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多伦多儿童肠移植纳入标准的国际多中心验证研究。

An international multicenter validation study of the Toronto listing criteria for pediatric intestinal transplantation.

机构信息

Group for Improvement of Intestinal Function and Treatment (GIFT), Transplant and Regenerative Medicine Centre, Hospital for Sick Children, Toronto, Canada.

New Zealand National Intestinal Failure and Rehabilitation Service (NZ-NIFRS), Starship Child Health, Auckland, New Zealand.

出版信息

Am J Transplant. 2022 Nov;22(11):2608-2615. doi: 10.1111/ajt.17150. Epub 2022 Aug 1.

Abstract

Deciding which patients would benefit from intestinal transplantation (IT) remains an ethical/clinical dilemma. New criteria* were proposed in 2015: ≥2 intensive care unit (ICU) admissions, loss of ≥3 central venous catheter (CVC) sites, and persistently elevated conjugated bilirubin (CB ≥ 75 μmol/L) despite 6 weeks of lipid modification strategies. We performed a retrospective, international, multicenter validation study of 443 children (61% male, median gestational age 34 weeks [IQR 29-37]), diagnosed with IF between 2010 and 2015. Primary outcome measure was death or IT. Sensitivity, specificity, NPV, PPV, and probability of death/transplant (OR, 95% confidence intervals) were calculated for each criterion. Median age at IF diagnosis was 0.1 years (IQR 0.03-0.14) with median follow-up of 3.8 years (IQR 2.3-5.3). Forty of 443 (9%) patients died, 53 of 443 (12%) were transplanted; 11 died posttransplant. The validated criteria had a high predictive value of death/IT; ≥2 ICU admissions (p < .0001, OR 10.2, 95% CI 4.0-25.6), persistent CB ≥ 75 μmol/L (p < .0001, OR 8.2, 95% CI 4.8-13.9). and loss of ≥3 CVC sites (p = .0003, OR 5.7, 95% CI 2.2-14.7). This large, multicenter, international study in a contemporary cohort confirms the validity of the Toronto criteria. These validated criteria should guide listing decisions in pediatric IT.

摘要

决定哪些患者将受益于肠移植(IT)仍然是一个伦理/临床难题。新的标准*于 2015 年提出:≥2 次重症监护病房(ICU)入院,≥3 个中心静脉导管(CVC)丢失,以及尽管进行了 6 周的脂质修饰策略,但结合胆红素(CB≥75μmol/L)持续升高。我们对 2010 年至 2015 年期间被诊断为 IF 的 443 名儿童(61%为男性,中位胎龄为 34 周[IQR 29-37])进行了回顾性、国际、多中心验证研究。主要结局测量是死亡或 IT。为每个标准计算了敏感性、特异性、NPV、PPV 和死亡/移植的概率(OR,95%置信区间)。IF 诊断时的中位年龄为 0.1 岁(IQR 0.03-0.14),中位随访时间为 3.8 年(IQR 2.3-5.3)。443 名患者中有 40 名(9%)死亡,443 名中有 53 名(12%)接受了移植;11 名在移植后死亡。验证标准对死亡/IT 有很高的预测价值;≥2 次 ICU 入院(p<0.0001,OR 10.2,95%CI 4.0-25.6),持续 CB≥75μmol/L(p<0.0001,OR 8.2,95%CI 4.8-13.9)和≥3 个 CVC 丢失(p=0.0003,OR 5.7,95%CI 2.2-14.7)。这项大型、多中心、国际研究在当代队列中证实了多伦多标准的有效性。这些验证标准应指导儿科 IT 的列表决策。

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