RATIONALE: Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells. PURPOSE: Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis.

Condition	Treatment or Intervention	Phase
Agnogenic Myeloid Metaplasia	Procedure: biological response modifier therapy  Procedure: growth factor antagonist therapy  Procedure: anti-cytokine therapy  Procedure: antiangiogenesis therapy  Drug: thalidomide	Phase II

Further Study Details: 

OBJECTIVES: I. Determine whether thalidomide is an effective therapeutic agent, in terms of anemia and/or organomegaly, in patients with myelofibrosis with myeloid metaplasia. II. Determine the effects of this regimen on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF and their respective receptors in these patients.

PROTOCOL OUTLINE: This is a multicenter study. Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy. Patients are followed every 6 months until 5 years from study entry.

PROJECTED ACCRUAL: A total of 18-30 patients will be accrued for this study within 2 years.

Ages Eligible for Study:  18 Years and above

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Histologically confirmed myelofibrosis with myeloid metaplasia

• Agnogenic myeloid metaplasia
• Post-polycythemic myeloid metaplasia
• Post-thrombocythemic myeloid metaplasia

No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis

No chromosomal translocation t(9;22) or bcr/abl gene rearrangement

Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood

Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly

--Prior/Concurrent Therapy--

Biologic therapy: At least 1 month since prior interferon, pirfenidone, anagrelide, or epoetin alfa

Chemotherapy: At least 1 month since prior hydroxyurea or other chemotherapy

Endocrine therapy: At least 1 month since prior corticosteroids or androgen derivatives

Radiotherapy: Not specified

Surgery: Not specified

--Patient Characteristics--

Age: 18 and over

Performance status: ECOG 0-2

Life expectancy: Not specified

Hematopoietic:

• See Disease Characteristics
• Absolute neutrophil count greater than 750/mm3
• Platelet count less than 400,000/mm3
• WBC less than 50,000/mm3

Hepatic:

• Bilirubin no greater than 2 mg/dL (if total bilirubin elevated, direct bilirubin must be normal)
• AST no greater than 3 times upper limit of normal (ULN)
• Alkaline phosphatase no greater than 3 times ULN

Renal:

• Creatinine no greater than 1.5 mg/dL OR
• Creatinine clearance at least 60 mL/min

Other:

• Not pregnant or nursing
• Negative pregnancy test
• Fertile women must use at least 1 highly active method AND 1 additional effective method of contraception for at least 4 weeks before study, during study, and for at least 4 weeks after study
• Fertile men must use effective contraception during study and for at least 4 weeks after study
• No uncontrolled infection
• No concurrent condition that would preclude study
• No peripheral neuropathy

Arizona
      CCOP - Scottsdale Oncology Program, Scottsdale,  Arizona,  85259-5404,  United States
Florida
      Mayo Clinic, Jacksonville,  Florida,  32224,  United States
Illinois
      CCOP - Carle Cancer Center, Urbana,  Illinois,  61801,  United States
      CCOP - Illinois Oncology Research Association, Peoria,  Illinois,  61602,  United States
Iowa
      CCOP - Cedar Rapids Oncology Project, Cedar Rapids,  Iowa,  52403-1206,  United States
      CCOP - Iowa Oncology Research Association, Des Moines,  Iowa,  50309-1016,  United States
      Siouxland Hematology-Oncology, Sioux City,  Iowa,  51101-1733,  United States
Kansas
      CCOP - Wichita, Wichita,  Kansas,  67214-3882,  United States
Minnesota
      CCOP - Metro-Minnesota, Saint Louis Park,  Minnesota,  55416,  United States
      CentraCare Health Plaza, Saint Cloud,  Minnesota,  56303,  United States
      Mayo Clinic Cancer Center, Rochester,  Minnesota,  55905,  United States
Nebraska
      CCOP - Missouri Valley Cancer Consortium, Omaha,  Nebraska,  68106,  United States
North Dakota
      CCOP - Merit Care Hospital, Fargo,  North Dakota,  58122,  United States
      Medcenter One Health System, Bismarck,  North Dakota,  58501,  United States
Pennsylvania
      Allegheny General Hospital, Pittsburgh,  Pennsylvania,  15212-4772,  United States
      CCOP - Geisinger Clinic and Medical Center, Danville,  Pennsylvania,  17822-2001,  United States
South Dakota
      CCOP - Sioux Community Cancer Consortium, Sioux Falls,  South Dakota,  57104,  United States
      Rapid City Regional Hospital, Rapid City,  South Dakota,  57709,  United States

Study chairs or principal investigators

Michelle A. Elliott,  Study Chair,  North Central Cancer Treatment Group   

Study ID Numbers:  CDR0000068367; NCCTG-N9982
Record last reviewed:  May 2003
Last Updated:  October 13, 2004
Record first received:  May 6, 2001
ClinicalTrials.gov Identifier:  NCT00015821
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08