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Phase 1/Phase 2
34 Years
Not Enrolling
Fanconi's Anemia

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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


--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

- 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

- 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:


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



Study ID:




Start Date:

February 2000

Completion Date:

Related Keywords:

  • Fanconi's Anemia
  • Anemia
  • Fanconi Anemia
  • Fanconi Syndrome



University of Minnesota Cancer Center Minneapolis, Minnesota  55455