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MDS Myelodysplastic syndromes

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Old Wed Dec 4, 2019, 07:17 PM
maggiemag maggiemag is offline
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Luspatercept

Hi everyone! You may already know this, but the drug Luspatercept has been FDA approved for treatment in beta thalassemia. Nope, not for low/intermediate risk MDS yet, but it’s approval for the one disease is very encouraging, I think. It has been shown to decrease transfusion dependence significantly in MDS. I think the target approval for MDS is April, 2020. So not that much longer to wait! It primarily was studied, I believe, for those MDS patients who have ring sideroblasts. I do not have those, so wonder if it will be strictly enforced. I asked my physician about that, and she thought there might be a way. We shall see! At any rate, we have some good news for a change.
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Margaret, age 68, dx MDS 5 q- 5/09- now RCMD; also MGUS. TP53 and TET2 mutations

Last edited by Neil Cuadra : Wed Dec 4, 2019 at 07:40 PM. Reason: removed extraneous linebreak
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Old Wed Dec 11, 2019, 07:24 AM
Pearl Pearl is offline
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Quote:
Originally Posted by maggiemag View Post
Hi everyone! You may already know this, but the drug Luspatercept has been FDA approved for treatment in beta thalassemia. Nope, not for low/intermediate risk MDS yet, but it’s approval for the one disease is very encouraging, I think. It has been shown to decrease transfusion dependence significantly in MDS. I think the target approval for MDS is April, 2020. So not that much longer to wait! It primarily was studied, I believe, for those MDS patients who have ring sideroblasts. I do not have those, so wonder if it will be strictly enforced. I asked my physician about that, and she thought there might be a way. We shall see! At any rate, we have some good news for a change.
Your post started us looking and the first group of posts at the link below are reports about Luspatercept from the very recent ASH conference.

http://acceleronpharma.com/science-p.../luspatercept/
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