scholarly journals The clinical effectiveness and cost-effectiveness of treatments for idiopathic pulmonary fibrosis: a systematic review and economic evaluation

2015 ◽  
Vol 19 (20) ◽  
pp. 1-336 ◽  
Author(s):  
Emma Loveman ◽  
Vicky R Copley ◽  
Jill Colquitt ◽  
David A Scott ◽  
Andy Clegg ◽  
...  

BackgroundIdiopathic 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.ObjectivesTo 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 sourcesEleven 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.MethodsTwo 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.ResultsFourteen studies were included in the review of clinical effectiveness, of which one evaluated azathioprine, threeN-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.LimitationsFew 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.ConclusionsFew 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.Study registrationThis study is registered as PROSPERO CRD42012002116.FundingThe National Institute for Health Research Health Technology Assessment programme.

2021 ◽  
Author(s):  
Fang Sun ◽  
Zhaochu Wu ◽  
Fengli Jiang ◽  
Mao Huang ◽  
Jine Wang ◽  
...  

Abstract Background Idiopathic pulmonary fibrosis (IPF) is a progressive, chronic, irreversible, and lethal interstitial lung disease (ILD) with unknown etiology occurring mainly in elderly. In recent years, the morbidity and mortality of IPF steady increase. However, there are currently no therapies that reverse fibrosis, the median survival time reported ranging from 2 to 5 years from diagnosis. The purpose of this systematic review and meta-analysis is to evaluate the effectiveness and safety of Chinese patent medicines as an adjuvant treatment in combination with Western medicines in the treatment of patients with IPF. Methods We will search electronic databases including PubMed, EMBASE, Cochrane Central, Web of Science, Chinese Biomedical Database (SinoMed), Chinese National Knowledge Infrastructure (CNKI), Chongqing VIP information (CQVIP), and Wanfang database using keywords related to IPF and Chinese patent medicine. Search was also carried out on ClinicalTrials.gov, International Clinical Trials Registry Platform (ICTRP) and the Chinese Clinical Trials Register (ChiCTR). Reference lists of literature included and relevant systematic reviews will be checked. Two independent reviewers will involved in screening studies, extracting data, and evaluating works. Any disagreements will be resolved by discussion or in consultation with a third reviewer. Data analysis will be conducted using the Review Manager software (version 5.4.1). The risk of bias of each study and the quality of evidence will be respectively assessed using Cochrane Risk of Bias tools and GRADE system. Discussion This review will integrate the current evidence and provide evidence regarding use of Chinese patent medicine as an adjuvant treatment in idiopathic pulmonary fibrosis to provide clarity regarding the potential benefits and best practice. Trial registration: PROSPERO CRD42021252806


BMJ Open ◽  
2019 ◽  
Vol 9 (5) ◽  
pp. e027849 ◽  
Author(s):  
Hiroyuki Kamiya ◽  
Ogee Mer Panlaqui

ObjectiveTo clarify clinical significance of the sole presence of autoantibodies for idiopathic pulmonary fibrosis (IPF) without any other symptoms or signs suggestive of autoimmune disease.DesignSystematic review and meta-analysisData sourcesMedline, EMBASE, Science Citation Index Expanded and Google Scholar were searched from 1 January 2002 through 12 February 2019.Eligibility criteria for selecting studiesPrimary studies addressing all-cause mortality and the development of a defined autoimmune disease for IPF with autoantibodies were included for the review.Data extraction and synthesisTwo reviewers extracted relevant data and assessed risk of bias independently. Meta-analysis was conducted using a random-effects model if three or more studies reported the same outcome for a certain autoantibody. The quality of evidence was assessed by the Grades of Recommendation, Assessment, Development and Evaluation system.ResultsOut of 4603 records retrieved nine studies were included in this review. All studies contained some risk of bias. Based on pooled data myeloperoxidase antineutrophil cytoplasmic antibody (MPO-ANCA) was significantly associated with microscopic polyangiitis incidence with risk ratio (RR) of 20.2 (95% CI: 7.22 to 56.4) and antinuclear antibody (ANA) was also significantly associated with the development of connective tissue diseases with RR of 7.11 (p=0.001) (10 cases in 157 patients with ANA) in one study. However, there was no significant association of autoantibodies with all-cause mortality aside from MPO-ANCA and proteinase 3-ANCA in one study each. MPO-ANCA was not demonstrated to be associated with all-cause mortality by meta-analysis. The quality of evidence was deemed as either low or very low.ConclusionsThe presence of autoantibodies such as MPO-ANCA and ANA was demonstrated to be associated with the development of some autoimmune diseases for patients with IPF although there was no difference of all-cause mortality. However, the results should be interpreted with caution due to low evidence level.PROSPERO registration numberCRD42017077336.


