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Three-year outcomes of cultured limbal epithelial allografts in aniridia and Stevens-Johnson syndrome evaluated using the Clinical Outcome Assessment in Surgical Trials assessment tool.
[aniridia]
Limbal
stem
cell
deficiency
(
LSCD
)
is
an
eye
disorder
in
which
the
stem
cells
responsible
for
forming
the
surface
skin
of
the
cornea
are
destroyed
by
disease
.
This
results
in
pain
,
loss
of
vision
,
and
a
cosmetically
unpleasant
appearance
.
Many
new
treatments
,
including
stem
cell
therapies
,
are
emerging
for
the
treatment
of
this
condition
,
but
assessment
of
these
new
technologies
is
severely
hampered
by
the
lack
of
biomarkers
for
this
disease
or
validated
tools
for
assessing
its
severity
.
The
aims
of
this
study
were
to
design
and
test
the
reliability
of
a
tool
for
grading
LSCD
,
to
define
a
set
of
core
outcome
measures
for
use
in
evaluating
treatments
for
this
condition
,
and
to
demonstrate
their
utility
.
This
was
achieved
by
using
our
defined
outcome
set
(
which
included
the
Clinical
Outcome
Assessment
in
Surgical
Trials
of
Limbal
stem
cell
deficiency
[
COASTL
]
tool
)
to
evaluate
the
3
-
year
outcomes
for
allogeneic
ex
vivo
cultivated
limbal
epithelial
transplantation
(
allo-
CLET
)
in
patients
who
had
bilateral
total
LSCD
secondary
to
aniridia
or
Stevens-
Johnson
syndrome
.
The
results
demonstrate
that
our
new
grading
tool
for
LSCD
,
the
COASTL
tool
,
is
reliable
and
repeatable
,
and
that
improvements
in
the
biomarkers
used
in
this
tool
correlate
positively
with
improvements
in
visual
acuity
.
The
COASTL
tool
showed
that
following
allo-
CLET
there
was
a
decrease
in
LSCD
severity
and
an
increase
in
visual
acuity
up
to
12
months
post-treatment
,
but
thereafter
LSCD
severity
and
visual
acuity
progressively
deteriorated
.
Diseases
Validation
Diseases presenting
"pain"
symptom
achondroplasia
acute rheumatic fever
adrenal incidentaloma
adrenomyeloneuropathy
aniridia
aromatase deficiency
carcinoma of the gallbladder
cholangiocarcinoma
coats disease
congenital diaphragmatic hernia
congenital toxoplasmosis
cushing syndrome
cutaneous mastocytosis
cystinuria
dedifferentiated liposarcoma
dentin dysplasia
dracunculiasis
dystrophic epidermolysis bullosa
epidermolysis bullosa simplex
erdheim-chester disease
erythropoietic protoporphyria
esophageal adenocarcinoma
esophageal carcinoma
esophageal squamous cell carcinoma
fabry disease
familial mediterranean fever
focal myositis
hirschsprung disease
hodgkin lymphoma, classical
holt-oram syndrome
junctional epidermolysis bullosa
kabuki syndrome
kindler syndrome
lamellar ichthyosis
liposarcoma
locked-in syndrome
lymphangioleiomyomatosis
malignant atrophic papulosis
neuralgic amyotrophy
oligodontia
oral submucous fibrosis
papillon-lefèvre syndrome
phenylketonuria
pleomorphic liposarcoma
primary hyperoxaluria type 1
proteus syndrome
pyomyositis
scrub typhus
sneddon syndrome
systemic capillary leak syndrome
thoracic outlet syndrome
trochlear dysplasia
typhoid
von hippel-lindau disease
waldenström macroglobulinemia
well-differentiated liposarcoma
wolf-hirschhorn syndrome
This symptom has already been validated