Goals of Treatment for Patients with MDS


Experts outline treatment goals for patients diagnosed with MDS.

Ryan Haumschild, PharmD, MS, MBA: Let’s jump into the treatment landscape of myelodysplastic syndrome. I think it would be good for us to review the goals of treatment and the quality of life considerations that are so important when selecting the different therapy options. First, Dr Mahmoudjafari, I’d like to start with you. What are the goals of treatment for myelodysplastic syndrome? If you could talk a little bit maybe about the hematological improvement we’re looking for, the cytogenetic response, and lastly, pull in that quality of life, which we know is so important to these patients.

Zahra Mahmoudjafari, PharmD, BCOP, DPLA: Absolutely. You know I think so far, my answers have been vague and I’m going to continue that trend. Our goals for the treatment for MDS are patient and disease specific. It’s more of an art than it is a science, so for our patients that are in the lower risk group, we’re seeking improvement in their hematologic parameters, minimizing their complications, and trying to maximize that quality of life like you mentioned. Whereas in our higher risk group, we’re going to try to implement more aggressive treatment strategies and try to improve their survival. We’re looking to not only maintain their quality of life but also try to achieve the cytogenetic responses that we’re looking for. Additionally, it’s not only important to consider our own [therapeutics], but then there’s also the patient’s individual goals and their other comorbidities to take into consideration. It’s really only then that we can tailor the treatment strategies. So again, it’s very patient-specific and dependent. We are looking for symptomatic improvement and again, minimizing the complications and then doing less harm as much as we can.

Ryan Haumschild, PharmD, MS, MBA: That hematological improvement that you discussed does pair well with the quality of life. If someone’s symptomatic or if they’re very transfusion dependent, it obviously affects the laboratory values that we’re monitoring but also their ability to get to these different transfusions or resolution of symptoms. I really appreciate you highlighting the two and tying them together. I think I want to build upon that with quality of life. Dr Mancini, there’s a lot of studies out there that measure quality of life and it’s an emerging area that I think our payers and provider colleagues continue to look at, but it’s essential within myelodysplastic syndrome. If you could talk us through what some of the quality of life assessments are that are valuable in myelodysplastic syndrome. How are you using these assessments in the real world in clinical practice with these patients?

Robert Mancini, PharmD, BCOP, FHOPA: As you said, Dr Haumschild, there have been several studies out there looking at health-related quality of life in patients with MDS. This can be both a functionally burdensome disease as well as treatment burden heavy disease. There are several assessments out there, but probably the most common are the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 or EORTC QLQ-C30. The functional assessment of cancer anemia or FACT-An scales. The EORTC’s 30-item questionnaire used to determine health-related quality of life in patients with cancer, not necessarily MDS-specific but it is a scale from 0 to 100 where it changes in score from 5 to 10, 10 to 20, or greater than 20, have been associated with small, moderate, or large changes in quality of life, respectively.

In 2019, there were10 supplemental items that were proposed to be added, specifically to make it more useful for patients with MDS and other hematologic malignancies. The FACT-An scale contains 27 items from a cancer-specific FACT-general scale, plus 13 fatigue-related items and 7 anemia-related or non–fatigue-related items. This one has been shown to be a valid measure of health-related quality of life changes over time in patients with advanced MDS. There are a few Heme- or MDS-specific ones out there that I’m aware of. The Hematologic Malignancy Patient Reported Outcome or HM-PRO, which is a newer tool that has been validated in the last few years to measure the impact of hematologic malignancies, including MDS, on patient quality of life and disease signs or symptoms. There are 2 MDS-specific instruments, the 33-item Quality of Life in Myelodysplasia or QUALMS. I think that’s a great acronym for that particular assessment. It talks about what’s bothering them. The 29-item Quality of Life E or QOL-E, both of which 1-100 item scales that have shown that changes in 5-10 on that score have become clinically meaningful impacts in quality of life for those patients. Now more research is coming to correlate these scale score changes with the clinical significance being a little vague and gray, like Dr Mahmoudjafari said, but the full data has not been published that I’m aware of. However, we hope to see how these changes correlate better with clinical significance and how we decide on treatments for our patients.

Ryan Haumschild, PharmD, MS, MBA: I think to have these validated scales is going to be so important. Therefore, we can replicate them across different trials. Maybe include them inside the EMR for any type of quality improvement projects around these patients and hopefully have better baselines to track patients against. I appreciate you going through the several different scales. It sounds like there’s a few options out there and more literature is being built up over time.

Transcript edited for clarity.

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