Zhu Yongbing, Wang Li, Xiang Yu, Wang Yaqin, Zhang Ai, Wang Yao, Yin Mengmeng, Dun Jianxin, Xu Yuting, Hu Qun, Yu Wen, Liu Aiguo
Department of Pediatrics, Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, People's Republic of China.
Drug Des Devel Ther. 2025 Jun 12;19:5059-5069. doi: 10.2147/DDDT.S513383. eCollection 2025.
Cytomegalovirus (CMV) infection represents a severe complication following hematopoietic cell transplantation (HCT), resulting in high mortality. The prevention of CMV reactivation is crucial for enhancing patient prognosis post-HCT. Letermovir prophylaxis has effectively reduced the incidence of clinically significant CMV infection (csCMVi) in adult HCT recipients. However, clinical data in pediatric patients remain limited.
We included 106 children who underwent HCT at our hospital between March 2019 and July 2024. The patients were grouped based on whether or not they received letermovir prophylaxis. By analyzing their general characteristics and laboratory findings, exploring the risk factors of csCMVi, and assessing the efficacy and safety of letermovir in pediatric patients.
Among the 106 patients, all patients were at high risk for CMV reactivation. Forty-four received letermovir prophylaxis, while 62 did not. CsCMVi occurred in 45 patients, with a significantly lower incidence in the letermovir group compared to the control group (5 [11.3%] vs 40 [64.5%], p < 0.001). Umbilical cord blood (UCB) was used in 7 patients (15.9%) in the letermovir group and in 1 patient in the control group ( < 0.05). There was no statistically significant difference in all-cause mortality between the two groups. Grade II-IV GvHD and the use of letermovir were associated with csCMVi, with letermovir identified as the only independent preventive factor for csCMVi during the first 100 days post-HCT, especially in patients with 4-5 risk factors of csCMVi. In patients with aplastic anemia, the incidence of csCMVi was notably lower in those who received letermovir prophylaxis. No patients in the study withdrew from treatment due to adverse reactions.
Letermovir is both effective and safe for CMV prophylaxis in pediatric patients following HCT, especially in patients with more risk factors of csCMVi. Grade II-IV GvHD increases the risk of csCMVi, while letermovir prophylaxis reduces the risk.
巨细胞病毒（CMV）感染是造血细胞移植（HCT）后的一种严重并发症，导致高死亡率。预防CMV重新激活对于改善HCT后患者的预后至关重要。来特莫韦预防已有效降低成年HCT受者中具有临床意义的CMV感染（csCMVi）的发生率。然而，儿科患者的临床数据仍然有限。
我们纳入了2019年3月至2024年7月期间在我院接受HCT的106名儿童。根据患者是否接受来特莫韦预防进行分组。通过分析他们的一般特征和实验室检查结果，探索csCMVi的危险因素，并评估来特莫韦在儿科患者中的疗效和安全性。
在这106名患者中，所有患者CMV重新激活的风险都很高。44名患者接受了来特莫韦预防，62名患者未接受。45名患者发生了csCMVi，来特莫韦组的发生率明显低于对照组（5例[11.3%]对40例[64.5%]，p<0.001）。来特莫韦组7名患者（15.9%）使用了脐带血（UCB），对照组1名患者使用了UCB（<0.05）。两组间全因死亡率无统计学显著差异。II-IV级移植物抗宿主病（GvHD）和来特莫韦的使用与csCMVi相关，来特莫韦被确定为HCT后前100天内csCMVi的唯一独立预防因素，尤其是在具有4-5个csCMVi危险因素的患者中。在再生障碍性贫血患者中，接受来特莫韦预防的患者csCMVi的发生率明显较低。研究中没有患者因不良反应退出治疗。
来特莫韦对HCT后的儿科患者预防CMV既有效又安全，尤其是在具有更多csCMVi危险因素的患者中。II-IV级GvHD增加了csCMVi的风险，而来特莫韦预防降低了该风险。
