Department of Pediatrics, Division of Pediatric Gastroenterology, Children's Hospital Western Ontario, Western University, London Health Sciences Centre, London, Ontario, Canada.
Alimentiv Inc, London, ON, Canada.
Inflamm Bowel Dis. 2024 Mar 1;30(3):357-369. doi: 10.1093/ibd/izad134.
Perianal fistulas and abscesses occur commonly as complications of pediatric Crohn's disease (CD). A validated imaging assessment tool for quantification of perianal disease severity and activity is needed to evaluate treatment response. We aimed to identify magnetic resonance imaging (MRI)-based measures of perianal fistulizing disease activity and study design features appropriate for pediatric patients.
Seventy-nine statements relevant to MRI-based assessment of pediatric perianal fistulizing CD activity and clinical trial design were generated from literature review and expert opinion. Statement appropriateness was rated by a panel (N = 15) of gastroenterologists, radiologists, and surgeons using modified RAND/University of California Los Angeles appropriateness methodology.
The modified Van Assche Index (mVAI) and the Magnetic Resonance Novel Index for Fistula Imaging in CD (MAGNIFI-CD) were considered appropriate instruments for use in pediatric perianal fistulizing disease clinical trials. Although there was concern regarding the use of intravascular contrast material in pediatric patients, its use in clinical trials was considered appropriate. A clinically evident fistula tract and radiologic disease defined as at least 1 fistula or abscess on pelvic MRI were considered appropriate trial inclusion criteria. A coprimary clinical and radiologic end point and inclusion of a patient-reported outcome were also considered appropriate.
Outcomes of treatment of perianal fistulizing disease in children must include MRI. Existing multi-item measures, specifically the mVAI and MAGNIFI-CD, can be adapted and used for children. Further research to assess the operating properties of the indices when used in a pediatric patient population is ongoing.
肛周瘘管和脓肿是小儿克罗恩病（CD）的常见并发症。需要一种经过验证的影像学评估工具来量化肛周疾病的严重程度和活动度，以评估治疗反应。我们旨在确定基于磁共振成像（MRI）的肛周瘘管疾病活动度的测量方法，并研究适合儿科患者的研究设计特征。
从文献回顾和专家意见中生成了 79 条与 MRI 评估小儿肛周瘘管 CD 活动度和临床试验设计相关的语句。通过修改后的 RAND/加州大学洛杉矶分校适宜性方法，由一组（N=15）胃肠病学家、放射科医生和外科医生对适宜性进行评分。
改良的 Van Assche 指数（mVAI）和磁共振新型 CD 瘘管成像指数（MAGNIFI-CD）被认为是小儿肛周瘘管疾病临床试验中合适的工具。尽管对小儿患者使用血管内对比剂存在担忧，但在临床试验中仍被认为是合适的。临床上明显的瘘管和影像学疾病（骨盆 MRI 上至少有 1 个瘘管或脓肿）被认为是合适的试验纳入标准。主要的临床和影像学终点以及患者报告结果的纳入也被认为是合适的。
治疗儿童肛周瘘管疾病的治疗结果必须包括 MRI。现有的多项目测量方法，特别是 mVAI 和 MAGNIFI-CD，可以进行改编并用于儿童。正在进行进一步的研究，以评估这些指数在儿科患者人群中的应用性能。
