Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: A phase II trial

Monia Marchetti, Giovanni Barosi, Francesca Balestri, Gianluca Viarengo, Sara Gentili, Sara Barulli, Jean Loup Demory, Fiorella Ilariucci, Antonio Volpe, Dominique Bordessoule, Alberto Grossi, Marie Caroline Le Bousse-Kerdiles, Andrea Caenazzo, Alessandro Pecci, Antonietta Falcone, Giorgio Broccia, Cesarina Bendotti, Fredric Bauduer, Francesco Buccisano, Brigitte Dupriez

Research output: Contribution to journalArticlepeer-review


Purpose: A phase II dose-escalation trial was conducted to ascertain low-dose thalidomide safety and response in patients with advanced myelofibrosis with myeloid metaplasia (MMM). Patients and Methods: Thalidomide was administered together with current therapy to 63 patients, starting at 50 mg daily and increasing to 400 mg as tolerated. Results: Half of the patients sustained daily doses more than 100 mg and the drop-out rate was 51% at 6 months: the drop-out rate was lower in patients with high baseline fatigue score. At efficacy analysis, anemia was ameliorated in 22% of the patients and transfusions were eliminated in 39% of transfusion-dependent patients. Platelet count increased by 50 × 109/L or more in 22% of patients with an initial count lower than 100 × 109/L. Splenomegaly decreased by more than 50% of the initial size in 19% of patients. Reduction of an overall disease severity score occurred in 31% of patients and was associated with a significant reduction of fatigue. Disease severity amelioration was independently predicted by a high baseline myeloproliferative index (ie, large splenomegaly, thrombocytosis, or leukocytosis). Conclusion: Low-dose thalidomide displays an acceptable toxicity profile and provides an objective and subjective advantage to a relevant portion of MMM patients.

Original languageEnglish
Pages (from-to)424-431
Number of pages8
JournalJournal of Clinical Oncology
Issue number3
Publication statusPublished - 2004

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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