Author(s):  
Elena Aloisio ◽  
Federica Braga ◽  
Chiara Puricelli ◽  
Mauro Panteghini

Abstract Objectives Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial disease with limited therapeutic options. The measurement of Krebs von den Lungen-6 (KL-6) glycoprotein has been proposed for evaluating the risk of IPF progression and predicting patient prognosis, but the robustness of available evidence is unclear. Methods We searched Medline and Embase databases for peer-reviewed literature from inception to April 2020. Original articles investigating KL-6 as prognostic marker for IPF were retrieved. Considered outcomes were the risk of developing acute exacerbation (AE) and patient survival. Meta-analysis of selected studies was conducted, and quantitative data were uniformed as odds ratio (OR) or hazard ratio (HR) estimates, with corresponding 95% confidence intervals (CI). Results Twenty-six studies were included in the systematic review and 14 were finally meta-analysed. For AE development, the pooled OR (seven studies) for KL-6 was 2.72 (CI 1.22–6.06; p=0.015). However, a high degree of heterogeneity (I2=85.6%) was found among selected studies. Using data from three studies reporting binary data, a pooled sensitivity of 72% (CI 60–82%) and a specificity of 60% (CI 52–68%) were found for KL-6 measurement in detecting insurgence of AE in IPF patients. Pooled HR (seven studies) for mortality prediction was 1.009 (CI 0.983–1.036; p=0.505). Conclusions Although our meta-analysis suggested that IPF patients with increased KL-6 concentrations had a significant increased risk of developing AE, the detection power of the evaluated biomarker is limited. Furthermore, no relationship between biomarker concentrations and mortality was found. Caution is also needed when extending obtained results to non-Asian populations.


2021 ◽  
pp. 193229682110124
Author(s):  
Aaron Drovandi ◽  
Shannon Wong ◽  
Leonard Seng ◽  
Benjamin Crowley ◽  
Chanika Alahakoon ◽  
...  

Background: Diabetes-related foot disease (DFD) management requires input from multiple healthcare professionals, and has worse outcomes for people living in remote localities by comparison to urban areas. Remotely delivered healthcare may reduce this disparity. This overview summarizes current evidence on the effectiveness, stakeholder perceptions, and cost-effectiveness of remotely delivered healthcare for DFD. Methods: A search of 5 databases was conducted to identify systematic reviews published between January 2000 and June 2020. Eligible reviews were those evaluating remotely delivered monitoring or management of patients at risk of or with active DFD, or clinicians managing these patients. Risk of bias was assessed using the AMSTAR-2 tool. Results: Eight reviews were eligible for inclusion, including 88 primary studies and 8509 participants, of which 36 studies involving 4357 participants evaluated remotely delivered monitoring or management of DFD. Only one review had a low risk of bias, with most reviews demonstrating limited search strategies and poor reporting of participants. Evidence on effectiveness was mixed, with meta-analyses demonstrating long-term ulcer healing and mortality were not significantly different between telehealth and standard care groups, although the lower-limb amputation rate was significantly decreased in one meta-analysis. Perceptions of telehealth by patients and clinicians were generally positive, whilst acknowledging limitations relating to access and use. Cost-effectiveness data were limited, with poor reporting preventing clear conclusions. Conclusions: Remotely delivered healthcare of DFD is well received by patients and clinicians, but its effectiveness is unclear. High quality trials are needed to evaluate the risks and benefits of remotely delivered DFD management.


2021 ◽  
Vol 15 ◽  
pp. 175346662110280
Author(s):  
Roberto Ariel Abeldaño Zuñiga ◽  
Ruth Ana María González-Villoria ◽  
María Vanesa Elizondo ◽  
Anel Yaneli Nicolás Osorio ◽  
David Gómez Martínez ◽  
...  

