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Interpreting evidence on percutaneous endoscopic gastrostomy versus laparoscopic gastrostomy in children. 儿童经皮内镜胃造口术与腹腔镜胃造口术的证据解释。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70410
Raanan Shamir, Hania Szajewska
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引用次数: 0
Stricture classification of pediatric esophageal strictures (SCOPES): A novel tool to predict response to endoscopic therapy. 儿童食管狭窄分类(SCOPES):一种预测内镜治疗反应的新工具。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70403
Brandon T Oby, Steven J Staffa, Peter D Ngo, Denis Chang, Michael A Manfredi, Jessica L Yasuda

Objectives: Esophageal anastomotic stricture is a common complication following esophageal atresia (EA) repair. While multiple endoscopic therapies exist, predicting individual stricture response remains challenging. Existing classification systems focus primarily on luminal diameter and do not account for complex physical characteristics of the stricture. We developed and evaluated a novel endoscopic scoring tool, stricture classification of pediatric esophageal strictures (SCOPES), to determine if physical features are predictive of treatment response. In addition to its predictive utility, SCOPES aims to standardize the description of and improve communication of stricture morphology across providers and institutions.

Methods: A retrospective cohort study was conducted on EA patients treated at a tertiary referral center between 2019 and 2024. Patients with ≥2 endoscopies and documented SCOPES scores were included. The SCOPES tool categorizes strictures based on diameter, length, scar band intrusion, symmetry, and presence of diverticula. Multivariable mixed-effects regression models were used to analyze associations between SCOPES characteristics and the primary outcome of number of therapeutic endoscopies within 6 months.

Results: Seventy patients (238 endoscopies) met inclusion criteria. In multivariable analysis, symmetric strictures and those with highly intrusive scar bands were significantly associated with a greater number of therapeutic endoscopies within 6 months. Two complications were observed, both managed without long-term sequelae.

Conclusions: The SCOPES classification tool identifies physical stricture features that significantly influence response to endoscopic therapy. Circumferential symmetry and scar tissue protuberance were associated with higher treatment burden, suggesting these characteristics may guide therapeutic decision-making. SCOPES may aid in individualizing endoscopic management of pediatric esophageal strictures and warrants prospective validation.

目的:食管吻合口狭窄是食管闭锁修复术后常见的并发症。虽然存在多种内窥镜治疗方法,但预测个体狭窄反应仍然具有挑战性。现有的分类系统主要集中在管径上,而没有考虑到狭窄的复杂物理特征。我们开发并评估了一种新的内镜评分工具——儿童食管狭窄分类(SCOPES),以确定身体特征是否能预测治疗反应。除了预测功能外,scope还旨在标准化结构形态的描述并改善提供者和机构之间的沟通。方法:对2019 - 2024年在某三级转诊中心就诊的EA患者进行回顾性队列研究。患者≥2次内窥镜检查和记录的SCOPES评分。scope工具根据直径、长度、瘢痕带侵入、对称性和憩室的存在对狭窄进行分类。采用多变量混合效应回归模型分析SCOPES特征与6个月内治疗性内窥镜检查次数主要结局之间的关系。结果:70例患者(238例)符合纳入标准。在多变量分析中,对称狭窄和高度侵入性瘢痕带与6个月内更多的治疗性内窥镜检查显著相关。观察到两例并发症,均无长期后遗症。结论:SCOPES分类工具识别了对内镜治疗反应有显著影响的物理结构特征。圆周对称和疤痕组织突出与较高的治疗负担相关,提示这些特征可能指导治疗决策。SCOPES可能有助于儿童食管狭窄的个体化内镜治疗,值得前瞻性验证。
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引用次数: 0
Pressure-flow analysis following peroral endoscopic myotomy in pediatric achalasia: Novel insights into esophageal emptying. 儿童贲门失弛缓症经口内窥镜肌切开术后的压力-流量分析:对食管排空的新见解。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70414
Renato Tambucci, Giulia Chiarazzo, Nathalie Rommel, Chiara Imondi, Tamara Caldaro, Paola De Angelis, Valerio Balassone

