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Blood, 1 September 2004, Vol. 104, No. 5, pp. 1266-1269. Prepublished online as a Blood First Edition Paper on May 20, 2004; DOI 10.1182/blood-2003-12-4333. This Article Full Text Full Text (PDF) All Versions of this Article: 2003-12-4333v1 104/5/1266    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map 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 Kuendgen, A. Articles by Gattermann, N. Search for Related Content PubMed PubMed Citation Articles by Kuendgen, A. Articles by Gattermann, N. Related Collections Neoplasia Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS Brief report Treatment of myelodysplastic syndromes with valproic acid alone or in combination with all-trans retinoic acid Andrea Kuendgen, Corinna Strupp, Manuel Aivado, Alf Bernhardt, Barbara Hildebrandt, Rainer Haas, Ulrich Germing, and Norbert Gattermann From the Department of Hematology, Oncology, and Clinical Immunology and the Institute of Genetics, Heinrich-Heine-University, Düsseldorf, Germany. Valproic acid (VPA) has been shown to inhibit histone deacetylase activity and to synergize with all-trans retinoic acid (ATRA) in the differentiation induction of acute myelogenous leukemia (AML) blasts in vitro. We treated 18 patients with myelodysplastic syndromes (MDS) and AML secondary to MDS (sAML/MDS) with VPA monotherapy (serum concentrations 346-693 µM [50-100 µg/mL]). Five patients received VPA and ATRA (80 mg/m2/d, days 1-7, every other week). Response according to international working group (IWG) criteria was observed in 8 patients (44%) on VPA monotherapy, including 1 partial remission. Median response duration was 4 months (range, 3-9 months). Four of 5 patients relapsing were treated with VPA + ATRA, 2 of them responding again. Among 5 patients receiving VPA + ATRA from the start, none responded according to IWG criteria, but 1 patient with sAML/MDS achieved a marked reduction in peripheral and marrow blasts. Thus, VPA is of therapeutic benefit for patients with MDS, and ATRA may be effective when added later. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: M. Dokmanovic, C. Clarke, and P. A. 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