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Retrospective Review Of Combined Sirolimus And Simvastatin Therapy In Lymphangioleiomyomatosis.
[lymphangioleiomyomatosis]
Background
.
Combined
simvastatin
and
sirolimus
therapy
reduce
TSC
2
-
null
lesions
and
alveolar
destruction
in
a
mouse
model
of
lymphangioleiomyomatosis
(
LAM
)
,
suggesting
that
therapy
with
both
drugs
may
benefit
LAM
patients
.
Methods
.
To
determine
whether
simvastatin
changed
the
prevalence
of
adverse
events
or
altered
the
therapeutic
effects
of
sirolimus
,
we
recorded
adverse
events
and
changes
in
lung
function
in
LAM
patients
treated
with
simvastatin
plus
sirolimus
(
n
=
14
)
,
sirolimus
(
n
=
44
)
,
or
simvastatin
(
n
=
20
)
.
Results
.
Sirolimus-related
adverse
events
in
the
simvastatin
plus
sirolimus
,
and
sirolimus
only
groups
,
were
64
and
66
%
for
stomatitis
,
50
and
52
%
for
diarrhea
,
50
and
45
%
for
peripheral
edema
,
36
and
61
%
for
acne
,
36
and
30
%
for
hypertension
,
29
and
27
%
for
proteinuria
,
29
and
27
%
for
leukopenia
,
and
21
and
27
%
for
hypercholesterolemia
.
The
frequency
of
simvastatin-related
adverse
events
in
the
simvastatin
,
and
simvastatin
plus
sirolimus
groups
were
60
and
50
%
for
arthralgias
,
and
35
and
36
%
for
myopathy
.
Before
simvastatin
plus
sirolimus
therapy
,
FEV
1
and
DLCO
yearly
rates
of
change
were
respectively
,
-
1
.
4
±
0
.
2
and
-
1
.
8
±
0
.
2
%
predicted
.
After
simvastatin
plus
sirolimus
therapy
,
these
rates
changed
to
+
1
.
2
±
0
.
5
(
p
=
0
.
635
)
and
+
0
.
3
±
0
.
4
%
predicted
,
respectively
(
p
=
0
.
412
)
.
In
44
patients
treated
with
sirolimus
alone
,
FEV
1
and
DLCO
rates
of
change
were
-
1
.
7
±
0
.
1
and
-
2
.
2
±
0
.
1
%
predicted
before
treatment
,
and
+
1
.
7
±
0
.
3
and
+
0
.
7
±
0
.
3
%
predicted
after
therapy
(
p
<
0
.
001
)
.
Conclusions
.
Therapy
with
sirolimus
and
simvastatin
does
not
increase
the
prevalence
of
drug
adverse
events
or
alter
the
therapeutic
effects
of
sirolimus
.
Combined
simvastatin
and
sirolimus
therapy
reduce
TSC
2
-
null
lesions
and
alveolar
destruction
in
a
mouse
model
of
lymphangioleiomyomatosis
(
LAM
)
,
suggesting
that
therapy
with
both
drugs
may
benefit
LAM
patients
.
To
determine
whether
simvastatin
changed
the
prevalence
of
adverse
events
or
altered
the
therapeutic
effects
of
sirolimus
,
we
recorded
adverse
events
and
changes
in
lung
function
in
LAM
patients
treated
with
simvastatin
plus
sirolimus
(
n
=
14
)
,
sirolimus
(
n
=
44
)
,
or
simvastatin
(
n
=
20
)
.
Sirolimus-related
adverse
events
in
the
simvastatin
plus
sirolimus
,
and
sirolimus
only
groups
,
were
64
and
66
%
for
stomatitis
,
50
and
52
%
for
diarrhea
,
50
and
45
%
for
peripheral
edema
,
36
and
61
%
for
acne
,
36
and
30
%
for
hypertension
,
29
and
27
%
for
proteinuria
,
29
and
27
%
for
leukopenia
,
and
21
and
27
%
for
hypercholesterolemia
.
The
frequency
of
simvastatin-related
adverse
events
in
the
simvastatin
,
and
simvastatin
plus
sirolimus
groups
were
60
and
50
%
for
arthralgias
,
and
35
and
36
%
for
myopathy
.
Before
simvastatin
plus
sirolimus
therapy
,
FEV
1
and
DLCO
yearly
rates
of
change
were
respectively
,
-
1
.
4
±
0
.
2
and
-
1
.
8
±
0
.
2
%
predicted
.
After
simvastatin
plus
sirolimus
therapy
,
these
rates
changed
to
+
1
.
2
±
0
.
5
(
p
=
0
.
635
)
and
+
0
.
3
±
0
.
4
%
predicted
,
respectively
(
p
=
0
.
412
)
.
In
44
patients
treated
with
sirolimus
alone
,
FEV
1
and
DLCO
rates
of
change
were
-
1
.
7
±
0
.
1
and
-
2
.
2
±
0
.
1
%
predicted
before
treatment
,
and
+
1
.
7
±
0
.
3
and
+
0
.
7
±
0
.
3
%
predicted
after
therapy
(
p
<
0
.
001
)
.
Therapy
with
sirolimus
and
simvastatin
does
not
increase
the
prevalence
of
drug
adverse
events
or
alter
the
therapeutic
effects
of
sirolimus
.
Diseases
Validation
Diseases presenting
"hypertension"
symptom
achondroplasia
acute rheumatic fever
adrenal incidentaloma
aniridia
aromatase deficiency
cadasil
child syndrome
cohen syndrome
congenital adrenal hyperplasia
congenital diaphragmatic hernia
cushing syndrome
cystinuria
erdheim-chester disease
erythropoietic protoporphyria
esophageal adenocarcinoma
fabry disease
familial hypocalciuric hypercalcemia
gm1 gangliosidosis
heparin-induced thrombocytopenia
hereditary cerebral hemorrhage with amyloidosis
holt-oram syndrome
homocystinuria without methylmalonic aciduria
hydrocephalus with stenosis of the aqueduct of sylvius
inclusion body myositis
kallmann syndrome
kindler syndrome
lamellar ichthyosis
lymphangioleiomyomatosis
pendred syndrome
primary effusion lymphoma
scrub typhus
severe combined immunodeficiency
sneddon syndrome
typhoid
von hippel-lindau disease
well-differentiated liposarcoma
werner syndrome
x-linked adrenoleukodystrophy
zellweger syndrome
This symptom has already been validated