Objective: Achalasia is a rare esophageal motility disorder in children. Peroral endoscopic myotomy (POEM) has emerged as a promising treatment yet standardized post-treatment evaluation remains lacking. While high-resolution manometry (HRM) is the diagnostic gold standard, its role post-treatment is limited. Pressure-flow analysis (PFA), derived from high-resolution impedance manometry (HRIM), offers a novel approach to assessing bolus transport and esophageal emptying. We aimed to evaluate PFA metrics in pediatric achalasia patients before and after POEM and assess its utility in monitoring post-treatment outcomes.

Methods: This retrospective study included 13 pediatric patients (8 males; mean age 13.3 ± 3.38 years) with achalasia treated with POEM at a tertiary referral center between 2016 and 2024. Inclusion required both pre- and post-POEM HRIM. HRM metrics were analyzed using Chicago Classification version 4.0 (CCv4.0), while PFA parameters, including impedance ratio (IR) and distension pressure during emptying (DPE), were calculated using the Swallow Gateway™ platform.

Results: All patients experienced symptomatic improvement, with the median Eckardt Symptom Score (ESS) significantly decreasing from 6 (range 2-10) to 0 (range 0-2; p = 0.0002). Following POEM, there was a significant reduction in median integrated relaxation pressure (IRP4s: 35.6-15.8 mmHg; p = 0.0002), although 7 out of 13 patients (53.8%) achieved IRP4s values below the upper limit of normal. Additionally, significant decreases were observed in median IR (0.65-0.52, p = 0.0002) and median DPE (28.9-17.3 mmHg, p = 0.0171). Subgroup analysis showed that improvements in IR and DPE occurred regardless of IRP4s normalization.

Conclusion: PFA enhances post-POEM evaluation in pediatric achalasia, offering an objective assessment of esophageal emptying beyond conventional pressure metrics. This study supports the potential role of PFA in post-treatment follow-up, warranting further validation in larger cohorts.

目的:贲门失弛缓症是一种罕见的儿童食管运动障碍。经口内窥镜下肌切开术(POEM)已成为一种很有前景的治疗方法,但标准化的治疗后评估仍然缺乏。虽然高分辨率测压(HRM)是诊断的金标准,但它在后处理中的作用有限。压力流分析(PFA)源于高分辨率阻抗测压(hrm),提供了一种评估大剂量输送和食管排空的新方法。我们的目的是评估儿童贲门失弛缓症患者在POEM前后的PFA指标,并评估其在监测治疗后结果中的效用。方法:本回顾性研究纳入2016年至2024年在三级转诊中心接受POEM治疗的13例贲门失弛缓症患儿(男性8例,平均年龄13.3±3.38岁)。包括诗前和诗后的hrm。使用Chicago Classification version 4.0 (CCv4.0)分析HRM指标,使用Swallow Gateway™平台计算PFA参数,包括阻抗比(IR)和排空过程中的膨胀压力(DPE)。结果:所有患者均出现症状改善,Eckardt症状评分(ESS)中位数从6(范围2-10)显著降低至0(范围0-2;p = 0.0002)。POEM治疗后,中位综合松弛压(IRP4s: 35.6-15.8 mmHg; p = 0.0002)显著降低,尽管13例患者中有7例(53.8%)的IRP4s值低于正常上限。此外,中位IR (0.65-0.52, p = 0.0002)和中位DPE (28.9-17.3 mmHg, p = 0.0171)显著降低。亚组分析显示,无论IRP4s是否正常化,IR和DPE都有所改善。结论:PFA增强了儿童贲门失弛缓症的poem后评估,提供了比传统压力指标更客观的食管排空评估。本研究支持PFA在治疗后随访中的潜在作用,需要在更大的队列中进一步验证。
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引用次数: 0
Interpreting evidence in pediatric gastrostomy: A continuing dialogue. 儿科胃造口术的证据解读:持续对话。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70409
Fabiola Cassaro, Pietro Impellizzeri, Carmelo Romeo, Salvatore Arena
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引用次数: 0
Long non-coding RNA-MIR3142HG as a novel biomarker for assessing disease activity in children with Crohn's disease. 长链非编码RNA-MIR3142HG作为评估克罗恩病儿童疾病活动性的新生物标志物
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70404
Xue Qiao, Weiying Zhang, Wenxin Chen, Min Yang, Jin Yang, Hui Yang

