Baclofen Does Not Significantly Improve Rumination Syndrome Symptoms in Children

Janice Khoo, MD

Credit: CHOC

A recent randomized controlled trial showed that baclofen for rumination syndrome was feasible and safe in children but did not significantly improve rumination syndrome symptoms and quality of life compared with placebo.¹

The research, led by Janice Khoo, MD, from the division of gastroenterology and the department of pediatrics at Nationwide Children’s Hospital in Columbus, Ohio, was presented at the 2024 Annual North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) Meeting in Hollywood, Florida, from November 7 to November 9, 2024.

Rumination syndrome, a disorder of gut-brain interaction characterized by postprandial and regurgitation, is typically treated with behavioral modification. Baclofen, a gamma-aminobutyric acid agonist used to treat muscle spasticity, can reduce rumination frequency, but not a lot of evidence exists on this. A retrospective review published in May 2023 suggested baclofen in addition to behavioral therapy can improve symptoms of rumination syndrome in children, but prospective, controlled studies were needed to replicate these findings.²

Khoo and colleagues sought to evaluate the feasibility, safety, and efficacy of baclofen in children with rumination syndrome^.1 Investigators conducted a randomized, double-blind, placebo-controlled study evaluating baclofen in 42 children aged 6 – 18 years (mean age: 13 years; 64% female) diagnosed with rumination syndrome in 2023 to 2024. The primary endpoint was the proportion of patients who had a clinical response, defined as vomiting ≤ 1 per week, at the 4-week follow-up.

Participants were randomized to receive either baclofen (0.5 mg/kg/day up to 15 mg/day divided 3 times daily) (n = 22) or placebo (n = 20) for 4 weeks, and during this time, patients continued to receive usual care as determined by their primary gastroenterologist. Participants also had to complete surveys at baseline, week 1, week 2, and week 4. The surveys included a novel rumination severity index (RumSI), the Pediatric Quality of Life Inventory (PedsQL), and questions on whether the symptoms bothered them or if they experienced adverse events.

The proportion of participants who experienced a clinical response after stating baclofen increased at week 2 (P = .008) and week 4 (P = .006). Novel rumination severity index scores improved at week 1 (P = .002), week 2 (P < .001), and week 4 (P < .001). Pediatric Quality of Life Inventory scores also improved at week 2 (P < .001) and week 4 (P < .001).

Investigators observed the proportion of patients who either reported no longer having symptoms or symptoms being only a little bothersome increased at week 2 (P = .004) and week 4 (P = .004). Patients on baclofen were 47% more likely to experience a clinical response and 70% more likely for symptoms to disappear o be only a little bothersome at week 4. Despite this finding, the team observed no statistically significant differences between the baclofen and placebo arms.

By the 4-week follow-up, 90% of the participants reported the use of behavioral skills for ruminations, with no significant difference between the number of people in the baclofen and placebo groups.

The study demonstrated that baclofen was safe, with reported adverse events of hiccups (n = 1), heartburn and decreased appetite (n = 1), and abdominal soreness (n = 1). Participants on placebo reported abdominal pain (n = 2), indigestion and rash (n = 1), decreased appetite (n = 1), fussiness and headache (n = 1). The patient in the placebo arm who reported fussiness and headache discontinued the study, and there was no discontinuation in the baclofen group.

“Although there was a greater numerical improvement in [rumination syndrome] symptoms and quality of life with baclofen versus placebo, this difference was not statistically significant,” investigators wrote. “Given the improvement we observed with baclofen and limited side effect profile, we recommend considering the addition of baclofen to behavioral treatment for children with [rumination syndrome].”

References

1. ^{Khoo, J, Kroon Van Diest, A, Sanchez, Raul, et al. A Randomized, Double-Blind, Placebo-Controlled Trial of Baclofen for Children with Rumination Syndrome.Presented at NASPHGAN 2024 in Hollywood, Florida, from November 7 – November 9, 2024.}
2. ^{Gupta SR, Lu PL, Vaz KH, Yacob D, Wall J, Van Diest AMK, Di Lorenzo C, Bali N. A Retrospective Review of Baclofen Treatment for Children with Rumination Syndrome at a Single Center. Paediatr Drugs. 2023 May;25(3):359-363. doi: 10.1007/s40272-023-00567-w. Epub 2023 Mar 21. PMID: 36943582.}