The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety

Döhner H, Weisdorf DJ, Bloomfield CD. Acute myeloid leukemia. N Engl J Med. 2015;373(12):1136–52.

Article  PubMed  Google Scholar 

Dong Y, Shi O, Zeng Q, Lu X, Wang W, Li Y, et al. Leukemia incidence trends at the global, regional, and national level between 1990 and 2017. Exp Hematol Oncol. 2020;9:14.

Article  PubMed  PubMed Central  Google Scholar 

Jani CT, Ahmed A, Singh H, Mouchati C, Al Omari O, Bhatt PS, et al. Burden of AML, 1990–2019: estimates from the Global Burden of Disease Study. JCO Glob Oncol. 2023;9:e2300229.

Article  PubMed  PubMed Central  Google Scholar 

Linet MS, Curtis RE, Schonfeld SJ, Vo JB, Morton LM, Dores GM. Survival of adult AML patients treated with chemotherapy in the US population by age, race and ethnicity, sex, calendar-year period, and AML subgroup, 2001–2019. EClinicalMedicine. 2024;71:102549.

Article  PubMed  PubMed Central  Google Scholar 

Nagel G, Weber D, Fromm E, Erhardt S, Lübbert M, Fiedler W, et al. Epidemiological, genetic, and clinical characterization by age of newly diagnosed acute myeloid leukemia based on an academic population-based registry study (AMLSG BiO). Ann Hematol. 2017;96(12):1993–2003.

Article  PubMed  PubMed Central  Google Scholar 

Hulegårdh E, Nilsson C, Lazarevic V, Garelius H, Antunovic P, Rangert Derolf Å, et al. Characterization and prognostic features of secondary acute myeloid leukemia in a population-based setting: a report from the Swedish Acute Leukemia Registry. Am J Hematol. 2015;90(3):208–14.

Article  PubMed  Google Scholar 

Martínez-Cuadrón D, Megías-Vericat JE, Serrano J, Martínez-Sánchez P, Rodríguez-Arbolí E, Gil C, et al. Treatment patterns and outcomes of 2310 patients with secondary acute myeloid leukemia: a PETHEMA registry study. Blood Adv. 2022;6(4):1278–95.

Article  PubMed  PubMed Central  Google Scholar 

Döhner H, Wei AH, Appelbaum FR, Craddock C, DiNardo CD, Dombret H, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140(12):1345–77.

Article  PubMed  Google Scholar 

Heuser M, Ofran Y, Boissel N, Brunet Mauri S, Craddock C, Janssen J, et al. Acute myeloid leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020;31(6):697–712.

Article  PubMed  CAS  Google Scholar 

Kayser S, Levis MJ. Updates on targeted therapies for acute myeloid leukaemia. Br J Haematol. 2022;196(2):316–28.

Article  PubMed  CAS  Google Scholar 

Bhansali RS, Pratz KW, Lai C. Recent advances in targeted therapies in acute myeloid leukemia. J Hematol Oncol. 2023;16(1):29.

Article  PubMed  PubMed Central  Google Scholar 

Wysota M, Konopleva M, Mitchell S. Novel therapeutic targets in acute myeloid leukemia (AML). Curr Oncol Rep. 2024;26(4):409–20.

Article  PubMed  PubMed Central  Google Scholar 

Liu H, Stanworth SJ, McPhail S, Bishton M, Rous B, Bacon A, et al. Impact of patient demographics on treatment outcomes in AML: a population-based registry in England, 2013–2020. Blood Adv. 2024;8(17):4593–605.

Article  PubMed  PubMed Central  Google Scholar 

Döhner H, Estey E, Grimwade D, Amadori S, Appelbaum FR, Büchner T, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424–47.

Article  PubMed  PubMed Central  Google Scholar 

Cloos J, Ossenkoppele GJ, Dillon R. Minimal residual disease and stem cell transplantation outcomes. Hematol Am Soc Hematol Educ Program. 2019;2019(1):617–25.

Article  Google Scholar 

Garcia-Manero G, Othus M, Pagel JM, Radich JP, Fang M, Rizzieri DA, et al. SWOG S1203: a randomized phase iii study of standard cytarabine plus daunorubicin (7 + 3) therapy versus idarubicin with high dose cytarabine (IA) with or without vorinostat (IA + V) in younger patients with previously untreated acute myeloid leukemia (AML). Blood. 2016;128(22):901.

Article  Google Scholar 

Löwenberg B, Ossenkoppele GJ, van Putten W, Schouten HC, Graux C, Ferrant A, et al. High-dose daunorubicin in older patients with acute myeloid leukemia. N Engl J Med. 2009;361(13):1235–48.

