The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.
Mobilization and collection of peripheral blood CD34+ cells from patients with Fanconi anemia
Abstract
A potential therapeutic option for patients with Fanconi anemia is collection of peripheral blood stem cells prior to the development of severe pancytopenia. These hematopoietic cells potentially could be infused when symptomatic bone marrow failure develops, as autologous rescue after chemotherapy in the event of leukemic transformation, or as targets for gene therapy. Eight patients with Fanconi anemia were mobilized with 10 μg/kg per day of granulocyte colony-stimulating factor (median, 10 ± 4 days) to determine the feasibility of collecting peripheral blood stem cells for future use. Six patients achieved a peripheral blood CD34+ count of ≥ 6/μL and underwent apheresis. The collection goal was 2 × 106 CD34+ cells/kg based on a predicted weight 5 years from the date of collection. A mean of 2.6 ± 0.9 × 106 CD34+ cells/kg of the weight at the time of collection were collected, which corresponded to 1.9 ± 0.4 × 106 CD34+cells/kg of the target weight. The collections required a mean of 4 ± 3 days (range, 2-8 days) of apheresis. Six of the 8 subjects had ≥ 1 × 106 CD34+ cells/kg cryopreserved based on both actual and target weights, and 4 subjects had ≥ 2 × 106 CD34+ cells/kg cryopreserved based on the target weight. These results suggest that some patients with Fanconi anemia can have adequate numbers of CD34+ cells mobilized and collected from the peripheral blood prior to the onset of severe bone marrow failure, but they may require an extended mobilization and multiple days of collection.
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“However, broad inter-individual variability of efficacy exists in normal healthy subjects, patients pre-treated with chemotherapy, and patients with Fanconi anemia[34,35,69,81].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [69]
In the citing paper
“Overall, the CD34+ collection yields in FA patients are below the expected yield of healthy individuals with a high degree of intra-patient variability.”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [69]
In the citing paper
“However, mobilization and collection was achieved only with extended G-CSF treatment and multiple days of apheresis, reducing the practical feasibility and raising questions regarding the ability of the CD34+ cells to engraft upon transplantation.”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [69]
In the citing paper
“In a second study of patients with FA, treatment with 10 μg/kg G-CSF for 6–14 days, resulted in the goal level of ≥ 6 CD34+ cells per μl peripheral blood being mobilized in six patients and the collection of 2 × 106 CD34+ cells/Kg of target weight in four of eight patients[69].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [69]
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Cited by
Stem Cell Collection and Gene Transfer in Fanconi Anemia
Kelly, P. F., Radtke, S., Kalle, C. V., Balcik, B., Bohn, K., Mueller, R., Schuesler, T., Haren, M., Reeves, L., Cancelas, J. A., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300033 · Cited by 180
Self-citationFinding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia
Müller, L. U. W., & Williams, D. A. (2009) · Mutation Research - Fundamental and Molecular Mechanisms of Mutagenesis · doi:10.1016/j.mrfmmm.2009.03.010 · Cited by 31
Self-citation
Metadata
- Authors
- Croop, J. M., Cooper, R., Fernandez, C., Graves, V., Kreissman, S., Hanenberg, H., Smith, F. O., & Williams, D. A.
- Affiliations
- From the Section of Pediatric Hematology/Oncology, Department of Pediatrics; Herman B Wells Center for Pediatric Research, Riley Hospital for Children; Pediatric Apheresis Program and Stem Cell Laboratory, Department of Pathology; Departments of Medicine, Microbiology, and Immunology; and the Howard Hughes Medical Institute, Indiana University School of Medicine, Indianapolis.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2001-11-15
- Volume
- 98
- Issue
- 10
- Pages
- 2917-2921
- ISSN
- 1528-0020
- 0006-4971
- Cited by
- 48
- References
- 1
- Language
- en
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