Efficacy of Coenzyme Q10 in the Treatment of Cyclic Vomiting Syndrome in Children

Research Article

J Pediatr & Child Health Care. 2021; 6(1): 1038.

Efficacy of Coenzyme Q10 in the Treatment of Cyclic Vomiting Syndrome in Children

Brezin F1, Wiedemann A1,2, Bansept C1, Albuisson E3, Renard E1,2 and Feillet F1,2*

1Reference Center for Inborn Errors of Metabolism of Nancy, France

2Inserm Ngere 1256, 3 UMDS, CHRU Nancy, France

3UMDS, CHRU Nancy, France

*Corresponding author: Francois Feillet, Pediatric Unit, University Children’s Hospital, CHU Brabois, Vandoeuvre Les Nancy, France

Received: February 27, 2021; Accepted: March 18, 2021; Published: March 25, 2021


Cyclic Vomiting Syndrome (CVS) is a chronic functional gastrointestinal disorder related to migraine, characterized by episodic nausea and vomiting. The treatment of CVS remains based on tricyclic antidepressants, triptans and antiepileptics. As mitochondriopathy has been involved in the pathophysiology of CVS, Coenzyme Q10 (CoQ10), a mitochondrial cofactor, has been used as the third line treatment in CVS. Considering the excellent safety profile of CoQ10, we decided to use it as the first line treatment in CVS. We retrospectively studied the evolution of 23 CVS patients who were treated for one year by CoQ10 alone. We recorded the characteristics of patients and their CVS history and compared data obtained the year before and the year following the prescription of CoQ10 treatment. We found a significant decrease in the number of vomiting episodes between the year before and the year after the start of CoQ10 (median [IQR]: 18.0 [15.75] vs. 3.00 [5.0]; p <0.001). This decrease persisted with time (2 and 3 years of treatment). The treatment was very efficient in 17/23 patients and did not decrease the number of vomiting episodes in 3 patients. Only one mild side effect related to the drug has been reported.

Conclusions: CoQ10 is an efficient and safe treatment of CVS and should be used as the first line treatment in this episodic syndrome related to migraine.

Keywords: Cyclic vomiting syndrome; Coenzyme Q10; Treatment; Migraine; Children


First described in 1806 [1,2], Cyclic Vomiting Syndrome (CVS) is still an underdiagnosed condition which belongs to the larger family of episodic syndromes related to migraines [3]. The latest Rome IV classification [4] defines CVS as stereotypical episodes of vomiting regarding onset (acute) and duration (less than 1 week), with 2 or more episodes in the past 6 months, occurring at least 1 week apart, with return to baseline health between cycles and which cannot be attributed to another condition. Its prevalence is estimated at 1.9% in children [1]. Some authors have evaluated this condition as being responsible for as much as 24 missed-school days per year for these children, or 15% of their childhood time, with an estimated yearly cost of $170,355. First and second-choice prophylactic treatments (Amitriptyline, Cyproheptadine, Propranolol, Pizotifen) are similar to migraine therapies but the safety profile of these drugs can be questionable as CVS is a functional disorder [6-8]. Some relations have been shown between migraine and mitochondrial DNA variants and CoQ10, which is a mitochondrial respiratory chain cofactor and has shown its efficacy (in association with L-carnitine) in the prevention of migraine in adults [9]. Boles published in 1997 an increased prevalence of two mitochondrial DNA polymorphisms similar to those found in migraine in patients with CVS [10] conducing them to try CVS treatment by Coenzyme Q10 (CoQ10) in association with L-carnitine and amitriptyline [11,12]. Considering that, compared to the other drugs used in CVS, CoQ10 has a very favorable safety profile [13] and after a very positive experience (complete disappearance of CVS in a very severe patient only treated by CoQ), we designed this study to evaluate the efficacy of CoQ10 in monotherapy as the first line treatment of CVS.


Study design

We conducted a monocenter, observational, retrospective study of children diagnosed for CVS and treated by CoQ10 in the reference center for inborn errors of metabolism of Nancy, France.


Eligible criteria: Diagnosis for CVS, age under 18 years, treatment by CoQ10 treatment for a planned duration of at least one year. There was no exclusion criterion.

