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[Cladribine is highly effective in the treatment of Langerhans cell histiocytosis and rare histiocytic disorders of the juvenile xanthogranuloma group].
[erdheim-chester disease]
Cladribine
(
2
-
chlorodeoxyadenosine
)
is
metabolised
and
phosphorylated
in
a
cell
up
to
2
-
chloroadenosine
triphosphate
which
is
the
actual
effective
form
of
the
drug
.
The
greatest
accumulation
of
2
-
chloroadenosine
triphosphate
is
in
the
most
active
cells
,
where
activating
(
phosphorylation
)
enzyme
,
deoxycytidine
kinase
,
has
the
highest
activity
,
whereas
inactivating
enzyme
(
dephosphorylation
)
,
cytoplasmic
5
-
nucleotidase
,
has
the
lowest
activity
.
A
very
good
ratio
of
the
both
enzymes
for
high
effectiveness
of
cladribine
is
in
resting
and
proliferating
lymphocytes
.
Therefore
,
cladribine
is
an
effective
medication
for
hairy
cell
leukemia
,
Waldenström
macroglo-bulinemia
but
also
for
chronic
-
B-
lymphocytic
leukemia
.
However
,
such
high
concentrations
of
2
-
chloroadenosine
triphosphate
are
reached
in
some
cells
of
histiocytic
lines
,
in
monocytes
and
also
in
Langerhans
dendritic
cells
.
That
's
why
cladribine
is
highly
effective
medication
in
treating
Langerhans
cell
histiocytosis
and
also
in
treating
diseases
of
the
juvenile
xanthogranuloma
group
.
In
the
paper
we
present
a
survey
of
published
experience
with
cladribine
in
patients
with
Langerhans
cell
histiocytosis
.
The
effectiveness
of
cladribine
in
the
childhood
form
of
Langerhans
cell
histiocytosis
is
investigated
only
in
1
multicentric
clinical
study
,
other
data
are
taken
from
single
case
reports
or
small
series
studies
.
Cladribine
was
used
in
60
adult
patients
altogether
and
in
51
of
them
(
85
%
)
treatment
response
(
CR
+
PR
)
was
achieved
.
In
the
group
of
childhood
patients
cladribine
was
used
in
182
cases
and
treatment
response
(
CR
+
PR
)
was
reached
in
110
(
60
.
4
%
)
thereof
.
One
possible
explanation
for
a
higher
number
of
therapy
responses
in
adults
is
lower
Langerhans
cell
histiocytosis
aggressiveness
in
adults
than
in
children
.
Another
explanation
is
the
fact
that
therapy
responses
in
adults
are
summarized
only
from
case
reports
and
smaller
cohorts
,
whereas
in
children
,
case
reports
and
also
results
of
a
prospective
randomized
clinical
study
are
included
.
Diseases
of
the
juvenile
xanthogranuloma
group
are
much
more
rare
than
Langerhans
cell
histiocytosis
and
so
the
number
of
publications
is
smaller
.
In
total
,
7
publications
describe
therapy
response
of
cladribine
in
some
of
the
juvenile
xanthogranuloma
forms
(
Erdheim-
Chester
disease
,
disseminated
juvenile
xanthogranuloma
and
localized
form
of
plane
xanthoma
type
)
.
Cladribine
was
also
effective
in
CNS
infiltration
by
Langerhans
cell
histiocytosis
cells
or
juvenile
xanthogranuloma
cells
.
Cladribine
is
a
highly
effective
medication
used
in
treating
Langerhans
cell
histiocytosis
.
It
is
very
good
tolerated
in
monotherapy
.
Therefore
,
it
is
suitable
for
initial
therapy
of
adults
with
multifocal
or
multisystem
form
of
Langerhans
cell
histiocytosis
.
Furthermore
,
it
has
the
use
in
treating
relapses
after
some
other
initial
therapy
.
According
to
published
experience
,
it
is
an
effective
drug
for
diseases
of
the
juvenile
xanthogranuloma
group
(
Erdheim-
Chester
disease
,
diffuse
juvenile
xanthogranuloma
and
also
Rosai-
Dorfman
disease
)
.
Diseases
Validation
Diseases presenting
"leukemia"
symptom
canavan disease
cholangiocarcinoma
cutaneous mastocytosis
erdheim-chester disease
esophageal adenocarcinoma
esophageal carcinoma
esophageal squamous cell carcinoma
hodgkin lymphoma, classical
junctional epidermolysis bullosa
kabuki syndrome
liposarcoma
monosomy 21
neuralgic amyotrophy
papillon-lefèvre syndrome
phenylketonuria
pleomorphic liposarcoma
pyomyositis
severe combined immunodeficiency
typhoid
waldenström macroglobulinemia
werner syndrome
wiskott-aldrich syndrome
x-linked adrenoleukodystrophy
This symptom has already been validated