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A random Abstract
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Biochemical and clinical response to hydroxocobalamin versus cyanocobalamin treatment in patients with methylmalonic acidemia and homocystinuria (cblC).
[homocystinuria without methylmalonic aciduria]
To
compare
the
therapeutic
effectiveness
of
hydroxocobalamin
and
cyanocobalamin
in
patients
with
combined
methylmalonic
acidemia
and
homocystinuria
.
Analysis
of
urine
methylmalonic
acid
,
plasma
homocystine
,
and
growth
of
two
unrelated
patients
with
cobalamin
C
disease
who
were
initially
receiving
cyanocobalamin
and
were
subsequently
switched
to
hydroxocobalamin
.
Each
patient
had
a
significant
decrease
in
urine
methylmalonic
acid
excretion
while
receiving
cyanocobalamin
,
but
levels
remained
at
least
10
times
normal
.
Cyanocobalamin
treatment
resulted
in
a
decrease
of
plasma
homocystine
to
near
normal
in
one
patient
but
had
no
effect
on
plasma
homocystine
in
the
second
patient
.
Each
patient
was
switched
to
hydroxocobalamin
and
urine
methylmalonic
acid
levels
decreased
to
the
limit
of
detection
.
Plasma
homocystine
values
while
taking
hydroxocobalamin
remained
<
5
nmol
/
ml
in
both
patients
.
In
patient
1
,
who
continued
to
receive
cyanocobalamin
therapy
for
more
than
1
year
,
growth
rates
(
height
,
weight
,
and
head
circumference
)
were
very
poor
.
After
initiation
of
hydroxocobalamin
,
growth
parameters
normalized
with
growth
rates
above
normal
.
Intramuscular
cyanocobalamin
treatment
is
inadequate
in
the
treatment
of
patients
with
cobalamin
C
disease
.
Appropriate
management
of
cobalamin
C
disease
should
include
only
the
hydroxocobalamin
form
of
cobalamin
.
