Zhang Meng, Chen Ningning, Luo Yanhui, Jia Chenguang, Zheng Jie, Cai Lixiao, Zhang Pan, Jing Yuanfang, Yang Wei, Qin Maoquan, Zhu Guanghua, Yang Jun
Department of Stem Cell Transplantation, Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics, Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Department of Hematology, Baoding Hospital of Beijing Children's Hospital, Capital Medical University, Baoding, China.
Transl Pediatr. 2025 Aug 31;14(8):1866-1872. doi: 10.21037/tp-2025-241. Epub 2025 Aug 25.
Letermovir (LET) is approved for the prophylaxis of cytomegalovirus (CMV) infection and disease in adult hematopoietic stem cell transplantation (HSCT) patients up to day 100. The use of LET in the pediatric HSCT population lacks extensive real-world research, especially for high-risk pediatric patients with CMV reactivation. The aim of our study was retrospectively analyzed the efficacy and safety of LET in high-risk pediatric patients with CMV activation, and conducted a preliminary exploration of its duration of use.
This was a single-center, retrospective study. A total of 123 patients under 18 years of age who were at high risk of CMV reactivation were included, with 61 of them receiving LET for prophylaxis. The median duration was 100 days (ranging from 52 to 298 days). The primary endpoint was the proportion of patients with clinically significant CMV infection (CsCMVi) at 24 weeks post-transplantation. The secondary endpoints were the proportion of patients with CsCMVi before week 14 and the timing of CMV infection. Additionally, we also assessed related adverse reactions.
The proportion of CMV reactivation at 24 weeks post-transplantation in the LET group was significantly lower than that in the control group (8.20% 43.55%, P<0.001). The secondary endpoint was the proportion of CMV reactivation in the LET group and it was also significantly lower than that of the control group at week 14 (6.56% 41.94%, P<0.001). The proportion of CMV reactivation at 24 weeks post-transplantation in patients who used LET for more than 100 days was significantly lower than that in the control group (5.89% 11.10%, P=0.009).
LET can effectively prevent CMV reactivation in patients under 18 years of age and demonstrates good safety. For pediatric patients at high risk of CMV reactivation, extending the duration of prophylaxis beyond 100 days may be beneficial.
来特莫韦（LET）已被批准用于预防成人造血干细胞移植（HSCT）患者至第100天的巨细胞病毒（CMV）感染和疾病。LET在儿科HSCT人群中的使用缺乏广泛的真实世界研究，尤其是对于有CMV再激活的高危儿科患者。我们研究的目的是回顾性分析LET在高危CMV激活儿科患者中的疗效和安全性，并对其使用持续时间进行初步探索。
这是一项单中心回顾性研究。共纳入123例18岁以下有CMV再激活高危风险的患者，其中61例接受LET预防。中位持续时间为100天（范围为52至298天）。主要终点是移植后24周时发生具有临床意义的CMV感染（CsCMVi）的患者比例。次要终点是第14周前发生CsCMVi的患者比例以及CMV感染的时间。此外，我们还评估了相关不良反应。
LET组移植后24周时CMV再激活的比例显著低于对照组（8.20%对43.55%，P<0.001）。次要终点是LET组在第14周时CMV再激活的比例，其也显著低于对照组（6.56%对41.94%，P<0.001）。使用LET超过100天的患者移植后24周时CMV再激活的比例显著低于对照组（5.89%对11.10%，P = 0.009）。
LET可有效预防18岁以下患者的CMV再激活，并显示出良好的安全性。对于有CMV再激活高危风险的儿科患者，将预防持续时间延长至100天以上可能有益。