Aims: Given the variability of previously reported results, this systematic review aims to determine the clinical effectiveness of convalescent plasma employed in the treatment of hospitalized patients diagnosed with COVID-19. Methods: We conducted a systematic review of controlled clinical trials assessing treatment with convalescent plasma for hospitalized patients diagnosed with SARS-CoV-2 infection. The outcomes were mortality, clinical improvement, and ventilation requirement. Results: A total of 51 studies were retrieved from the databases. Five articles were finally included in the data extraction and qualitative and quantitative synthesis of results. The overall risk of bias in the reviewed articles was established at low-risk only in two trials. The meta-analysis suggests that there is no benefit of convalescent plasma compared with standard care or placebo in reducing the overall mortality and the ventilation requirement. However, there could be a benefit for the clinical improvement in patients treated with plasma. Conclusion: Current results led to assume that the convalescent plasma transfusion cannot reduce the mortality or ventilation requirement in hospitalized patients diagnosed with SARS-CoV-2 infection. More controlled clinical trials conducted with methodologies that ensure a low risk of bias are still needed. The reviews of this paper are available via the supplemental material section.


BMJ Open ◽  
2021 ◽  
Vol 11 (12) ◽  
pp. e050004
Author(s):  
Wenjuan Wu ◽  
Lingxiao Qiu ◽  
Jizhen Wu ◽  
Xueya Liu ◽  
Guojun Zhang

ObjectivesIdiopathic pulmonary fibrosis (IPF) has been defined as a distinctive type of chronic fibrotic disease, characterised by a progressive decline in lung function and a common histological pattern of interstitial pneumonia. To analyse the efficacy and safety of pirfenidone in the treatment of IPF, a systematic review and meta-analysis was performed.DesignThis is a meta-analysis study.ParticipantsPatients were diagnosed as IPF.InterventionsUse of pirfenidone.Primary and secondary outcomeProgression-free survival (PFS), acute exacerbation and worsening of IPF and Impact on adverse events.MeasuresThe inverse variance method for the random-effects model was used to summarise the dichotomous outcomes, risk ratios and 95% CIs.ResultsA total of 9 randomised controlled trials with 1011 participants receiving pirfenidone and 912 controls receiving placebo were summarised. The pooled result suggested a statistically significant difference inall-cause mortality after pirfenidone use, with a summarised relative ratio of 0.51 (p<0.01). Longer PFS was observed in patients receiving pirfenidone compared with those who were given placebo (p<0.01). The IPF groups presented a high incidence of adverse events with a pooled relative ratio of 3.89 (p<0.01).ConclusionsPirfenidone can provide survival benefit for patients with IPF. Pirfenidone treatment was also associated with a longer PFS, a lower incidence of acute exacerbation and worsening of IPF.


2020 ◽  
pp. 106002802096445
Author(s):  
Enrica Di Martino ◽  
Alessio Provenzani ◽  
Patrizio Vitulo ◽  
Piera Polidori

Background: The comparative efficacy of pirfenidone, nintedanib, and pamrevlumab in slowing the rate of forced vital capacity (FVC) decline and mortality in patients with idiopathic pulmonary fibrosis (IPF) is unknown. Objective: To perform a systematic review and meta-analysis (MA) of these drugs for IPF. Methods: We searched CENTRAL, PubMed, EMBASE, ClincalTrials.gov, and the World Health Organization’s registry databases up to March 2020. Phase II/III randomized controlled trials in adults with IPF were eligible. The random-effect model was implemented calculating the effect size and respective 95% CI as Cohen’s d for change from baseline FVC (in percentage predicted and liters) and odds ratio (OR) for 10% reduction in FVC and all-cause mortality (ACM). Results: Six studies were included in the MA. For change from baseline in percentage predicted FVC, the MA indicated that the 3 drugs were more effective than placebo (pirfenidone: d=3.30%, 95% CI=2.15-4.45; nintedanib: d=3.15%, 95% CI=2.35-3.95; pamrevlumab: d=4.30%, 95% CI=0.45-8.15). These results are superimposable to those relating to change from baseline FVC in liters (pirfenidone: d=0.09L, 95% CI=0.04-0.14; nintedanib: d=0.13L, 95% CI=0.10-0.16; pamrevlumab: d=0.20L, 95% CI=0.05-0.35). Each drug had a positive effect on 10% reduction in FVC (pirfenidone: OR=0.57, 95% CI=0.45-0.74; nintedanib: OR=0.66, 95% CI=0.51-0.85; pamrevlumab: OR=0.24, 95% CI=0.08-0.73), but only pirfenidone showed an effect on ACM (OR=0.50; 95% CI=0.31-0.83). Conclusion and Relevance: This MA provided encouraging results on pamrevlumab efficacy in slowing the decline in FVC compared with pirfenidone and nintedanib. Actually, in phase 3, it could become a potential IPF treatment.


