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Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

Phase 2
Not Enrolling
Myelodysplastic Syndrome

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Trial Information

Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

MDS is a disease that produces low blood counts and may cause anemia, infections, and/or
bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are
drugs that stimulate the production of red cells and white cells. Prednisone and
cyclosporin are drugs that work against MDS by affecting your immune system.

Before you can start treatment on this study, you will have what are called "screening
tests". These tests will help the doctor decide if you are eligible to take part in the
study. You will have a complete medical history and physical exam. Routine blood tests
(between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To
collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic
and a small amount of bone marrow is withdrawn through a large needle. Women who are able
to have children must have a negative blood or urine pregnancy test.

If you are found to be eligible to take part in this study, you will receive erythropoietin
as an injection under the skin once a week and G-CSF as an injection under the skin 1-2
times a week for as long as you respond well to treatment. You will take prednisone by
mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.

During this study, you will need to visit your doctor for a physical exam and measurement of
your vital signs. The frequency of doctor visits will vary depending on your physical
condition, but will be required at least once every 3 months.

Blood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks
of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples
will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples
will also be taken to check cells related to the disease before, during, and after
completion of this study.

You will be taken off study if the disease gets worse or intolerable side effects occur.

This is an investigational study. All drugs are FDA approved and commercially available.
Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.

Inclusion Criteria:

1. Patients with MDS and (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (>/=
1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent
chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts
< 5% must have an indication for therapy, such as transfusion needs, symptomatic
anemia or Hb < 11g/dl, platelets < 100 x 10 9/L, or granulocytes < 10 9/L.

2. Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function
(bilirubin of < 2mg/dl) and renal function (creatinine < 2mg/dl). Adequate cardiac
functions (New York Heart Association (NYHA) cardiac III-IV excluded)

3. Signed informed consent

Exclusion Criteria:

1. Nursing and pregnant females are excluded. Women of childbearing potential should
practice effective methods of contraception. Should a woman become pregnant or
suspect she is pregnant while participating in this study, she should inform her
treating physician immediately.

2. Patients with active and uncontrolled infections.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Response

Outcome Description:

Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.

Outcome Time Frame:

Response evaluation within first 3 months from start of therapy, then every 3 to 6 months

Safety Issue:


Principal Investigator

Gautam Borthakur, MBBS

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

June 2004

Completion Date:

June 2009

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic Syndrome
  • MDS
  • Erythropoietin
  • Darbepoetin alfa
  • Aranesp
  • Erythropoiesis stimulating protein
  • G-CSF
  • Filgrastim
  • Neupogen
  • Prednisone
  • Cyclosporin A
  • Sandimmune
  • CYA
  • Cyclosporine
  • Myelodysplastic Syndromes
  • Preleukemia



U.T.M.D. Anderson Cancer Center Houston, Texas  77030