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Preclinical and clinical evidence of activity of pazopanib in solitary fibrous tumour.
[severe combined immunodeficiency]
To
explore
the
activity
of
pazopanib
in
solitary
fibrous
tumour
(
SFT
)
.
In
a
preclinical
study
,
we
compared
the
activity
of
pazopanib
,
sorafenib
,
sunitinib
,
regorafenib
,
axitinib
and
bevacizumab
in
a
dedifferentiated-
SFT
(
DSFT
)
xenotransplanted
into
Severe
Combined
Immunodeficiency
(
SCID
)
mice
.
Antiangiogenics
were
administered
at
their
reported
optimal
doses
when
mean
tumour
volume
(
TV
)
was
80
mm
(
3
)
.
Drug
activity
was
assessed
as
TV
inhibition
percentage
(
TVI
%
)
.
From
May
2012
,
six
consecutive
patients
with
advanced
SFT
received
pazopanib
,
on
a
national
name-based
programme
.
In
one
case
sunitinib
was
administered
after
pazopanib
failure
.
In
the
xenograft
model
,
pazopanib
showed
the
lowest
antitumour
activity
(
21
%
TVI
)
,
while
regorafenib
was
the
most
active
(
95
%
TVI
)
.
Sorafenib
,
bevacizumab
,
sunitinib
were
markedly
active
(
78
/
70
/
65
%
TVI
)
.
Axitinib
was
marginally
active
(
51
%
TVI
)
.
In
the
retrospective
case-series
,
three
patients
carried
malignant
-
SFT
(
MSFT
)
,
three
DSFT
.
Best
Response
Evaluation
Criteria
in
Solid
Tumour
(
RECIST
)
responses
were
:
three
stable
disease
(
SD
)
,
all
MSFT
,
three
progressive
disease
(
PD
)
,
all
DSFT
,
corresponding
to
one
partial
response
(
PR
)
,
two
SD
,
three
PD
by
Choi
criteria
.
Median-progression-free
survival
was
3
months
(
range
1
-
15
)
.
In
one
patient
,
sunitinib
was
started
after
pazopanib
failure
,
with
a
response
.
In
dedifferentiated-
SFT
xenograft
pazopanib
induced
a
marginal
antitumour
activity
,
while
regorafenib
appeared
the
most
active
and
promising
agent
.
When
administered
in
patients
,
pazopanib
showed
a
modest
activity
in
terms
of
tumour
growth
stabilisation
,
observed
only
in
non-dedifferentiated
cases
.
Diseases
Validation
Diseases presenting
"solitary fibrous tumour"
symptom
severe combined immunodeficiency
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