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

VL - 22

SP - 424

EP - 431

JO - Journal of Clinical Oncology

JF - Journal of Clinical Oncology

SN - 0732-183X

IS - 3

ER -