Lv Bo, He Lan, Liu Nan, Li Chunfu, Peng Zhiyong, Bu Chaoke, Liu Huaying, Liao Jianyun
Hematology Department, Dongguan Taixin Hospital·Nanfang-Chunfu Children's Institute of Hematology & Oncology, Dongguan, China.
Pediatr Transplant. 2025 Jun;29(4):e70096. doi: 10.1111/petr.70096.
In this prospective observational study, we aim to investigate the role of cidofovir (CDV) as prophylactic treatment in preventing BK virus (BKV)-associated hemorrhagic cystitis (HC) in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT).
This study enrolled 28 patients who received prophylactic treatment with CDV following allogeneic hematopoietic stem cell transplantation (Allo-HSCT) and 25 Allo-HSCT patients who received conventional supportive care, which included hydration and analgesics, from January 2020 to January 2022. Patients in the CDV prophylactic treatment group received intravenous injections of CDV at a dose of 1 mg/kg once a week for 4 weeks. PCR was used to detect the copy number of BKV (BKV-DNA) in the urine of all study participants at weeks 1, 2, 3, 4, and 5 after HSCT.
From the third week onward, the BKV copy numbers in the CDV prophylactic treatment group were significantly lower than those in the conventional treatment group at subsequent time points. The proportion of patients with acute graft-versus-host disease (aGVHD), cytomegalovirus infection, and BKV copy numbers was significantly lower in the CDV group compared to the conventional treatment group. Additionally, the proportion of patients receiving CDV prophylactic treatment was significantly higher in the non-HC group. Receiver operating characteristic curve analysis indicated that BKV copy numbers could predict the occurrence and severity of HC. Furthermore, logistic regression analysis identified aGVHD, BKV copy numbers, and CDV prophylactic treatment as risk factors for the occurrence of HC in patients after Allo-HSCT.
We identified for the first time in the literature that prophylactic treatment with CDV could significantly reduce the incidence of BKV-associated hemorrhagic cystitis.
在这项前瞻性观察性研究中，我们旨在探讨西多福韦（CDV）作为预防性治疗在预防接受异基因造血干细胞移植（HSCT）的儿科患者中BK病毒（BKV）相关出血性膀胱炎（HC）的作用。
本研究纳入了2020年1月至2022年1月期间接受异基因造血干细胞移植（Allo-HSCT）后接受CDV预防性治疗的28例患者以及25例接受包括补液和止痛在内的常规支持治疗的Allo-HSCT患者。CDV预防性治疗组的患者每周静脉注射一次剂量为1mg/kg的CDV，共4周。在HSCT后第1、2、3、4和5周，使用聚合酶链反应（PCR）检测所有研究参与者尿液中BKV（BKV-DNA）的拷贝数。
从第三周起，CDV预防性治疗组在后续时间点的BKV拷贝数显著低于常规治疗组。与常规治疗组相比，CDV组急性移植物抗宿主病（aGVHD）、巨细胞病毒感染和BKV拷贝数的患者比例显著更低。此外，非HC组接受CDV预防性治疗的患者比例显著更高。受试者工作特征曲线分析表明，BKV拷贝数可预测HC的发生和严重程度。此外，逻辑回归分析确定aGVHD、BKV拷贝数和CDV预防性治疗是Allo-HSCT后患者发生HC的危险因素。
我们在文献中首次发现，CDV预防性治疗可显著降低BKV相关出血性膀胱炎的发生率。