Objectives: This study aimed to investigate the potential of long non-coding RNAs (lncRNA)-MIR3142HG as a novel biomarker for assessing disease activity in pediatric Crohn's disease (CD).

Methods: A total of 119 children suspected of having CD who visited the outpatient department of the affiliated Children's Hospital of Nanjing Medical University from September 2022 to March 2025 and were classified into CD and non-CD groups based on clinical diagnosis. Serum and intestinal mucosal samples were collected, and lncRNA-MIR3142HG expression was quantified using real-time quantitative reverse transcription polymerase chain reaction (RT-qPCR). Clinical activity and endoscopic activity were similarly assessed using the pediatric Crohn's disease activity index (PCDAI) and simplified endoscopic score for CD (SES-CD). Correlations between lncRNA-MIR3142HG levels and clinical indices, including Chiu's histopathological score and Occludin protein expression, were analyzed, with receiver operating characteristic curve area under the curve values calculated for evaluation efficacy.

Results: LncRNA-MIR3142HG expression was significantly elevated in the CD group compared to non-CD and control groups (p < 0.05). Intestinal mucosal lncRNA-MIR3142HG was positively correlated with PCDAI, SES-CD, and Chiu's scores (p < 0.05) and negatively with Occludin expression (p < 0.05). Serum lncRNA-MIR3142HG also showed positive correlations with PCDAI and Chiu's scores (p < 0.05) and a negative correlation with Occludin (p < 0.05).

Conclusions: This study indicated that both serum and intestinal mucosal lncRNA-MIR3142HG, alone or combined with fecal calprotectin, may be useful for evaluating clinical and endoscopic activity in pediatric CD, with combined assessments yielding superior efficacy.

目的:本研究旨在探讨长链非编码rna (lncRNA)-MIR3142HG作为评估儿童克罗恩病(CD)疾病活动性的新生物标志物的潜力。方法:选取南京医科大学附属儿童医院于2022年9月至2025年3月门诊就诊的119例疑似CD患儿,根据临床诊断分为CD组和非CD组。采集血清和肠黏膜标本,采用实时定量逆转录聚合酶链反应(RT-qPCR)定量lncRNA-MIR3142HG的表达。临床活动性和内镜活动性同样采用儿童克罗恩病活动性指数(PCDAI)和简化CD内镜评分(SES-CD)进行评估。分析lncRNA-MIR3142HG水平与Chiu’s组织病理学评分、Occludin蛋白表达等临床指标的相关性,计算曲线值下的受试者工作特征曲线面积,评价疗效。结果:与非CD组和对照组相比,LncRNA-MIR3142HG在CD组中的表达显著升高(p)。结论:本研究表明,血清和肠黏膜LncRNA-MIR3142HG单独或与粪便钙保护蛋白联合,可能有助于评估儿童CD的临床和内镜活性,联合评估效果更好。
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引用次数: 0
Autoimmune gastritis in children: A prospective cohort with serologic screening in at-risk patients. 儿童自身免疫性胃炎:对高危患者进行血清学筛查的前瞻性队列研究
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70408
Andrea Chiaro, Flavia Parrinello, Camilla Marazzi, Alice Zucconi, Marta Bassi, Maria Grazia Calevo, Giampaola Pesce, Nicola Minuto, Roberto Gastaldi, Maurizio Miano, Paolo Gandullia

