Background/Objectives: The objective of this study was to analyze the clinical and laboratory features, management, outcomes, and complications of PRES in children with malignancies or following hematopoietic cell transplantation (HCT).Methods: This was a multicenter retrospective analysis of PRES episodes diagnosed between 2014 and 2022 in Polish pediatric hematology and oncology (PHO) centers and HCT units. The study included 438 patients treated for malignancy or post-HCT: 120 with PRES (study group) and 318 without PRES (control group).Results: PRES was diagnosed in children aged 1.7–16.5 years (median = 7.7 years). The most common underlying diagnosis was ALL (76.7%; n = 92). Symptoms of PRES included disturbances of consciousness (84.2%), seizures (80.0%), hypertension (74.2%), apathy (64.2%), abdominal pain (45.0%), visual disturbances (28.3%), and headaches (26.7%). Electrolyte abnormalities were observed in 75.0% of children, most commonly hyponatremia (49.2%) and hypokalemia (37.5%). Children with PRES were more likely to require admission to the intensive care unit (ICU) than controls (50.0% vs. 29.6%,p< 0.001). The most frequent long-term complications of PRES were hypertension (22.5%) and epilepsy (20.8%). Among PHO patients, those with PRES had significantly lower DFS (76.7% vs. 93.7%,p< 0.001) and OS (79.2% vs. 93.4%,p< 0.001). In the HCT group, PRES was also associated with lower DFS (40.0% vs. 83.3%,p= 0.012) and OS (40.0% vs. 77.8%,p= 0.047).Conclusions: PRES is a significant complication of oncological and transplant treatment in children. Its occurrence was associated with worse overall and disease-free survival. We proposed a predictive index for PRES, diagnostic criteria, and a revised name for this syndrome.
背景/目的：本研究旨在分析恶性肿瘤患儿及造血细胞移植（HCT）后患儿发生可逆性后部脑病综合征（PRES）的临床特征、实验室指标、治疗方案、预后及并发症情况。 方法：本研究为多中心回顾性分析，收集2014年至2022年间波兰儿科血液肿瘤中心及HCT单元确诊的PRES病例。研究共纳入438例接受恶性肿瘤治疗或HCT术后患儿，其中120例发生PRES（研究组），318例未发生PRES（对照组）。 结果：PRES患儿年龄范围为1.7-16.5岁（中位年龄7.7岁）。最常见的原发病为急性淋巴细胞白血病（76.7%；n=92）。PRES临床表现包括意识障碍（84.2%）、癫痫发作（80.0%）、高血压（74.2%）、淡漠状态（64.2%）、腹痛（45.0%）、视觉障碍（28.3%）及头痛（26.7%）。75.0%的患儿存在电解质紊乱，以低钠血症（49.2%）和低钾血症（37.5%）最为常见。与对照组相比，PRES患儿更需重症监护治疗（50.0% vs. 29.6%，p<0.001）。PRES最常见的远期并发症为高血压（22.5%）和癫痫（20.8%）。在儿科血液肿瘤患者中，PRES患儿的无病生存率（76.7% vs. 93.7%，p<0.001）和总生存率（79.2% vs. 93.4%，p<0.001）显著降低。在HCT组中，PRES同样与较低的无病生存率（40.0% vs. 83.3%，p=0.012）和总生存率（40.0% vs. 77.8%，p=0.047）相关。 结论：PRES是儿童肿瘤治疗及移植治疗的重要并发症，其发生与较低的总生存率和无病生存率相关。本研究提出了PRES预测指数、诊断标准及该综合征的修订命名建议。
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