Relapsed/refractory (R/R) acute myeloid leukemia (AML) is a largely unmet medical need, owing to the lack of standardized, effective treatment approaches, resulting in an overall dismal outcome. The only curative option for R/R AML patients is allogeneic hematopoietic stem cell transplantation (HSCT) which is only applicable in a fraction of patients due to the scarce efficacy and high toxicity of salvage regimens. Recently, a number of targeted agents with relatively favorable toxicity profiles have been explored in clinical trials for R/R AML patients. The Bcl-2 inhibitor venetoclax, in combination with hypomethylating agents or low dose cytarabine, has produced impressive results for newly diagnosed AML, while its role in R/R disease is not well defined yet. We retrospectively analyzed the clinical outcomes of 47 R/R AML patients treated with venetoclax-based regimens between March 2018 and December 2020 at our institution. Overall, we report a composite complete response rate of 55% with an overall acceptable toxicity profile. Outcomes were particularly favorable for NPM1 mutated patients, unlike for FLT3-ITD positive patients irrespective of NPM1 status. For patients treated with intention to transplant, the procedure could be finally performed in 54%. These findings suggest a role for venetoclax-based regimens in R/R AML patients and support the design of prospective studies.

Venetoclax-Based Regimens for Relapsed/Refractory Acute Myeloid Leukemia in a Real-Life Setting: A Retrospective Single-Center Experience / Piccini, Matteo; Pilerci, Sofia; Merlini, Marta; Grieco, Pietro; Scappini, Barbara; Bencini, Sara; Peruzzi, Benedetta; Caporale, Roberto; Signori, Leonardo; Pancani, Fabiana; Vannucchi, Alessandro Maria; Gianfaldoni, Giacomo. - In: JOURNAL OF CLINICAL MEDICINE. - ISSN 2077-0383. - ELETTRONICO. - 10:(2021), pp. 1684-1695. [10.3390/jcm10081684]

Venetoclax-Based Regimens for Relapsed/Refractory Acute Myeloid Leukemia in a Real-Life Setting: A Retrospective Single-Center Experience

Piccini, Matteo;Pilerci, Sofia;Merlini, Marta;Grieco, Pietro;Scappini, Barbara;Bencini, Sara;Peruzzi, Benedetta;Caporale, Roberto;Signori, Leonardo;Pancani, Fabiana;Vannucchi, Alessandro Maria;Gianfaldoni, Giacomo
2021

Abstract

Relapsed/refractory (R/R) acute myeloid leukemia (AML) is a largely unmet medical need, owing to the lack of standardized, effective treatment approaches, resulting in an overall dismal outcome. The only curative option for R/R AML patients is allogeneic hematopoietic stem cell transplantation (HSCT) which is only applicable in a fraction of patients due to the scarce efficacy and high toxicity of salvage regimens. Recently, a number of targeted agents with relatively favorable toxicity profiles have been explored in clinical trials for R/R AML patients. The Bcl-2 inhibitor venetoclax, in combination with hypomethylating agents or low dose cytarabine, has produced impressive results for newly diagnosed AML, while its role in R/R disease is not well defined yet. We retrospectively analyzed the clinical outcomes of 47 R/R AML patients treated with venetoclax-based regimens between March 2018 and December 2020 at our institution. Overall, we report a composite complete response rate of 55% with an overall acceptable toxicity profile. Outcomes were particularly favorable for NPM1 mutated patients, unlike for FLT3-ITD positive patients irrespective of NPM1 status. For patients treated with intention to transplant, the procedure could be finally performed in 54%. These findings suggest a role for venetoclax-based regimens in R/R AML patients and support the design of prospective studies.
2021
10
1684
1695
Piccini, Matteo; Pilerci, Sofia; Merlini, Marta; Grieco, Pietro; Scappini, Barbara; Bencini, Sara; Peruzzi, Benedetta; Caporale, Roberto; Signori, Leonardo; Pancani, Fabiana; Vannucchi, Alessandro Maria; Gianfaldoni, Giacomo
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Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1257178
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