Objectives: Autoimmune gastritis (AIG) is a chronic immune-mediated condition characterized by the destruction of the gastric oxyntic mucosa. Though commonly diagnosed in adults, its occurrence in pediatric patients with autoimmune comorbidities is increasingly reported. This study aims to determine the prevalence of AIG in children with Type 1 diabetes, autoimmune thyroiditis, primary immune regulatory disorders, or refractory anemia (RA), and to assess the diagnostic performance of anti-parietal cell antibodies (APCAs) and anti-intrinsic factor antibodies (AIFAs) in this group of patients.

Methods: We conducted a prospective study in 203 children aged 4-17 years. All underwent laboratory screening for APCAs and AIFAs. Those who tested positive for at least one of the two antibodies were referred for upper endoscopy with gastric biopsies for histological confirmation. A control group of 26 patients with dyspeptic symptoms and negative histology was also evaluated.

Results: Nineteen patients tested positive for APCAs and/or AIFAs (9.4%). Among them, nine (47.4%) had histologically confirmed AIG. Additionally, two patients with RA and negative serology were diagnosed with AIG based on histology. Thus, a total of 11 patients (5.4%) were diagnosed with AIG. The sensitivity and specificity of antibody testing for AIG were 81.8% and 77.9%, respectively.

Conclusions: AIG may be underdiagnosed in children with autoimmune conditions. Our findings support the integration of serological screening with histological assessment to enhance early recognition. The combined use of APCAs and AIFAs demonstrates good diagnostic accuracy, offering a valuable tool for timely diagnosis and improved management in this at-risk pediatric population.

目的:自身免疫性胃炎(AIG)是一种以胃氧合粘膜破坏为特征的慢性免疫介导疾病。虽然常见于成人,但其在患有自身免疫性合并症的儿科患者中的发生率越来越多。本研究旨在确定患有1型糖尿病、自身免疫性甲状腺炎、原发性免疫调节障碍或难治性贫血(RA)的儿童AIG的患病率,并评估抗壁细胞抗体(APCAs)和抗内因子抗体(AIFAs)在这组患者中的诊断效果。方法:我们对203名4-17岁儿童进行了前瞻性研究。所有患者均接受了apca和aifa的实验室筛查。对两种抗体中至少一种检测呈阳性的患者进行上胃镜检查和胃活检以进行组织学证实。对照组26例有消化不良症状且组织学阴性的患者也进行了评估。结果:19例患者APCAs和/或AIFAs检测呈阳性(9.4%)。其中9例(47.4%)经组织学证实为AIG。另外,2例RA血清阴性患者经组织学诊断为AIG。因此,共有11例(5.4%)患者被诊断为AIG。抗体检测对AIG的敏感性为81.8%,特异性为77.9%。结论:患有自身免疫性疾病的儿童AIG可能未被充分诊断。我们的研究结果支持血清学筛查与组织学评估的结合,以提高早期识别。联合使用APCAs和AIFAs显示出良好的诊断准确性,为及时诊断和改善这一高危儿科人群的管理提供了有价值的工具。
{"title":"Autoimmune gastritis in children: A prospective cohort with serologic screening in at-risk patients.","authors":"Andrea Chiaro, Flavia Parrinello, Camilla Marazzi, Alice Zucconi, Marta Bassi, Maria Grazia Calevo, Giampaola Pesce, Nicola Minuto, Roberto Gastaldi, Maurizio Miano, Paolo Gandullia","doi":"10.1002/jpn3.70408","DOIUrl":"https://doi.org/10.1002/jpn3.70408","url":null,"abstract":"<p><strong>Objectives: </strong>Autoimmune gastritis (AIG) is a chronic immune-mediated condition characterized by the destruction of the gastric oxyntic mucosa. Though commonly diagnosed in adults, its occurrence in pediatric patients with autoimmune comorbidities is increasingly reported. This study aims to determine the prevalence of AIG in children with Type 1 diabetes, autoimmune thyroiditis, primary immune regulatory disorders, or refractory anemia (RA), and to assess the diagnostic performance of anti-parietal cell antibodies (APCAs) and anti-intrinsic factor antibodies (AIFAs) in this group of patients.</p><p><strong>Methods: </strong>We conducted a prospective study in 203 children aged 4-17 years. All underwent laboratory screening for APCAs and AIFAs. Those who tested positive for at least one of the two antibodies were referred for upper endoscopy with gastric biopsies for histological confirmation. A control group of 26 patients with dyspeptic symptoms and negative histology was also evaluated.</p><p><strong>Results: </strong>Nineteen patients tested positive for APCAs and/or AIFAs (9.4%). Among them, nine (47.4%) had histologically confirmed AIG. Additionally, two patients with RA and negative serology were diagnosed with AIG based on histology. Thus, a total of 11 patients (5.4%) were diagnosed with AIG. The sensitivity and specificity of antibody testing for AIG were 81.8% and 77.9%, respectively.</p><p><strong>Conclusions: </strong>AIG may be underdiagnosed in children with autoimmune conditions. Our findings support the integration of serological screening with histological assessment to enhance early recognition. The combined use of APCAs and AIFAs demonstrates good diagnostic accuracy, offering a valuable tool for timely diagnosis and improved management in this at-risk pediatric population.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147499065","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Antibiotics in the first week of life are not associated with functional gastrointestinal disorders at 9-12 years of age. 生命第一周使用抗生素与9-12岁时的功能性胃肠道疾病无关。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-23 DOI: 10.1002/jpn3.70402
Nora C Carpay, Kim Kamphorst, Ruurd M van Elburg, Arine M Vlieger

