Venetoclax-Based Regimens for Relapsed/Refractory Acute Myeloid Leukemia in a Real-Life Setting: A Retrospective Single-Center Experience.
Matteo PicciniSofia PilerciMarta MerliniPietro GriecoBarbara ScappiniSara BenciniBenedetta PeruzziRoberto CaporaleLeonardo SignoriFabiana PancaniAlessandro Maria VannucchiGiacomo GianfaldoniPublished in: Journal of clinical medicine (2021)
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.
Keyphrases
- acute myeloid leukemia
- newly diagnosed
- allogeneic hematopoietic stem cell transplantation
- end stage renal disease
- ejection fraction
- low dose
- clinical trial
- prognostic factors
- healthcare
- acute lymphoblastic leukemia
- randomized controlled trial
- high dose
- metabolic syndrome
- type diabetes
- smoking cessation
- hodgkin lymphoma
- replacement therapy