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Long-term results of the phase II trial of the oral mTOR inhibitor everolimus (RAD001) in relapsed or refractory Waldenstrom Macroglobulinemia.
[waldenström macroglobulinemia]
Everolimus
is
an
oral
raptor
mTOR
inhibitor
and
has
shown
activity
in
patients
with
Waldenstrom
's
macroglobulinemia
(
WM
)
.
This
study
examines
a
large
cohort
of
patients
with
relapsed
/
refractory
WM
with
long
-term
follow
up
for
survival
.
Patients
were
eligible
if
they
had
measurable
disease
,
a
platelet
count
>
75
,
000
x
10
(
6
)
/
L
,
an
absolute
neutrophil
count
>
1
,
000
x
10
(
6
)
/
L
.
Patients
received
everolimus
10
mg
PO
daily
and
were
evaluated
monthly
.
A
success
was
defined
as
a
complete
or
partial
response
(
PR
)
;
minor
responses
(
MR
)
were
recorded
and
considered
to
be
of
clinical
benefit
.
Sixty
patients
were
enrolled
and
treated
.
The
overall
response
rate
(
ORR
)
was
50
%
(
all
PR
)
;
the
clinical
benefit
rate
including
MR
or
better
was
73
%
(
95
%
CI
:
60
-
84
%
)
with
23
%
MR
.
The
median
time
to
response
for
patients
who
achieved
PR
was
2
months
(
range
,
1
-
26
)
.
The
median
duration
of
response
has
not
been
reached
and
median
progression-free
survival
(
PFS
)
was
21
months
.
Grade
3
or
higher
toxicities
(
at
least
possibly
related
to
everolimus
)
were
observed
in
67
%
of
patients
.
The
most
common
grade
3
or
4
toxicities
were
anemia
(
27
%
)
,
leukopenia
(
22
%
)
,
and
thrombocytopenia
(
20
%
)
.
Other
nonhematological
toxicities
were
diarrhea
(
5
%
)
,
fatigue
(
8
%
)
,
stomatitis
(
8
%
)
and
pulmonary
toxicity
(
5
%
)
.
Everolimus
has
a
high
single
-
agent
activity
of
73
%
including
MR
,
with
a
progression
free
survival
of
21
months
,
indicating
that
this
agent
is
active
in
relapsed
/
refractory
WM
.
Diseases
Validation
Diseases presenting
"stomatitis"
symptom
kindler syndrome
lymphangioleiomyomatosis
oral submucous fibrosis
waldenström macroglobulinemia
This symptom has already been validated