Objectives: Dysbiosis due to early-life antibiotics may contribute to the development of functional gastrointestinal disorders (FGIDs). This follow-up study of a birth cohort primarily investigates the association between antibiotic treatment in the first week of life and the presence of FGIDs at 9-12 years. Secondarily, it examines whether a history of infantile colic or current food allergy is associated with FGIDs.

Methods: A prospective observational birth cohort of 436 term-born infants was followed up at the age of 9-12 years; 151 received intravenous antibiotics in the first week of life due to suspected early onset sepsis (AB+) and 285 did not (AB-). Participants filled out questionnaires on the presence of FGIDs (Rome IV questionnaire) and food allergies, and FGID diagnoses were reported by general practitioners. Statistical analyses included chi-squared tests and multivariable logistic regression.

Results: 306 of 388 eligible participants (79.5%) participated in the follow-up study: 109 (35.6%) AB+ and 197 (64.4%) AB-. FGID prevalence at 9-12 years was similar in AB+ and AB- (any FGID: odds ratio [OR] 1.083, 95% confidence interval [CI] 0.608-1.932). Infantile colic was not significantly associated with FAPDs after adjusting for confounders (adjusted OR 2.007, 95% CI 0.978-4.003, p = 0.051). Children with a food allergy were more likely to have a functional abdominal pain disorder (FAPD) (adjusted OR 4.028, 95% CI 1.532-10.286).

Conclusions: No statistically significant association was observed between first-week antibiotics or infantile colic and FGIDs at 9-12 years of age, but FAPDs were significantly more prevalent in children with food allergies.

