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Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study.
[cutaneous mastocytosis]
Treatment
options
for
patients
suffering
from
indolent
forms
of
mastocytosis
remain
inadequate
with
the
hyperactivation
of
mast
cells
responsible
for
many
of
the
disease
's
systemic
manifestations
.
Masitinib
is
a
potent
and
highly
selective
oral
tyrosine
kinase
inhibitor
.
A
combined
inhibition
of
c-
Kit
and
Lyn
make
it
particularly
efficient
in
controlling
the
activity
of
mast
cells
and
therefore
,
of
potential
therapeutic
benefit
in
mastocytosis
.
Masitinib
was
administered
to
25
patients
diagnosed
as
having
systemic
or
cutaneous
mastocytosis
with
related
handicap
(
i
.
e
.
,
disabilities
associated
with
flushes
,
depression
,
pruritus
and
quality-of-life
)
at
the
initial
dose
levels
of
3
or
6
mg
/
kg
/
day
over
12
weeks
.
In
accordance
with
the
AFIRMM
study
,
response
was
based
upon
change
of
clinical
symptoms
associated
with
patient
handicap
at
week
12
relative
to
baseline
,
regardless
of
disease
subtype
.
Improvement
was
observed
in
all
primary
endpoints
at
week
12
including
a
reduction
of
flushes
,
Hamilton
rating
,
and
pruritus
as
compared
with
baseline
by
64
%
(
P
=
0
.
0005
)
,
43
%
(
P
=
0
.
0049
)
,
and
36
%
(
P
=
0
.
0077
)
,
respectively
.
An
overall
clinical
response
was
observed
in
14
/
25
patients
(
56
%
;
[
95
%
CI
=
37
%
-
75
%
]
)
,
with
sustainable
improvement
observed
throughout
an
extension
phase
(
>
60
weeks
)
.
Common
adverse
events
were
edema
(
44
%
)
,
nausea
(
44
%
)
,
muscle
spasms
(
28
%
)
,
and
rash
(
28
%
)
,
the
majority
of
which
were
of
mild
or
moderate
severity
with
a
significant
decline
in
frequency
observed
after
12
weeks
of
treatment
.
One
patient
experienced
a
serious
adverse
event
of
reversible
agranulocytosis
.
Masitinib
is
a
promising
treatment
for
indolent
forms
of
mastocytosis
with
handicap
and
indicates
acceptable
tolerability
for
long
-term
treatment
regimens
.
Diseases
Validation
Diseases presenting
"depression"
symptom
22q11.2 deletion syndrome
achondroplasia
adrenal incidentaloma
adrenomyeloneuropathy
alexander disease
aromatase deficiency
cadasil
child syndrome
classical phenylketonuria
congenital adrenal hyperplasia
congenital toxoplasmosis
cushing syndrome
cutaneous mastocytosis
dracunculiasis
fabry disease
familial hypocalciuric hypercalcemia
gm1 gangliosidosis
hereditary cerebral hemorrhage with amyloidosis
krabbe disease
locked-in syndrome
malignant atrophic papulosis
oligodontia
oral submucous fibrosis
phenylketonuria
sneddon syndrome
triple a syndrome
trochlear dysplasia
von hippel-lindau disease
This symptom has already been validated