NCCN Guidelines Directing MDS Treatment Strategies

EP: 1.Prevalence and Types of Myelodysplastic Syndromes (MDS)

EP: 2.Risk Factors for Developing MDS

EP: 3.The Impact of MDS Diagnosis on Patients’ Quality of Life

EP: 4.Excluding Differential Diagnoses: MDS vs AML

EP: 5.Goals of Treatment for Patients with MDS

EP: 6.Utilizing Supportive Care in Patients with MDS

EP: 7.Evaluating MDS Treatment Pathways

EP: 8.Monitoring for Treatment Toxicity in Patients with MDS

EP: 9.Role of Oral Agents in MDS Treatment Strategy

EP: 10.Barriers to Optimal Care for Patients Diagnosed with MDS

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EP: 11.NCCN Guidelines Directing MDS Treatment Strategies

EP: 12.Trajectory of MDS Treatment Landscape

Ryan Haumschild, PharmD, MS, MBA: When we talk about treatments, there’s a variety of approaches. We talked about some of the most common ones. Mr Mancini, because you talked about it earlier, I want to direct this question to you. Because we know that NCCN [National Comprehensive Cancer Network] Guidelines are established, and there are clear criteria there in terms of how we treat unique patient populations. Do you see it reflecting real-world practice? Does the NCCN differ from real-world practice? If so, why? Talk to us a little about how you see practitioners adopting these guidelines into everyday treatment decisions.

Robert Mancini, PharmD, BCOP, FHOPA: NCCN Guidelines are pretty clean for some categories of MDS [myelodysplastic syndrome], whereas others are a more of a gray area. Lower-risk MDS is pretty straightforward. You’re treating the symptomatology. You’re treating what lineages are specifically affected. With symptomatic anemia, those guidelines are straightforward with 1 exception. If you look at the patient’s del(5q) symptomatic anemia and lenalidomide following the NCCN Guidelines, that’s straightforward. That’s what most people are going to do. If you look at the next category, your options are ESA [erythropoietin-stimulating agent] vs luspatercept. That’s where I start to see the first differences with the NCCN Guidelines. If you look at luspatercept, for example, the indication is after failing an erythropoietin-stimulating agent. You have to fail an ESA and have a certain number of transfusions. Though if you look at the guidelines, that’s where serum EPO [erythropoietin] comes into play. The guidelines outline where it says serum EPO should factor into whether you use an ESA or luspatercept. Pathophysiologically, that makes perfect sense.

Unfortunately, the FDA indication creates issues for us in terms of what the insurance is going to approve based on that. I hear it all the time in tumor board. It’s like their EPO level is 550 mU/mL, they’ve never had an ESA, but insurance is going to let us go to luspatercept in the front line. The guidelines help cover that, but we still face those battles sometimes. We start to take in some of these pathophysiological considerations beyond the guidelines and the indications for the medications. You have to factor that in and then jump through the hoops to get those products approved, which are the best products for our patients. You look at that situation. High risk is straightforward.

Then you have intermediate-risk patients with MDS. That’s where I see the greatest debate. You start to bring in the age and the fitness of the patient. In 1 situation, you may have providers pushing for a transplant. In other situations, you may have them pushing for symptomatic care. Unfortunately, you have a gray area within the stratification of patients with MDS. That sometimes makes NCCN Guidelines hard to follow.

Ryan Haumschild, PharmD, MS, MBA: Great. Ms Mahmoudjafari, you have a lot of real-world experience, and you also know the NCCN Guidelines. Are you seeing real-world practice differ from NCCN Guidelines in your area?

Zahra Mahmoudjafari, PharmD, BCOP, DPLA: Yes. One challenge with NCCN Guidelines is that they have to be black and white. One reason why I’ve been drawn to hematology for so long is that the reality of these patients is very gray. We don’t have a robust trial or thousands of patients in a 1-path-fits-all. It’s important that the treatment is individualized. We have to take the entire patient into consideration. I think of patient care as not only efficacy and safety but also financial toxicity as a third pillar. That’s something that NCCN is working toward by building value into its guidelines. I definitely think they do the best that they can, but there’s still the element of art when it comes to taking care of patients with hematologic malignancies.

Transcript edited for clarity.