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Abiraterone acetate to lower androgens in women with classic 21-hydroxylase deficiency.
[congenital adrenal hyperplasia]
Chronic
supraphysiological
glucocorticoid
therapy
controls
the
androgen
excess
of
21
-
hydroxylase
deficiency
(
21
OHD
)
but
contributes
to
the
high
prevalence
of
obesity
,
glucose
intolerance
,
and
reduced
bone
mass
in
these
patients
.
Abiraterone
acetate
(
AA
)
is
a
prodrug
for
abiraterone
,
a
potent
CYP
17
A
1
inhibitor
used
to
suppress
androgens
in
the
treatment
of
prostate
cancer
.
The
objective
of
the
study
was
to
test
the
hypothesis
that
AA
added
to
physiological
hydrocortisone
and
9
α-fludrocortisone
acetate
corrects
androgen
excess
in
women
with
21
OHD
without
causing
hypertension
or
hypokalemia
.
This
was
a
phase
1
dose-escalation
study
.
The
study
was
conducted
at
university
clinical
research
centers
.
We
screened
14
women
with
classic
21
OHD
taking
hydrocortisone
12
.
5
-
20
mg
/
d
to
enroll
six
participants
with
serum
androstenedione
greater
than
345
ng
/
dL
(
>
12
nmol
/
L
)
.
AA
was
administered
for
6
days
at
100
or
250
mg
every
morning
with
20
mg
/
d
hydrocortisone
and
9
α-fludrocortisone
acetate
.
The
primary
endpoint
was
normalization
of
mean
predose
androstenedione
on
days
6
and
7
(
<
230
ng
/
dL
[
<
8
nmol
/
L
)
]
in
greater
than
80
%
of
participants
.
Secondary
end
points
included
serum
17
-
hydroxyprogesterone
and
testosterone
(
T
)
,
electrolytes
,
plasma
renin
activity
,
and
urine
androsterone
and
etiocholanolone
glucuronides
.
With
100
mg
/
d
AA
,
mean
predose
androstenedione
fell
from
764
to
254
ng
/
dL
(
26
.
7
-
8
.
9
nmol
/
L
)
.
At
250
mg
/
d
AA
,
mean
androstenedione
normalized
in
five
participants
(
83
%
)
and
decreased
from
664
to
126
ng
/
dL
(
23
.
2
-
4
.
4
nmol
/
L
)
,
meeting
the
primary
end
point
.
Mean
androstenedione
declined
further
during
day
6
to
66
and
38
ng
/
dL
(
2
.
3
and
1
.
3
nmol
/
L
)
at
100
and
250
mg
/
d
,
respectively
.
Serum
T
and
urinary
metabolites
declined
similarly
.
Abiraterone
exposure
was
strongly
negatively
correlated
with
mean
androstenedione
.
Hypertension
and
hypokalemia
were
not
observed
.
AA
100
-
250
mg
/
d
added
to
replacement
hydrocortisone
normalized
several
measures
of
androgen
excess
in
women
with
classic
21
OHD
and
elevated
serum
androstenedione
.
Diseases
Validation
Diseases presenting
"obesity"
symptom
acute rheumatic fever
adrenal incidentaloma
aniridia
aromatase deficiency
carcinoma of the gallbladder
cohen syndrome
congenital adrenal hyperplasia
cushing syndrome
cystinuria
esophageal adenocarcinoma
esophageal carcinoma
esophageal squamous cell carcinoma
familial hypocalciuric hypercalcemia
familial mediterranean fever
heparin-induced thrombocytopenia
kabuki syndrome
monosomy 21
phenylketonuria
primary hyperoxaluria type 1
sneddon syndrome
werner syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
This symptom has already been validated