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用于预测胆道闭锁患者行 Kasai 手术后早期死亡或肝移植的便捷列线图。

A convenient nomogram for predicting early death or liver transplantation after the Kasai procedure in patients with biliary atresia.

机构信息

Department of Pediatric Surgery, Children's Hospital of Nanjing Medical University, Nanjing, 210008, China.

School of Pediatrics, Nanjing Medical University, Nanjing, 211166, China.

出版信息

Langenbecks Arch Surg. 2024 Jan 8;409(1):30. doi: 10.1007/s00423-023-03216-5.

Abstract

PURPOSE

Many patients with biliary atresia (BA) after the Kasai procedure (KP) progress to death or require liver transplantation to achieve long-term survival; however, most cases of death/liver transplantation (D/LT) occur in the early period after KP (usually within 1 year). This study was designed to construct a convenient nomogram for predicting early D/LT in patients with BA after KP.

METHODS

A BA cohort was established in May 2017, and up to May 2023, 112 patients with 1-5 years of follow-up were enrolled in the study and randomly (ratio, 3:1) divided into a training cohort for constructing a nomogram (n = 84) and a validation cohort (n = 28) for externally validating the discrimination and calibration. The training cohort was divided into two groups: the early D/LT group (patients who died or had undergone LT within 1 year after KP [n = 35]) and the control group (patients who survived through the native liver more than 1 year after KP [n = 49]). Multivariate logistic regression and stepwise regression were applied to detect variables with the best predictive ability for the construction of the nomogram. The discrimination and calibration of the nomogram were internally and externally validated.

RESULTS

The Kaplan-Meier (K-M) curve showed an actual 1-year native liver transplantation (NLS) rate of 57.1% and an estimated 2-year NLS rate of 55.2%. By multivariate regression and stepwise regression, age at KP, jaundice clearance (JC) speed 1 month after KP, early-onset PC (initial time < 36.5 days) after KP, sex, aspartate aminotransferase-to-platelet ratio index (APRI), and weight at KP were identified as the independent variables with the best ability to predict early D/LT and were used to construct a nomogram. The developed nomogram based on these independent variables showed relatively good discrimination and calibration according to internal and external validation.

CONCLUSION

Most D/LTs were early D/LTs that occurred within 1 year after KP. The established nomogram based on predictors, including sex, weight at the KP, the APRI, age at the KP, JC speed 1 month after the KP, and early PC, may be useful for predicting early D/LT and may be helpful for counseling BA patients about patient prognosis after KP. This study was retrospectively registered at ClinicalTrials.gov (NCT05909033) in June 2023.

摘要

目的

许多胆道闭锁(BA)患儿在施行Kasai 手术后(KP)会进展为死亡或需要进行肝移植才能长期生存;然而,大多数死亡/肝移植(D/LT)事件发生在 KP 后早期(通常在 1 年内)。本研究旨在构建一个用于预测 KP 后 BA 患儿早期 D/LT 的简便列线图。

方法

2017 年 5 月建立了 BA 队列,截至 2023 年 5 月,共纳入了 112 例随访 1-5 年的患者,将其随机(比例为 3:1)分为构建列线图的训练队列(n=84)和验证队列(n=28)进行外部验证,以评估其区分度和校准度。训练队列分为两组:早期 D/LT 组(KP 后 1 年内死亡或进行 LT 的患者[n=35])和对照组(KP 后 1 年以上通过自身肝脏存活的患者[n=49])。采用多变量逻辑回归和逐步回归检测用于构建列线图的具有最佳预测能力的变量。通过内部和外部验证评估列线图的区分度和校准度。

结果

Kaplan-Meier(K-M)曲线显示实际 1 年的原发性肝移植(NLS)率为 57.1%,估计 2 年的 NLS 率为 55.2%。通过多变量回归和逐步回归,KP 时的年龄、KP 后 1 个月时的黄疸消退速度、KP 后早期出现的原发性胆管炎(初始时间<36.5 天)、性别、天门冬氨酸氨基转移酶/血小板比值指数(APRI)和 KP 时的体重被确定为预测早期 D/LT 的最佳能力的独立变量,并用于构建列线图。基于这些独立变量建立的列线图在内部和外部验证中显示出较好的区分度和校准度。

结论

大多数 D/LT 是 KP 后 1 年内发生的早期 D/LT。基于包括性别、KP 时的体重、APRI、KP 时的年龄、KP 后 1 个月时的黄疸消退速度和早期原发性胆管炎在内的预测因子建立的列线图,可能有助于预测早期 D/LT,并可能有助于为 KP 后 BA 患儿提供有关患者预后的咨询。本研究于 2023 年 6 月在 ClinicalTrials.gov(NCT05909033)进行了回顾性注册。

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