Endoscopic pyloromyotomy for the treatment of severe and refractory gastroparesis: a pilot, randomised, sham-controlled trial

Abstract

Objective: Endoscopic pyloromyotomy (G-POEM) is a minimally invasive treatment option with promising uncontrolled outcome results in patients with gastroparesis.

Design: In this prospective randomised trial, we compared G-POEM with a sham procedure in patients with severe gastroparesis. The primary outcome was the proportion of patients with treatment success (defined as a decrease in the Gastroparesis Cardinal Symptom Index (GCSI) by at least 50%) at 6 months. Patients randomised to the sham group with persistent symptoms were offered cross-over G-POEM.

Results: The enrolment was stopped after the interim analysis by the Data and Safety Monitoring Board prior to reaching the planned sample of 86 patients. A total of 41 patients (17 diabetic, 13 postsurgical, 11 idiopathic; 46% male) were randomised (21 G-POEM, 20-sham). Treatment success rate was 71% (95% CI 50 to 86) after G-POEM versus 22% (8-47) after sham (p=0.005). Treatment success in patients with diabetic, postsurgical and idiopathic gastroparesis was 89% (95% CI 56 to 98), 50% (18-82) and 67% (30-90) after G-POEM; the corresponding rates in the sham group were 17% (3-57), 29% (7-67) and 20% (3-67).Median gastric retention at 4 hours decreased from 22% (95% CI 17 to 31) to 12% (5-22) after G-POEM and did not change after sham: 26% (18-39) versus 24% (11-35). Twelve patients crossed over to G-POEM with 9 of them (75%) achieving treatment success.

Conclusion: In severe gastroparesis, G-POEM is superior to a sham procedure for improving both symptoms and gastric emptying 6 months after the procedure. These results are not entirely conclusive in patients with idiopathic and postsurgical aetiologies.

Trial registration number: NCT03356067; ClinicalTrials.gov.

Keywords: GASTRIC EMPTYING; GASTROPARESIS.

Conflict of interest statement

Competing interests: None declared.

© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1

Flowchart demonstrating screening, enrolment, randomisation, follow-up and cross-over procedure with the subsequent follow-up. Eligible patients from the two centres were randomly assigned to either G-POEM or the sham procedure consisting of endoscopic examination under general anaesthesia. The length of the follow-up was 6 months when the treatment allocation was revealed. A total of 12 patients, who did not have treatment success after the sham procedure and agreed with a cross-over endoscopic pyloromyotomy (G-POEM), underwent the procedure and were followed up for another 6 months. The intention to treat (ITT) analysis comprises 41 patients, the per protocol (PP) analysis 39 patients.

Figure 2

Treatment success at 6 months after the assigned procedure (main outcome), after the crossover G-POEM (A) and treatment success in sub-groups by aetiology of gastroparesis (B). The plot shows rates of treatment success with 95% CIs, where the clinical success is defined as reduction of the total Gastroparesis Cardinal Symptom Index (GCSI) score by at least 50% from the baseline. For the cross-over endoscopic pyloromyotomy (G-POEM), GCSI at 6 months after the sham procedure was considered as baseline. The results analysed on the intention to treat (ITT) population (N=41, N-Di-G-POEM=9, N-Di-Sham=8, N-PS-G-POEM=6, N-PS-Sham=7, N-Id-G-POEM=6, N-Id-Sham=5, 1 GCSI value (2 %) imputed in diabetic GP patient in the sham group) are supplemented by the main outcome analysis on the per protocol (PP) population (N=39).

Figure 3

Evolution of the Gastroparesis Cardinal Symptom Index (GCSI) total score. Point estimates of medians with 95% CIs calculated on the intention to treat (ITT) population are shown for patients after the endoscopic pyloromyotomy (G-POEM) procedure (green circles, N=21), sham procedure (blue triangles, N=20, imputed 1 value (5 %) for 3 months and 1 value (5 %) for 6 months), and cross-over G-POEM procedure (purple squares, N=12). For the cross-over G-POEM group, the value at 6 months reflects only the data for the patients in this group (who subsequently underwent the cross-over G-POEM procedure). The GCSI score may range from 0 (no symptoms) to 5 (maximally severe symptoms).

Figure 4

Evolution of gastric retention at 4 hours after meal ingestion on a standardised sulphur colloid solid-phase gastric emptying study (scintigraphy). Point estimates of medians with 95% CIs are shown for patients after the G-POEM procedure (green circles, N=21, imputed 2 values (10 %) for 3 months), sham procedure (blue triangles, N=20, imputed 1 value (5 %) for 3 months), and cross-over GPOEM procedure (purple squares, N=12). For the cross-over G-POEM group, the value at 3 months reflects only the data for the patients in this group (who subsequently underwent the cross-over G-POEM procedure). GES, gastric emptying study; G-POEM, endoscopic pyloromyotomy.