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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
"fatigue"
symptom
benign recurrent intrahepatic cholestasis
cholangiocarcinoma
congenital adrenal hyperplasia
cowden syndrome
cutaneous mastocytosis
dedifferentiated liposarcoma
dracunculiasis
erdheim-chester disease
esophageal carcinoma
fabry disease
familial hypocalciuric hypercalcemia
familial mediterranean fever
hydrocephalus with stenosis of the aqueduct of sylvius
inclusion body myositis
kabuki syndrome
lymphangioleiomyomatosis
neuralgic amyotrophy
pleomorphic liposarcoma
systemic capillary leak syndrome
triple a syndrome
typhoid
von hippel-lindau disease
waldenström macroglobulinemia
x-linked adrenoleukodystrophy
This symptom has already been validated