Article  PubMed  Google Scholar 

Kantarjian H, O’Brien S, Cortes J, Giles F, Faderl S, Jabbour E, et al. Results of intensive chemotherapy in 998 patients age 65 years or older with acute myeloid leukemia or high-risk myelodysplastic syndrome: predictive prognostic models for outcome. Cancer. 2006;106(5):1090–8.

Article  PubMed  Google Scholar 

Juliusson G, Antunovic P, Derolf A, Lehmann S, Möllgård L, Stockelberg D, et al. Age and acute myeloid leukemia: real world data on decision to treat and outcomes from the Swedish Acute Leukemia Registry. Blood. 2009;113(18):4179–87.

Article  PubMed  CAS  Google Scholar 

Atallah E, Cortes J, O’Brien S, Pierce S, Rios MB, Estey E, et al. Establishment of baseline toxicity expectations with standard frontline chemotherapy in acute myelogenous leukemia. Blood. 2007;110(10):3547–51.

Article  PubMed  CAS  Google Scholar 

Zeidan AM, Podoltsev NA, Wang X, Zhang C, Bewersdorf JP, Shallis RM, et al. Patterns of care and clinical outcomes with cytarabine-anthracycline induction chemotherapy for AML patients in the United States. Blood Adv. 2020;4(8):1615–23.

Article  PubMed  PubMed Central  Google Scholar 

Pratz KW, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Döhner H, et al. Long-term follow-up of VIALE-A: venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemia. Am J Hematol. 2024;99(4):615–24.

Article  PubMed  CAS  Google Scholar 

U.S. Department of Health and Human Services, Food and Drug Administration Oncology Center of Excellence, Center for Drug Evaluation and Research, and Center for Biologics Evaluation and Research. Guidance for industry: acute myeloid leukemia: developing drugs and biological products for treatment. 2022. https://www.fda.gov/media/162362/download. Accessed 2 Sept 2024.

Aldoss I, Pullarkat V, Stein AS. Venetoclax-containing regimens in acute myeloid leukemia. Ther Adv Hematol. 2021;12:2040620720986646.

Article  PubMed  PubMed Central  CAS  Google Scholar 

DiNardo CD, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Wei AH, et al. Azacitidine and venetoclax in previously untreated acute myeloid leukemia. N Engl J Med. 2020;383(7):617–29.

Article  PubMed  CAS  Google Scholar 

Pratz KW, DiNardo CD, Selleslag D, Li J, Yamamoto K, Konopleva M, et al. Cytopenia management in patients with newly diagnosed acute myeloid leukemia treated with venetoclax plus azacitidine in the VIALE-A study. Blood. 2020;136(Suppl 1):51–3.

Article  Google Scholar 

Dicko A, Mayer LD, Tardi PG. Use of nanoscale delivery systems to maintain synergistic drug ratios in vivo. Expert Opin Drug Deliv. 2010;7(12):1329–41.

Article  PubMed  CAS  Google Scholar 

Tolcher AW, Mayer LD. Improving combination cancer therapy: the CombiPlex® development platform. Future Oncol. 2018;14(13):1317–32.

Article  PubMed  CAS  Google Scholar 

Mayer LD, Harasym TO, Tardi PG, Harasym NL, Shew CR, Johnstone SA, et al. Ratiometric dosing of anticancer drug combinations: controlling drug ratios after systemic administration regulates therapeutic activity in tumor-bearing mice. Mol Cancer Ther. 2006;5(7):1854–63.

Article  PubMed  CAS  Google Scholar 

Ma L, Kohli M, Smith A. Nanoparticles for combination drug therapy. ACS Nano. 2013;7(11):9518–25.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Liboiron BD, Louie AC, Mayer LD. Nanoscale complexes: a novel nanotechnology-based platform to optimize combination cancer therapies: rational development & improved delivery using CombiPlex®. Drug Dev Deliv. 2016;16(1):34–9.

Google Scholar 

VYXEOS® (daunorubincin and cytarabine) liposome for injection, for intravenous use [prescribing information]. Palo Alto: Jazz Pharmaceuticals, Inc.; 2022.

VYXEOS® (daunorubicin and cytarabine liposome for injection) product monograph. Mississauga: Jazz Pharmaceuticals Canada, Inc.; 2023.

Vyxeos liposomal (44 mg/100 mg powder for concentrate for solution for infusion) summary of product characteristics. European Medicines Agency: Jazz Pharmaceuticals Ireland, Ltd.; 2024.

Vyxeos liposomal (44 mg/100 mg powder for concentrate for solution for infusion) summary of product characteristics. Electronic Medicines Compendium: Jazz Pharmaceuticals UK, Ltd.; 2025.

Lancet JE, Uy GL, Cortes JE, Newell LF, Lin TL, Ritchie EK, et al. CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia. J Clin Oncol. 2018;36(26):2684–92.

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