■患有某些罕见疾病的儿科患者患严重呼吸道合胞病毒(RSV)感染的风险增加。然而,日本目前没有在这些患者中预防性使用抗RSV抗体(帕利珠单抗).
■这是世界上第一个多中心,不受控制,开放标签,2019年7月28日至2021年9月24日在日本7家医疗机构进行了II期临床试验,以调查疗效,安全,帕利珠单抗在新生儿招募的23名受试者中的药代动力学,婴儿,或24个月或更小的儿童患有以下任何一种疾病:肺发育不全,气道狭窄,先天性食管闭锁,遗传性代谢病,或者神经肌肉疾病。以15mg/kg以30天的间隔肌内施用至少四个连续剂量的帕利珠单抗。
■23名受试者完成了研究。没有受试者需要因RSV住院治疗。不良事件(AE)与最新访谈表格中描述的事件术语没有显着差异。5例严重不良事件需要计划外住院,但没有RSV感染就解决了。在整个研究期间维持帕利珠单抗的治疗有效浓度。
■帕利珠单抗可能具有良好的耐受性,可有效预防严重呼吸道感染引起的严重呼吸道症状和住院,表明本研究中包括的儿科患者的预防性使用。
■日本医学研究与发展机构(AMED),赠款编号19lk0201097h0001(至MM),20lk0201097h0002(至MM),21lk0201097h0003(至MM),和22lk0201097h0004(至MM)。AMED在这项研究的执行中没有任何作用,分析和解释数据,或提交结果的决定。
UNASSIGNED: Pediatric patients with certain rare diseases are at increased risk of severe respiratory syncytial virus (RSV) infection. However, the prophylactic use of anti-RSV antibody (palivizumab) in these patients is not indicated at present in Japan.
UNASSIGNED: This first-in-the-world multicenter, uncontrolled, open-label, phase II clinical trial was carried out between 28 July 2019 and 24 September 2021 at seven medical institutions in Japan to investigate the efficacy, safety, and pharmacokinetics of palivizumab in 23 subjects recruited from among neonates, infants, or children aged 24 months or younger who had any of the following conditions: pulmonary hypoplasia, airway stenosis, congenital esophageal atresia, inherited metabolic disease, or neuromuscular disease. At least four continuous doses of palivizumab were administered intramuscularly at 15 mg/kg at intervals of 30 days.
UNASSIGNED: Twenty-three enrolled subjects completed the study. No subject required hospitalization for RSV. Adverse events (AE) did not notably differ from the event terms described in the latest interview form. Five severe AEs required unplanned hospitalization, but resolved without RSV infection. Therapeutically effective concentrations of palivizumab were maintained throughout the study period.
UNASSIGNED: Palivizumab might be well tolerated and effective in preventing serious respiratory symptoms and hospitalization due to severe RSV infection, indicating the prophylactic use in the pediatric patients included in this study.
UNASSIGNED: Japan Agency for Medical Research and Development (AMED), grant numbers 19lk0201097h0001 (to MM), 20lk0201097h0002 (to MM), 21lk0201097h0003 (to MM), and 22lk0201097h0004 (to MM). AMED did not have any role in the execution of this study, analysis and interpretation of the data, or the decision to submit the results.