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The effect of rilonacept versus placebo on health-related quality of life in patients with poorly controlled familial Mediterranean fever.
[familial mediterranean fever]
To
examine
the
effect
of
rilonacept
on
the
health-related
quality
of
life
(
HRQoL
)
in
patients
with
poorly
controlled
familial
Mediterranean
fever
(
FMF
)
.
As
part
of
a
randomized
,
double
-blinded
trial
comparing
rilonacept
and
placebo
for
the
treatment
of
FMF
,
patients
/
parents
completed
the
modified
Child
Health
Questionnaire
(
CHQ
)
at
baseline
,
and
at
the
start
and
end
of
each
of
4
treatment
courses
,
2
each
with
rilonacept
and
placebo
.
Fourteen
subjects
were
randomized
;
mean
age
was
24
.
4
±
11
.
8
years
.
At
baseline
the
physical
HRQoL
score
was
significantly
less
(
24
.
2
±
49
.
5
)
but
the
psychosocial
score
was
similar
to
the
population
norm
(
49
.
5
±
10
.
0
)
.
There
were
significant
improvements
in
most
HRQoL
concepts
after
rilonacept
but
not
placebo
.
Significant
differences
between
rilonacept
and
placebo
were
found
in
the
physical
(
33
.
7
±
16
.
4
versus
23
.
7
±
14
.
5
,
P
=
0
.
021
)
but
not
psychosocial
scores
(
51
.
4
±
10
.
3
versus
49
.
8
±
12
.
4
,
P
=
0
.
42
)
.
The
physical
HRQoL
was
significantly
impacted
by
the
treatment
effect
and
patient
global
assessment
.
Treatment
with
rilonacept
had
a
beneficial
effect
on
the
physical
HRQoL
in
patients
with
poorly
controlled
FMF
and
was
also
significantly
related
to
the
patient
global
assessment
.
This
trial
is
registered
with
ClinicalTrials
.
gov
Identifier
NCT
00582907
.
Diseases
Validation
Diseases presenting
"fever"
symptom
22q11.2 deletion syndrome
acute rheumatic fever
alexander disease
allergic bronchopulmonary aspergillosis
canavan disease
carcinoma of the gallbladder
child syndrome
congenital toxoplasmosis
cushing syndrome
cystinuria
dracunculiasis
erdheim-chester disease
esophageal adenocarcinoma
esophageal carcinoma
familial mediterranean fever
focal myositis
hodgkin lymphoma, classical
lamellar ichthyosis
legionellosis
locked-in syndrome
malignant atrophic papulosis
neonatal adrenoleukodystrophy
neuralgic amyotrophy
oculocutaneous albinism
papillon-lefèvre syndrome
pyomyositis
pyruvate dehydrogenase deficiency
scrub typhus
severe combined immunodeficiency
sneddon syndrome
systemic capillary leak syndrome
triple a syndrome
typhoid
waldenström macroglobulinemia
wolf-hirschhorn syndrome
This symptom has already been validated