Morioka Ichiro, Kakei Yasumasa, Omori Takashi, Nozu Kandai, Fujioka Kazumichi, Takahashi Naoto, Yoshikawa Tetsushi, Moriuchi Hiroyuki, Ito Yoshinori, Oka Akira
Department of Pediatrics and Child Health, Nihon University School of Medicine, Tokyo 173-8610, Japan.
Department of Oral and Maxillofacial Surgery, Kobe University Graduate School of Medicine, Kobe 650-0017, Japan.
J Clin Med. 2022 Jun 21;11(13):3582. doi: 10.3390/jcm11133582.
Our aims were to determine the clinical impact of oral valganciclovir (VGCV) in infants aged ≤2 months with congenital cytomegalovirus (CMV) disease and evaluate the efficacy of VGCV when initiated beyond the neonatal period. The multicenter, single-arm, open-label clinical trial was conducted in Japan. Twenty-five infants aged ≤2 months with congenital CMV disease involving the central nervous system were enrolled and treated with VGCV for 6 months. The primary endpoint was the change in the whole blood CMV load before and after treatment. The secondary endpoint was the change in the auditory brainstem response (ABR) before and after treatment. Changes in ABR were assessed between the younger and older age groups (≤ and >30 days at treatment initiation). Of the 25 patients, one was excluded owing to epilepsy before VGCV administration. The median change in the CMV DNA level in whole blood was −246.0 IU/mL. The best ear and total ear assessments based on ABR were categorized as (improved + unchanged) after treatment for 100% and 93.8%, respectively. No differences in hearing efficacy were observed between the younger and older age groups. Oral VGCV is a potential therapeutic option for treating infants aged ≤2 months with congenital CMV disease.
我们的目的是确定口服缬更昔洛韦（VGCV）对年龄≤2个月的先天性巨细胞病毒（CMV）病婴儿的临床影响，并评估在新生儿期之后开始使用VGCV的疗效。这项多中心、单臂、开放标签的临床试验在日本进行。25名年龄≤2个月、患有累及中枢神经系统的先天性CMV病的婴儿入组，并接受VGCV治疗6个月。主要终点是治疗前后全血CMV载量的变化。次要终点是治疗前后听性脑干反应（ABR）的变化。在较年轻和较年长年龄组（开始治疗时≤30天和>30天）之间评估ABR的变化。25例患者中，1例因在给予VGCV前患有癫痫而被排除。全血中CMV DNA水平的中位数变化为−246.0 IU/mL。治疗后，基于ABR的最佳耳和全耳评估分别有100%和93.8%被归类为（改善+未改变）。较年轻和较年长年龄组之间未观察到听力疗效的差异。口服VGCV是治疗年龄≤2个月的先天性CMV病婴儿的一种潜在治疗选择。
