Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemia

References

• Larson RA. Is secondary leukemia an independent poor prognostic factor in acute myeloid leukemia? Best Pract Res Clin Haematol. 2007;20(1):29–37.
   PubMed Web of Science ®Google Scholar
• Granfeldt Ostgard LS, Medeiros BC, Sengelov H. Epidemiology and clinical significance of secondary and therapy-related acute myeloid leukemia: a national population-based cohort study. J Clin Oncol. 2015;33:3641–3649.
   PubMed Web of Science ®Google Scholar
• Hulegardh E, Nilsson C, Lazarevic V, 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;90:208–214.
   PubMed Web of Science ®Google Scholar
• American Cancer Society. Key statistics for acute myeloid leukemia (AML). [cited 2019 Mar 28]. Available from: https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html
   Google Scholar
• Miesner M, Haferlach C, Bacher U, et al. Multilineage dysplasia (MLD) in acute myeloid leukemia (AML) correlates with MDS-related cytogenetic abnormalities and a prior history of MDS or MDS/MPN but has no independent prognostic relevance: a comparison of 408 cases classified as “AML not otherwise specified” (AML-NOS) or “AML with myelodysplasia-related changes” (AML-MRC). Blood. 2010;116:2742–2751.
   PubMed Web of Science ®Google Scholar
• Schoch C, Kern W, Schnittger S, et al. Karyotype is an independent prognostic parameter in therapy-related acute myeloid leukemia (t-AML): an analysis of 93 patients with t-AML in comparison to 1091 patients with de novo AML. Leukemia. 2004;18(1):120–125.
   PubMed Web of Science ®Google Scholar
• Leith CP, Kopecky KJ, Godwin J, et al. Acute myeloid leukemia in the elderly: assessment of multidrug resistance (MDR1) and cytogenetics distinguishes biologic subgroups with remarkably distinct responses to standard chemotherapy. A Southwest Oncology Group study. Blood. 1997;89(9):3323–3329.
   PubMed Web of Science ®Google Scholar
• Grimwade D, Hills RK, Moorman AV, et al. Refinement of cytogenetic classification in acute myeloid leukemia: determination of prognostic significance of rare recurring chromosomal abnormalities among 5876 younger adult patients treated in the United Kingdom Medical Research Council trials. Blood. 2010;116(3):354–365.
   PubMed Web of Science ®Google Scholar
• Xu XQ, Wang JM, Gao L, et al. Characteristics of acute myeloid leukemia with myelodysplasia-related changes: a retrospective analysis in a cohort of Chinese patients. Am J Hematol. 2014;89(9):874–881.
   PubMed Web of Science ®Google Scholar
• Bhatia S. Therapy-related myelodysplasia and acute myeloid leukemia. Semin Oncol. 2013;40(6):666–675.
   PubMed Web of Science ®Google Scholar
• Kayser S, Dohner K, Krauter J, et al. The impact of therapy-related acute myeloid leukemia (AML) on outcome in 2853 adult patients with newly diagnosed AML. Blood. 2011;117(7):2137–2145.
   PubMed Web of Science ®Google Scholar
• Lim WS, Tardi PG, Dos Santos N, et al. Leukemia-selective uptake and cytotoxicity of CPX-351, a synergistic fixed-ratio cytarabine:daunorubicin formulation, in bone marrow xenografts. Leuk Res. 2010;34(9):1214–1223.
   PubMed Web of Science ®Google Scholar
• Tardi P, Johnstone S, Harasym N, et al. In vivo maintenance of synergistic cytarabine:daunorubicin ratios greatly enhances therapeutic efficacy. Leuk Res. 2009;33(1):129–139.
   PubMed Web of Science ®Google Scholar
• Lancet JE, Uy GL, Cortes JE, 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):2684–2692.
   PubMed Web of Science ®Google Scholar
• Faderl S, Uy GL, Schiller GJ, et al. Outcome in older patients with newly diagnosed high-risk/secondary AML (sAML) who achieve remission with CPX-351 versus 7 + 3 induction. Presented at: International Symposium on Acute Leukemias XVII (ISAL XVII); 2019 February 24–27; Munich, Germany. Poster #39.
   Google Scholar
• VYXEOS^® (daunorubicin and cytarabine injection), solution for intravenous use [package insert]. Palo Alto (CA): Jazz Pharmaceuticals, Inc.; 2019.
   Google Scholar
• European Medicines Agency, Committee for Medicinal Products for Human Use (CHMP). Vyxeos daunorubicin/cytarabine. Opinion. [cited 2019 Sep 12]. Available from: https://www.ema.europa.eu/medicines/human/summaries-opinion/vyxeos
   Google Scholar
• Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391–2405.
   PubMed Web of Science ®Google Scholar
• Dohner H, Estey EH, Amadori S, et al. Diagnosis and management of acute myeloid leukemia in adults: recommendations from an international expert panel, on behalf of the European LeukemiaNet. Blood. 2010;115:453–474.
   PubMed Web of Science ®Google Scholar
• Szotkowski T, Rohon P, Zapletalova L, et al. Secondary acute myeloid leukemia - a single center experience. Neoplasma. 2010;57(2):170–178.
   PubMed Web of Science ®Google Scholar
• Feldman EJ, Lancet JE, Kolitz JE, et al. First-in-man study of CPX-351: a liposomal carrier containing cytarabine and daunorubicin in a fixed 5:1 molar ratio for the treatment of relapsed and refractory acute myeloid leukemia. J Clin Oncol. 2011;29(8):979–985.
   PubMed Web of Science ®Google Scholar
• Lancet JE, Cortes JE, Hogge DE, et al. Phase 2 trial of CPX-351, a fixed 5:1 molar ratio of cytarabine/daunorubicin, vs cytarabine/daunorubicin in older adults with untreated AML. Blood. 2014;123(21):3239–3246.
   PubMed Web of Science ®Google Scholar
• Feldman EJ, Kolitz JE, Trang JM, et al. Pharmacokinetics of CPX-351; a nano-scale liposomal fixed molar ratio formulation of cytarabine: daunorubicin, in patients with advanced leukemia. Leuk Res. 2012;36(10):1283–1289.
   PubMed Web of Science ®Google Scholar
• Ryan DH, Uy GL, Cortes JE, et al. Efficacy and safety of CPX-351 versus 7 + 3 in a subgroup of older patients with newly diagnosed acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) enrolled in a phase 3 study. Blood. 2018;132(1):1425.
   Google Scholar