TY - JOUR
T1 - Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial.
AU - Marchetti, Monia
AU - Barosi, Giovanni
AU - Balestri, Francesca
AU - Viarengo, Gianluca
AU - Gentili, Sara
AU - Barulli, Sara
AU - Demory, Jean-Loup
AU - Ilariucci, Fiorella
AU - Volpe, Antonio
AU - Bordessoule, Dominique
AU - Le Bousse-Kerdiles, Marie Caroline
AU - Caenazzo, Andrea
AU - Pecci, Alessandro
AU - Falcone, Antonietta
AU - Broccia, Giorgio
AU - Bendotti, Cesarina
AU - Bauduer, Fredric
AU - Buccisano, Francesco
AU - Dupriez, Brigitte
N1 - 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
PY - 2004
Y1 - 2004
N2 - 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.
AB - 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.
U2 - 10.1200/JCO.2004.08.160
DO - 10.1200/JCO.2004.08.160
M3 - Journal article
C2 - 14752066
SN - 0732-183X
VL - 22
SP - 424
EP - 431
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 3
ER -