Lisai-Goldstein Yaara, Focht Gili, Orlanski-Meyer Esther, Yogev Dotan, Lev-Tzion Raffi, Ledder Oren, Assa Amit, Navas-López Victor Manuel, Baldassano Robert N, Otley Anthony, Shouval Dror S, Griffiths Anne M, Turner Dan, Atia Ohad
Juliet Keidan Institute of Pediatric Gastroenterology Hepatology and Nutrition, Shaare Zedek Medical Center, The Hebrew University of Jerusalem, Eisenberg R&D Authority, P.O. Box 3235, 9103102, Jerusalem, Israel.
Pediatric Gastroenterology and Nutrition Unit, Regional University Hospital of Málaga, Málaga, Spain.
Dig Dis Sci. 2025 Jan;70(1):333-339. doi: 10.1007/s10620-024-08732-y. Epub 2024 Nov 27.
To advance personalized medicine in pediatric Crohn's disease (CD), we aimed to explore the utility of serological biomarkers in predicting response to anti-tumor necrosis factor (TNF).
Children with CD were enrolled at initiation of anti-TNF and followed prospectively at 4 and 12 months thereafter, as well as at last follow-up. At baseline, 10 serological markers of the "PROMETHEUS® IBD sgi Diagnostic test" were measured, including pANCA, ASCA IgG and IgA, anti-CBir1, anti-OmpC, anti-A4-Fla2, anti-Fla-X, SAA, ICAM-1 and VCAM-1. The primary outcome was sustained steroid-free remission (SSFR, i.e. clinical remission without steroids at both 4 and 12 months) and the secondary outcome was primary non-response (PNR).
Of the 72 included children (mean age, 12.8 ± 3.1 years; median disease duration, 6.4 months [IQR 2.5-17.3]), 42 (58%) were treated with adalimumab and 30 (42%) with infliximab. PNR was noted in 20 (28%) children and failure to achieve SSFR in 36 (50%). The most common positive serological markers were SAA (86%) and ICAM-1 (82%). In univariate analyses, none of the serological markers achieved statistical significance in association with SSFR or with PNR. In multivariable analysis, positivity of ASCA IgG (OR 3.3 [95%CI 0.8-14.4]) and pANCA (OR 5.3 [95%CI 0.9-48]) were the closest to achieving significance in predicting SSFR, with fair predictive performance for the model (AUC 0.67 [95%CI 0.55-0.80]).
The serological markers tested here have limited utility in predicting response to anti-TNF treatment. Further studies with larger sample sizes are needed to confirm the utility of ASCA IgG and pANCA.
为推动儿童克罗恩病(CD)的个性化医疗,我们旨在探索血清生物标志物在预测抗肿瘤坏死因子(TNF)反应中的效用。
CD患儿在开始抗TNF治疗时入组,并在之后的4个月和12个月以及末次随访时进行前瞻性随访。在基线时,检测了“PROMETHEUS® IBD sgi诊断测试”的10种血清标志物,包括抗中性粒细胞胞浆抗体(pANCA)、抗酿酒酵母抗体IgG和IgA(ASCA IgG和IgA)、抗CBir1、抗OmpC、抗A4-Fla2、抗Fla-X、血清淀粉样蛋白A(SAA)、细胞间黏附分子-1(ICAM-1)和血管细胞黏附分子-1(VCAM-1)。主要结局为持续无类固醇缓解(SSFR,即4个月和12个月时均无类固醇的临床缓解),次要结局为原发无反应(PNR)。
纳入的72例儿童(平均年龄12.8±3.1岁;疾病持续时间中位数6.4个月[四分位间距2.5 - 17.3])中,42例(58%)接受阿达木单抗治疗,30例(42%)接受英夫利昔单抗治疗。20例(28%)儿童出现PNR,36例(50%)未实现SSFR。最常见的阳性血清标志物是SAA(86%)和ICAM-1(82%)。在单变量分析中,没有血清标志物与SSFR或PNR的关联达到统计学显著性。在多变量分析中,ASCA IgG阳性(比值比[OR] 3.3 [95%置信区间(CI)0.8 - 14.4])和pANCA阳性(OR 5.3 [95%CI 0.9 - 48])在预测SSFR方面最接近具有显著性,该模型的预测性能一般(曲线下面积[AUC] 0.67 [95%CI 0.55 - 0.80])。
此处检测的血清标志物在预测抗TNF治疗反应方面效用有限。需要进一步开展更大样本量的研究以确认ASCA IgG和pANCA的效用。