Management
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VLCAD Nutrition Management Guidelines
First Edition
February 2019, v.1.0
Current version: v.1.1
Updated: February 2019
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Nutrition Recommendations
Question
3. For individuals with VLCAD, do other specific nutrient supplementation or other treatment modalities (i.e. L-carnitine, triheptanoin, bezafibrates) improve outcomes? 
Conclusion Statement
Derived from evidence and consensus based clinical practice

The use of L-carnitine, triheptanoin, bezafibrate, riboflavin and coenzyme Q-10 (CoQ10) have all been reported in treatment of individuals with very-long chain acyl-coA dehydrogenase deficiency (VLCAD). The supplements are often used in conjunction with nutritional interventions such as restriction of long-chain fat (LCF) intake and supplementation of medium chain triglycerides (MCT).  The use of L-carnitine continues to be controversial in individuals with VLCAD. There are case reports of its successful use in conjunction with other nutritional therapies, and when dosages are reported, the most common range is 25 to100 mg/kg/d to maintain normal free carnitine concentrations. The concern raised by experts is whether supplementation of L-carnitine may increase the production of long-chain acylcarnitines and promote toxic effects, particularly in cardiomyopathy. However, supplementation of L-carnitine alone, without other nutritional interventions, does not provide a clinical benefit.

Triheptanoin (C7), currently only available in clinical trials, is being studied as a substitute for MCT and preliminary studies have shown improvement in cardiac status. Dosages of triheptanoin vary widely - 15-35% of total calories or 2.6-4 g/kg/day are commonly reported. In addition, there have been reports of gastrointestinal (GI) distress. 

Bezafibrates have been trialed, often without showing a positive effect, and require further study. Supplementation with CoQ10 and iboflavin have been reported in conjunction with other nutrition interventions, but also need further study to determine efficacy.

Recommendation 3.1

In a well individual with VLCAD, L-carnitine supplementation may not be needed, unless free carnitine concentration is <10 µmol/L. If low, consider a starting dose for L-carnitine of 10-25 mg/kg/d and titrate as needed based on lab monitoring.

Strength of Recommendation:
Insufficient EvidenceConsensusWeakFairStrong
Clinical Action:
ConditionalImperative
Recommendation 3.2

Triheptanoate (C7) is available only in clinical trials or as an investigational new drug (IND); therefore, recommendations for its general use in VLCAD cannot be made at this time.

Strength of Recommendation:
Insufficient EvidenceConsensusWeakFairStrong
Clinical Action:
ConditionalImperative
Recommendation 3.3

Bezafibrates are an experimental treatment for VLCAD and should not be used outside of a clinical trial. 

Strength of Recommendation:
Insufficient EvidenceConsensusWeakFairStrong
Clinical Action:
ConditionalImperative
Recommendation 3.4

There is insufficient evidence on the effectiveness of CoQ10 or other vitamin supplements in VLCAD; therefore, recommendations for their use cannot be made at this time.

Strength of Recommendation:
Insufficient EvidenceConsensusWeakFairStrong
Clinical Action:
ConditionalImperative
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