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Stem cell transplantation for primary immunodeficiency diseases: the North American experience.
[wiskott-aldrich syndrome]
This
review
describes
recent
studies
on
outcomes
after
allogeneic
hematopoietic
cell
transplantation
for
primary
immunodeficiency
in
North
America
,
including
severe
combined
immunodeficiency
(
SCID
)
,
Wiskott-
Aldrich
syndrome
and
chronic
granulomatous
disease
.
Using
uniform
diagnostic
criteria
,
the
Primary
Immune
Deficiency
Treatment
Consortium
described
the
baseline
characteristics
of
newly
diagnosed
infants
with
SCID
in
North
America
.
Analysis
of
outcomes
of
hematopoietic
cell
transplantation
for
SCID
in
North
America
from
2000
to
2009
showed
that
young
infants
,
and
older
infants
without
active
infection
,
had
excellent
survival
irrespective
of
type
of
donor
or
transplant
approach
with
regard
to
conditioning
.
Although
pretransplant
conditioning
with
chemotherapy
had
a
clear
and
strong
negative
impact
on
survival
in
infants
with
active
infection
at
the
time
of
transplant
,
among
survivors
,
conditioning
was
associated
with
improved
immune
reconstitution
.
However
,
the
potential
late
effects
of
conditioning
in
these
infants
remain
to
be
characterized
.
Advances
in
transplant
outcomes
for
Wiskott-
Aldrich
syndrome
and
chronic
granulomatous
disease
support
the
strategy
of
early
transplantation
before
the
onset
of
severe
complications
;
additional
multicenter
studies
are
needed
to
fully
define
optimal
approaches
.
The
formation
of
the
Primary
Immune
Deficiency
Treatment
Consortium
,
a
multiinstitutional
North
American
consortium
,
has
contributed
to
our
understanding
of
outcomes
after
transplant
for
primary
immunodeficiency
.
Diseases
Validation
Diseases presenting
"chronic granulomatous disease"
symptom
achondroplasia
allergic bronchopulmonary aspergillosis
alpha-thalassemia
junctional epidermolysis bullosa
omenn syndrome
pyomyositis
severe combined immunodeficiency
wiskott-aldrich syndrome
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