Diagnosis: VLCAD (very long chain acyl-Coenzyme A dehydrogenase) deficiency Treatment: Dojolvi SGM The insurer denied the Dojolvi SGM The denial is upheld. The patient is a girl with a diagnosis of very long chain acyl-Coenzyme A dehydrogenase (VLCAD) deficiency. She has frequent episodes of vomiting and headache and was recently diagnosed with cyclic vomiting syndrome. She did have episodes of mild hypoglycemia when younger. She is now clinically stable with respect to her VLCAD (very long chain acyl-Coenzyme A dehydrogenase) without rhabdomyolysis or myoglobinuria, although she does have muscle pains, and is treated with medium chain triglyceride oil and betaquik. However, she is variably or not compliant with carnitine or MCT (mediumchain triglyceride) Oil supplements. Plasma carnitine levels are normal. Other medical problems include abdominal pain, adjustment disorder with anxious mood, ADHD (attention deficit hyperactivity disorder), chromosomal abnormalities of chromosome 7 and 16, excessive sweating, fatigue, constipation, gait difficulty, hyperhidrosis, learning disability, scoliosis, self-injurious behavior, and urinary retention. She does not have cardiomyopathy. Molecular genetic testing demonstrated one pathogenic variant and one variant of uncertain significance in the VLCAD (very long chain acyl-Coenzyme A dehydrogenase) gene. The patient's provider has requested Dojolvi, an alternative to medium chain triglycerides, as a dietary supplement in her to hopefully improve her compliance with dietary management of her VLCAD (very long chain acyl-Coenzyme A dehydrogenase), and her general clinical state and prevent episodes of hypoglycemia and/or rhabdomyolysis. No, the requested Dojolvi is not medically necessary. While Dojolvi is the only FDA (United States Food and Drug Administration)-approved dietary supplement for VLCAD (very long chain acyl-Coenzyme A dehydrogenase), it is not necessary for all patients with this disorder of long chain fatty acid oxidation. This young woman does not have active current rhabdomyolysis, muscle disease directly caused by deficiency of this enzyme of long chain fatty acid oxidation, hypoglycemia, or cardiomyopathy. Therefore, given the mild to moderate clinical presentation of her disease, replacement of medium chain triglyceride supplements with Doljovi is not medically necessary, clinically appropriate, and is not supported by the current treatment guidelines for VLCAD (very long chain acyl-Coenzyme A dehydrogenase) patients with mild to moderate disease. Given that this individual has only mild to moderate VLCAD (very long chain acyl-Coenzyme A dehydrogenase) deficiency that is largely asymptomatic and well-controlled on MCT (mediumchain triglyceride) supplements, Dojolvi supplementation is not medically necessary or clinically appropriate in her specific clinical context. As such, health plan acted reasonably with sound medical judgment and in the best interest of the patient in this specific clinical context and scenario.

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