Venetoclax plus daunorubicin and cytarabine for newly diagnosed acute myeloid leukemia: results of a phase 1b study

I Ioannis Mantzaris (1Montefiore Medical Center, Bronx, United States) M Mendel Goldfinger (2Montefiore Einstein Comprehensive Cancer Center, Bronx, United States) M Matan Uriel (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) A Aditi Shastri N Nishi Shah (2Winship Cancer Institute of Emory University, Atlanta, United States) K Kira Gritsman N Noah S. Kornblum (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) L Lauren Shapiro (Tulane University, New Orleans, Louisiana, United States) R Roberto Alejandro Sica (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) A Anne Munoz (2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States) N Nicole Chambers (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) A Aradhika Dhawan (2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States) J Jhannine Alyssa Verceles (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) K Karen Fehn (2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States) B Balda Tirone (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) L Lamisha Shah (21Montefiore Medical Center, Department of Oncology, New York, United States) S Shaunmonique Clark (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) C Chenxin Zhang M Mimi Kim D Dennis L. Cooper (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY) A Amit Verma M Marina Konopleva E Eric J. Feldman (1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY)

Abstract

Abstract Venetoclax (Ven), when combined with intensive chemotherapy, shows promise for untreated acute myeloid leukemia (AML), but its integration with the 7+3 regimen remains underexplored. In a phase 1b study, we assessed the safety and efficacy of Ven with daunorubicin and cytarabine in patients with newly diagnosed AML. A total of 34 patients (median age, 59 years; 62% non-White) received Ven at escalating durations (8, 11, or 14 days). Adverse events included febrile neutropenia (100%), sepsis (29%), and enterocolitis (23.5%), but there were no induction deaths. The median recovery times for neutrophils (>1.0 × 103/μL) and platelets (>100 × 103/μL) were less than 30 days. Composite complete remission was achieved in 85.3% of patients, and 86.2% were negative for measurable residual disease (MRD). Responses spanned all European Leukemia Net 2022 risk categories. With a median follow-up of 9.6 (2-20) months, the median duration of response, event-free survival, and overall survival were not reached. Ven (400 mg), when combined with 7+3 chemotherapy, was safe and effective in achieving MRD-negative remissions across all durations. Ven dose optimization is being explored in the expansion phase of this trial. Future multicenter studies should confirm our findings. This trial was registered at clinicaltrials.gov as #NCT05342584.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 17
Published April 24, 2025
Pages 1870-1875
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (23)

I

Ioannis Mantzaris

1Montefiore Medical Center, Bronx, United States

M

Mendel Goldfinger

2Montefiore Einstein Comprehensive Cancer Center, Bronx, United States

M

Matan Uriel

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

A

Aditi Shastri

N

Nishi Shah

2Winship Cancer Institute of Emory University, Atlanta, United States

K

Kira Gritsman

N

Noah S. Kornblum

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

L

Lauren Shapiro

Tulane University, New Orleans, Louisiana, United States

R

Roberto Alejandro Sica

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

A

Anne Munoz

2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States

N

Nicole Chambers

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

A

Aradhika Dhawan

2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States

J

Jhannine Alyssa Verceles

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

K

Karen Fehn

2Montefiore Einstein Comprehensive Cancer Center, Albert Einstein College of Medicine, Bronx, United States

B

Balda Tirone

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

L

Lamisha Shah

21Montefiore Medical Center, Department of Oncology, New York, United States

S

Shaunmonique Clark

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

C

Chenxin Zhang

M

Mimi Kim

D

Dennis L. Cooper

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY

A

Amit Verma

M

Marina Konopleva

E

Eric J. Feldman

1Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY