Zhu Jiajie, Chen Huifen, Meng Lingdu, Jiang Jingying, Yang Yifan, Wang Junfeng, Ren Xue, Kong Fanyang, Dong Rui, Chen Gong, Zheng Shan
Department of Pediatric Surgery, Children's Hospital of Fudan University, Shanghai Key Laboratory of Birth Defect and Key Laboratory of Neonatal Disease, Ministry of Health, 399 Wan Yuan Road, Shanghai, 201102, People's Republic of China.
J Gastroenterol. 2025 Jun;60(6):783-793. doi: 10.1007/s00535-025-02234-y. Epub 2025 May 5.
Accurately predicting the prognosis of biliary atresia (BA) prior to Kasai portoenterostomy (KPE) remains a challenge. The identification of the specific BA population that may benefit from primary liver transplantation (pLT) instead of KPE remains elusive.
A total of 196 BA patients and 31 age-matched non-BA cholestasis patients were recruited. BA patients were divided into training (February 2018-February 2019) and validation (March 2019-December 2021) cohorts. C-index was applied to evaluate the utility of indicators and models.
Serum fibroblast growth factor 19 (FGF19) was elevated in BA patients [95.67 (58.97-140.6) vs. 58.73 (43.59-85.35) pg/ml, P = 0.0003]. Constructed with FGF19, GGT, DBIL, and ALB, nomogram A demonstrated optimal C-index in training (0.767 ± 0.039) and validation (0.721 ± 0.062) cohorts with ideal consistency in predicting 1-year NLS after KPE as well as potential clinically utility in BA patients with an age at KPE ≤ 60 days. Leveraging the risk score (RS) developed with nomogram A, our findings revealed a notable decrease in 2-year NLS after KPE among BA patients with a preoperative RS > 1.36, and the patients with a preoperative RS > 2.6 appear to be potential candidates for pLT [2-year NLS after KPE: 0% (training cohort), 21.4% (validation cohort); specificity = 100% and sensitivity = 22.2%].
Nomogram A demonstrated significant efficacy in preoperatively predicting NLS in early BA. BA patients (age at KPE ≤ 60 days) with a RS > 2.6 may potentially benefit from pLT.
在进行肝门空肠吻合术(KPE)之前准确预测胆道闭锁(BA)的预后仍然是一项挑战。确定可能从原位肝移植(pLT)而非KPE中获益的特定BA患者群体仍然很困难。
共招募了196例BA患者和31例年龄匹配的非BA胆汁淤积患者。BA患者被分为训练队列(2018年2月至2019年2月)和验证队列(2019年3月至2021年12月)。应用C指数评估指标和模型的效用。
BA患者血清成纤维细胞生长因子19(FGF19)升高[95.67(58.97 - 140.6)对58.73(43.59 - 85.35)pg/ml,P = 0.0003]。由FGF19、γ-谷氨酰转移酶(GGT)、直接胆红素(DBIL)和白蛋白(ALB)构建的列线图A在训练队列(0.767±0.039)和验证队列(0.721±0.062)中显示出最佳C指数,在预测KPE后1年无肝生存(NLS)方面具有理想的一致性,并且对KPE时年龄≤60天的BA患者具有潜在的临床实用性。利用列线图A得出的风险评分(RS),我们的研究结果显示,术前RS>1.36的BA患者KPE后2年NLS显著降低,术前RS>2.6的患者似乎是pLT的潜在候选者[KPE后2年NLS:0%(训练队列),21.4%(验证队列);特异性 = 100%,敏感性 = 22.2%]。
列线图A在术前预测早期BA的NLS方面显示出显著疗效。RS>2.6的BA患者(KPE时年龄≤60天)可能从pLT中获益。