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Several investigators have studied the use of
medium-chain triglycerides (MCT) in CPT II deficiency.

In
1997, Schaefer et al. assessed the effects of an MCT emulsion of 0.5 g/kg
body weight in one CPT II patient and two controls.
Plasma ketone production was similar in patient and controls after MCT loading, but in contrast to controls, the patient developed a significant transient medium-chain dicarboxylic aciduria.
Because CPT is involved in the transport of medium-chain fatty acids, and in fact, accounts for 70% of total cellular carnitine octanoyl-transferase (COT) activity, the investigators concluded that any dose of MCT over 0.5 g/kg body weight would probably overload the uptake mechanism. They recommend a careful adjustment of the dose so as not to exceed the limited CPT transport capacity for medium-chain fatty acids.
In
1981, DiDonato et al.reported a CPT II patient who was unable to build up
ketones when fed long-chain triglycerides but showed prompt ketogenic response when fed MCT.
In
1991, Scott et al. reported a CPT II deficiency patient with high triglycerides. The patient was treated with MCT. |
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