Royal Manchester Children's Hospital, Manchester, UK.
Paediatric Respiratory Medicine, University of Manchester, Manchester, UK.
J Med Econ. 2020 Dec;23(12):1640-1652. doi: 10.1080/13696998.2020.1836923. Epub 2020 Oct 27.
Respiratory syncytial virus (RSV) is a common cause of respiratory infection in infants and severe infection can result in hospitalization. The passive immunization, palivizumab, is used as prophylaxis against RSV, however, use in the UK is restricted to populations at high risk of hospitalization. This study assesses the cost-effectiveness (CE) of palivizumab in premature infants with and without risk factors for hospitalization (congenital heart disease [CHD], bronchopulmonary dysplasia [BPD]).
A decision tree model, based on earlier CE analyses, was updated using data derived from targeted literature reviews and advice gained from a Round Table meeting. All costs were updated to 2019 prices. One-way and probabilistic sensitivity analyses were performed to assess the degree of uncertainty surrounding the results.
Palivizumab is dominant (i.e. clinically superior and cost saving) when used in premature infants born ≤35 weeks gestational age (wGA) without CHD or BPD and aged <6 months at the start of the RSV season, infants aged <24 months with CHD and infants aged <24 months requiring treatment for BPD within the last 6 months.
One-way sensitivity analysis suggests that these results are highly sensitive to the efficacy of prophylaxis, number of doses, impact of long-term respiratory sequalae, rate of hospitalization and mortality due to RSV. A conservative approach has been taken toward long-term respiratory sequalae due to uncertainty around epidemiology and etiology and a lack of recent cost and utility data.
Palivizumab prophylaxis is cost-effective in preventing severe RSV infection requiring hospital admission in a wider population than currently recommended in UK guidelines. Prophylaxis in premature infants born <29 wGA, 29-32 wGA and 33-35 wGA without CHD or BPD aged <6 months at the start of the RSV season is not funded under current guidance, however, prophylaxis has been demonstrated to be cost-effective in this analysis.
呼吸道合胞病毒（RSV）是婴儿呼吸道感染的常见病因，严重感染可导致住院。帕利珠单抗作为 RSV 的被动免疫制剂，被用于预防 RSV，但在英国的使用仅限于有住院高风险的人群。本研究评估了帕利珠单抗在有和无住院风险因素（先天性心脏病[CHD]、支气管肺发育不良[BPD]）的早产儿中的成本效益（CE）。
基于早期的 CE 分析，使用来自靶向文献综述的数据和圆桌会议获得的建议，更新了决策树模型。所有成本均更新至 2019 年价格。进行了单因素和概率敏感性分析，以评估结果的不确定性程度。
在无 CHD 或 BPD 的≤35 周胎龄（GA）早产儿和 RSV 季节开始时<6 月龄、有 CHD 的<24 月龄和在过去 6 个月内因 BPD 接受治疗的<24 月龄婴儿中，使用帕利珠单抗具有优势（即临床效果更好且节省成本）。
单因素敏感性分析表明，这些结果对预防效果、预防剂量、长期呼吸道后遗症的影响、因 RSV 导致的住院率和死亡率高度敏感。由于对流行病学和病因学的不确定性以及缺乏近期成本和效用数据，对长期呼吸道后遗症采取了保守的方法。
帕利珠单抗预防可在比英国指南目前建议更广泛的人群中预防需要住院治疗的严重 RSV 感染，具有成本效益。在无 CHD 或 BPD 的<29 周 GA、29-32 周 GA 和 33-35 周 GA 早产儿和 RSV 季节开始时<6 月龄的婴儿中，目前的指南不资助预防，但本分析表明预防具有成本效益。
