Failure of three novel regimens to improve outcome for patients with relapsed or refractory acute myeloid leukaemia: a report from the Eastern Cooperative Oncology Group

Br J Haematol. 2010 Jan;148(2):217-25. doi: 10.1111/j.1365-2141.2009.07917.x. Epub 2009 Oct 5.


The treatment of relapsed acute myeloid leukaemia (AML) remains unsatisfactory. We conducted a phase II randomized trial where patients received intermediate-dose cytarabine for 4 d followed by gemtuzumab ozogamicin on day 5 (Arm A), or combined with liposomal daunorubicin for 3 d (Arm B), or cytarabine given for 5 d combined with cyclophosphamide for 3 d and topotecan by continuous infusion for 5 d (Arm C). Eligible patients had primary refractory AML, a first relapse after a remission of <1 year, or a second or greater relapse. The primary objective of this trial was attainment of a conventional complete remission (CR) or a CR without platelet recovery (CRp) in at least 40% of patients. The CR/CRp rates for the 82 eligible patients were 3/26 (12%) in Arm A, 2/29 (7%) in Arm B, and 1/27 (4%) in Arm C. No patients who had relapsed within 6 months of initial CR or who had suffered multiple relapses responded. More than 95% of patients subsequently died of AML. No unexpected toxicities were encountered. We conclude that none of these three regimens were effective enough in the treatment of high-risk relapsed or refractory AML to warrant further study. This trial was registered at as #NCT00005962.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Aminoglycosides / therapeutic use
  • Antibodies, Monoclonal / therapeutic use
  • Antibodies, Monoclonal, Humanized
  • Antimetabolites, Antineoplastic / therapeutic use*
  • Antineoplastic Agents / therapeutic use*
  • Cyclophosphamide / therapeutic use
  • Cytarabine / therapeutic use
  • Daunorubicin / administration & dosage
  • Daunorubicin / therapeutic use
  • Drug Therapy, Combination
  • Female
  • Gemtuzumab
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / mortality
  • Liposomes
  • Male
  • Middle Aged
  • Recurrence
  • Remission Induction
  • Survival Analysis
  • Topotecan / therapeutic use


  • Aminoglycosides
  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Antimetabolites, Antineoplastic
  • Antineoplastic Agents
  • Immunosuppressive Agents
  • Liposomes
  • Cytarabine
  • Topotecan
  • Cyclophosphamide
  • Gemtuzumab
  • Daunorubicin

Associated data