We identified 34 patients with achalasia (mean age at time of POEM 14.1 (2.5) years, 18 female (54.5%)) that underwent POEM before the age of 18 years at the AUMC between October 2015 and April 2022 (Table 1). One patient was later diagnosed with pseudo-achalasia based on a malignancy, this patient is excluded from the analysis. The mean age at achalasia diagnosis was 12.09 (2.69) years. Patients reported a mean Eckardt score of 6.29 (2.05) at time of diagnosis, all Eckardt scores were ≥ 3. HRM data was available in 32 (97.0%) of 33 patients at time of diagnosis and showed a median integrated relaxation pressure (IRP-4) of 36.4 (26.0-50.4) mmHg (normal < 15mmHg). Based on HRM and according to the Chicago classification of Esophageal Motility Disorders (version 3.0) the following achalasia subtypes were seen; 12 (36.4%) patients had subtype I, 19 (57.6%) patients had subtype II, one (3.0%) patient had subtype III achalasia and in one (3.0%) patient the type of achalasia was unknown. Twenty-nine of 33 patients (87.8%) had received a different treatment prior to POEM. This was PD (20 [60.6%] of 33), LHM (1 [3.0%] of 33) PD + LHM (7 [21.2%] of 33) or PD + Botox (1 [3.0%] of 33). The time between diagnosis and POEM was 12 (6–26) months.

The POEM procedure was technically successful in all patients. In 27 [81.8%] of 33 patients a myotomy of the anterior side was done and 6 [18.2%] patients received a posterior myotomy. The mean length of the myotomy was 9.38 (2.08) cm and the median length of myotomy extended in the stomach was 3 [2, 3, 4] cm. The median duration time of the procedure was 52 (42–61) minutes. The median hospital stay was one (1–1) night.

No major complications occurred during the POEM procedure or in the thirty days thereafter. Four intra-procedural events occurred. Three (9.1%) patients had minor intra-procedural bleedings that could be resolved with coagulation during the procedure. In one (3.0%) patient a small mucosal tear occurred that could be closed with one clip during the procedure.

The mean follow-up duration of all patients was 33 (25) months. During follow-up 23 [69.7%] of 33 patients did not need retreatment and 3 (9.1%) patients were lost to follow-up. A log-rank survival curve for the proportion of patients that did not need retreatment is shown in Fig. 1. Of the seven patients that needed retreatment, four (57.1%) were treated with PD only and received the first dilation 6, 8, 10 and 12 months after POEM. One [14.3%] patient received PD 3 months and LHM 13 months after POEM. One [14.3%] patient received a re-POEM 42 months after the initial POEM. And one [14.3%] patient received a re-POEM at 18 months followed by PD 29 months after the first POEM.

Log-rank survival curves for the proportion of patients that did not need retreatment

At 3 months after POEM a follow-up visit was completed in 28 (84.8%) of 33 patients. 27 patients (96.4%) did not need additional treatment. Five (17.9%) of these patients reported an Eckardt score > 3. One patient was retreated with PD within 3 months after treatment with POEM. Endoscopy was performed in 10 (30.3%) of 33 patients off PPI and showed LA grade A esophagitis in one (10.0%) patient and LA grade B esophagitis in 2 (20%) patients.

Follow-up data at 1-year after treatment with POEM was available in 21 (77.8%) of 27 patients. In the other six of the 33 patients included in the trial POEM was performed less than one year ago. 18 (85.7%) of these 21 patients did not need additional treatment after POEM. Five (23.8%) patients reported an Eckardt score > 3, three of these of them patients received treatment with PD between 3 months and 1 year after POEM, one of these patients needed a second dilation series.

In total 29 (87.9%) of 33 patients were included for additional follow-up questionnaires and interview. Despite multiple attempts, the other 4 patients could not be reached. At time of additional follow-up, patients had a mean current age of 16.66 (2.79) years at time of follow-up. The mean Eckardt score was 2.43 (1.75); 13 (44.8%) of 29 patients reported an Eckardt score > 3. An overview of the Eckardt subscores can be found in Table 2. At the time of additional follow-up 6 (18.2%) patients reported a GERDQ of 8 or higher, 12 (26.4%) patients were using PPI daily. Two (6.9%) of 29 indicated coughing.

Fourteen (77.8%) out of 18 patients younger than 18 years of age completed the Kidscreen-52 and disease specific QoL (DSQoL). On the Kidscreen-52 our cohort did not score significantly different on any domain compared to the population norms, except for the ‘bullying’ domain where achalasia treated patients scored better (i.e. were bullied less often, p = 0.013). An overview of the scores on the different subdomains can be found in supplement 1. A total median pediatric achalasia-specific DSQoL score of 21 (14.8–26.3) was reported. The subdomains swallowing problems, friends and family and feelings scored 8 (4.8–16), 5.5 (1.8–11.3) and 7.5 (1.8–12) respectively.

Six (54.5%) of the 11 patients older than 18 years old at time of the additional follow-up completed the achalasia-specific HRQoL with a median score of 17 (12.8–22.5). The SF-36 showed a mental component score of 44.2 (35.8–51.0) and physical component score of 51.9 (41.2–58.3).

We performed a post-hoc analysis where we created multiple subgroups. First the cohort was divided based on Eckardt score; one group had an Eckardt score ≤ 3 and the other group an Eckardt score of > 3 at the additional follow-up.

In the patients who were younger than 18 years of age at the time of the additional follow-up and had an Eckardt score > 3, we found a significantly lower score on the Kidscreen-52 physical well-being subscore (44.7 (32.7–47.1) vs. 52.4 (47.1–59.4) p = 0.011) and moods and emotions subscore (42.5 (37.8–49.1) vs. 51.3 (47.2–54.0) p = 0.038) compared to the group of children with an Eckardt score ≤ 3, Patients younger than 18 years old with an Eckardt score above three also scored significantly higher on the total achalasia-specific DSQoL score (indicating more symptoms) (35 (17–46) vs. 16 [8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21] p = 0.017) and on the friends and family subdomain (11 [7, 8, 9, 10, 11, 12, 13, 14, 15, 16] vs. 3 [1, 2, 3, 4, 5, 6] p = 0.017) compared to patients with an Eckardt score below three.

In patients older than 18 years of age we did not find a significant difference on the SF-36 mental component score (47.7 (40.7–51.6) vs. 38.4 (28.0-50.9), p = 1.000) and physical component score (53.0 (41.4–58.5) vs. 50.8 (40.8–58.2), p = 0.700) or the achalasia-specific HRQoL score (22 (18–24) vs. 13 [12, 13, 14, 15, 16], p = 0.100) comparing patients with an Eckardt score of > 3 and patients with an Eckardt score below three.

Additionally, we performed a post hoc analysis where the cohort was divided in a subgroup of patients that were retreated after POEM and patients that did not receive retreatment. This did not result in a significant difference in quality of life on all questionnaires between both subgroups. Furthermore, we divided the cohort is a subgroup who were primarily treated with POEM and patients who had POEM after PD and/or LHM. We did not find a difference in quality of life.

We compared our results to a previous study evaluating the pediatric achalasia population in our center. This study consisted of 87 patients (mean age 11.4 (3.4), 60% male) diagnosed with achalasia < 18 years old between 1990 and 2013. Initial treatment was PD in 79% and LHM in 21% patients. Retreatment was necessary more often after PD compared to LHM (88% vs. 22%). A full overview of the demographics can be found in the original publication [1]. The percentage of patients that participated in the additional follow-up (83% vs. 78%) and the number of patients with an Eckardt score of > 3 (44.5% vs. 45%) was comparable in the previous cohort vs. this study.

Patients younger than 18 years at the time of the additional follow-up completed the Kidscreen and achalasia-specific DSQoL questionnaires. When comparing the Kidscreen domains in POEM treated patients with patients treated by PD or LHM, we noticed that POEM treated patients scored significantly worse on ‘self-perception’ (44.6 (40.2–53.0) vs. 53.2 (48.7–60.5) p = 0.036) and ‘school’ (49.6 (44.6–54.8) vs. 56.4 (50.8–61.9), p = 0.018). The total pediatric achalasia-specific DSQoL score did not significantly differ between the two cohorts (21 (14.8–36.3) vs. 17.5 (8–29), p = 0.341). Figure 2.

Health related quality of life after PD, LHM and POEM in children

Patients of 18 years and older at the time of the additional follow-up completed the SF-36 and achalasia-specific HRQoL questionnaires. When comparing the scores of these questionnaires to the previous cohort of patients treated with PD or LHM we did not see a significant difference in achalasia-specific HRQoL (17 (12.8–22.5) vs. 19 [15, 16, 17, 18, 19, 20, 21, 22], p = 0.713) and SF-36 physical component score (51.9 (41.2–58.3) vs. 53 (49.1–57.0), p = 0.849) between the current and the previous cohort (Fig. 3). However the SF-36 mental component score (44.2 (35.8–51.0) vs. 53.1 (47.7–57.5), p = 0.036) was significantly lower in patients treated with POEM compared to patients treated with PD and LHM.

Health related quality of life after PD, LHM and POEM in adults