Thalidomide, Prednisone, and Cyclophosphamide in Treating Patients With Myelofibrosis and Myeloid Metaplasia
RATIONALE: Giving thalidomide together with prednisone and cyclophosphamide may lessen symptoms caused by myelofibrosis and myeloid metaplasia.
PURPOSE: This phase II trial is studying the side effects and how well giving thalidomide together with prednisone and cyclophosphamide works in treating patients with myelofibrosis and myeloid metaplasia.
Chronic Myeloproliferative Disorders
Other: immunohistochemistry staining method
Other: laboratory biomarker analysis
|Study Design:||Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of the Combination of Low-Dose Thalidomide, Prednisone, and Oral Cyclophosphamide ("TPC") in the Therapy of Myelofibrosis With Myeloid Metaplasia (MMM)|
- Confirmed response, defined as a complete or partial response in ≥ 1 of 3 response categories (i.e., anemia, thrombocytopenia, or splenomegaly or hepatomegaly) [ Designated as safety issue: No ]
- Constitutional symptom status and bone marrow morphology [ Designated as safety issue: No ]
- Overall survival [ Designated as safety issue: No ]
- Progression-free survival [ Designated as safety issue: No ]
- Time to progression [ Designated as safety issue: No ]
- Duration of response [ Designated as safety issue: No ]
- Toxicity as measured by NCI CTC v 2.0 [ Designated as safety issue: Yes ]
|Study Start Date:||October 2004|
|Study Completion Date:||October 2006|
|Primary Completion Date:||October 2006 (Final data collection date for primary outcome measure)|
- 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 angiogenesis.
- 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 toxicity.
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 angiogenesis.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00445900
|Study Chair:||Ruben A. Mesa, MD||Mayo Clinic|