目的:早期抗生素导致的生态失调可能导致功能性胃肠道疾病(fgid)的发展。这项对出生队列的随访研究主要调查了出生后第一周抗生素治疗与9-12岁时fgid存在之间的关系。其次,它检查婴儿绞痛史或当前的食物过敏是否与fgid有关。方法:对436例9-12岁足月新生儿进行前瞻性观察性随访;151例因怀疑早发性脓毒症(AB+)在出生后第一周接受静脉注射抗生素治疗,285例未接受静脉注射抗生素治疗(AB-)。参与者填写关于FGID存在的调查问卷(罗马IV调查问卷)和食物过敏,FGID诊断由全科医生报告。统计分析包括卡方检验和多变量logistic回归。结果:388名符合条件的参与者中有306人(79.5%)参加了随访研究,其中109人(35.6%)为AB+, 197人(64.4%)为AB-。9-12岁时,AB+和AB-的FGID患病率相似(任何FGID:优势比[OR] 1.083, 95%可信区间[CI] 0.608-1.932)。调整混杂因素后,婴儿绞痛与FAPDs无显著相关性(调整OR 2.007, 95% CI 0.978-4.003, p = 0.051)。食物过敏的儿童更有可能出现功能性腹痛障碍(FAPD)(校正OR 4.028, 95% CI 1.532-10.286)。结论:第一周抗生素或婴儿绞痛与9-12岁fgid之间没有统计学意义上的关联,但FAPDs在食物过敏儿童中更为普遍。
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引用次数: 0
Children and adolescents with disorders of gut-brain interaction with comorbid hypermobility and orthostatic intolerance have worse outcomes. 儿童期和青少年期肠-脑相互作用障碍合并多动和直立性不耐受的结果更差。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-19 DOI: 10.1002/jpn3.70417
Neha Santucci, Austin VonAxelson, Jesse Li, Kahleb Graham, Jennifer Hardy, Megan Miller, Rashmi Sahay

Objectives: Disorders of gut-brain interaction (DGBI) affect about 40% of children and are often comorbid with hypermobility spectrum disorders (HSDs) and orthostatic intolerance (OI). However, how these comorbidities impact outcomes in pediatric DGBI is not well understood. This study aimed to compare outcomes in DGBI patients with HSD, OI, both, or neither.

Methods: We reviewed records of patients aged 9-21 years from a multidisciplinary DGBI clinic. Patients met Rome IV criteria for DGBI and had documented HSD and/or OI diagnoses from specialists in gastroenterology, rheumatology, genetics, cardiology, adolescent medicine, and others. HSD terms included Ehlers-Danlos syndrome, hypermobile Ehlers-Danlos syndrome, and generalized hypermobility; OI terms included postural orthostatic tachycardia syndrome, dysautonomia, and orthostatic hypotension. Clinical data included the following validated questionnaires: abdominal pain index, nausea severity scale, functional disability inventory, patient health questionnaire-9 (Depression), children somatization inventory, pediatric insomnia severity index, pain catastrophizing scale for children, and screen for child anxiety related disorders (Anxiety). We compared DGBI patients with both HSD and OI, those with either disorder, and those without.

Results: Of 175 patients, 46% had HSD and 43% had OI. Patients with both HSD and OI had significantly worse nausea, depression, disability, and somatization scores than others (p < 0.01). HSD and OI groups individually also showed worse outcomes than non-HSD/non-OI groups. Moderate correlations were found between depression and anxiety in OI and nausea and disability in HSD.

Conclusions: Comorbid HSD and OI worsen DGBI symptoms. Accurate diagnosis and treatment are critical to improving outcomes due to shared autonomic dysfunction.

