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ASH 2025 | Venetoclax in combination with hypomethylating agents in adult patients with newly diagnosed CBF-AML

Christoph Röllig, MD, University Hospital Carl Gustav Carus, Dresden, Germany, discusses a study investigating venetoclax in combination with hypomethylating agents in adult patients with newly diagnosed core-binding factor acute myeloid leukemia (CBF-AML). Dr Röllig highlights findings from the study that suggest these patients perform in an intermediate prognostic group when treated with this combination. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.

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Transcript

So with this work we try to answer a clinically very important question. Patients with CBF-AML are normally considered as having favorable risk when they are treated with intensive chemotherapy. Because of that rather good prognosis, these patients were excluded from trials establishing the venetoclax hypomethylating agents combination as a standard of care for unfit patients ineligible for intensive chemotherapy...

So with this work we try to answer a clinically very important question. Patients with CBF-AML are normally considered as having favorable risk when they are treated with intensive chemotherapy. Because of that rather good prognosis, these patients were excluded from trials establishing the venetoclax hypomethylating agents combination as a standard of care for unfit patients ineligible for intensive chemotherapy. And because of that, we don’t really know how well patients with CBF-AML respond to a Ven-Aza combination. In order to find out, we looked at the literature and there are some publications on patient collections but these patients are in general quite young and they did not receive Ven-Azafor a long period of time but they went on to chemotherapy consolidation or allogeneic stem cell transplantation so they’re not really representative for the patient population we were interested in. And to answer that question, we, which means Klaus Metzeler from the University Hospital in Leipzig and Leo Ruhnke from my hospital at the University in Dresden, gathered data or patient data together from registries from international collaborating AML groups. Seven study groups actually provided data and we could put together a number of 53 patients with CBF-AML being treated with Ven-Aza or with venetoclax-based combinations. What we found is that these patients actually have a good response rate of around 70%. We found that there is no difference between inversion 16 or translocation 8-21 patients, which has been published before. And in general, the median overall survival was around 12 months, which is when you look at the different prognostic groups in less intensively treated patients, this is rather an intermediate number. So CBF-AML patients treated with Ven-Aza combinations seem to perform in a rather intermediate prognostic group rather than being favorable as when treated with intensive chemotherapy. These are the main findings of our trial.

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