Rare Diseases Symptoms Automatic Extraction
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High-dose methotrexate for the treatment of relapsed central nervous system erdheim-chester disease.
[erdheim-chester disease]
Erdheim-
Chester
disease
(
ECD
)
is
a
rare
multisystem
non-
Langerhans
histiocytosis
.
CNS
involvement
is
a
major
complication
,
which
is
often
rapidly
progressive
and
confers
a
poor
prognosis
.
However
,
treatment
of
CNS
ECD
is
difficult
due
to
poor
CNS
penetrance
by
the
most
effective
chemotherapeutic
drugs
commonly
used
in
this
disorder
(
e
.
g
.
,
interferon
and
cladribine
)
.
We
describe
a
case
of
a
60
-
year
-old
lady
with
a
5
-
year
history
of
stable
systemic
ECD
who
presented
with
new
brainstem
lesions
and
rapid
,
steroid-refractory
neurological
deterioration
which
required
immediate
intervention
.
High
-dose
methotrexate
was
chosen
due
to
its
rapid
onset
of
action
and
excellent
CNS
penetration
.
Her
neurological
deterioration
was
quickly
arrested
with
significant
functional
improvement
,
which
was
sustained
for
4
months
with
consolidation
doses
of
high
-dose
methotrexate
.
Subsequent
treatment
with
cladribine
and
interferon
did
not
confer
any
appreciable
clinical
improvement
.
High
-dose
methotrexate
is
effective
in
controlling
rapidly
progressive
CNS
ECD
and
should
be
considered
as
a
salvage
agent
prior
to
commencement
of
more
definitive
treatment
.