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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
"a serious adverse event of reversible agranulocytosis"
symptom
cutaneous mastocytosis
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