Longterm Outcomes of Individuals With Anorectal Malformations
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- Bowel function score
研究概览
简要总结
This is a cross-sectional observational study with the aim to assess longterm outcomes in a large cohort of individuals with anorectal malformations. Patients managed for anorectal malformations at Karolinska University Hospital, Stockholm, Sweden, Stockholm 1994-2017 will be reviewed and they will be invited to respond to validated questionnaires evaluating bowel function (>4 years of age), urinary tract function (>4 years of age), sexual function (>18 years of age) and quality of life (>8 years of age). Data will be analyzed with descriptive and comparative statistics.
详细描述
Background Anorectal malformations comprise a spectrum of congenital anomalies, that range from mild anterior displacement of the anus to very complex anomalies, involving both the hindgut and the urogenital tract. The birth prevalence in Sweden is approximately 1:3000 (1). Associated anomalies are encountered in 50-60 % of individuals with anorectal malformation. Anorectal malformations are associated with tethered cord in 20-30% of patients, which may impair bowel and urinary tract function. Development of surgical techniques and neonatal care have improved survival of children with anorectal malformations over the last decades. The focus of care has shifted from survival to optimizing bowel and urinary tract function as well as reducing the negative impact on quality of life. Current surgical techniques were introduced during the early 1980's. The outcomes compare favorably with older techniques, although there is still limited information on the outcomes up to adult age. Particularly, there are limited data of composite outcomes of bowel function, urinary tract function, sexual function and quality of life in comparison to age- and gender-matched controls from the normal population. Newborn infants are often screened for tethered cord with ultrasound. It is unclear if infants with normal ultrasound also need an MRI at a later stage to exclude spinal cord anomalies. Tethered cord is frequently asymptomatic. There are conflicting data with respect to prophylactic de-tethering.
Aims The overall aim of this project was to assess composite outcomes, bowel function, urinary tract function, sexual function, and quality of life in a cross-sectional study of children with ARM managed at Karolinska University Hospital.
Specific aims
- To assess if bowel function, urinary tract function, sexual function, and quality of life is impaired in children with ARM compared with normative data.
- To assess if bowel function, urinary tract function, sexual function and quality of life correlate
- To assess if individuals with ARM and tethered cord benefit from detethering.
- To assess if ultrasound of the spinal cord and MRI are equal imaging modalities to diagnose tethered cord.
Methods Study design Cross-sectional observational study. The STROBE Statement for reporting of observational studies will be used (2).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 4 Years 至 28 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individual who has undergone surgical management for anorectal malformation at Karolinska University Hospital 1994 to 2017.
排除标准
- •Individual not providing informed consent.
结局指标
主要结局
Bowel function score
时间窗: 3 years
Bowel function score, BFS, \>4 years of age (Rintala et al. J Pediatr Surg 1995;30:491-4)
Urinary tract function
时间窗: 3 years
Urinary tract function evaluated with a modification of Danish Prostatic Symptom Score, \>4 years of age (Kyrklund et al Dis Colon Rectum 2012;55:671-6)
Sexual function
时间窗: 3 years
Sexual function evaluated with Profile of Female Sexual Function in females \>18 years of age and with IIEF in males \>18 years of age
次要结局
未报告次要终点
研究者
Tomas Wester
Professor
Karolinska Institutet