Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial.
Research output: Contribution to journal › Journal article › Research › peer-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 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.
Original language | English |
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Journal | Journal of Clinical Oncology |
Volume | 22 |
Issue number | 3 |
Pages (from-to) | 424-31 |
Number of pages | 7 |
ISSN | 0732-183X |
DOIs | |
Publication status | Published - 2004 |
Bibliographical note
Keywords: Adolescent; Adult; Aged; Aged, 80 and over; Anemia; Female; Humans; Immunosuppressive Agents; Leukopenia; Male; Middle Aged; Myelofibrosis; Myeloid Metaplasia; Platelet Count; Safety; Severity of Illness Index; Splenomegaly; Thalidomide; Thrombocytopenia; Treatment Outcome
ID: 8463393