The clinical effectiveness and cost-effectiveness of treatments for idiopathic pulmonary fibrosis: a systematic review and economic evaluation
The clinical effectiveness and cost-effectiveness of treatments for idiopathic pulmonary fibrosis: a systematic review and economic evaluation
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DOI：
10.3310/hta19200
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发表时间：
2015-03-01
影响因子：
3.6
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中科院分区：
文献类型：
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背景：特发性肺纤维化（IPF）是一种限制生命的肺部疾病，通常影响60岁以上的人群。主要症状是呼吸急促和咳嗽，随着疾病的发展，对日常生活产生相当大的影响。目的：基于经济模型，对IPF的临床有效性进行系统评价，并对IPF治疗的成本-效果进行分析，该经济模型由成本-效果和生活质量的系统评价提供信息。数据来源：11个电子书目数据库，包括MEDLINE、EMBASE、Web of Science、科克伦图书馆和评论与传播中心数据库，从数据库建立到2013年7月进行了检索。方法：由两名评价者独立筛选系统评价的参考文献，提取并检查纳入研究的数据，并评估其偏倚风险。就干预措施的选择咨询了一个咨询小组，直到就资格达成共识。采用荟萃分析进行了叙述性综述，并进行了网络荟萃分析（NMA）。开发了一种决策分析马尔可夫模型，以估计IPF药物治疗的成本-效果。参数值来自NMA和系统综述。进行了单变量和概率敏感性分析。模型的视角是NHS和个人社会服务，成本和健康福利的贴现率为3.5%。14项研究被纳入临床有效性审查，其中1项评价了硫唑嘌呤、3项N-乙酰半胱氨酸（NAC）（单药或联合用药）、4项吡非尼酮、1项BIBF 1120、1项西地那非、1项沙利度胺、2项肺康复和1项疾病管理项目。研究质量总体良好，偏倚风险较低。目前的证据表明，一些治疗似乎是临床有效的。模型基础病例结果显示，与最佳支持治疗相比，五种药物治疗的生存率增加，成本增加。一般的建议，不能作出其成本效益，由于在evidencebased.Limitations的限制：很少有直接比较的治疗方法。通过NMA进行了间接比较;但是，建议在解释这些结果时谨慎。与经济模型相关，假设药物治疗对预测的用力肺活量下降的相对百分比具有恒定的影响。结论：很少有干预措施对IPF具有统计学显着影响，并且缺乏关于姑息治疗方法的研究。需要研究症状控制干预措施的效果，特别是肺康复和沙利度胺。其他研究重点包括一项关于吸入性NAC治疗的随机对照试验，以及正在进行的研究结果报告后的最新证据合成。
Background: Idiopathic pulmonary fibrosis (IPF) is a life-limiting lung disease that generally affects people over 60 years old. The main symptoms are shortness of breath and cough, and as the disease progresses there is a considerable impact on day-to-day life. Few treatments are currently available.Objectives: To conduct a systematic review of clinical effectiveness and an analysis of cost-effectiveness of treatments for IPF based on an economic model informed by systematic reviews of cost-effectiveness and quality of life.Data sources: Eleven electronic bibliographic databases, including MEDLINE, EMBASE, Web of Science, and The Cochrane Library and the Centre for Reviews and Dissemination databases, were searched from database inception to July 2013. Reference lists of relevant publications were also checked and experts consulted.Methods: Two reviewers independently screened references for the systematic reviews, extracted and checked data from the included studies and appraised their risk of bias. An advisory group was consulted about the choice of interventions until consensus was reached about eligibility. A narrative review with meta-analysis was undertaken, and a network meta-analysis (NMA) was performed. A decision-analytic Markov model was developed to estimate cost-effectiveness of pharmacological treatments for IPF. Parameter values were obtained from NMA and systematic reviews. Univariate and probabilistic sensitivity analyses were undertaken. The model perspective is NHS and Personal Social Services, and discount rate is 3.5% for costs and health benefits.Results: Fourteen studies were included in the review of clinical effectiveness, of which one evaluated azathioprine, three N-acetylcysteine (NAC) (alone or in combination), four pirfenidone, one BIBF 1120, one sildenafil, one thalidomide, two pulmonary rehabilitation, and one a disease management programme. Study quality was generally good, with a low risk of bias. The current evidence suggests that some treatments appear to be clinically effective. The model base-case results show increased survival for five pharmacological treatments, compared with best supportive care, at increased cost. General recommendations cannot be made of their cost-effectiveness owing to limitations in the evidence base.Limitations: Few direct comparisons of treatments were identified. An indirect comparison through a NMA was performed; however, caution is recommended in the interpretation of these results. In relation to the economic model, there is an assumption that pharmacological treatments have a constant effect on the relative rate of per cent predicted forced vital capacity decline.Conclusions: Few interventions have any statistically significant effect on IPF and a lack of studies on palliative care approaches was identified. Research is required into the effects of symptom control interventions, in particular pulmonary rehabilitation and thalidomide. Other research priorities include a well-conducted randomised controlled trial on inhaled NAC therapy and an updated evidence synthesis once the results of ongoing studies are reported.