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B型肠道神经元发育异常患者的长期随访:一项观察性、前瞻性和对比性研究方案

Long-term follow-up of patients with intestinal neuronal dysplasia type B: Protocol for an observational, ambispective, and comparative study.

作者信息

Lourenção Pedro Luiz Toledo de Arruda, Ortolan Erika Veruska Paiva, Rosa Laura Luiza Minelli, Angelini Marcos Curcio, Terra Simone Antunes, Rodrigues Maria Aparecida Marchesan

机构信息

Discipline of Pediatric Surgery-Department of Surgery and Orthopedics Botucatu Medical School, São Paulo State University (UNESP), São Paulo, Brazil Department of Pathology, Botucatu Medical School, São Paulo State University (UNESP), São Paulo, Brazil.

出版信息

Medicine (Baltimore). 2017 Jul;96(28):e7485. doi: 10.1097/MD.0000000000007485.

Abstract

Intestinal neuronal dysplasia type B (IND-B) is a pathological entity of the group of gastrointestinal neuromuscular diseases characterized by complex alterations in the enteric nervous system. Patients typically present with intestinal constipation, sometimes complicated by episodes of intestinal obstruction. The 2 therapeutic modalities include conservative clinical treatment and surgical treatment. Nevertheless, the results of the different therapeutic modalities are conflicting, and follow-up studies are scarce and include only a limited number of patients.This is a single-center, ambispective, observational, longitudinal, and comparative follow-up study to compare the results of conservative clinical and surgical treatments in patients with IND-B. Sixty-three patients (<15 years) who received this diagnosis will be included. These patients will be divided into 2 groups according to the type of treatment that they previously received: 29 patients in the surgical treatment group and 34 patients in the conservative treatment group. Previous data will be recovered from the medical records of the study patients, including signs and symptoms present at the time of diagnosis, particularly those related to bowel habits, and treatments undergone. Later, these patients will be invited to participate in a semistructured interview during which aspects related to the long-term functional results of the bowel habit and quality of life will be investigated after a minimum interval of 5 years posttreatment.This project aims to assess the long-term clinical evolution of patients diagnosed with IND-B and compare the results obtained following conservative clinical and surgical treatments.This protocol will provide sufficient data to analyze the long-term clinical outcome obtained through the 2 treatment modalities proposed for patients with IND-B.

摘要

B型肠道神经元发育异常(IND-B)是胃肠神经肌肉疾病组中的一种病理实体,其特征是肠神经系统存在复杂改变。患者通常表现为肠道便秘,有时并发肠梗阻。两种治疗方式包括保守临床治疗和手术治疗。然而,不同治疗方式的结果相互矛盾,随访研究稀缺且仅纳入有限数量的患者。这是一项单中心、双前瞻性、观察性、纵向和比较性随访研究,旨在比较IND-B患者保守临床治疗和手术治疗的结果。将纳入63名确诊为此病的(15岁以下)患者。这些患者将根据他们之前接受的治疗类型分为两组:手术治疗组29例患者,保守治疗组34例患者。既往数据将从研究患者的病历中获取,包括诊断时出现的体征和症状,特别是与排便习惯相关的症状,以及接受的治疗。之后,将邀请这些患者参加半结构化访谈,在治疗后至少5年的间隔期后,调查与排便习惯的长期功能结果和生活质量相关的方面。该项目旨在评估IND-B确诊患者的长期临床演变,并比较保守临床治疗和手术治疗后获得的结果。本方案将提供足够的数据来分析通过为IND-B患者提出的两种治疗方式所获得的长期临床结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb21/5515763/da50d47326b8/medi-96-e7485-g002.jpg

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