Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients

Over the past two decades, the prognosis in adolescents and young adults (AYAs) diagnosed with acute myeloid leukemia (AML) has significantly improved. The standard intensive cytotoxic treatment approach for AYAs with AML, consisting of induction chemotherapy with anthracycline/cytarabine combinatio...

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Main Authors: Elena Chatzikalil, Kleoniki Roka, Panagiotis T. Diamantopoulos, Efthymia Rigatou, Georgia Avgerinou, Antonis Kattamis, Elena E. Solomou
Format: Article
Language:English
Published: MDPI AG 2024-04-01
Series:Journal of Clinical Medicine
Subjects:
Online Access:https://www.mdpi.com/2077-0383/13/7/2046
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author Elena Chatzikalil
Kleoniki Roka
Panagiotis T. Diamantopoulos
Efthymia Rigatou
Georgia Avgerinou
Antonis Kattamis
Elena E. Solomou
author_facet Elena Chatzikalil
Kleoniki Roka
Panagiotis T. Diamantopoulos
Efthymia Rigatou
Georgia Avgerinou
Antonis Kattamis
Elena E. Solomou
author_sort Elena Chatzikalil
collection DOAJ
description Over the past two decades, the prognosis in adolescents and young adults (AYAs) diagnosed with acute myeloid leukemia (AML) has significantly improved. The standard intensive cytotoxic treatment approach for AYAs with AML, consisting of induction chemotherapy with anthracycline/cytarabine combination followed by consolidation chemotherapy or stem cell transplantation, has lately been shifting toward novel targeted therapies, mostly in the fields of clinical trials. One of the most recent advances in treating AML is the combination of the B-cell lymphoma 2 (Bcl-2) inhibitor venetoclax with hypomethylating agents, which has been studied in elderly populations and was approved by the Food and Drug Administration (FDA) for patients over 75 years of age or patients excluded from intensive chemotherapy induction schemas due to comorbidities. Regarding the AYA population, venetoclax combination therapy could be a therapeutic option for patients with refractory/relapsed (R/R) AML, although data from real-world studies are currently limited. Venetoclax is frequently used by AYAs diagnosed with advanced hematologic malignancies, mainly acute lymphoblastic leukemia and myelodysplastic syndromes, as a salvage therapeutic option with considerable efficacy and safety. Herein, we aim to summarize the evidence obtained from clinical trials and observational studies on venetoclax use in AYAs with AML. Based on the available evidence, venetoclax is a safe and effective therapeutic option for R/R AML AYA patients. However, further research in larger cohorts is needed to confirm these data, establishing the benefits of a venetoclax-based regimen for this special population.
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spelling doaj.art-16ad913ff63842b78ac4a0024b3007a72024-04-12T13:21:24ZengMDPI AGJournal of Clinical Medicine2077-03832024-04-01137204610.3390/jcm13072046Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult PatientsElena Chatzikalil0Kleoniki Roka1Panagiotis T. Diamantopoulos2Efthymia Rigatou3Georgia Avgerinou4Antonis Kattamis5Elena E. Solomou6Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceDivision of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceFirst Department of Internal Medicine, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceDivision of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceDivision of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceDivision of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 11527 Athens, GreeceDepartment of Internal Medicine, University of Patras Medical School, 26500 Rion, GreeceOver the past two decades, the prognosis in adolescents and young adults (AYAs) diagnosed with acute myeloid leukemia (AML) has significantly improved. The standard intensive cytotoxic treatment approach for AYAs with AML, consisting of induction chemotherapy with anthracycline/cytarabine combination followed by consolidation chemotherapy or stem cell transplantation, has lately been shifting toward novel targeted therapies, mostly in the fields of clinical trials. One of the most recent advances in treating AML is the combination of the B-cell lymphoma 2 (Bcl-2) inhibitor venetoclax with hypomethylating agents, which has been studied in elderly populations and was approved by the Food and Drug Administration (FDA) for patients over 75 years of age or patients excluded from intensive chemotherapy induction schemas due to comorbidities. Regarding the AYA population, venetoclax combination therapy could be a therapeutic option for patients with refractory/relapsed (R/R) AML, although data from real-world studies are currently limited. Venetoclax is frequently used by AYAs diagnosed with advanced hematologic malignancies, mainly acute lymphoblastic leukemia and myelodysplastic syndromes, as a salvage therapeutic option with considerable efficacy and safety. Herein, we aim to summarize the evidence obtained from clinical trials and observational studies on venetoclax use in AYAs with AML. Based on the available evidence, venetoclax is a safe and effective therapeutic option for R/R AML AYA patients. However, further research in larger cohorts is needed to confirm these data, establishing the benefits of a venetoclax-based regimen for this special population.https://www.mdpi.com/2077-0383/13/7/2046Bcl-2 inhibitorvenetoclaxacute myeloid leukemiatargeted therapyadolescentsyoung adults
spellingShingle Elena Chatzikalil
Kleoniki Roka
Panagiotis T. Diamantopoulos
Efthymia Rigatou
Georgia Avgerinou
Antonis Kattamis
Elena E. Solomou
Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
Journal of Clinical Medicine
Bcl-2 inhibitor
venetoclax
acute myeloid leukemia
targeted therapy
adolescents
young adults
title Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
title_full Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
title_fullStr Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
title_full_unstemmed Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
title_short Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients
title_sort venetoclax combination treatment of acute myeloid leukemia in adolescents and young adult patients
topic Bcl-2 inhibitor
venetoclax
acute myeloid leukemia
targeted therapy
adolescents
young adults
url https://www.mdpi.com/2077-0383/13/7/2046
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