ESH MDS 2024 | Emerging treatment options for patients with ESA-refractory MDS: luspatercept & imetelstat
Eva Hellström-Lindberg, MD, PhD, Karolinska University Hospital Huddinge, Stockholm, Sweden, outlines potential therapeutic options for patients with myelodysplastic syndromes (MDS) who have failed first-line treatment with erythropoiesis-stimulating agents (ESAs). The timing of ESA treatment is highly consequential for treatment response, with implementation prior to the onset of transfusion dependency resulting in a response rate twice as high as that seen in ESA-treated patients who are already transfusion-dependent. Prof. Hellström-Lindberg comments on luspatercept as a promising option for SF3B1-mutated MDS and highlights that imetelstat is also showing encouraging results in ongoing investigations. This interview took place at the European School of Hematology 9th Translational Research Conference on Myelodysplastic Syndromes (ESH MDS) in Budapest, Hungary.
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Transcript (edited for clarity)
So one thing is that, if you give ESAs before the onset of a permanent transfusion need, the average response rate will be twice as high as if you started after they have started to become transfusion-dependent. So that’s one strategy. It will also last longer.
When patients fail, it depends a bit on whether they also have a progress of disease in terms of transforming to a more high-risk disease...
So one thing is that, if you give ESAs before the onset of a permanent transfusion need, the average response rate will be twice as high as if you started after they have started to become transfusion-dependent. So that’s one strategy. It will also last longer.
When patients fail, it depends a bit on whether they also have a progress of disease in terms of transforming to a more high-risk disease. Then, of course, they need other types of treatment- chemotherapy, azacitidine, transplant, and so on. If they are still in the low-risk phase, it’s usually because the stem cell pool has been completely invaded by the mutated clone. We have luspatercept, of course, it’s licensed at the European level but still is not easy to get hold of because, in many individual countries, it has not been approved with regard to the pricing. But luspatercept could be an option, in particular in the SF3B1-mutated patients, perhaps also outside. Another interesting option is imetelstat which acts in a totally different way.If things hold now in novel studies, it seems to actually reduce the clone in comparison to remaining residual wild-type cells. So that would be an action that is more like what you see with lenalidomide in del5q minus patients. So that may be something. It will not have a huge response rate, but it’s definitely something that will be an option for other patients. There are many other drugs in trial. I’m a bit…yeah…let’s see, let’s see. Just because it’s shown in a Phase I to II trial, it doesn’t mean it will hold. So many other options have not really been met with expectations, but I would say luspatercept and imetelstat are two new options that we have to find the role for.
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