Real World Evidence en Pacientes pediátricos Con Enfermedad de Crohn Activa
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 58
- 试验地点
- 15
- 主要终点
- Effectiveness of the CDED + NEP
研究概览
简要总结
This prospective study aims to provide real-world evidence of the effectiveness of the NEP + CDED diet in paediatric patients with Crohn's disease under conditions of routine clinical practice.
详细描述
Pediatric Crohn's disease is a chronic inflammatory disorder of the gastrointestinal tract that impacts growth and quality of life. Exclusive enteral nutrition (EEN) is the standard dietary treatment to induce remission, although its use is limited by low acceptability and strict dietary restrictions.
The combination of partial enteral nutrition (PEN) with the Crohn's Disease Exclusion Diet (CDED) represents a more tolerable alternative that reduces exposure to harmful dietary components and has shown efficacy in inducing clinical remission in previous studies.
This observational, prospective, multicenter study will evaluate the effectiveness of CDED+PEN in routine clinical practice in pediatric patients (8-18 years) with active Crohn's disease. The primary objective is to assess the proportion of patients achieving clinical remission (wPCDAI ≤ 12.5) and mucosal healing (MINI Index < 8), steroid-free at weeks 6, 12, and 24, and anti-TNF-free at week 24.
Secondary objectives include assessing health-related quality of life (IMPACT-III), treatment satisfaction and gastrointestinal tolerance, treatment persistence, and estimation of healthcare resource utilization and associated costs. The study is observational, descriptive, and non-interventional with respect to drugs, and will be conducted in pediatric patients initiating CDED+PEN as part of standard clinical care. Treatment prescription will be independent of study participation, and patients who receive the indication may be invited to participate after providing informed consent together with their parents or legal guardians.
Primary outcome measures include the frequency of patients in steroid-free clinical remission at weeks 6, 12, and 24, anti-TNF-free remission at week 24, and the proportion achieving mucosal healing (MINI Index < 8) at the same time points. Secondary analyses will include quality of life scores (IMPACT-III), distribution of questionnaire responses on satisfaction and tolerance, treatment persistence assessed by survival analysis, and cost estimates based on healthcare resource use.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with Crohn's disease (diagnosis based on clinical, laboratory, endoscopic, radiological, and histological criteria, according to the revised Porto criteria of the ESPGHAN for the diagnosis of inflammatory bowel disease in children and adolescents);
- •Aged between 8 and 18 years;
- •Who are about to start treatment with NEP + CDED, i.e. CDED-naive patients who may have previously received treatment with NEE;
- •With active Crohn's disease defined by the Weighted Paediatric Crohn's Disease Activity Index (wPCDAI) ≥ 12.5;
- •With data collection available for the following 6 months;
- •Able to follow the study protocol or whose parent/caregiver is able to adhere to the study protocol, according to the investigator's perspective.
排除标准
- •Patients with ulcerative colitis and/or unclassified inflammatory bowel disease.
- •Patients with more than 36 months of disease progression.
- •Penetrating (B3) and/or non-inflammatory stenosing (B2) pattern.
- •Active perianal disease.
- •Presence of extraintestinal manifestations.
- •Patients who, in the investigator's opinion, will require surgical treatment or initiation of steroids or biological drugs (anti-TNF, vedolizumab or ustekinumab) within the next 3 months and for whom nutritional support is indicated exclusively as a bridge. Patients who are undergoing biological treatment but discontinue treatment due to non-response and initiate CDED+NEP are not excluded.
结局指标
主要结局
Effectiveness of the CDED + NEP
时间窗: At week 24
Measurement of absolute and relative frequencies of patients in anti-TNF-free remission
Mucosal healing
时间窗: Weeks 6, 12, 24
The relative and absolute frequencies of patients who have obtained a score of \<8 on the MINI Index will be calculated; this value implies healing of the mucosa.
次要结局
- Health-related quality of life(Weeks 6, 12, 24)
- Satisfaction and acceptability with nutritional treatment(Weeks 6, 12, 24)
- Gastrointestinal tolerance(Weeks 6, 12, 24)
- Persistence with the diet(Weeks 6, 12, 24)
- Healthcare resource use and associated costs(Weeks 6, 12, 24)