Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Kyoto, Japan.
Pediatr Int. 2020 Jun;62(6):677-682. doi: 10.1111/ped.14157. Epub 2020 Jun 1.
Palivizumab is a prophylactic drug used for severe respiratory syncytial virus (RSV) infection in young children with high-risk factors, such as prematurity and congenital heart diseases. Although Japan expanded the use of palivizumab for Down syndrome (DS) in 2013 regardless of additional high-risk factors, there is not enough evidence to support its use. We analyzed health insurance claim data from the JMDC (Tokyo, Japan) to investigate the prescription pattern and efficacy of palivizumab in children with DS.
We enrolled children of ≤24 months of age with DS at the start of the RSV epidemic season (2007-2015). We compared the risk of RSV-related admission with and without palivizumab prophylaxis using mixed effects logistic regression models accounting for patient-level clustering.
Of 632 children, 30% (81/268) and 83% (303/364) received palivizumab before and after the expanded program (2007-2012 and 2013-2015), respectively. Among children with DS but without additional high-risk factors (n = 135), palivizumab use surged from 0% (0/62) to 73% (53/73). In the whole study population, RSV-related hospitalization occurred in 4.2% (16/384) patients with prophylaxis and 6.0% (15/248) patients without prophylaxis. The multivariate analysis revealed that palivizumab was associated with reduced RSV-related hospitalization (odds ratio: 0.41, 95% confidence interval: 0.18-0.92, P = 0.03).
Virtually all children with DS in Japan have now received palivizumab, the use of which was associated with a reduction in RSV-related hospitalization in DS. Further evidence is required to clarify whether palivizumab prophylaxis should be risk-tailored or universal for DS.
帕利珠单抗是一种预防性药物，用于有早产和先天性心脏病等高危因素的幼儿严重呼吸道合胞病毒（RSV）感染。尽管日本在 2013 年扩大了帕利珠单抗在唐氏综合征（DS）中的使用范围，无论是否有其他高危因素，但目前尚无足够的证据支持其使用。我们分析了来自 JMDC（日本东京）的医疗保险索赔数据，以研究帕利珠单抗在 DS 儿童中的使用模式和疗效。
我们招募了在 RSV 流行季节（2007-2015 年）开始时年龄≤24 个月的 DS 儿童。我们使用混合效应逻辑回归模型，考虑到患者水平的聚类，比较了有和没有帕利珠单抗预防的 RSV 相关住院的风险。
在 632 名儿童中，2007-2012 年和 2013-2015 年分别有 30%（81/268）和 83%（303/364）的儿童接受了帕利珠单抗预防（扩大方案前和后）。在没有其他高危因素的 DS 儿童中（n=135），帕利珠单抗的使用率从 0%（0/62）飙升至 73%（53/73）。在整个研究人群中，接受预防治疗的 384 名患者中有 4.2%（16/384）发生了 RSV 相关住院，未接受预防治疗的 248 名患者中有 6.0%（15/248）发生了 RSV 相关住院。多变量分析显示，帕利珠单抗与 RSV 相关住院的减少相关（比值比：0.41，95%置信区间：0.18-0.92，P=0.03）。
现在，日本几乎所有的 DS 儿童都接受了帕利珠单抗治疗，该治疗与 DS 中 RSV 相关住院的减少有关。需要进一步的证据来阐明 DS 中帕利珠单抗预防是否应针对风险进行个体化或普遍化。
