Clinical trial NCT00520468
Conditions: Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndrome MDS Erythropoietin Darbepoetin alfa Aranesp Erythropoiesis stimulating protein G-CSF Filgrastim Neupogen Prednisone Cyclosporin A Sandimmune CYA Cyclosporine
Sponsor: M.D. Anderson Cancer Center
Start date: 2004-06-01; Completion date: 2009-06-01
Enrollment: 15 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

Brief summary:
Objectives:

Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).

Secondary: To evaluate response duration, survival and side effects of the treatment.

Detailed description:
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a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle.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.

Arms: arm 1: Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice a week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Interventions: intervention 1: Erythropoietin intervention 2: Cyclosporin A intervention 3: G-CSF intervention 4: Prednisone
Intervention descriptions: intervention 1: 40,000 units injected under the skin (SQ) weekly intervention 2: 300 mg (tablets) by mouth daily for 6 months intervention 3: 300 mcg injected under the skin (SQ) two times per week intervention 4: 60 mg per day for 7 days, taper over 1 month

Protocol documents: