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Huemer, F; Melchardt, T; Jansko, B; Wahida, A; Jilg, S; Jost, PJ; Klieser, E; Steiger, K; Magnes, T; Pleyer, L; Greil-Ressler, S; Rass, C; Greil, R; Egle, A.
Durable remissions with venetoclax monotherapy in secondary AML refractory to hypomethylating agents and high expression of BCL-2 and/or BIM.
Eur J Haematol. 2019; 102(5):437-441 Doi: 10.1111/ejh.13218 (- Case Report) [OPEN ACCESS]
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Jost Philipp

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Acute myeloid leukemia (AML) is a disease of the elderly population and survival remains poor after failure of hypomethylating agents (HMA). The BCL-2 inhibitor venetoclax demonstrated activity as monotherapy and in combination with chemotherapy or HMA in AML. In this case series, patients with secondary AML (sAML) not eligible for intensive chemotherapy and refractory to HMA were treated with venetoclax within a named patient program at our tertiary cancer center in Salzburg, Austria. Between April 2017 and September 2018, seven patients with sAML received venetoclax therapy. Two out of seven patients achieved a complete remission upon venetoclax initiation with a PFS of 505 days and 352 days and another patient achieved complete peripheral blood blast clearing within nine days after start of venetoclax. Among the venetoclax responders, primary refractory disease to prior HMA therapy was documented, 2 patients harbored IDH1/IDH2 mutations and one patient had an antecedent myeloproliferative neoplasm. High BCL-2 and/or BIM expression in myeloblasts was found in venetoclax responders and response was significantly associated with overall survival (responders: 364 days versus non-responders: 24 days, P = 0.018). Venetoclax monotherapy is safe and is able to induce durable responses in elderly patients with secondary AML after treatment failure with HMA. © 2019 The Authors. European Journal of Haematology Published by John Wiley & Sons Ltd.
Find related publications in this database (using NLM MeSH Indexing)
Aged -
Aged, 80 and over -
Antineoplastic Agents - therapeutic use
Bcl-2-Like Protein 11 - genetics
Bcl-2-Like Protein 11 - metabolism
Biomarkers, Tumor -
Bridged Bicyclo Compounds, Heterocyclic - therapeutic use
DNA Methylation - drug effects
Drug Resistance, Neoplasm -
Female -
Gene Expression Regulation, Leukemic - drug effects
Humans -
Leukemia, Myeloid, Acute - diagnosis
Leukemia, Myeloid, Acute - drug therapy
Leukemia, Myeloid, Acute - genetics
Male -
Proto-Oncogene Proteins c-bcl-2 - genetics
Proto-Oncogene Proteins c-bcl-2 - metabolism
Remission Induction -
Retreatment -
Sulfonamides - therapeutic use
Treatment Outcome -

Find related publications in this database (Keywords)
hypomethylating agents
myeloproliferative neoplasm
secondary acute myeloid leukemia
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