Thalidomide, Prednisone, and Cyclophosphamide in Treating Patients With Myelofibrosis and Myeloid Metaplasia

Phase 2

Completed

• Conditions: Chronic Myeloproliferative Disorders; Secondary Myelofibrosis

• Registration Number: NCT00445900
• Lead Sponsor: Mayo Clinic

Brief Summary

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.

Detailed Description

OBJECTIVES:

Primary

* 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.

Secondary

* 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.

Study Timeline

• Study Start: 2004-10
• Primary Completion: 2006-10
• Study Completion: 2006-10
• Study First Submitted: 2007-03-07
• Study First Submitted QC: 2007-03-07
• Study First Posted: 2007-03-09
• Status Verified: 2011-03
• Last Update Submitted: 2011-03-16
• Last Update Posted: 2011-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Confirmed response, defined as a complete or partial response in ≥ 1 of 3 response categories (i.e., anemia, thrombocytopenia, or splenomegaly or hepatomegaly)

Secondary Outcome Measures

Name	Time	Method
Toxicity as measured by NCI CTC v 2.0
Constitutional symptom status and bone marrow morphology
Overall survival
Progression-free survival
Time to progression
Duration of response