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A 2-year randomised placebo-controlled trial of doxycycline for lymphangioleiomyomatosis.
[lymphangioleiomyomatosis]
Lymphangioleiomyomatosis
(
LAM
)
is
characterised
by
lung
cysts
and
airflow
obstruction
.
Matrix
metalloproteinases
have
been
implicated
in
lung
destruction
in
LAM
.
We
performed
a
randomised
,
double
-blind
trial
,
comparing
the
matrix
metalloproteinases
inhibitor
doxycycline
with
placebo
on
the
progression
of
LAM
.
23
females
with
LAM
were
randomised
to
doxycycline
100
mg
daily
for
3
months
followed
by
200
mg
daily
for
21
months
,
or
matched
placebo
.
Lung
function
,
exercise
capacity
,
quality
of
life
and
matrix
metalloproteinases
levels
were
measured
.
21
patients
completed
6
months
of
treatment
,
17
completed
1
year
of
treatment
and
15
completed
2
years
of
treatment
.
Eight
withdrew
from
the
trial
due
,
four
due
to
a
pneumothorax
and
four
because
of
other
reasons
.
The
mean
±
sd
decline
in
FEV
1
,
the
primary
endpoint
,
did
not
differ
between
the
groups
being
-
90
±
154
mL
·
year
(
-
1
)
in
the
placebo
group
and
-
123
±
246
mL
·
year
(
-
1
)
in
the
doxycycline
group
(
difference
-
32
.
5
,
95
%
CI
-
213
-
148
;
p
=
0
.
35
)
.
Doxycycline
had
no
effect
upon
vital
capacity
,
gas
transfer
,
shuttle
walk
distance
or
quality
of
life
.
Urine
matrix
metalloproteinases-
9
measurements
were
lower
with
doxycycline
treatment
(
p
=
0
.
03
)
.
Although
with
limited
numbers
we
can
not
completely
exclude
an
effect
of
doxycycline
,
the
lack
of
effect
on
any
outcome
makes
it
unlikely
that
doxycycline
has
a
useful
effect
in
LAM
.
Diseases
Validation
Diseases presenting
"primary endpoint"
symptom
allergic bronchopulmonary aspergillosis
congenital adrenal hyperplasia
dedifferentiated liposarcoma
heparin-induced thrombocytopenia
lymphangioleiomyomatosis
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