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Blood, 15 November 2001, Vol. 98, No. 10, pp. 2917-2921
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Mobilization and collection of peripheral blood
CD34+ cells from patients with
Fanconi anemia
James M. Croop,
Ryan Cooper,
Christine Fernandez,
Vicki Graves,
Susan Kreissman,
Helmut Hanenberg,
Franklin O. Smith, and
David A. Williams
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.
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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