with hematologic malignancies and cancer but especially for MDS.

Ryan Haumschild, PharmD, MS, MBA: Thank you. To our viewing audience, we hope you found this Pharmacy Times® Peer Exchange to be rich and informative.

Transcript edited for clarity.

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