scholarly journals The assessment of new drugs for asthma and COPD: a Delphi study examining the perspectives of Italian payers and clinicians

2016 ◽  
Vol 11 ◽  
Author(s):  
Silvia Sommariva ◽  
Aureliano P. Finch ◽  
Claudio Jommi
Keyword(s):  
Delphi Study ◽  
Treatment Options ◽  
Airflow Obstruction ◽  
Real Life ◽  
New Drugs ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Important Place ◽  
Consensus Document ◽  
Critical Issues

Background: Asthma and chronic obstructive pulmonary disease (COPD) are disorders of the lungs characterized by airflow obstruction, inflammation and tissue remodeling. Management of patients with these diseases is complex and the improvement of diagnostic-therapeutic strategies represents a critical challenge for the healthcare system. In this context, investigating the criteria and information needed for an appropriate and effective evaluation of incoming treatment options is crucial to ensure that clinicians and policy-makers are provided with the best available evidence to make decisions aimed at improving patient outcomes. Therefore, the objective of this study was to investigate the degree of agreement among Health Technology Assessment (HTA) experts on issues crucial to the evaluation of new drugs for asthma and COPD and to appropriately manage the clinical pathway for patients. Methods: This research was conducted using an e-Delphi technique organized in three subsequent rounds and involving a panel of ten experts (six regional and local payers and four clinicians). Panelists were asked to comment in written form on a set of statements, explaining qualitatively the extent to which they agreed or disagreed with the assertions. Statements were subsequently modified and resubmitted for assessment. Results: Panelists expressed their opinions during each round and, after round III, a consensus document was finalized. The degree of consensus was high among experts and concerned five main topics: (a) the need to address current unmet needs of patients with asthma or COPD, (b) the importance of further studies and real-life information in the evaluation of treatments, (c) existing evidence and evidence needed to assess drugs, (d) critical issues in obtaining a positive evaluation from regional and local authorities for new treatments to be included in regional formularies and to have an important place in therapeutic categories, and (e) the major obstacles to the appropriate administration of drugs and management of patients. Conclusion: The final document highlights that no proof of difference among drugs exists, that evidence on final endpoints (and particularly on mortality) should be strengthened and that actions regarding risk factors, appropriate diagnosis, patient staging and adherence to therapy are particularly important for a better clinical management.

scholarly journals Clinically relevant subgroups in COPD and asthma

2015 ◽  
Vol 24 (136) ◽  
pp. 283-298 ◽  
Author(s):  
Alice M. Turner ◽  
Lilla Tamasi ◽  
Florence Schleich ◽  
Mehmet Hoxha ◽  
Ildiko Horvath ◽  
...  
Keyword(s):  
Clinical Characteristics ◽  
Treatment Options ◽  
New Drugs ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
The Past ◽  
Asthma Patients ◽  
Prognostic Implications ◽  
Changes Over Time ◽  
Over Time

As knowledge of airways disease has grown, it has become apparent that neither chronic obstructive pulmonary disease (COPD) nor asthma is a simple, easily defined disease. In the past, treatment options for both diseases were limited; thus, there was less need to define subgroups. As treatment options have grown, so has our need to predict who will respond to new drugs. To date, identifying subgroups has been largely reported by detailed clinical characterisation or differences in pathobiology. These subgroups are commonly called “phenotypes”; however, the problem of defining what constitutes a phenotype, whether this should include comorbid diseases and how to handle changes over time has led to the term being used loosely.In this review, we describe subgroups of COPD and asthma patients whose clinical characteristics we believe have therapeutic or major prognostic implications specific to the lung, and whether these subgroups are constant over time. Finally, we will discuss whether the subgroups we describe are common to both asthma and COPD, and give some examples of how treatment might be tailored in patients where the subgroup is clear, but the label of asthma or COPD is not.

scholarly journals Mechanisms, Pathophysiology and Currently Proposed Treatments of Chronic Obstructive Pulmonary Disease

2021 ◽  
Vol 14 (10) ◽  
pp. 979
Author(s):  
Sarah de Oliveira Rodrigues ◽  
Carolina Medina Coeli da Cunha ◽  
Giovanna Martins Valladão Soares ◽  
Pedro Leme Silva ◽  
Adriana Ribeiro Silva ◽  
...  
Keyword(s):  
Chronic Obstructive Pulmonary Disease ◽  
Disease Progression ◽  
Pulmonary Disease ◽  
Inhaled Corticosteroids ◽  
Treatment Options ◽  
Airflow Obstruction ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Inflammatory Microenvironment ◽  
Cell Functions

Chronic obstructive pulmonary disease (COPD) is one of the leading global causes of morbidity and mortality. A hallmark of COPD is progressive airflow obstruction primarily caused by cigarette smoke (CS). CS exposure causes an imbalance favoring pro- over antioxidants (oxidative stress), leading to transcription factor activation and increased expression of inflammatory mediators and proteases. Different cell types, including macrophages, epithelial cells, neutrophils, and T lymphocytes, contribute to COPD pathophysiology. Alteration in cell functions results in the generation of an oxidative and inflammatory microenvironment, which contributes to disease progression. Current treatments include inhaled corticosteroids and bronchodilator therapy. However, these therapies do not effectively halt disease progression. Due to the complexity of its pathophysiology, and the risk of exacerbating symptoms with existing therapies, other specific and effective treatment options are required. Therapies directly or indirectly targeting the oxidative imbalance may be promising alternatives. This review briefly discusses COPD pathophysiology, and provides an update on the development and clinical testing of novel COPD treatments.