Data collection

We recorded the data of all patients followed for CVS in our center (n=26). 3 patients were excluded because or treatment refusal. Finally, 23 patients were included in the study.

Inclusion was retrospective and data was collected from patients’ medical records from December 1st, 2016 to January 31st, 2019. All data were then anonymized. We collected familial and patient medical history (including personal and maternal migraine history) and yearly auxological data (weight, height and Body Mass Index (BMI)) expressed in z-score. The characteristics of CVS included age of onset, diagnosis and treatment by CoQ10 and the number of episodes per year. The severity of vomiting crises was subjectively evaluated by the parents. The statistical analysis was performed between data of the year before and the year following the prescription of CoQ10 treatment. The long-term efficacy of the treatment has also been evaluated for the patients who have been treated longer than one year. We also collected the safety data and the number of hospitalizations related to vomiting crises before and after CoQ10 treatment.


The primary objective was to demonstrate a reduction (of at least 50%) in the number of vomiting crisis under CoQ10 treatment. The secondary objectives were the evaluation of episodes intensity and long-term efficacy for patients treated more than one year. The nutritional status was estimated by yearly weight, height and BMI. The number of hospitalizations before and after treatment was recorded as all the CoQ10 side effects to assess the safety profile of coQ10 in this indication (Table 1).

Statistical analyses

Statistical analysis was performed using IBM SPSS Statistics v22 (IBM Corp.). According to their nature and distributions, variables are expressed with median and Interquartile Range (IQR) or frequencies and percentages. The non-parametric Wilcoxon signed-rank test was used to compare two related samples as before and after the use of CoQ10. Spearman’s Rho was used to study correlations between two parameters. The statistical significance level was set at 5%.


Patients ad CVS description

We identified 26 patients with CVS according to Rome IV definition in our center. 3 patients were not included because of treatment refusal. 23 patients (15 males and 8 females) accepted to be treated by Coenzyme Q10. The 23 patients were referred to our center by their pediatricians or their general practitioners. Before being referred to our center, one patient was seen by a pediatric endocrinologist, one by a pediatric allergist and the last patient by a pediatric gastroenterologist, a child psychiatrist and a psychologist. Median age at onset of CVS was 4.92 years [IQR 6.86], median age at diagnosis was 9.81 years [IQR 6.18] and median age at treatment was 10.00 years [IQR 7.00]. The median delay from the onset of symptoms to treatment is 2.5 years [IQR 4.02].

Past medical history included allergies (pollen, ash, dust mite, n=1), milk hypersensitivity (n=1), mild anorexia nervosa (n=1), mild delay of acquisitions (n=1) and hypospadias (n=1). In one case, CVS was associated with a complex syndrome including Hirschsprung’s disease, facial dysmorphism, psychomotor retardation, asymmetry of the temporal lobes, and thoraco-lumbar scoliosis. This patient was suspected to have a mitochondrial disease or Goldberg-Shprintzen syndrome without genetic proof to date. For 7 patients a trigger of crisis was described. This trigger was stress for 4 patients, infection for 2 patients and fatigue for the 7th patient.

We found a history of migraine in 13/23 patients (57%), and in 12/23 of mothers (52%). In 7 cases children and mothers have migraine symptoms. Two paternal history of migraine was reported in our cohort. One family provides a detailed family tree with multiple occurrence of migraine with maternal transmission which could be compatible with an mtDNA transmission (Figure 1). No significant association was found between personal or familial history of migraine (with and without) and the age of onset of CVS, respectively: personal migraine history (2.78 years [IQR 5.08] vs 7.0 years [IQR 6.33], p=0.08) and maternal migraine history (5.01 years (IQR 6.62] vs 4.34 years [IQR 7.06], p=0.78) or with the number of vomiting crises per year before treatment respectively: personal migraine history (with vs without; 18.0 [IQR 17.25] vs. 18.0 [IQR 12.0], p=0.90) and maternal migraine history (with vs without; 18.0 (IQR 27.0] vs. 18.0 [IQR 15.75], p=0.52).