Vendemini Francesca, De Lorenzo Paola, Romani Francesca, Malandrin Sandro Maria Ivano, Verna Marta, Bonanomi Sonia, Valsecchi Maria Grazia, Lucchini Giovanna, Balduzzi Adriana
Department of Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.
Pediatrics and Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.
Transplant Cell Ther. 2025 Sep;31(9):699.e1-699.e11. doi: 10.1016/j.jtct.2025.05.021. Epub 2025 May 26.
CMV reactivation represents one of the most frequent infectious complications post HSCT. Letermovir is a viral terminate inhibitor which has been approved in adults for the prophylaxis of post HSCT CMV reactivation. Its use in pediatric HSCT recipient was recently approved by US FDA but data on the use of letermovir in children remain limited.
OBJECTIVE(S): We aimed at comparing the incidence of CMV reactivation in a more recent cohort of pediatric HSCT recipients receiving primary or secondary prohylaxis with letermovir with a previous cohort of children receiving no prophylaxis. We analyzed the risk factors for CMV reactivation among IgG seropositive recipients and the role of CMV reactivation on treatment related mortality/overall survival.
This is a single center retrospective study which enrolled all consecutive patients aged <21 years who underwent allogeneic HSCT between 1 January 1, 2014 and October 31, 2023 at the pediatric HSCT Unit of the Fondazione IRCCS San Gerardo dei Tintori in Monza RESULTS: 287 patients who received 308 HSCT were analyzed. Three months cumulative incidence of CMV reactivation was 29.5% (95% CI: 24.3-35) in the standard cohort versus 3.7% (95% CI: 0.3-16.3) in the cohort of patients receiving letermovir as primary prophylaxis (P = .0029). The use of letermovir as well as the use of a HLA-identical donor with no serotherapy, bone marrow as a source of stem cell and the absence of acute GVHD were statistically significant protective factors against CMV reactivation at multivariate analysis. At 3 months after discontinuation of preemptive therapy, the cumulative incidence of CMV reactivation was 0% for the 15 patients receiving letermovir as secondary prophylaxis versus 34.7% (SE 5.8, 95% CI: 23.7-46.0) for the 72 patients not receiving secondary prophylaxis.
Letermovir is safe and efficacious in preventing CMV reactivation in pediatric patients undergoing HSCT in both primary and secondary prophylaxis.
巨细胞病毒（CMV）再激活是异基因造血干细胞移植（HSCT）后最常见的感染并发症之一。来特莫韦是一种病毒终止抑制剂，已被批准用于成人预防HSCT后的CMV再激活。其在儿科HSCT受者中的应用最近获得了美国食品药品监督管理局（FDA）的批准，但关于来特莫韦在儿童中使用的数据仍然有限。
我们旨在比较最近一组接受来特莫韦一级或二级预防的儿科HSCT受者与先前一组未接受预防的儿童中CMV再激活的发生率。我们分析了IgG血清学阳性受者中CMV再激活的危险因素以及CMV再激活对治疗相关死亡率/总生存率的作用。
这是一项单中心回顾性研究，纳入了2014年1月1日至2023年10月31日期间在蒙扎的圣杰拉尔多·德伊廷托里基金会儿科HSCT科接受异基因HSCT的所有连续年龄<21岁的患者。结果：分析了287例接受308次HSCT的患者。标准队列中CMV再激活的3个月累积发生率为29.5%（95%CI：24.3 - 35），而接受来特莫韦作为一级预防的队列中为3.7%（95%CI：0.3 - 16.3）（P = 0.0029）。在多变量分析中，使用来特莫韦以及使用无血清疗法的HLA相同供体、骨髓作为干细胞来源和无急性移植物抗宿主病（GVHD）是预防CMV再激活的统计学显著保护因素。在抢先治疗停药3个月后，15例接受来特莫韦作为二级预防的患者中CMV再激活的累积发生率为0%，而72例未接受二级预防的患者中为34.7%（标准误5.8，95%CI：23.7 - 46.0）。
来特莫韦在预防接受HSCT的儿科患者的CMV再激活方面，无论是一级预防还是二级预防，都是安全有效的。
