Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial
- Authors:
-
- Marchetti, Monia ;
- Barosi, Giovanni ;
- Balestri, Francesca ;
- Viarengo, Gianluca ;
- Gentili, Sara ;
- Barulli, Sara ;
- Demory, Jean-Loup ;
- Ilariucci, Fiorella ;
- Volpe, Antonio ;
- Bordessoule, Dominique ;
- Le Bousse-Kerdiles, Marie Caroline ;
- Caenazzo, Andrea ;
- Pecci, Alessandro ;
- Falcone, Antonietta ;
- Broccia, Giorgio ;
- Bendotti, Cesarina ;
- Bauduer, Fredric ;
- Buccisano, Francesco ;
- Dupriez, Brigitte
- DOI:
- 10.1200/jco.2004.08.160
- Abstract:
- 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 x 10(9)/L or more in 22% of patients with an initial count lower than 100 x 10(9)/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.
- Type:
- Journal article
- Language:
- English
- Published in:
- Journal of Clinical Oncology, 2004, Vol 22, Issue 3, p. 424-31
- Main Research Area:
- Medical science
- Submission year:
- 2004
- ID:
- 108722459
