A randomized phase 3 study of tipifarnib compared with best supportive care, including hydroxyurea, in the treatment of newly diagnosed acute myeloid leukemia in patients 70 years or older

Jean Luc Harousseau, Giovanni Martinelli, Wieslaw W. Jedrzejczak, Joseph M. Brandwein, Dominique Bordessoule, Tamas Masszi, Gert J. Ossenkoppele, Julia A. Alexeeva, Gernot Beutel, Johan Maertens, Maria Belen Vidriales, Hervé Dombret, Xavier Thomas, Alan K. Burnett, Tadeusz Robak, Nuriet K. Khuageva, Anatoly K. Golenkov, Elena Tothova, Lars Mollgard, Youn C. ParkAnnick Bessems, Peter De Porre, Angela J. Howes

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Abstract

This phase 3, multicenter, open-label study evaluated the efficacy and safety of tipifarnib compared with best supportive care (BSC), including hydroxyurea, as first-line therapy in elderly patients (≥70 years) with newly diagnosed, de novo, or secondary acute myeloid leukemia. A total of 457 patients were enrolled with 24% 80 years of age or older. Tipifarnib 600 mg orally twice a day was administered for the first 21 consecutive days, in 28-day cycles. The primary endpoint was overall survival. The median survival was 107 days for the tipifarnib arm and 109 days for the BSC arm. The hazard ratio (tipifarnib vs BSC) for overall survival was 1.02 (P value by stratified log-rank test, .843). The complete response rate for tipifarnib in this study (8%) was lower than that observed previously, but with a similar median duration of 8 months. The most frequent grade 3 or 4 adverse events were cytopenias in both arms, slightly more infections (39% vs 33%), and febrile neutropenia (16% vs 10%) seen in the tipifarnib arm. The results of this randomized study showed that tipifarnib treatment did not result in an increased survival compared with BSC, including hydroxyurea. This trial was registered at www.clinicaltrials.gov as #NCT00093990.

Original languageEnglish
Pages (from-to)1166-1173
Number of pages8
JournalBlood
Volume114
Issue number6
DOIs
Publication statusPublished - Nov 19 2009

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ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Harousseau, J. L., Martinelli, G., Jedrzejczak, W. W., Brandwein, J. M., Bordessoule, D., Masszi, T., Ossenkoppele, G. J., Alexeeva, J. A., Beutel, G., Maertens, J., Vidriales, M. B., Dombret, H., Thomas, X., Burnett, A. K., Robak, T., Khuageva, N. K., Golenkov, A. K., Tothova, E., Mollgard, L., ... Howes, A. J. (2009). A randomized phase 3 study of tipifarnib compared with best supportive care, including hydroxyurea, in the treatment of newly diagnosed acute myeloid leukemia in patients 70 years or older. Blood, 114(6), 1166-1173. https://doi.org/10.1182/blood-2009-01-198093