Reinfusion of Autologous Bone Marrow, Cord Blood and Peripheral Blood Progenitor Cells from Patients with Fanconi Anemia
Date Last Modified: 2002-11-12
Date First Published: 2002-07-08
Basic Study Information
| Type of Trial | Status of Trial | Age Range (yrs.) | Sponsor of Trial | Protocol IDs |
|---|
| Treatment | Active | Not specified | CCHMC | |
Objectives
- To determine the toxicities associated with the reinfusion of autologous bone marrow, cord blood or peripheral blood progenitor cells from patients with Fanconi anemia (FA) who have developed cytopenias.
- To determine the efficacy of reinfusion of autologous bone marrow, cord blood or peripheral blood progenitor cells as determined by transitory increased bone marrow cellularity, bone marrow CFU and improved peripheral blood counts.
Entry Criteria
Disease Characteristics:
- All patients with Fanconi anemia demonstrated by a positive test for increased sensitivity to chromosomal breakage with mitomycin C or diepoxybutane.
Prior/Concurrent Therapy:
Biologic therapy:
- Prior collection of autologous bone marrow, mobilized peripheral blood or umbilical cord blood. Sources of autologous stem cells will not be collected as part of this study.
Chemotherapy:
Endocrine therapy:
- No initiation of androgens and hematopoietic growth factors during the study period. Patients may receive androgens during the study period only if they have received these for > 3 months prior to study enrollment.
Radiotherapy:
Surgery:
Other:
Patient Characteristics:
Age:
Performance status:
Life expectancy:
Hematopoietic:
- Peripheral blood cytopenias defined as:
- Absolute neutrophil count < 500/mm3 –OR-
- Platelet count < 20,000/mm3 –OR-
- Hemoglobin < 8 gm/dl
Hepatic:
Renal:
Cardiovascular:
Pulmonary:
Neurologic:
Other:
- Physical or emotional status adequate to insure protocol compliance and follow-up.
- Signed informed consent or ability to undergo informed consent process by the patient or legally authorized representative.
- Not enrolled concurrently in any other study using an investigational drug.
- No malignancies.
- HIV negative.
Projected Accrual
A total of approximately 5 patients will be accrued for this study.
Outline
The purpose of this protocol is to reinfuse autologous bone marrow, cord blood or peripheral blood hematopoietic progenitor cells from children with Fanconi anemia who have developed cytopenias. Patients with Fanconi anemia will have already undergone a bone marrow harvest, cord blood collection, or peripheral blood progenitor cell mobilization and cryopreservation according to other investigational protocols. This protocol will allow analysis of data pertaining to the side effects of progenitor cell reinfusion and the effect on BM cellularity, CD34+ cell number, colony-forming units (CFU) and peripheral blood counts following infusion. This protocol does not have curative intent. It is anticipated that these patients will require allogeneic hematopoietic stem cell transplantation subsequent to participation in this trial.
