Kim Jin Kyu, Szymanski Konrad M, Misseri Rosalia, Reddy Pramod, Strine Andrew, Vanderbrink Brian A, Mcleod Daryl, Jayanthi Rama, Davis-Dao Carol, Khoury Antoine, Comella Assia, Taghavi Kiarash, Kattini Ribal, Wang Zhan Tao Peter, Dave Sumit, Boswell Timothy, Bicknell Brenton T, Merguerian Paul, Santos Joana Dos, Lorenzo Armando J, Rickard Mandy
Riley Hospital for Children, Indianapolis, IN, USA; The Hospital for Sick Children, Toronto, ON, Canada.
Riley Hospital for Children, Indianapolis, IN, USA.
J Pediatr Urol. 2025 Jul 17. doi: 10.1016/j.jpurol.2025.07.015.
Congenital lower urinary tract obstruction due to posterior urethral valves (PUV) often results in chronic kidney disease (CKD) and renal replacement therapy (RRT). The Posterior Urethral Valve Risk of Chronic Kidney Disease (PURK) score, a validated prognostic tool, uses clinical variables at presentation to predict development of CKD stage 3 or higher. However, its role in predicting progression to RRT remains unexplored.
This study evaluates the utility of the PURK score for RRT risk stratification internationally, aiming to facilitate early counseling and management for affected families.
Patients with posterior urethral valves (PUV) presenting before age 1 were analyzed from internal and external databases (9 institutions), excluding early RRT events (<14 days). PURK scores were calculated. ROC curves assessed predictive accuracy and Kaplan-Meier survival analysis stratified risk groups. External validation involved data from eight international centers.
Internal validation from 183 PUV patients at a single center, with a median follow-up of 8.1 years, was analyzed. This showed significant differences in PURK score distribution between patients who progressed to RRT (15.1 %) and those who did not. Kaplan-Meier curves identified low- (0-1, 0 %), intermediate- (2-3, 9.6 %), and high-risk groups (≥4, 37.5 %) for RRT progression. AUROC values demonstrated good predictive ability for 1-, 5-, and 10-year RRT events (0.798-0.839). External validation of 265 patients confirmed these findings, with excellent AUROC values for 1-, 5-, and 10-year RRT events (0.868-0.885) and consistent survival stratification.
We identified clear risk groups for progression to RRT using survival analysis, with those with high PURK score being much more likely to progress to RRT compared to lower scores. Being able to prognosticate patients into different risk groups is important as it can guide frequency of follow up and early interventions including alpha-blockers, anticholinergics, or clean intermittent catheterization for children with higher risk profiles.
The PURK score is a reliable tool for early prediction and risk stratification of progression to RRT in PUV patients.
后尿道瓣膜（PUV）导致的先天性下尿路梗阻常引发慢性肾脏病（CKD）及肾脏替代治疗（RRT）。后尿道瓣膜慢性肾脏病风险（PURK）评分作为一种经过验证的预后工具，利用患者就诊时的临床变量来预测3期及以上CKD的发生。然而，其在预测进展至RRT方面的作用尚未得到探索。
本研究评估PURK评分在国际上对RRT风险分层的效用，旨在为受影响家庭提供早期咨询和管理。
对来自内部和外部数据库（9个机构）中1岁前出现后尿道瓣膜（PUV）的患者进行分析，排除早期RRT事件（<14天）。计算PURK评分。ROC曲线评估预测准确性，Kaplan-Meier生存分析对风险组进行分层。外部验证采用来自8个国际中心的数据。
对单中心183例PUV患者进行内部验证，中位随访时间为8.1年。结果显示，进展至RRT的患者（15.1%）和未进展至RRT的患者在PURK评分分布上存在显著差异。Kaplan-Meier曲线确定了RRT进展的低风险组（0 - 1分，0%）、中风险组（2 - 3分，9.6%）和高风险组（≥4分，37.5%）。AUROC值显示对1年、5年和10年RRT事件具有良好的预测能力（0.798 - 0.839）。对265例患者的外部验证证实了这些结果，1年、5年和10年RRT事件的AUROC值优异（0.868 - 0.885），生存分层一致。
我们通过生存分析确定了进展至RRT的明确风险组，与低分患者相比，PURK评分高的患者进展至RRT的可能性要大得多。能够将患者分为不同风险组很重要，因为它可以指导随访频率和早期干预，包括对高风险儿童使用α受体阻滞剂、抗胆碱能药物或清洁间歇性导尿。
PURK评分是早期预测PUV患者进展至RRT并进行风险分层的可靠工具。
