Thalidomide in Treating Patients With Myelodysplastic Syndrome

National Cancer Institute (NCI)

Status and phase

Completed

Phase 2

Conditions

Refractory Anemia With Excess Blasts in Transformation

Refractory Anemia With Excess Blasts

de Novo Myelodysplastic Syndromes

Refractory Anemia With Ringed Sideroblasts

Previously Treated Myelodysplastic Syndromes

Refractory Anemia

Secondary Myelodysplastic Syndromes

Chronic Myelomonocytic Leukemia

Treatments

Drug: thalidomide

Other: laboratory biomarker analysis

Study type

Interventional

Funder types

NIH

Identifiers

NCT00015990

N998B

NCI-2012-01856

U10CA025224 (U.S. NIH Grant/Contract)

CDR0000068580 (Registry Identifier)

Details and patient eligibility

About

Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome

Full description

OBJECTIVES:

I. Determine whether thalidomide improves cytopenias in patients with myelodysplastic syndromes.

II. Determine the toxicity of this regimen in these patients. III. Determine whether this regimen down regulates the peripheral blood levels of tumor necrosis factor alpha, interferon gamma, and interleukin-12 and whether these changes correlate with clinical response in these patients.

IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution and whether these changes correlate with clinical response in these patients.

V. Determine the effect of this regimen on bone marrow microvessel density and whether these effects correlate with clinical response in these patients.

OUTLINE: This is a multicenter study. Patients are stratified according to prognosis (favorable vs unfavorable). (Favorable stratum closed to accrual 12/28/01)

Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.

Patients are followed every 6 months for 1 year and then annually for 4 years.

PROJECTED ACCRUAL: A total of 20-58 patients (10-29 per stratum) will be accrued for this study within 20 months. (Favorable stratum closed to accrual 12/28/01)

Enrollment

29 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Pre-transfusion hemoglobin =< 10 g/dL
Pre-transfusion platelet count =< 50,000/μL
Absolute neutrophil count < 1000/μL
Total bilirubin ≤ 1.5 x UNL
Alkaline phosphatase ≤ 3 x UNL
AST ≤ 3 x UNL
Creatinine ≤ 1.5 x UNL
A diagnosis of MDS as demonstrated in the bone marrow; any subtypes are eligible including:
- Refractory anemia (cytopenia)
- Refractory anemia with ringed sideroblasts
- Chronic myelomonocytic leukemia
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- Unclassified MDS
Patients with refractory anemia with excess blasts in transformation who are not candidates for (or who decline) induction chemotherapy are eligible; those patients who were candidates for (and accepted) induction chemotherapy should have failed at least 1 chemotherapy regimen prior to entry
Patients who are candidates for marrow transplantation should have this option discussed prior to study entry

Exclusion criteria

Any of the following as this regimen may be harmful to a developing fetus or nursing child:
- Pregnant women
- Nursing women
- Women of childbearing potential or their sexual partners who are unwilling to employ 2 adequate methods of contraception (condoms, diaphragm, birth control pills, injections, intrauterine device [IUD], surgical sterilization, subcutaneous implants, or abstinence, etc.)
Peripheral neuropathy (by history or clinical exam)
Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (< 20 mg/d) for reasons other than MDS are allowed
Uncontrolled infections