Phase II Study of the Combination of Low-Dose Thalidomide, Prednisone, and Oral Cyclophosphamide ("TPC") in the Therapy of Myelofibrosis With Myeloid Metaplasia (MMM)
- Determine the benefit of thalidomide, prednisone, and cyclophosphamide in alleviating
disease-associated anemia, thrombocytopenia, and/or splenomegaly in patients with
myelofibrosis with myeloid metaplasia (MMM).
- Determine the benefit of this regimen in palliating four hypercatabolic constitutional
symptoms (i.e., weight loss, fatigue, drenching night sweats, and unexplained fevers)
in these patients.
- Determine the toxicity profile of this regimen in these patients.
- Determine the effect of this regimen on leukocyte count.
- Determine the effect of this regimen on bone marrow histology, including microvessel
density and reticulin fibrosis.
- Determine the effect of this regimen on intramedullary and urinary markers of
- Determine the effect of this regimen on circulating myeloid progenitor cells by
quantifying CD34+ cells.
OUTLINE: Patients receive oral thalidomide, oral prednisone, and oral cyclophosphamide (TPC)
once daily on days 1-28. Treatment repeats every 28 days for 3 courses. After 3 courses (3
months) of treatment, patients who respond to TPC therapy may receive oral thalidomide alone
once daily for up to 3 months in the absence of disease progression or unacceptable
Patients undergo bone marrow aspirate and biopsy prior to study entry, 6 months after
starting therapy, and then every 6 months for up to 3 years. Samples are analyzed by
microvessel density/angiogenesis studies (i.e., CD34 immunohistochemical and vascular
endothelium-specific staining) to determine the effect of therapy on markers of bone marrow
After completion of study therapy, patients are followed every 6 months for up to 3 years.
PROJECTED ACCRUAL: A total of 22 patients will be accrued for this study.
Masking: Open Label, Primary Purpose: Treatment
Confirmed response, defined as a complete or partial response in ≥ 1 of 3 response categories (i.e., anemia, thrombocytopenia, or splenomegaly or hepatomegaly)
Ruben A. Mesa, MD
United States: Federal Government