Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Long-term outcome of monoclonal (type 1) cryoglobulinemia.
[waldenström macroglobulinemia]
The
aim
of
this
study
is
to
investigate
long
-term
outcome
of
symptomatic
type
1
cryoglobulinemia
(
CG
)
and
its
determinants
.
Retrospective
cohort
study
was
conducted
in
two
French
University
Hospitals
.
Patients
with
type
1
CG
were
identified
using
laboratory
databases
.
Inclusion
criterion
was
the
presence
of
persistent
symptoms
of
CG
.
Among
227
screened
patients
,
36
were
included
.
Skin
or
vasomotor
symptoms
were
the
most
frequent
features
(
75
%
)
.
Nephropathy
and
neuropathy
occurred
in
30
%
and
47
%
of
cases
,
respectively
.
The
underlying
B
cell
disease
(
BCD
)
was
a
nonmalignant
monoclonal
gammopathy
(
NMMG
)
in
13
(
36
%
)
and
a
hematologic
malignancy
(
HM
)
in
23
(
64
%
;
Waldenstrom
macroglobulinemia
(
WM
)
in
12
,
low
-grade
non-
Hodgkin
lymphoma
(
NHL
)
in
6
,
multiple
myeloma
(
MM
)
in
4
,
and
chronic
lymphocytic
leukemia
in
1
patient
.
Severe
manifestations
affected
half
the
patients
and
were
more
frequent
with
IgG
(
82
vs
.
30
%
(
P
=
0
.
006
)
)
.
At
last
follow-up
,
64
%
of
patients
had
suffered
no
hematologic
manifestation
.
Potent
chemotherapeutic
regimens
were
mainly
used
in
HM
.
For
patients
with
NMMG
,
WM
,
or
NHL
,
fludarabine
or
rituximab-based
regimens
appeared
to
yield
better
responses
.
Five
-
year
survival
rate
was
82
%
.
In
multivariate
analysis
,
mortality
was
significantly
higher
in
older
patients
(
HR
:
1
.
17
per
year
[
95
%
CI
:
1
.
06
-
1
.
28
]
,
P
=
0
.
001
)
and
those
with
nephropathy
(
HR
:
8
.
9
[
95
%
CI
:
1
.
9
-
43
]
,
P
=
0
.
006
)
.
Kidney
disease
,
infections
,
Richter
's
transformation
,
and
second
malignancies
were
important
sources
of
morbi-mortality
.
Despite
its
limitations
,
this
series
provide
novel
information
regarding
type
1
CG
.
Further
studies
are
needed
to
improve
its
management
.
To
date
,
therapeutic
strategy
should
be
tailored
according
to
patient
's
characteristics
(
age
,
comorbidities
,
underlying
BCD
)
,
and
therapeutic
target
.
Diseases
Validation
Diseases presenting
"long-term outcome"
symptom
aniridia
cholangiocarcinoma
classical phenylketonuria
cystinuria
erythropoietic protoporphyria
esophageal squamous cell carcinoma
fabry disease
hirschsprung disease
homocystinuria without methylmalonic aciduria
hydrocephalus with stenosis of the aqueduct of sylvius
inclusion body myositis
kabuki syndrome
krabbe disease
oligodontia
papillon-lefèvre syndrome
primary hyperoxaluria type 1
trochlear dysplasia
von hippel-lindau disease
waldenström macroglobulinemia
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom