Peroral Endoscopic myotomy (POEM) in pediatric achalasia: a retrospective cohort on institutional experience and quality of life.

IF 3.5 2区 医学 Q2 GENETICS & HEREDITY Orphanet Journal of Rare Diseases Pub Date : 2025-01-25 DOI:10.1186/s13023-025-03565-y
Thijs Kuipers, Carlijn Mussies, Aaltje Lei, Gwen M C Masclee, Marc A Benninga, Paul Fockens, Barbara A J Bastiaansen, Albert J Bredenoord, Michiel P van Wijk
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Abstract

Background: Achalasia is a rare esophageal motility disorder with an estimated annual incidence of 1-5/100.000 and a mean age at diagnosis > 50 years of age. Only a fraction of the patients has an onset during childhood (estimated incidence of 0.1-0.18/ 100.000 children per year). No curative treatment is currently available. Peroral Endoscopic Myotomy (POEM) is a widely accepted treatment option to improve symptoms in adults. Studies evaluating safety and efficacy of POEM in children are scarce and no data exist regarding the quality of life in patients after POEM.

Methods: We evaluated the effectiveness and safety of POEM in a cohort of children that was treated for achalasia and we prospectively evaluated their quality of life. We compared the results to a previous cohort evaluating Pneumatic Dilation (PD) and Laparoscopic Heller's Myotomy (LHM) in children with achalasia.

Results: Thirty-three achalasia patients (age at time of POEM 14.1(± 2.5) years, 54.5% female) were included. Twenty-nine (87.8%) percent had received previous treatment (PD (n = 20); LHM (n = 1); PD + LHM (n = 7); PD + Botox (n = 1). POEM was technically successful in all patients and no major complications occurred. Mean follow-up duration was 33 (± 25) months. Twenty three (70%) patients did not need retreatment after POEM during the follow up period. Quality of life after POEM did not differ from the population norms. Patients with an Eckardt score > 3 had a significantly worse general (Kidscreen-52: physical score 44.7 vs. 52.4; p = 0.011; mental score: 42.5 vs. 51.3; p = 0.038) and disease specific (35 vs. 16; p = 0.017) quality of life compared to those with an Eckardt ≤ 3. The SF-36 mental health component score was significantly lower (44.2 vs. 53.1; p = 0.036) in patients treated with POEM compared to those treated with PD and LHM. These lower scores could be related to a selection bias, as more severe patients received POEM, and other influences such as the Corona pandemic. However, the overall, quality of life after POEM was not significantly different to PD and LHM.

Conclusion: POEM is an effective and safe treatment for achalasia in children. Quality of life after POEM is comparable to the results obtained after PD and Heller.

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经口内窥镜下肌切开术(POEM)治疗儿童失弛缓症:一项关于机构经验和生活质量的回顾性队列研究。
背景:贲门失弛缓症是一种罕见的食管运动障碍,估计年发病率为1-5/ 100,000,诊断时平均年龄为50岁。只有一小部分患者在儿童期发病(估计发病率为每年每10万名儿童0.1-0.18例)。目前尚无有效的治疗方法。经口内窥镜下肌切开术(POEM)是一种广泛接受的治疗选择,以改善成人的症状。评估POEM在儿童中的安全性和有效性的研究很少,也没有关于POEM后患者生活质量的数据。方法:我们评估了POEM在一组接受失弛缓症治疗的儿童中的有效性和安全性,并前瞻性地评估了他们的生活质量。我们将结果与先前评估气动扩张(PD)和腹腔镜Heller's肌切开术(LHM)治疗贲门失弛缓症儿童的队列进行比较。结果:纳入33例贲门失弛缓症患者(POEM时年龄14.1(±2.5)岁,女性54.5%)。29人(87.8%)曾接受过治疗(PD (n = 20);LHM (n = 1);PD + LHM (n = 7);PD +肉毒杆菌(n = 1)。POEM在技术上是成功的,没有发生重大并发症。平均随访时间33(±25)个月。随访期间,23例(70%)患者经POEM治疗后不需要再治疗。POEM后的生活质量与人口标准没有差异。Eckardt评分为bb0.3的患者总体评分明显较差(Kidscreen-52:身体评分44.7比52.4;p = 0.011;心理得分:42.5 vs. 51.3;P = 0.038)和疾病特异性(35 vs. 16;p = 0.017),与Eckardt≤3的患者相比,其生活质量明显降低。SF-36心理健康成分得分显著较低(44.2比53.1;p = 0.036),与PD和LHM治疗的患者相比。这些较低的分数可能与选择偏差有关,因为更严重的患者接受了POEM,以及其他影响,如冠状病毒大流行。然而,总体而言,POEM术后的生活质量与PD和LHM无显著差异。结论:POEM是一种安全有效的治疗小儿失弛缓症的方法。POEM治疗后的生活质量与PD和Heller治疗后的结果相当。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases 医学-医学:研究与实验
CiteScore
6.30
自引率
8.10%
发文量
418
审稿时长
4-8 weeks
期刊介绍: Orphanet Journal of Rare Diseases is an open access, peer-reviewed journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on clinical trial outcome reports, either positive or negative, and articles on public health issues in the field of rare diseases and orphan drugs. The journal does not accept case reports.
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