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Blood, 15 May 2007, Vol. 109, No. 10, pp. 4158-4163. Prepublished online as a Blood First Edition Paper on January 30, 2007; DOI 10.1182/blood-2006-07-035725. This Article Full Text (PDF) Supplemental Appendix All Versions of this Article: blood-2006-07-035725v1 109/10/4158    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Fenaux, P. Articles by Kurzrock, R. Search for Related Content PubMed PubMed Citation Articles by Fenaux, P. Articles by Kurzrock, R. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted July 17, 2006 Accepted January 8, 2007 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, Rene Kerstens, Peter De Porre, and Razelle Kurzrock* Hopital Avicenne, Paris, France Rush Cancer Institute, Chicago, IL, United States Kings College, London, United Kingdom St. Johannes Hospital, Duisbug, Germany Heinrich Heine University, Dusseldorf, Germany The University of Texas M.D. Anderson Cancer Center, Houston, TX, United States The Indiana University Cancer Center, Indianapolis, IN, United States Johnson & Johnson Pharmaceutical Research & Development, Beerse, Belgium * Corresponding author; email: rkurzroc{at}mdanderson.org. This multicenter phase 2 study evaluated the use of tipifarnib (ZARNESTRA®, R115777) in patients with poor-risk 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 (CR) and 14 (17%) hematologic improvements; 37 patients (45%) had stable disease (modified International Working Group criteria, 2006). Among 12 CRs, the median response duration was 11.5 months (range, 2.0 to 21.9), the median time to progression was 12.4 months (range, 3.9 to 23.8) and 7 were still alive at time of analysis (all > 3 year). Median overall survival was 11.7 months (95% CI, 9.4 to 15.0). Grade 3-4 neutropenia (18%) and thrombocytopenia (32%) were the most common treatmentrelated adverse events; severe non-hematological 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. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: J. E. Karp, B. D. Smith, I. Gojo, J. E. Lancet, J. Greer, M. Klein, L. Morris, M. J. Levis, S. D. Gore, J. J. Wright, et al. Phase II Trial of Tipifarnib as Maintenance Therapy in First Complete Remission in Adults with Acute Myelogenous Leukemia and Poor-Risk Features Clin. Cancer Res., May 15, 2008; 14(10): 3077 - 3082. [Abstract] [Full Text] [PDF] D. Hong, L. Ye, R. Gagel, L. Chintala, A. K. El Naggar, J. Wright, and R. Kurzrock Medullary thyroid cancer: targeting the RET kinase pathway with sorafenib/tipifarnib Mol. Cancer Ther., May 1, 2008; 7(5): 1001 - 1006. [Abstract] [Full Text] [PDF] R. Kurzrock, H. M. Kantarjian, M. A. Blascovich, C. Bucher, S. Verstovsek, J. J. Wright, S. R. Pilat, J. E. Cortes, E. H. Estey, F. J. Giles, et al. Phase I Study of Alternate-Week Administration of Tipifarnib in Patients with Myelodysplastic Syndrome Clin. Cancer Res., January 15, 2008; 14(2): 509 - 514. [Abstract] [Full Text] [PDF]

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