Elacytarabine has single-agent activity in patients with advanced acute myeloid leukaemia.
AffiliationDepartment of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, TX 77230, USA.
MetadataShow full item record
AbstractElacytarabine is a novel cytotoxic nucleoside analogue, independent of nucleoside transporters (e.g. human Equilibrative Nucleoside Transporter 1 [hENT1]) for cell uptake, and mechanisms of action similar to those of cytarabine. This Phase II study assessed the efficacy and safety of elacytarabine in patients with advanced stage acute myeloid leukaemia (AML). Patients received 2000 mg/m(2) per d continuously i.v. during days 1-5 every 3 weeks. Patients were matched by six risk factors with historical controls; remission rate (assessed after 1 or 2 cycles) and 6-month survival were compared. Sixty-one patients, median age 58 years, were enrolled; 52% had five or six risk factors. The remission rate was 18% (95% confidence interval: 9-30%) vs. 4% in controls (P < 0·0001), 6-month survival rate was 43%, median overall survival was 5·3 months (vs. 1·5 months); 10 patients (16%) were referred for stem cell transplantation after treatment. Side effects were predictable and manageable. The most common grade 3/4 non-haematological adverse events were febrile neutropenia, hypokalemia, fatigue, hyponatraemia, dyspnoea and pyrexia. Thirty-day all-cause mortality, after start of treatment, was 13% vs. 25% in controls. Elacytarabine has monotherapy activity in patients with advanced AML. This study provides proof-of-concept that lipid esterification of nucleoside analogues is clinically relevant.
CitationElacytarabine has single-agent activity in patients with advanced acute myeloid leukaemia. 2012, 158 (5):581-8 Br J Haematol
JournalBritish Journal of Haematology
- A phase II study of elacytarabine in combination with idarubicin and of human equilibrative nucleoside transporter 1 expression in patients with acute myeloid leukemia and persistent blasts after the first induction course.
- Authors: Rizzieri D, Vey N, Thomas X, Huguet-Rigal F, Schlenk RF, Krauter J, Kindler T, Gjertsen BT, Blau IW, Jacobsen TF, Johansen M, Bergeland T, Gianella-Borradori A, Krug U
- Issue date: 2014 Sep
- Elacytarabine (CP-4055) in the treatment of acute myeloid leukemia.
- Authors: DiNardo CD, O'Brien S, Gandhi VV, Ravandi F
- Issue date: 2013 Aug
- Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia (QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial.
- Authors: Cortes JE, Khaled S, Martinelli G, Perl AE, Ganguly S, Russell N, Krämer A, Dombret H, Hogge D, Jonas BA, Leung AY, Mehta P, Montesinos P, Radsak M, Sica S, Arunachalam M, Holmes M, Kobayashi K, Namuyinga R, Ge N, Yver A, Zhang Y, Levis MJ
- Issue date: 2019 Jul
- Idarubicin, cytarabine, and nivolumab in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a single-arm, phase 2 study.
- Authors: Ravandi F, Assi R, Daver N, Benton CB, Kadia T, Thompson PA, Borthakur G, Alvarado Y, Jabbour EJ, Konopleva M, Takahashi K, Kornblau S, DiNardo CD, Estrov Z, Flores W, Basu S, Allison J, Sharma P, Pierce S, Pike A, Cortes JE, Garcia-Manero G, Kantarjian HM
- Issue date: 2019 Sep
- Elacytarabine in relapsed/refractory acute myeloid leukaemia: an evaluation of clinical efficacy, pharmacokinetics, cardiac safety and effects on lipid profile.
- Authors: Knapper S, Chevassut T, Duarte R, Bergua JM, Salamero O, Johansen M, Jacobsen TF, Hals PA, Rasch W, Gianella-Borradori A, Smith M
- Issue date: 2014 Mar