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Myelodysplastic Syndrome: Goals of Therapy


A review of the goals of therapy in managing myelodysplastic syndrome and instances in which patients might go untreated.


Bart Scott, MD, MS: When I see a patient with newly diagnosed MDS [myelodysplastic syndrome], 1 of the most important discussions we have is this: what are the goals of treatment? For each individual patient it may be different, and I see 3 broad outlines in what the goals of care are. One would be, how can we make your quality of life better? Can we help with your fatigue? Can we help with your bleeding and bruising? Can we help decrease your rates of infection so you can have more quality time with your loved ones and be better able to enjoy life?

Another big category would be, are we able to cure your disease? If I see a young patient or an older patient without a lot of comorbidities, then we would have a discussion about whether we should consider stem-cell transplant. That discussion is driven not only by their age and by their comorbidities but also by the severity of their underlying MDS. We tend to consider cure through allogeneic transplants in patients who have higher-risk disease.

Finally, the third category of patients would be, are we able to prolong your life? Are we looking at treatments that wouldn’t necessarily cure your disease but could potentially prolong your life? When I think about goals of care for patients, I would fit it into 3 broad categories: improving quality of life, potentially curing patients, and then finally prolonging survival.

I also think it’s important that, while those are the 3 broad categories, there can also be a lot of overlap between those, so they’re not necessarily mutually exclusive.

What percentage of patients go untreated with MDS? There have been publications that have looked at that. We have registry data, we have patient-internet-based surveys, and we have physician-based surveys. It depends on the risk of MDS, so if you look at lower-risk MDS, about 24% of patients go untreated; if you look at higher-risk MDS, about 5% of patients go untreated. If you look broadly at the group of patients who go untreated, the median duration of what we call a watch-and-wait approach is approximately 13 to 20 months.

Why might a patient be chosen for a watch-and-wait approach? Part of it is risk. If they’re lower risk and you’re less concerned about them having mortality from their disease, they may not have a lot of symptom burden from their disease. Maybe they don’t need any therapeutic intervention because the physician feels they wouldn’t have any clinical benefit from it. That’s why you see about 24% of patients with lower intermediate risk disease not receiving therapeutic intervention.

If you look at the higher-risk patients, with about 5% going untreated, that could be because they may have a lot of comorbidities that the physician is considering; that potentially they’re not a candidate for treatment; or maybe the comorbidities are so severe that they don’t feel like they would benefit from the treatment. Finally, there are some patients who are offered treatment options but decide that they would not want to pursue those options and would rather receive transfusions or antibiotics, for example.

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