Roth Joshua D, Bowen Diana, Fuchs Molly E, Gargollo Patricio C, Gottlich Harrison, Hains David S, Strine Andrew C, Szymanski Konrad M
Riley Children's Health at Indiana University Health, USA.
Lurie Children's Hospital of Chicago, USA.
J Pediatr Urol. 2025 Feb;21(1):41-46. doi: 10.1016/j.jpurol.2024.10.016. Epub 2024 Oct 23.
While most children with classic bladder exstrophy (CBE) are born with normal kidneys, some experience renal deterioration in adulthood. Little is known about the incidence of end-stage and chronic kidney disease (ESKD and CKD, respectively) in this population. Our group has recently published on surgical outcomes in a multi-institutional cohort of 216 people with CBE. Our aim was to describe the incidence of ESKD and prevalence of CKD in this cohort of people with CBE.
We retrospectively reviewed records of patients with CBE followed at five tertiary care centers described previously. The primary outcome was incidence of ESKD, defined as permanent peritoneal/hemodialysis or renal transplantation. The secondary outcome was prevalence of CKD stage 3 or higher (CKD3+, estimated glomerular filtration rate [eGFR]<60 ml/min/1.73 m) at the last appointment. Creatinine-based eGFRs were calculated using the CKD-EPI Creatinine Equation (adults) and the Schwartz formula (children). Survival analysis and Fisher's exact test were used.
A total of 201 patients (93 % of the original cohort) had renal function data available (63 % male). Four patients who had a primary urinary diversion remained diverted at a median follow-up of 20.1 years. None developed ESKD and one developed CKD3+. The remaining 197 patients had a primary bladder closure. At a median follow-up of 18.8 years old, 12 were diverted, 108 were augmented and 77 were neither. Three patients developed ESKD (1.5 %) at a median age of 23.4 years (1 hemodialysis, 2 transplantation). On survival analysis, the risk of ESKD was 0 % at 10 years, 1 % at 20 years and 5 % at 30 years (Figure 1). This was higher than the risk of 0.003 % at 21 years of age in the general population (p < 0.001). The median age of 141 individuals with eGFR data was 21.6 years old (65 % male). No children, 4 % of adolescents and 8 % of adults had CKD3+ (p = 0.45). On exploratory analyses, prevalence of CKD3+ did not differ by center or birth year (p ≥ 0.99).
The risk of ESKD and CKD among patients with CBE is not insignificant and appears to be more common than the general population. The potential role of modifiable contributing factors, such as increased bladder outlet resistance, warrants further investigation. Reliable long-term follow up is needed in this population to monitor for ESKD and CKD.
虽然大多数患有典型膀胱外翻（CBE）的儿童出生时肾脏正常，但一些人在成年后会出现肾功能恶化。关于这一人群中终末期肾病和慢性肾病（分别为ESKD和CKD）的发病率知之甚少。我们的研究小组最近发表了一项关于216例CBE患者的多机构队列手术结果的研究。我们的目的是描述该CBE患者队列中ESKD的发病率和CKD的患病率。
我们回顾性地查阅了之前描述的五个三级医疗中心随访的CBE患者的记录。主要结局是ESKD的发病率，定义为永久性腹膜透析/血液透析或肾移植。次要结局是上次就诊时CKD 3期或更高分期（CKD3+，估计肾小球滤过率[eGFR]<60 ml/min/1.73 m²）的患病率。基于肌酐的eGFR使用CKD-EPI肌酐方程（成人）和Schwartz公式（儿童）计算。采用生存分析和Fisher精确检验。
共有201例患者（占原始队列的93%）有可用的肾功能数据（63%为男性）。4例接受了初次尿流改道的患者在中位随访20.1年时仍处于改道状态。无人发展为ESKD，1人发展为CKD3+。其余197例患者进行了初次膀胱闭合术。在中位随访18.8岁时，12例进行了尿流改道，108例进行了膀胱扩大术，77例既未进行尿流改道也未进行膀胱扩大术。3例患者（1.5%）在中位年龄23.4岁时发展为ESKD（1例血液透析，2例肾移植）。生存分析显示，ESKD的风险在10年时为0%，20年时为1%，30年时为5%（图1）。这高于一般人群21岁时0.003%的风险（p<0.001）。141例有eGFR数据的个体的中位年龄为21.6岁（65%为男性）。儿童中无CKD3+，青少年中4%有CKD3+，成人中8%有CKD3+（p=0.45）。探索性分析显示，CKD3+的患病率在不同中心或出生年份之间无差异（p≥0.99）。
CBE患者中ESKD和CKD的风险并非微不足道，而且似乎比一般人群更常见。可改变的促成因素（如膀胱出口阻力增加）的潜在作用值得进一步研究。这一人群需要可靠的长期随访以监测ESKD和CKD。
