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[Efficacy and safety of a phenylalanine-free amino acid-based enteral formula (AA-PKU2) in 1-8 year-old children with phenylketonuria: a prospective, open, self-controlled and multi-center study in China].
[phenylketonuria]
To
evaluate
the
efficacy
and
safety
of
a
phenylalanine-free
amino
acid-based
enteral
formula
(
AA
-
PKU
2
)
in
the
treatment
of
children
with
phenylketonuria
(
PKU
)
aged
1
-
8
years
.
A
prospective
,
open
,
self-controlled
,
multi-center
trial
was
performed
,
enrolling
121
PKU
children
(
1
-
8
years
in
age
)
consecutively
between
July
,
2009
and
May
,
2011
.
Enteral
nutrition
therapy
was
administered
for
32
weeks
.
The
data
on
blood
phenylalanine
(
PHE
)
levels
,
metal
development
,
weight
,
height
,
head
circumference
,
serum
nutritional
biomarkers
(
total
protein
,
pre-
albumin
,
albumin
,
total
cholesterol
,
total
triglyceride
,
low
-density
lipoprotein
cholesterol
,
high
-density
lipoprotein
cholesterol
)
,
and
measurements
from
routine
blood
and
urine
examinations
and
from
renal
and
hepatic
function
tests
were
collected
before
the
therapy
and
at
8
weeks
and
32
weeks
after
the
therapy
and
were
comparatively
analyzed
.
The
mean
blood
PHE
level
at
8
and
32
weeks
of
AA
-
PKU
2
treatment
was
353
±
253
and
361
±
280
µmol
/
L
respectively
,
significantly
lower
than
that
before
the
treatment
(
487
±
327
µmol
/
L
;
P
<
0
.
01
)
.
The
difference
in
intelligence
quotient
scores
before
and
after
AA
-
PKU
2
treatment
was
not
significant
(
P
>
0
.
05
)
when
assessed
by
the
Gesell
tests
in
children
aged
1
-
4
years
but
significant
(
P
<
0
.
01
)
when
assessed
by
WPPSI
or
WISR-R
tests
in
children
over
4
years
.
The
average
height
,
weight
and
head
circumference
at
8
and
32
weeks
after
treatment
were
significantly
increased
as
compared
to
these
measurements
before
treatment
(
P
<
0
.
01
)
with
absolute
levels
similar
to
those
in
the
control
children
.
In
contrast
,
the
mean
values
of
total
protein
,
pre-
albumin
,
albumin
,
total
cholesterol
,
total
triglyceride
,
low
-density
lipoprotein
cholesterol
and
high
-density
lipoprotein
cholesterol
at
both
time
points
were
not
different
either
from
those
prior
to
the
treatment
or
from
those
in
the
control
children
.
Mild
diarrhea
was
the
adverse
events
associated
with
AA
-
PKU
2
treatment
,
which
occurred
in
3
(
2
.
5
%
)
cases
.
All
these
3
patients
fully
recovered
without
treatment
.
The
phenylalanine-free
amino
acid-based
formula
,
AA
-
PKU
2
,
is
effective
and
safe
in
controlling
blood
PHE
levels
and
improving
mental
development
with
adequate
nutritional
support
in
PKU
.
Diseases
Validation
Diseases presenting
"serum nutritional biomarkers"
symptom
phenylketonuria
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