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Outcome of adult acute myeloid leukemia patients with extramedullary disease and treatment with venetoclax/hypomethylating agents: acute myeloid leukemia

We evaluated response to venetoclax/hypomethylating agents (HMA) in 46 patients with acute myeloid leukemia (AML) characterized by extramedullary disease. The median age of these patients was 65 years (range, 19-81). The patients had a median of two sites of extramedullary disease (range, 1-5) and 3...

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Main Authors: Kayser, Sabine (Author) , Sanber, Khaled (Author) , Marconi, Giovanni (Author) , Mattei, Agnese (Author) , Luskin, Marlise R. (Author) , Kelkar, Amar (Author) , Cerrano, Marco (Author) , Kristensen, Daniel Tuyet (Author) , Roug, Anne Stidsholt (Author) , Sartor, Chiara (Author) , Giglio, Fabio (Author) , Riva, Marta (Author) , Rizzo, Lorenzo (Author) , Saraceni, Francesco (Author) , Guerzoni, Selene (Author) , Lessi, Federica (Author) , Borlenghi, Erika (Author) , Levis, Mark J. (Author) , Schlenk, Richard Friedrich (Author) , Jain, Tania (Author) , Papayannidis, Cristina (Author)
Format: Article (Journal)
Language:English
Published: February 2025
In: Haematologica
Year: 2025, Volume: 110, Issue: 2, Pages: 378-384
ISSN:1592-8721
DOI:10.3324/haematol.2024.285985
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.3324/haematol.2024.285985
Verlag, kostenfrei, Volltext: https://haematologica.org/article/view/haematol.2024.285985
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Author Notes:Sabine Kayser, Khaled Sanber, Giovanni Marconi, Agnese Mattei, Marlise R. Luskin, Amar Kelkar, Marco Cerrano, Daniel Tuyet Kristensen, Anne Stidsholt Roug, Chiara Sartor, Fabio Giglio, Marta Riva, Lorenzo Rizzo, Francesco Saraceni, Selene Guerzoni, Federica Lessi, Erika Borlenghi, Mark J. Levis, Richard F. Schlenk, Tania Jain and Christina Papayannidis
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Summary:We evaluated response to venetoclax/hypomethylating agents (HMA) in 46 patients with acute myeloid leukemia (AML) characterized by extramedullary disease. The median age of these patients was 65 years (range, 19-81). The patients had a median of two sites of extramedullary disease (range, 1-5) and 35 (76%) had concurrent bone marrow involvement. Twenty (43%) patients had high-risk genetic features according to the European LeukemiaNet 2022 classification. Twenty-nine (63%) had relapsed or were refractory after intensive chemotherapy, including 13 (28%) who had undergone prior allogeneic hematopoietic cell transplantation. Patients received a median of two cycles of venetoclax/HMA (range, 1-31). Twenty (43%) patients achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi) after venetoclax/HMA and five (11%) achieved a partial remission (PR). Six patients were subsequently consolidated with allogeneic hematopoietic cell transplantation (CR/CRi, N=4; PR, N=2). The median follow-up was 49.1 months (95% confidence interval [95% CI]: 26.1 months - not reached) and the median overall survival was 6.4 months (95% CI: 5.1-11 months). One-year and 2-year overall survival rates were 29.3% (95% CI: 18.6-46.2%) and 12.3% (95% CI: 5.5-27.6%), respectively. Age, with a cutoff of 60 years, did not have an impact on overall survival (P=0.90). Relapse occurred in 12 of 20 (60%) patients who achieved CR/CRi after venetoclax/ HMA treatment. Of those, all except one succumbed to their disease. Six (30%) patients were in CR/CRi at last follow-up and two (10%) died in CR. In our cohort of patients with AML with extramedullary disease with high-risk features, treatment with venetoclax/HMA resulted in an encouraging overall response rate of 54% with a CR/CRi rate of 43.5%. However, venetoclax/ HMA alone may not be effective in maintaining disease control.
Item Description:Online veröffentlicht: 3. Oktober 2024
Gesehen am 14.07.2025
Physical Description:Online Resource
ISSN:1592-8721
DOI:10.3324/haematol.2024.285985

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