Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow. It should also be less harmful (toxic). Researchers wish to test whether this approach can overcome the graft failure often seen when bone marrow or peripheral blood stem cells from an unrelated donor are used. Researchers also will look at whether the procedure is less toxic than a conventional bone marrow transplant (BMT).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fred Hutchinson Cancer Research Center
• Collaborators: National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Cyclosporine; Cyclosporins; Fludarabine; Fludarabine phosphate; Mycophenolate mofetil; Mycophenolic Acid; Vidarabine

Criteria

Inclusion Criteria:

• Any patient with marrow failure and increased chromosome fragility as determined in the diepoxybutane (DEB) or mitomycin C test

• Any patient with Fanconi anemia (FA) with marrow failure meeting the following criteria:

• Granulocyte count < 0.2 x 10^9/L

• Platelet count < 20 x 10^9/L

• Hemoglobin < 8 g/dl

• Corrected reticulocyte count <1%

• Any patient with FA as determined by DEB fragility, who has life-threatening marrow failure involving a single hematopoietic lineage

• Any patient with FA and pre-existing cytogenetic abnormality including hematopoietic malignancy (myelodysplastic syndromes [MDS] or acute myeloid leukemia [AML]) in remission

• DONOR: Unrelated Donors who are prospectively: Matched for human lymphocyte antigen (HLA)-DRB1 and DQB1 alleles (must be defined by high resolution typing); only a single allele disparity will be allowed for HLA -A, B, or C as defined by high resolution typing

• DONOR: HLA typing will be performed at the highest level of resolution available at the time of transplant

Exclusion Criteria:

• Evidence for hematopoietic malignancy in relapse

• Heart or lung disease that would prevent compliance with conditioning and GvHD regimen or would severely limit the probability of survival

• Human immunodeficiency virus (HIV) seropositive patients

• Females who are pregnant or breastfeeding, or unwilling to use contraceptive techniques during and for the 12 months following treatment

• DONOR: Donors who by DEB testing are found to have FA

• DONOR: Donors who test positive in the lymphocytotoxic crossmatch assay

• DONOR: Donors who are HIV positive

• DONOR: Donors who for other medical or psychological reasons are not suitable as donors