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Blood, Vol. 95 No. 4 (February 15), 2000:
pp. 1195-1198
Stem cell transplantation in patients with severe
congenital neutropenia without evidence of leukemic
transformation
C. Zeidler,
K. Welte,
Y. Barak,
F. Barriga,
A. A. Bolyard,
L. Boxer,
G. Cornu,
M. J. Cowan,
D. C. Dale,
T. Flood,
M. Freedman,
H. Gadner,
H. Mandel,
R. J. O'Reilly,
U. Ramenghi,
A. Reiter,
R. Skinner,
C. Vermylen, and
J. E. Levine
From the Medizinische Hochschule, Hannover, Germany; Kaplan Medical
Center, Rehovot, Israel; Catholic University of Chile, Santiago, Chile;
University of Washington, Seattle, WA; University of Michigan, Ann
Arbor, MI; Université Catholique de Louvain, Cliniques
Universitaires de Saint-Luc, Brussels, Belgium; University of
California, San Francisco, CA; Sir James Spence Institute of Child
Health, Newcastle upon Tyne, UK; Hospital for Sick Children, Toronto,
Ontario, Canada; St. Anna Kinderspital, Vienna, Austria; Rambam Medical
Center, Haifa, Israel; Memorial Sloan Kettering Cancer Center, New
York, NY; Dipartimento di Scienze Pediatriche, Universita di Torino,
Turin, Italy.
Severe congenital neutropenia (CN) (Kostmann syndrome) is a
hematologic disorder characterized by a maturation arrest of
myelopoiesis at the promyelocyte/myelocyte stage of development. This
arrest results in severe neutropenia leading to absolute neutrophil
counts (ANC) below 0.2 × 109/L associated with severe
bacterial infections from early infancy. Data on over 300 patients with
CN collected by the Severe Chronic Neutropenia International Registry
(SCNIR) beginning in 1994 indicate that more than 90% of these
patients respond to recombinant human granulocyte-colony stimulating
factor (r-HuG-CSF) treatment with an ANC greater than
1.0 × 109/L. For patients who are refractory to
r-HuG-CSF treatment and continue to have severe and often
life-threatening bacterial infections, hematopoietic stem cell
transplantation is the only currently available treatment. We report on
a total of 11 patients with CN reported to the SCNIR who underwent
transplantation for reasons other than malignant transformation between
1976 and 1998. Of these patients, 8 were nonresponders or showed only
partial response to r-HuG-CSF treatment with ongoing infections.
Results from these patients suggest that transplantation of stem cells
from an HLA-identical sibling is beneficial for patients refractory to
r-HuG-CSF.
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