2015 ◽  
Vol 10 (2) ◽  
pp. 150
Author(s):  
Joanne L. Jordan

A Review of: Westphal, A., Kriston, L., Hölzel, L.P., Härter, M., & von Wolff, A. (2014). Efficiency and contribution of strategies for finding randomized controlled trials: a case study from a systematic review on therapeutic interventions of chronic depression. Journal of Public Health Research, 3(2), 177. doi: 10.4081/jphr.2014.177 Abstract Objective – To evaluate the efficiency and contribution of additional searching strategies for finding randomized controlled trials (RCTs) in a systematic review. Design – A methodological case study. Setting – Biomedical literature. Methods – A sensitive search (defined as “the ratio of the number of relevant reports identified to the total number of relevant reports in existence”) was conducted of electronic databases, Cochrane CENTRAL database, MEDLINE, EMBASE, PsycInfo, CINAHL, BIOSIS, and Web of Science databases (Science and Social Science Citation Indexes). The following additional searching strategies were conducted: hand-searching contents of relevant journals (Archives of General Psychiatry, Journal of Consulting and Clinical Psychology, and Journal of Affective Disorders), citation tracking (forwards tracking using Social Science and Science Citation Index and backwards tracking by looking through reference lists of included studies), screening reference lists of relevant systematic reviews, searching clinical trials registers (ClinicalTrials.gov and ICTRP registers), and contacting first authors of included studies to find any similar unpublished studies. The number of articles identified by each of these methods was recorded and screened for inclusion in the systematic review. The authors calculated what they labelled as the ‘efficiency’ of each searching strategy (the number of included studies identified by the search method as a proportion of the full text articles screened) and the ‘contribution’ of the search strategies (the ratio of included studies identified by that method to the final number of included studies in the systematic review). The methodological quality of each included study was assessed using the Cochrane Risk of Bias Tool, which is a critical appraisal checklist used to judge the study’s value in the systematic review. The meta-analysis in the systematic review was conducted with and without the studies identified by the additional searching strategies to assess their impact on the review’s findings. Main Results – In total 50 studies were identified, 42 from electronic database searches and 8 from additional search strategies. As illustrated by the results in Table 1, the most useful additional search strategy was screening reference lists of relevant systematic reviews. Journal hand-searching and contacting authors also contributed to the review. Of the eight studies identified by the additional search strategies none were judged to have a low risk of bias (four had high risk of bias and four were unclear). Of the 42 included studies from electronic searches only 11 were judged to have a low risk of bias, whereas 9 studies had a high risk of bias and 22 were unclear. Excluding the eight studies retrieved from additional search strategies in the systematic review meta-analysis did not influence the results on the effectiveness of the different interventions for chronic depression. These studies were found to be indexed correctly on the electronic databases, but were not identified in the initial search. Conclusion – Additional search strategies, especially screening reference lists of systematic reviews and hand-searching relevant journals, retrieved a substantial number of relevant studies for a systematic review of interventions for treating chronic depression. However, results of the review’s meta-analysis did not differ when these additional studies (rated as either high or unclear risk of bias) were not included and search methods were time consuming. It might be reasonable to rely on electronic searching strategies when resources for conducting a systematic review are limited or when doing a “rapid review.” The benefits and limitations of additional search strategies should be considered particularly when resources or time for conducting a systematic review are limited. If the electronic database search is sensitive and includes the Cochrane CENTRAL database additional search strategies may not be necessary, but these findings should be tested in other research areas.


2019 ◽  
Vol 8 (1) ◽  
pp. 55-61 ◽  
Author(s):  
Aristeidis Skandamis ◽  
Chara Kani ◽  
Sophia L. Markantonis ◽  
Kyriakos Souliotis

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