Rare Diseases Symptoms Automatic Extraction
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A random Abstract
Our Project
Our Team
A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.
[severe combined immunodeficiency]
For
infants
with
SCID
,
the
ideal
conditioning
regimen
before
allogeneic
HCT
would
omit
cytotoxic
chemotherapy
to
minimize
short
-
and
long
-term
complications
.
We
performed
a
prospective
pilot
trial
with
G-CSF
plus
plerixafor
given
to
the
host
to
mobilize
HSC
from
their
niches
.
We
enrolled
six
patients
who
received
CD
34
-
selected
haploidentical
cells
and
one
who
received
T
-
replete
matched
unrelated
BM
.
All
patients
receiving
G-CSF
and
plerixafor
had
generally
poor
CD
34
(
+
)
cell
and
Lin
(
-
)
CD
34
(
+
)
CD
38
(
-
)
CD
9
0
(
+
)
CD
4
5
RA
(
-
)
HSC
mobilization
,
and
developed
donor
T
cells
,
but
no
donor
myeloid
or
B-
cell
engraftment
.
Although
well
tolerated
,
G-CSF
plus
plerixafor
alone
failed
to
overcome
physical
barriers
to
donor
engraftment
.