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Ultralow-dose Dexamethasone to Preserve Endogenous Cortisol Stress Response in Nonclassical Congenital Adrenal Hyperplasia: A New Promising Treatment.
[congenital adrenal hyperplasia]
Nonclassical
congenital
adrenal
hyperplasia
(
CAH
)
is
characterized
by
sufficient
cortisol
and
aldosterone
production
at
the
cost
of
androgen
overproduction
.
Hydrocortisone
or
dexamethasone
in
supraphysiological
doses
are
current
treatment
;
however
,
their
downside
is
suppression
of
endogenous
cortisol
production
resulting
in
corticosteroid
dependency
.
We
aimed
to
treat
children
with
nonclassical
CAH
with
a
ultralow-dose
dexamethasone
to
normalize
androgen
levels
,
without
a
detrimental
effect
on
endogenous
cortisol
production
.
We
recruited
five
patients
diagnosed
with
nonclassical
CAH
on
the
basis
of
clinical
presentation
,
biochemical
analyses
,
and
genetic
testing
.
Anthropometric
as
well
as
biochemical
parameters
and
bone
age
were
measured
on
a
regular
basis
.
During
treatment
,
an
adrenocorticotropin
(
ACTH
)
stimulation
test
was
performed
.
Outcome
measures
were
normalization
of
androgens
and
deceleration
of
the
bone
age
advancement
with
sufficient
endogenous
cortisol
response
.
Androgen
levels
were
normalized
in
all
patients
resulting
in
a
deceleration
of
the
bone
age
advancement
.
Cortisol
stress
response
remained
normal
in
four
out
of
five
patients
.
Only
one
patient
needed
hydrocortisone
stress
dosing
.
According
to
this
case
series
,
it
seems
that
ultralow-dose
dexamethasone
in
treatment
of
nonclassical
CAH
would
be
a
promising
novel
treatment
strategy
.
The
advantage
of
this
treatment
strategy
is
that
adverse
effects
of
hyperandrogenism
can
be
reversed
while
preserving
the
endogenous
cortisol
stress
response
.