目的:肠脑相互作用障碍(DGBI)影响约40%的儿童,通常与多动谱障碍(HSDs)和直立性不耐受(OI)合并症。然而,这些合并症如何影响儿童DGBI的预后尚不清楚。本研究的目的是比较DGBI患者合并HSD、OI、两者都有或两者都没有的结果。方法:我们回顾了来自多学科DGBI诊所的9-21岁患者的记录。患者符合DGBI的Rome IV标准,并经胃肠病学、风湿病学、遗传学、心脏病学、青少年医学等专家诊断为HSD和/或OI。HSD术语包括ehers - danlos综合征、过度活动型ehers - danlos综合征和广泛性过度活动症;成骨不全术语包括体位性心动过速综合征、自主神经异常和体位性低血压。临床资料包括以下有效问卷:腹痛指数、恶心严重程度量表、功能障碍量表、患者健康问卷-9(抑郁)、儿童躯体化量表、儿童失眠严重程度指数、儿童疼痛灾难化量表、儿童焦虑相关障碍筛查(焦虑症)。我们比较了同时伴有HSD和OI的DGBI患者,有任何一种疾病的患者和没有这种疾病的患者。结果:175例患者中,46%为HSD, 43%为成骨不全。同时患有HSD和OI的患者的恶心、抑郁、残疾和躯体化评分明显高于其他患者(p)。准确的诊断和治疗对于改善共享自主神经功能障碍的预后至关重要。
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引用次数: 0
Neurodevelopmental assessment at 24 months in infants with esophageal atresia: A prospective cohort study. 食道闭锁婴儿24个月时神经发育评估:一项前瞻性队列研究。
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-19 DOI: 10.1002/jpn3.70406
Julia Tagmouti, Manon Midavaine, Victor Sartorius, Nicolas Vinit, Elise Leroy-Terquem, Sylvie Beaudoin, Elsa Kermorvant-Duchemin, Véronique Rousseau, Alexandre Lapillonne

Objective: To evaluate neurodevelopment at 24 months in infants surgically treated for esophageal atresia (EA), using the ages and stages questionnaire (ASQ), and identify perinatal and early-life factors associated with developmental delay.

Methods: Infants with EA were prospectively enrolled in a structured multidisciplinary follow-up program. Total ASQ scores ≤185 were considered indicative of significant neurodevelopmental delay, while scores >220 reflected reassuring neurodevelopment. Five skill domains (communication, gross motor skills, fine motor skills, problem solving, and social skills) were also assessed. Clinical, surgical, and growth data were analyzed to identify associated risk factors.

Results: Of the 74 infants included, 13 patients (18%) had an ASQ score ≤185. Lower ASQ scores were significantly associated with prematurity, prolonged hospitalization, multiple surgeries, poor weight gain at 1 and 2 years, and feeding disorders. After adjusting for gestational age and genetic anomalies, prolonged hospitalization remained associated with lower ASQ scores, and higher weight-for-age at 1 and 2 years with better ASQ scores. Communication and social skills were the most severely affected, with approximately 80% of infants in the ASQ ≤ 185 group scoring below -2 standard deviations.

Conclusion: Although most children with EA demonstrated normal development at 2 years, a minority experienced significant delays, especially in communication and social skills. Early screening and targeted multidisciplinary follow-up are essential to support optimal outcomes in this high-risk population.

目的:采用年龄和分期问卷(ASQ)评估手术治疗食管闭锁(EA)婴儿24月龄时的神经发育,并确定与发育迟缓相关的围产期和早期生活因素。方法:对患有EA的婴儿进行前瞻性的多学科随访研究。ASQ总分≤185分被认为是神经发育明显迟缓的指标,而总分>220分则是神经发育稳定的指标。五个技能领域(沟通、大动作技能、精细动作技能、问题解决能力和社交技能)也被评估。分析临床、手术和生长数据以确定相关的危险因素。结果:纳入的74例婴儿中,13例(18%)ASQ评分≤185。较低的ASQ评分与早产、住院时间延长、多次手术、1岁和2岁时体重增加不佳以及喂养障碍显著相关。在调整胎龄和遗传异常后,延长住院时间仍与较低的ASQ评分相关,1岁和2岁时较高的年龄体重与较好的ASQ评分相关。沟通和社交技能受到的影响最为严重,ASQ≤185组中约80%的婴儿得分低于-2个标准差。结论:虽然大多数EA儿童在2岁时表现出正常的发育,但少数儿童表现出明显的延迟,特别是在沟通和社交技能方面。早期筛查和有针对性的多学科随访对于支持这一高危人群的最佳结果至关重要。
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引用次数: 0
Quality of life for pediatric patients with short bowel syndrome-associated intestinal failure treated with teduglutide. 特杜葡肽治疗短肠综合征相关肠衰竭患儿的生活质量
IF 2.6 3区 医学 Q3 GASTROENTEROLOGY & HEPATOLOGY Pub Date : 2026-03-19 DOI: 10.1002/jpn3.70358
Danielle Wendel, Paul W Wales, Samuel Kocoshis, Robert Venick, Beth Carter, Valeria Cohran, Cheney Matthews, Bram P Raphael, Ian Robinson, Susan Hill

