ASH 2023 | Long-term evaluation of luspatercept in ESA-intolerant/refractory LR-MDS: MEDALIST follow-up study

Actually, I must say that we are very happy of the results we obtained for our lower-risk MDS patients, because we have now the possibility to induce and obtain a transfusion independence in a very high proportion of patients. The use of luspatercept, that is an active trap molecule, has demonstrated activity in inducing transfusion independence longer than eight weeks in around 49% of patients if you consider the long-term therapy with this drug. So this drug is given subcutaneously every three weeks, and the comparison with placebo indicated that patients really had a meaningful clinical advantage. 75 patients who were treated and followed for a long time, almost six years, with the the long follow-up after the first considerations, so in an extension phase and then a rollover phase, they obtain, as I mentioned, 49% of transfusion independence longer than eight weeks and 30% longer than 16 weeks. This is really, I want to reiterate, a meaningful objective that was reached. 

And then, if you consider the patients who had a transfusion independence longer than one year, which is interrupted transfusion independence, we also have observed quite a high proportion of these patients, among the ones who achieved eight weeks transfusion independence, almost half of them had such a long transfusion independence. This means that we are really eliminating transfusion independence for a substantial period for all of them. 

And I want to just stress the fact that these were lower-risk MDS patients with transfusion dependence and with ring sideroblasts. So it’s a specific subtype of MDS, and this is what we have now with a long-term period follow-up. But luspatercept has been approved in the US also for first line treatment, and this is also for patients without ring sideroblasts, so for all lower-risk MDS patients who experience transfusion dependence.