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帕利珠单抗预防小儿骨髓移植后呼吸道合胞病毒致死率:一项决策分析模型

Palivizumab prophylaxis to prevent respiratory syncytial virus mortality after pediatric bone marrow transplantation: a decision analysis model.

作者信息

Thomas Neal J, Hollenbeak Christopher S, Ceneviva Gary D, Geskey Joseph M, Young Mark J

机构信息

Department of Pediatrics, Division of Pediatric Critical Care Medicine, Penn State Children's Hospital, The Pennsylvania State University College of Medicine, Hershey, Pennsylvania 17033, USA.

出版信息

J Pediatr Hematol Oncol. 2007 Apr;29(4):227-32. doi: 10.1097/MPH.0b013e3180437ded.

Abstract

OBJECTIVE

Palivizumab, a monoclonal antibody against respiratory syncytial virus (RSV), has been demonstrated to be safe and effective in young children, but evidence is lacking as to whether palivizumab is effective in preventing RSV-induced morbidity and mortality in children who are immunosuppressed after bone marrow transplantation (BMT). As a randomized, double-blind, placebo-controlled trial is lacking, we chose to examine this issue with the use of decision analysis methodology.

METHODS

A decision tree was designed to determine mortality from RSV-related lung disease in children who received palivizumab after BMT. Probabilities were derived by meta-analysis methodology on the basis of the available literature. Sensitivity analyses were performed across a broad range of biologically plausible probabilities to judge the robustness of the results of the model.

RESULTS

The model revealed that there is a 10% increase in survival in BMT patients who receive palivizumab. The absolute survival rate increased from 83% to 92%. A practitioner would need to treat 12 children to save 1 post-BMT child from dying from RSV-related lung disease.

CONCLUSIONS

Decision analysis modeling demonstrates a decrease in mortality in pediatric BMT patients with the addition of palivizumab to protect against RSV-related lung disease. A well-designed, randomized controlled trial is necessary.

摘要

目的

帕利珠单抗是一种抗呼吸道合胞病毒(RSV)的单克隆抗体,已被证明对幼儿安全有效,但对于帕利珠单抗在预防骨髓移植(BMT)后免疫抑制儿童的RSV诱导的发病和死亡方面是否有效,尚缺乏证据。由于缺乏随机、双盲、安慰剂对照试验,我们选择使用决策分析方法来研究这个问题。

方法

设计了一个决策树,以确定BMT后接受帕利珠单抗治疗的儿童因RSV相关肺部疾病导致的死亡率。概率是根据现有文献通过荟萃分析方法得出的。在广泛的生物学上合理的概率范围内进行敏感性分析,以判断模型结果的稳健性。

结果

该模型显示,接受帕利珠单抗治疗的BMT患者生存率提高了10%。绝对生存率从83%提高到了92%。一名医生需要治疗12名儿童,才能使1名BMT后儿童免于死于RSV相关肺部疾病。

结论

决策分析模型表明,在儿科BMT患者中添加帕利珠单抗以预防RSV相关肺部疾病可降低死亡率。有必要进行一项精心设计的随机对照试验。

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