University of Colorado Hospital, Department of Transplant Surgery, Aurora, CO; Johns Hopkins Hospital, Department of Pediatric Surgery, Baltimore, MD; Ann and Robert H. Lurie Children's Hospital of Chicago, Division of Pediatric Surgery, Chicago, IL; University Children's Hospital of Basel, Department of Pediatric Surgery, Basel, Switzerland; Colorado Children's Hospital, Department of Pediatric Surgery, Aurora, CO.
University of Colorado Hospital, Department of Transplant Surgery, Aurora, CO; Johns Hopkins Hospital, Department of Pediatric Surgery, Baltimore, MD; Ann and Robert H. Lurie Children's Hospital of Chicago, Division of Pediatric Surgery, Chicago, IL; University Children's Hospital of Basel, Department of Pediatric Surgery, Basel, Switzerland; Colorado Children's Hospital, Department of Pediatric Surgery, Aurora, CO.
J Pediatr Surg. 2020 Sep;55(9):1802-1808. doi: 10.1016/j.jpedsurg.2020.03.017. Epub 2020 Mar 30.
Increasingly, for pediatric patients with short bowel syndrome (SBS), intestinal lengthening procedures such as serial transverse enteroplasty (STEP) are being offered with the hope of improving patients' chances for achieving enteral autonomy. However, it remains unclear to what extent STEP reduces the long-term need for intestinal transplant or improves survival.
Based on existing literature, a decision analytic Markov state transition model was created to simulate the life of 1,000 pediatric SBS patients. Two simulations were modeled: 1) No STEP: patients were listed for transplant once medical management failed and 2) STEP: patients underwent STEP therapy and subsequent transplant listing if enteral autonomy was not achieved. Sensitivity analysis of small bowel length and anatomy was completed. Base case patients were defined as neonates with a small bowel length of 30cm.
For base case patients with an ostomy and a NEC SBS etiology, STEP was associated with increased rates of enteral autonomy after 10 years for patients with an ICV (53.9% [STEP] vs. 51.1% [No STEP]) and without an ICV (43.4% [STEP] vs. 36.3% [No STEP]). Transplantation rates were also reduced following STEP therapy for both ICV (17.5% [STEP] vs. 18.2% [No STEP]) and non-ICV patients (20.2% [STEP] vs. 22.1% [No STEP]). 10-year survival was the highest in the (+) STEP and (+) ICV group (85.4%) and lowest in the (-) STEP and (-) ICV group (83.3%).
For SBS patients, according to our model, STEP increases rates of enteral autonomy, reduces need for intestinal transplantation, and improves long-term survival.
Economic/Decision Analysis or Modeling Studies LEVEL OF EVIDENCE: Level III.
越来越多患有短肠综合征（SBS）的儿科患者接受了肠延长手术，如连续横向肠成形术（STEP），以期提高患者实现肠内自主的机会。然而，肠延长手术在多大程度上降低了长期肠移植的需求或提高了生存率仍不清楚。
基于现有文献，创建了一个决策分析马尔可夫状态转移模型，以模拟 1000 名儿科 SBS 患者的生活。模拟了两种情况：1）无 STEP：一旦药物治疗失败，患者就被列入移植名单；2）STEP：患者接受 STEP 治疗，如果未能实现肠内自主，则随后被列入移植名单。对小肠长度和解剖结构进行了敏感性分析。基础病例患者定义为小肠长度为 30cm 的新生儿。
对于患有肠造口术和 NEC SBS 病因的基础病例患者，STEP 与肠内自主的更高发生率相关，在 10 年内，有或无颅内血管异常（ICV）的患者分别为 53.9%（STEP）和 51.1%（无 STEP）；无 ICV 的患者分别为 43.4%（STEP）和 36.3%（无 STEP）。在接受 STEP 治疗后，ICV（17.5% [STEP] 与 18.2% [无 STEP]）和非 ICV 患者（20.2% [STEP] 与 22.1% [无 STEP]）的移植率也有所降低。10 年生存率最高的是（+）STEP 和（+）ICV 组（85.4%），最低的是（-）STEP 和（-）ICV 组（83.3%）。
根据我们的模型，对于 SBS 患者，STEP 提高了肠内自主的比率，降低了肠移植的需求，并提高了长期生存率。
经济/决策分析或建模研究
三级。
