A multicenter phase 2 study of the farnesyltransferase inhibitor tipifarnib in intermediate- to high-risk myelodysplastic syndrome

Pierre Fenaux, Azra Raza, Ghulam J. Mufti, Carlo Aul, Ulrich Germing, Hagop Kantarjian, Larry Cripe, René Kerstens, Peter De Porre, Razelle Kurzrock

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92 Scopus citations

Abstract

This multicenter phase 2 study evaluated the use of tipifarnib (R115777) in patients with poor-risk myelodysplastic syndrome (MDS; French-American-British classification). Patients (n = 82) received tipifarnib 300 mg orally twice daily for the first 21 days of each 28-day cycle. Twenty-six patients (32%) responded to tipifarnib: 12 (15%) complete responses (CRs) and 14 (17%) hematologic improvements; 37 patients (45%) had stable disease (modified International Working Group criteria, 2006). Among the 12 CRs, the median response duration was 11.5 months (range, 2.0-21.9 months), the median time to progression was 12.4 months (range, 3.9-23.8 months), and 7 were still alive at time of analysis (all > 3 years). Median overall survival was 11.7 months (95% CI, 9.4-15.0). Grade 3-4 neutropenia (18%) and thrombocytopenia (32%) were the most common treatment-related adverse events; severe nonhematologic adverse events were rarely reported. In this study, durable responses and acceptable side effects were observed. Tipifarnib is an active agent for the treatment of patients with intermediate- to high-risk MDS.

Original languageEnglish (US)
Pages (from-to)4158-4163
Number of pages6
JournalBlood
Volume109
Issue number10
DOIs
StatePublished - May 15 2007

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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    Fenaux, P., Raza, A., Mufti, G. J., Aul, C., Germing, U., Kantarjian, H., Cripe, L., Kerstens, R., De Porre, P., & Kurzrock, R. (2007). A multicenter phase 2 study of the farnesyltransferase inhibitor tipifarnib in intermediate- to high-risk myelodysplastic syndrome. Blood, 109(10), 4158-4163. https://doi.org/10.1182/blood-2006-07-035725