MCAD deficiency
Medium-chain
acyl-CoA
dehydrogenase deficiency (MCAD deficiency) is a rare, inherited disease
where the
body is unable to break down fat properly.
MCAD
deficiency can cause lethargy and a lack of energy and can lead to
serious complications such as seizures, coma and even death.
What exactly happens?
If
one doesn't eat for a long time, the body will burn body fat in order
to produce energy. Body fat is broken down to fatty acids and those
fatty acids are in their turn broken down to shorter fatty acids until
they are completely broken down or metabolised. With each step some
energy is released.
People
with MCAD deficiency cannot break down fatty acids in a normal
way because one of the enzymes necessary for this is not functioning
properly. This enzyme is called the MCAD enzyme.
As
a result medium chain fatty acids are accumulated in the body.
If the body wants to break down fat quickly (because one hasn't eaten
in
a while or has an infection) then a high concentration of medium chain
fatty acids is accumulated. This is poisonous, and can lead to serious
complications.
At
the same time these people cannot get energy from breaking down
medium chain fats, so they are mostly reliant on sugars for their
energy production.
A lack of energy can in its turn cause serious complications such as
low blood sugar.
See
the chapter on metabolism
for more information.
Causes
MCAD
deficiency is an inborn, inherited disease.
A
baby has a 25 % chance of inheriting the disease from his parents if
both parents are carriers of the disease.
See
the chapter on genetics
for more information.
Diagnosis
In
Belgium every newborn child is screened for a number of inherited
diseases, among which MCAD deficiency.
If
it is suspected that a baby has MCAD deficiency there are some other
examinations possible to confirm the diagnosis.
See
the chapter on diagnosis
for more information.
Outlook and treatment
Once
the diagnosis is confirmed, patients with MCAD deficiency can live a
relatively normal life as long as they take a few simple precautions.
They
shouldn't go too long without eating and should be put on a
healthy, low fat diet.
Some
extra care is necessary in case of illness and/or vomiting. In
that case they should be admitted to the hospital to be treated with IV
glucose.
See
the chapter on treatment
for more information.
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The content of this website is not
validated by doctors, scientists or geneticists. |
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