A prospective study of intrapyloric botulinum toxin and EndoFLIP in children with nausea and vomiting.

[BACKGROUND] Intrapyloric botulinum toxin injection (IPBI) is used to treat nausea and vomiting in children, but no prospective pediatric studies exist. The aims of the current study were to assess the efficacy of IPBI in children with refractory nausea or vomiting and to use EndoFLIP as a biomarker of IPBI response.

[METHODS] This was a prospective observational cohort study of pediatric patients undergoing IPBI for refractory nausea, vomiting, or feeding difficulties at a tertiary center. Patients completed validated questionnaires at baseline and 1, 2, and 3 months after IPBI. A subset of patients >10 years old underwent pyloric EndoFLIP at the time of IPBI. Symptoms were compared pre- and post-IPBI, and EndoFLIP measurements were assessed in relation to baseline characteristics and response to IPBI.

[KEY RESULTS] Forty-five patients (mean age 14.2 ± 6.0 years) received IPBI, and 23 of those patients underwent EndoFLIP. Twenty-nine patients (64%) had symptomatic improvement at 1-month follow-up, including improvements in overall GI symptoms (p = 0.003), nausea and vomiting (p = 0.009), and discomfort when eating (p = 0.006). Symptomatic improvements lasted up to 3 months. There was a trend towards lower pyloric distensibility in patients with delayed versus normal gastric emptying (4.5 ± 3.8 mm /mmHg in delayed vs. 8.9 ± 6.6 mm /mmHg in normal, p = 0.09), though there were no differences in EndoFLIP measurements between IPBI responders and non-responders (p > 0.05).

[CONCLUSIONS AND INFERENCES] The majority of patients responded to IPBI, with significant improvements seen in nausea, vomiting, and discomfort. Pyloric EndoFLIP tended to distinguish baseline delays in gastric emptying, but it did not predict IPBI response in this patient cohort.