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

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.

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Journal
Haematologica
Published
2024-10-03
DOI
https://doi.org/10.3324/haematol.2024.285985
Citations
4
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
1.82
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article

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

Anne Stidsholt Roug, Sabine Käyser, Selene Guerzoni, Chiara Sartor et al.
4 citations
Haematologica
Acute Myeloid Leukemia Research
1.82
article

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

Anne Stidsholt Roug, Sabine Käyser, Selene Guerzoni, Chiara Sartor, Daniel Tuyet Kristensen, Marlise Rachael Luskin, A. Mattei, Giovanni Marconi, Erika Borlenghi, Federica Lessi, Lorenzo Rizzo, Marco Cerrano, Cristina Papayannidis, Tania Jain, Khaled Sanber, Richard F. Schlenk, Amar H. Kelkar, Fabio Giglio, Francesco Saraceni, Mark J. Levis, Marta Riva
article en
4 citations

Abstract

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.

HaematologicaVol. 110(2)
Johns Hopkins University (US), Heidelberg University (DE), Aalborg University Hospital (DK), University Hospital Heidelberg (DE), Aarhus University Hospital (DK), Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT), Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedali Riuniti di Ancona (IT), Azienda Ospedaliero Universitaria Ospedali Riuniti (IT), National Center for Tumor Diseases (DE), Dana-Farber Cancer Institute (US), Istituto di Ematologia di Bologna (IT), Presidio Ospedaliero (IT), Azienda Ospedale - Università Padova (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Sidney Kimmel Comprehensive Cancer Center (US), European Institute of Oncology (IT)
Good health and well-being
Openalex Percentile: Top 14%
Acute Myeloid Leukemia Research
1.82
DOIOpen Access PDFOpenAlex
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