Rare Diseases Symptoms Automatic Extraction
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Trabectedin in soft tissue sarcomas.
[liposarcoma]
Trabectedin
(
Yondelis
®
;
PharmaMar
,
Madrid
,
Spain
)
,
a
synthetic
anticancer
agent
originally
isolated
from
the
Caribbean
tunicate
,
Ecteinascidia
turbinata
,
is
currently
approved
in
more
than
70
countries
worldwide
for
the
treatment
of
soft
tissue
sarcoma
(
STS
)
.
Trabectedin
is
an
isoquinoline
alkylating
agent
that
,
unlike
other
alkylating
agents
,
binds
in
the
DNA
minor
groove
to
initiate
cytotoxic
activity
.
Other
multitarget
mechanisms
of
action
of
trabectedin
include
important
effects
within
the
tumor
microenvironment
;
in
particular
,
trabectedin
possesses
indirect
anti-
inflammatory
and
anti-angiogenic
activity
via
tumor
-associated
macrophages
and
high
-specificity
modulation
of
various
transcription
factors
.
The
clinical
efficacy
of
trabectedin
,
administered
intravenously
over
24
h
every
3
weeks
,
has
been
demonstrated
in
several
studies
in
patients
with
STS
.
In
the
Phase
II
STS
-
201
trial
,
270
patients
with
liposarcoma
or
leiomyosarcoma
were
randomized
to
receive
trabectedin
1
.
5
mg
/
m
(
2
)
given
as
a
24
-
h
intravenous
(
iv
.
)
infusion
every
3
weeks
or
as
a
weekly
regimen
(
0
.
58
mg
/
m
(
2
)
;
3
-
h
iv
.
infusion
for
three
consecutive
weeks
in
a
4
-
week
cycle
)
.
There
was
a
statistically
significant
and
clinically
relevant
27
%
reduction
in
the
risk
of
disease
progression
(
primary
end
point
)
with
trabectedin
given
as
a
24
-
h
infusion
q
3
w
(
p
=
0
.
0302
)
with
an
overall
survival
rate
at
12
months
of
60
%
.
Trabectedin
was
generally
well
tolerated
;
the
most
frequently
reported
severe
adverse
events
were
neutropenia
(
47
%
of
patients
)
and
elevated
transaminases
(
47
%
)
.
Overall
,
the
majority
of
adverse
events
were
mild
to
moderate
and
,
despite
a
long
duration
of
exposure
to
trabectedin
in
some
patients
,
no
cumulative
toxicities
were
experienced
.
Diseases
Validation
Diseases presenting
"primary end point"
symptom
cadasil
congenital adrenal hyperplasia
cushing syndrome
dedifferentiated liposarcoma
heparin-induced thrombocytopenia
liposarcoma
You can validate or delete this automatically detected symptom
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