In a study led by researchers at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine and funded by Daiichi Sankyo, patients recently diagnosed with a common and aggressive form of acute myeloid leukemia reported having improved quality of life when a newly approved drug was part of the treatment plan.

The results of the study were released during an oral presentation [1] at the 65th ASH Annual Meeting and Exposition, the American Society of Hematology’s conference held December 9 – 12, 2023 in San Diego, California.

The report has been identified by the American Society of Hematology (ASH) as a clinically relevant abstract.

Mutations in several genes have been implicated in the development of acute myeloid leukemia, or AML, a type of cancer that starts in the blood-forming cells of bone marrow. About 30% of all cases result from mutations in a gene called FLT3 – FMS-like tyrosine kinase 3 – and the majority of these involve “internal tandem duplication,” or ITD.

The new drug, quizartinib (Vanflyta®; Daiichi Sankyo), an oral, highly potent, selective, type 2 FLT3 inhibitor, specifically targets this FLT3-ITD mutation, which is found in about 25% of all AML cases and is associated with poor survival.

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“In previous research, we showed that patients treated with quizartinib had improved survival compared with patients who didn’t receive the drug. This study further demonstrated that patients treated with quizartinib had better quality of life over time and that the side effects from the drug did not negatively affect the improved quality of life,” said Dr. Mikkael A. Sekeres, MD, Professor of Medicine and Chief of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, and former Chair of the Oncologic Drugs Advisory Committee of the US Food and Drug Administration (FDA). was also senior author of the abstract presented at ASH. Photo courtesy: 2023 Clutch Content Partners. Used with permission.

Improved survival
“In previous research, we showed that patients treated with quizartinib had improved survival compared with patients who didn’t receive the drug,” said Mikkael A. Sekeres, MD, a Sylvester researcher, chief of the Division of Hematology, and senior author of the abstract presented at the annual meeting of the American Society of Hematology

“This study further demonstrated that patients treated with quizartinib had better quality of life over time and that the side effects from the drug did not negatively affect the improved quality of life,” Sekeres added.

One focus of Sekeres’ research is acute myeloid leukemia in older adults.

Combination therapy
Quizartinib was recently approved by the U.S. Food and Drug Administration (FDA) to be used in combination with chemotherapy (Cytarabine, Daunorubicin and Idarubicin) for the first and second phases of treatment – called induction and consolidation phases – for adult patients with newly diagnosed FLT3-ITD-positive AML.

It also can be used alone as maintenance therapy and has been approved for similar applications in the European Union and Japan.

Patient-reported quality-of-life outcomes
The current study, looking specifically at patient-reported quality-of-life outcomes, is a continuation Ian exploratory endpoint ) of findings coming from QuANTUM-First (NCT02668653), a global, Phase 3 randomized clinical trial that evaluated the safety and effectiveness of the drug.

The authors said QuANTUM-First is the first study to explore the impact of an FLT3-inhibitor as first-line therapy on patient-reported outcomes. Patients treated with quizartinib who achieve complete remission remain in remission longer, continue treatment longer, and have better quality of life than those who do not receive the drug.

The purpose of this study is to compare the effect of quizartinib versus placebo (administered with standard induction and consolidation chemotherapy, then administered as continuation therapy for up to 36 cycles) on overall survival in subjects with FLT3-internal tandem duplication (ITD) positive AML.

“Not only did we find that patients treated with quizartinib lived longer, and they lived better, but those who went on to have a bone marrow transplant had particularly improved quality of life,” noted Sekeres, who was a co-author of the QuANTUM-First findings reported earlier this year in The Lancet. [2]

Sekeres said the research team plans to conduct additional studies combining quizartinib with different chemotherapy regimens used to treat patients who have FLT3-positive AML to see if it provides similar improvements in health related quantity and quality of life (hrQoL).

Based on the results from the QuANTUM-First trial, the investigators concluded that quizartinib provides a new, effective, and generally well tolerated treatment option for adult patients with FLT3-ITD-positive newly diagnosed AML.

The current abstract’s first author, Esther Natalie Oliva, MD, a hematologist and researcher at Grande Ospedale Metropolitano Bianchi Melacrino in Reggio Calabria, Italy, presented the findings earlier today.

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Clinical trial
Quizartinib With Standard of Care Chemotherapy and as Continuation Therapy in Patients With Newly Diagnosed FLT3-ITD (+) Acute Myeloid Leukemia (AML) (QuANTUM-First) – ClinicalTrials.gov ID NCT02668653

Highlights of prescribing information
Quizartinib (Vanflyta®; Daiichi Sankyo)[Prescribing Information]

Reference
[1] Oliva EN, Unni S, Cottone F, Vashi R, Li X, Cortes J, Sekeres MA. 918 Patient-Reported Outcomes in Acute Myeloid Leukemia Patients with FLT3-ITD Mutation Receiving Quizartinib Vs. Standard Chemotherapy: Results from the Quantum-First Trial. Oral and Poster Abstracts. December 11, 2023: 4:00 PM. Clinical Relevant Abstract.
[2] Erba HP, Montesinos P, Kim HJ, Patkowska E, Vrhovac R, Žák P, Wang PN, Mitov T, Hanyok J, Kamel YM, Rohrbach JEC, Liu L, Benzohra A, Lesegretain A, Cortes J, Perl AE, Sekeres MA, Dombret H, Amadori S, Wang J, Levis MJ, Schlenk RF; QuANTUM-First Study Group. Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023 May 13;401(10388):1571-1583. doi: 10.1016/S0140-6736(23)00464-6. Epub 2023 Apr 25. Erratum in: Lancet. 2023 Oct 14;402(10410):1328. PMID: 37116523.

Feature image: General Views of attendees during the day at the American Society of Hematology. Photo courtesy: © 2018 ASH/Scott Morgan. Used with permission.

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