Paeds Vivas · genetics-dysmorphology-and-metabolism
Fatty-acid oxidation disorders — branching viva
Branching viva on the fatty-acid oxidation disorders: recognising the hypoketotic hypoglycaemia signature, delivering the intravenous-dextrose emergency protocol, localising the defect with plasma acylcarnitines, and locking in long-term management with fasting avoidance, a sick-day plan and triheptanoin for long-chain defects.
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Opening framework
My framework has four layers. First, the recognition — a child with fasting- or illness-induced hypoketotic hypoglycaemia is a fatty-acid oxidation disorder until proven otherwise, and the bedside ketone measurement is the test that decides it. Second, the emergency protocol — IV 10 percent dextrose immediately to shut off lipolysis, collect the critical sample in parallel, and do not wait for the enzyme or molecular diagnosis. Third, the biochemistry — the carnitine shuttle and the beta-oxidation spiral, and each block produces a recognisable acylcarnitine signature. Fourth, the family — these are autosomal recessive, newborn screening detects most, and a written sick-day plan is what keeps the child safe. [1]
References5ShowHide
- [1]Merritt JL 2nd, Norris M, Kanungo S. Fatty acid oxidation disorders. Ann Transl Med, 2018.PMID 30740404
- [2]Vockley J Long-chain fatty acid oxidation disorders and current management strategies. Am J Manag Care, 2020.PMID 32840329
- [5]Wilcken B. Fatty acid oxidation disorders: outcome and long-term prognosis. J Inherit Metab Dis, 2010.PMID 20049534
- [6]Derks TG, Reijngoud DJ, Waterham HR, et al. The natural history of medium-chain acyl CoA dehydrogenase deficiency in the Netherlands: clinical presentation and outcome. J Pediatr, 2006.PMID 16737882
- [9]Vockley J, Burton B, Berry GT, Longo N, et al. Triheptanoin for the treatment of long-chain fatty acid oxidation disorders: Final results of an open-label, long-term extension study. J Inherit Metab Dis, 2023.PMID 37276053