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HLA-haploidentical transplantations for primary immunodeficiencies: a single-center experience.
[omenn syndrome]
SCID
is
characterized
by
profound
deficiencies
of
T
and
B
lymphocytes
.
HSCT
is
the
only
curative
treatment
for
children
with
SCID
.
The
clinical
characteristics
and
outcome
of
30
HLA-haploidentical
transplantations
in
18
patients
(
15
SCID
,
two
Omenn
syndrome
,
and
one
MHC
Class
II
deficiency
)
are
reported
here
.
The
age
of
patients
at
diagnosis
ranged
from
one
and
half
to
nine
months
(
median
:
four
months
)
.
The
median
time
was
one
month
between
the
diagnosis
and
the
time
of
the
initial
transplantation
.
Infused
CD
34
+
stem
cell
dose
was
ranged
between
7
and
94
.
2
×
10
(
6
)
/
kg
.
Nine
of
18
patients
were
found
to
be
positive
for
CMV
antigenemia
at
diagnosis
;
therefore
,
none
of
them
received
a
conditioning
regimen
.
The
most
common
complication
was
graft
failure
(
61
%
)
,
so
repeated
transplantations
(
two
to
four
)
were
performed
in
seven
patients
.
The
mean
time
of
lymphoid
engraftment
was
17
.
5
days
(
median
:
16
,
range
:
11
-
29
days
)
.
Ten
of
15
SCID
(
67
%
)
patients
survived
with
a
stable
complete
donor
chimerism
.
However
,
all
three
non-
SCID
patients
died
.
In
conclusion
,
in
the
absence
of
a
matched
family
donor
,
HLA-haploidentical
transplantation
from
parental
donors
represents
a
readily
available
treatment
option
especially
for
patients
with
SCID
,
offering
a
high
chance
of
cure
.