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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
"immunodeficiency"
symptom
adrenal incidentaloma
allergic bronchopulmonary aspergillosis
cushing syndrome
dracunculiasis
hirschsprung disease
hodgkin lymphoma, classical
homocystinuria without methylmalonic aciduria
kabuki syndrome
legionellosis
malignant atrophic papulosis
oculocutaneous albinism
omenn syndrome
papillon-lefèvre syndrome
primary effusion lymphoma
primary hyperoxaluria type 1
pyomyositis
severe combined immunodeficiency
sneddon syndrome
werner syndrome
wiskott-aldrich syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
This symptom has already been validated