A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia

DOI PDF 被引用文献10件
  • Ruben A. Mesa
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • David P. Steensma
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Animesh Pardanani
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Chin-Yang Li
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Michelle Elliott
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Scott H. Kaufmann
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Gregory Wiseman
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Leigh A. Gray
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Georgene Schroeder
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Terra Reeder
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Jerome B. Zeldis
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.
  • Ayalew Tefferi
    From the Division of Hematology and Internal Medicine, Cancer Center Statistics Unit, Division of Hematopathology, and Division of Nuclear Medicine, Mayo Clinic, Rochester, MN; and Celgene, Warren, NJ.

抄録

<jats:p>Single-agent thalidomide (THAL) at “conventional” doses (> 100 mg/d) has been evaluated in myelofibrosis with myeloid metaplasia (MMM) based on its antiangiogenic properties and the prominent neoangiogenesis that occurs in MMM. THAL monotherapy at such doses produces approximately a 20% response rate in anemia but is poorly tolerated (an adverse dropout rate of > 50% in 3 months). To improve efficacy and tolerability, we prospectively treated 21 symptomatic patients (hemoglobin level < 10 g/dL or symptomatic splenomegaly) with MMM with low-dose THAL (50 mg/d) along with a 3-month oral prednisone (PRED) taper (beginning at 0.5 mg/kg/d). THAL-PRED was well tolerated in all enrolled patients, with 20 patients (95%) able to complete 3 months of treatment. An objective clinical response was demonstrated in 13 (62%) patients, all improvements in anemia. Among 10 patients who were dependent on erythrocyte transfusions, 7 (70%) improved and 4 (40%) became transfusion independent. Among 8 patients with thrombocytopenia (platelet count < 100 × 109/L), 6 (75%) experienced a 50% or higher increase in their platelet count. In 4 of 21 patients (19%), spleen size decreased by more than 50%. Responses observed were mostly durable after discontinuation of the PRED. The dose of THAL in this study (50 mg/d) was better tolerated than the higher doses used in previous studies. Adverse events associated with corticosteroid therapy were mild and transient. Clinical responses did not correlate with improvements in either intramedullary fibrosis or angiogenesis. THAL-PRED is well tolerated and preliminarily appears to be a promising drug regimen for treating cytopenias in patients with MMM.</jats:p>

収録刊行物

  • Blood

    Blood 101 (7), 2534-2541, 2003-04-01

    American Society of Hematology

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