N/A
34 Years
Both
Fanconi's Anemia
Trial Information
PROTOCOL OUTLINE: Donor bone marrow, peripheral blood, or umbilical cord blood is processed
to harvest CD34+ cells.
Patients receive preparative cytoreductive therapy comprising total body irradiation on day
-6; cyclophosphamide IV over 2 hours on days -5 to -2; fludarabine IV over 30 minutes on
days -5 to -2; methylprednisone IV on days -5 to 24; anti-thymocyte globulin IV over 4-6
hours on days -5 to -1; cyclosporine IV over 2 hours every 12 hours (every 8 hours for
patients less than 40 kg in weight) on days -3 to 180, and then tapering in the absence of
graft-versus-host disease; hematopoietic cell transplantation on day 0; and filgrastim
(G-CSF) IV starting on day 1 and continuing until blood counts recover.
Patients are followed at days 60, 90, and 180, and then annually for 3 years.
Inclusion Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Patients with a diagnosis of Fanconi's anemia AND Aplastic anemia OR Myelodysplastic
syndrome OR Acute leukemia with or without chromosomal anomalies Aplastic anemia as
defined by having at least one of the following: Platelet count less than 20,000/mm3
Absolute neutrophil count less than 500/mm3 Hemoglobin less than 8 g/dL
- Myelodysplastic syndrome with multilineage dysplasia With or without chromosomal
anomalies
- Hematologic malignancy (e.g., acute myeloid leukemia, acute lymphoblastic leukemia,
or B-cell non-Hodgkin's lymphoma)
- No greater than 30% blasts in bone marrow or greater than 5% blasts in peripheral
blood
- No active CNS leukemia at time of transplantation
- Must have an HLA A, B, DRB1 identical or 1 antigen mismatched related (nonsibling) or
unrelated bone marrow, peripheral blood, or umbilical cord blood donor
--Prior/Concurrent Therapy--
- Radiotherapy: No prior radiotherapy that would preclude total body irradiation
- Surgery: Not specified
--Patient Characteristics--
- Performance status: Karnofsky 70-100% OR Lansky 50-100%
- Hematopoietic: See Disease Characteristics
- Hepatic: No hepatic failure (e.g., coagulopathy or ascites)
- Renal: Creatinine clearance at least 40 mL/min
- Cardiovascular: Ejection fraction at least 45%
- Other: No active uncontrolled infection within one week of transplantation No
malignant solid tumor (e.g., squamous cell carcinoma of the head, neck, or cervix)
within 2 years of transplantation Not pregnant or nursing Negative pregnancy test
Fertile patients must use effective contraception
Type of Study:
Interventional
Study Design:
Primary Purpose: Treatment
Principal Investigator
John E. Wagner, Jr.
Investigator Role:
Study Chair
Investigator Affiliation:
University of Minnesota - Clinical and Translational Science Institute
Authority:
Unspecified
Study ID:
199/15109
NCT ID:
NCT00005898
Start Date:
February 2000
Completion Date:
Related Keywords:
- Fanconi's Anemia
- Anemia
- Fanconi Anemia
- Fanconi Syndrome
Name | Location |
|---|---|
| University of Minnesota Cancer Center | Minneapolis, Minnesota 55455 |
