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Mycophenolate mofetil: a novel immunosuppressant in the treatment of dystrophic epidermolysis bullosa, a randomized controlled trial.
[dystrophic epidermolysis bullosa]
No
effective
treatment
has
been
found
for
epidermolysis
bullosa
dystrophica
(
EBD
)
.
To
evaluate
the
efficacy
and
safety
mycophenolate
mofetil
(
MMF
)
in
treating
EBD
.
T
his
randomized
controlled
double
-blinded
study
included
35
patients
with
severe
generalized
EBD
.
Patients
were
randomly
divided
into
two
groups
:
group
I
(
18
patients
)
received
cyclosporine
therapy
(
5
mg
/
kg
/
day
)
and
group
II
(
17
patients
)
received
MMF
therapy
(
500
-
1500
mg
/
day
)
.
Clinical
assessment
was
made
weekly
for
3
months
from
the
start
of
the
treatment
.
Patients
were
assessed
by
measuring
the
extent
of
the
disease
,
the
%
of
improvement
,
assessing
the
number
of
new
blister
formation
and
the
time
of
complete
healing
of
new
blisters
.
Side
effects
were
recorded
when
detected
.
The
%
of
improvement
in
the
disease
extent
was
statistically
significantly
higher
(
p
=
0
.
009
)
in
group
I
(
mean
±
SD
:
59
.
21
±
22
.
676
)
than
in
group
II
(
mean
±
SD
:
44
.
03
±
25
.
71
)
.
As
regards
the
number
of
new
blisters
and
the
rate
of
healing
of
blisters
,
there
was
no
statistically
significant
difference
between
both
groups
(
p
=
0
.
693
and
0
.
404
,
respectively
)
.
No
serious
side
effects
were
reported
.
MMF
seems
to
be
a
good
therapeutic
option
for
the
long
-term
treatment
of
EBD
,
it
can
be
a
good
alternative
for
patients
who
can
not
tolerate
cyclosporine
.