Objectives: The complex care needs of pediatric patients with short bowel syndrome-associated intestinal failure (SBS-IF) can negatively impact the health-related quality of life (HRQoL) of patients and their caregivers. We assessed the impact of teduglutide on HRQoL of pediatric patients with SBS-IF.

Methods: Two long-term extension (LTE) studies (NCT02949362, NCT02954458) assessed HRQoL over 96 weeks using the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales and the PedsQL Family Impact Module. Patients included in this analysis received teduglutide in one of three randomized Phase 3 parent studies and the respective LTE study. We assessed changes in HRQoL over time, as well as the impact of attaining enteral autonomy on HRQoL and predictors of change in HRQoL.

Results: In pediatric patients with SBS-IF receiving teduglutide (n = 69), the total mean (standard deviation) patient-reported PedsQL score was 75.0 (16.2). Higher mean number of stools per day at LTE study baseline (p = 0.03) predicted increased patient-reported HRQoL. Longer teduglutide exposure (p = 0.047), increasing length of remnant small intestine (p = 0.034), and older age (p = 0.026) predicted higher caregiver-proxy-reported HRQoL. Mean PedsQL scores remained stable over 96 weeks regardless of patients achieving enteral autonomy.

Conclusions: HRQoL of pediatric patients with SBS-IF treated with teduglutide was generally stable over 96 weeks. Predictors of improved HRQoL included higher baseline stool number, longer teduglutide treatment duration, greater length of small intestine, and older age. To better understand these observations, future studies should assess HRQoL before initiation of treatment.

目的:小儿短肠综合征相关性肠衰竭(SBS-IF)患者复杂的护理需求会对患者及其护理者的健康相关生活质量(HRQoL)产生负面影响。我们评估了替杜葡肽对小儿SBS-IF患者HRQoL的影响。方法:两项长期延伸(LTE)研究(NCT02949362, NCT02954458)使用儿科生活质量量表(PedsQL) 4.0通用核心量表和PedsQL家庭影响模块评估96周的HRQoL。该分析中纳入的患者在三个随机iii期亲本研究之一和各自的LTE研究中接受了teduglutide。我们评估了HRQoL随时间的变化,以及获得肠内自主对HRQoL的影响和HRQoL变化的预测因素。结果:在接受teduglutide治疗的SBS-IF儿童患者中(n = 69),患者报告的PedsQL评分的总平均值(标准差)为75.0(16.2)。在LTE研究基线时,较高的每天平均排便次数(p = 0.03)预示着患者报告的HRQoL增加。teduglutide暴露时间越长(p = 0.047),残余小肠长度越长(p = 0.034),年龄越大(p = 0.026),护理者代理报告的HRQoL越高。无论患者是否实现肠内自主,平均PedsQL评分在96周内保持稳定。结论:替杜葡肽治疗小儿SBS-IF患者的HRQoL在96周内总体稳定。HRQoL改善的预测因子包括更高的基线粪便数量、更长的teduglutide治疗时间、更长的小肠长度和年龄。为了更好地理解这些观察结果,未来的研究应该在开始治疗前评估HRQoL。
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Journal of Pediatric Gastroenterology and Nutrition
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