Abstract

BackgroundCyclic vomiting syndrome (CVS), defined by recurrent stereotypical episodes of nausea and vomiting, is a relatively-common disabling and historically difficult-to-treat condition associated with migraine headache and mitochondrial dysfunction. Limited data suggests that the anti-migraine therapies amitriptyline and cyproheptadine, and the mitochondrial-targeted cofactors co-enzyme Q10 and L-carnitine, have efficacy in episode prophylaxis.MethodsA retrospective chart review of 42 patients seen by one clinician that met established CVS diagnostic criteria revealed 30 cases with available outcome data. Participants were treated on a loose protocol consisting of fasting avoidance, co-enzyme Q10 and L-carnitine, with the addition of amitriptyline (or cyproheptadine in those < 5 years) in refractory cases. Blood level monitoring of the therapeutic agents featured prominently in management.ResultsVomiting episodes resolved in 23 cases, and improved by > 75% and > 50% in three and one additional case respectively. Among the three treatment failures, two could not tolerate amitriptyline (as was also the case in the child with only > 50% efficacy) and one had multiple congenital gastrointestinal anomalies. Excluding the latter case, substantial efficacy (> 75% response) was 26/29 at the start of treatment, and 26/26 in those able to tolerate the regiment, including high dosages of amitriptyline.ConclusionOur data suggest that a protocol consisting of mitochondrial-targeted cofactors (co-enzyme Q10 and L-carnitine) plus amitriptyline (or possibly cyproheptadine in preschoolers) coupled with blood level monitoring is highly effective in the prevention of vomiting episodes.

Highlights

  • Cyclic vomiting syndrome (CVS), defined by recurrent stereotypical episodes of nausea and vomiting, is a relatively-common disabling and historically difficult-to-treat condition associated with migraine headache and mitochondrial dysfunction

  • Amitriptyline (Elavil®), a tricyclic “antidepressant” frequently used to treat migraine, is the most widely prescribed prophylactic medication used for the treatment of CVS, with response rates varying from 52-73% in open-label and subject recall-based studies in children and adults [reviewed in 5]

  • In a recent consensus statement, amitriptyline was recommended as the first-line treatment choice for CVS prophylaxis in children and adolescents age 5 years and older, while cyproheptadine is recommended in younger children [1]

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Summary

Introduction

Cyclic vomiting syndrome (CVS), defined by recurrent stereotypical episodes of nausea and vomiting, is a relatively-common disabling and historically difficult-to-treat condition associated with migraine headache and mitochondrial dysfunction. Limited data suggests that the anti-migraine therapies amitriptyline and cyproheptadine, and the mitochondrial-targeted cofactors co-enzyme Q10 and L-carnitine, have efficacy in episode prophylaxis. Cyclic vomiting syndrome (CVS) is characterized by recurrent identical episodes of nausea and vomiting, with the absence of these symptoms between episodes [1]. Amitriptyline (Elavil®), a tricyclic “antidepressant” frequently used to treat migraine, is the most widely prescribed prophylactic medication used for the treatment of CVS, with response rates varying from 52-73% in open-label and subject recall-based studies in children and adults [reviewed in 5]. In a recent consensus statement, amitriptyline was recommended as the first-line treatment choice for CVS prophylaxis in children and adolescents age 5 years and older, while cyproheptadine is recommended in younger children [1]

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