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Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
[omenn syndrome]
Pediatric
patients
with
SCID
constitute
medical
emergencies
.
In
the
absence
of
an
HLA-identical
hematopoietic
stem
cell
(
HSC
)
donor
,
mismatched
related-donor
transplantation
(
MMRDT
)
or
unrelated-donor
umbilical
cord
blood
transplantation
(
UCBT
)
are
valuable
treatment
options
.
To
help
transplantation
centers
choose
the
best
treatment
option
,
we
retrospectively
compared
outcomes
after
175
MMRDTs
and
74
UCBTs
in
patients
with
SCID
or
Omenn
syndrome
.
Median
follow-up
time
was
83
months
and
58
months
for
UCBT
and
MMRDT
,
respectively
.
Most
UCB
recipients
received
a
myeloablative
conditioning
regimen
;
most
MMRDT
recipients
did
not
.
UCB
recipients
presented
a
higher
frequency
of
complete
donor
chimerism
(
P
=
.
04
)
and
faster
total
lymphocyte
count
recovery
(
P
=
.
04
)
without
any
statistically
significance
with
the
preparative
regimen
they
received
.
The
MMRDT
and
UCBT
groups
did
not
differ
in
terms
of
T
-
cell
engraftment
,
CD
4
(
+
)
and
CD
3
(
+
)
cell
recoveries
,
while
Ig
replacement
therapy
was
discontinued
sooner
after
UCBT
(
adjusted
P
=
.
02
)
.
There
was
a
trend
toward
a
greater
incidence
of
grades
II
-IV
acute
GVHD
(
P
=
.
06
)
and
more
chronic
GVHD
(
P
=
.
03
)
after
UCBT
.
The
estimated
5
-
year
overall
survival
rates
were
62
%
±
4
%
after
MMRDT
and
57
%
±
6
%
after
UCBT
.
For
children
with
SCID
and
no
HLA-identical
sibling
donor
,
both
UCBT
and
MMRDT
represent
available
HSC
sources
for
transplantation
with
quite
similar
outcomes
.