scholarly journals Chronic Obstructive Pulmonary Disease

2016 ◽  
Vol 11 (4) ◽  
pp. 296-302 ◽  
Author(s):  
Sean O’Reilly
Keyword(s):  
Tobacco Smoke ◽  
Treatment Options ◽  
Airflow Obstruction ◽  
Clinical History ◽  
Pulmonary Function Testing ◽  
Accurate Diagnosis ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Common Cause ◽  
Function Testing

COPD is a common, preventable, and treatable disease characterized by persistent airflow obstruction associated with enhanced inflammation in the airways and the lung in response to noxious particles or gases. Clinical history and pulmonary function testing are necessary for accurate diagnosis. While exposure to tobacco smoke remains a common cause, other etiologies and underlying genetic predisposition play significant roles. Treatment options are numerous and should be individualized based on symptoms and exacerbation frequency.

scholarly journals Enhanced Transcriptomic Resilience following Increased Alternative Splicing and Differential Isoform Production between Air Pollution Conurbations

Atmosphere ◽  
2021 ◽  
Vol 12 (8) ◽  
pp. 959
Author(s):  
Shengkai Pan ◽  
Xiaokai Feng ◽  
Daniel Pass ◽  
Rachel A. Adams ◽  
Yusong Wang ◽  
...  
Keyword(s):  
Air Pollution ◽  
Alternative Splicing ◽  
Real Life ◽  
Chronic Obstructive ◽  
Translational Efficiency ◽  
Ambient Particulate Matter ◽  
Obstructive Pulmonary Disease ◽  
Adverse Health Outcomes ◽  
Membrane Bound ◽  
Migration Capacity

Adverse health outcomes caused by ambient particulate matter (PM) pollution occur in a progressive process, with neutrophils eliciting inflammation or pathogenesis. We investigated the toxico-transcriptomic mechanisms of PM in real-life settings by comparing healthy residents living in Beijing and Chengde, the opposing ends of a well-recognised air pollution (AP) corridor in China. Beijing recruits (BRs) uniquely expressed ~12,000 alternative splicing (AS)-derived transcripts, largely elevating the proportion of transcripts significantly correlated with PM concentration. BRs expressed PM-associated isoforms (PMAIs) of PFKFB3 and LDHA, encoding enzymes responsible for stimulating and maintaining glycolysis. PMAIs of PFKFB3 featured different COOH-terminals, targeting PFKFB3 to different sub-cellular functional compartments and stimulating glycolysis. PMAIs of LDHA have longer 3′UTRs relative to those expressed in Chengde recruits (CRs), allowing glycolysis maintenance by enhancing LDHA mRNA stability and translational efficiency. PMAIs were directly regulated by different HIF-1A and HIF-1B isoforms. BRs expressed more non-functional Fas isoforms, and a resultant reduction of intact Fas proportion is expected to inhibit the transmission of apoptotic signals and prolong neutrophil lifespan. BRs expressed both membrane-bound and soluble IL-6R isoforms instead of only one in CRs. The presence of both IL-6R isoforms suggested a higher migration capacity of neutrophils in BRs. PMAIs of HIF-1A and PFKFB3 were downregulated in Chronic Obstructive Pulmonary Disease patients compared with BRs, implying HIF-1 mediated defective glycolysis may mediate neutrophil dysfunction. PMAIs could explain large variances of different phenotypes, highlighting their potential application as biomarkers and therapeutic targets in PM-induced diseases, which remain poorly elucidated.

scholarly journals Protease-Specific Biomarkers to Analyse Protease Inhibitors for Emphysema Associated with Alpha 1-Antitrypsin Deficiency. An Overview of Current Approaches

2021 ◽  
Vol 22 (3) ◽  
pp. 1065
Author(s):  
Simona Viglio ◽  
Elisabeth G. Bak ◽  
Iris G. M. Schouten ◽  
Paolo Iadarola ◽  
Jan Stolk
Keyword(s):  
Serine Proteases ◽  
Lung Parenchyma ◽  
New Drugs ◽  
Chronic Obstructive ◽  
Augmentation Therapy ◽  
Lung Function Decline ◽  
Obstructive Pulmonary Disease ◽  
Antitrypsin Deficiency ◽  
Novel Biomarkers ◽  
Alpha1 Antitrypsin

As a known genetic cause of chronic obstructive pulmonary disease (COPD), alpha1-antitrypsin deficiency (AATD) can cause severe respiratory problems at a relatively young age. These problems are caused by decreased or absent levels of alpha1-antitrypsin (AAT), an antiprotease which is primarily functional in the respiratory system. If the levels of AAT fall below the protective threshold of 11 µM, the neutrophil-derived serine proteases neutrophil elastase (NE) and proteinase 3 (PR3), which are targets of AAT, are not sufficiently inhibited, resulting in excessive degradation of the lung parenchyma, increased inflammation, and increased susceptibility to infections. Because other therapies are still in the early phases of development, the only therapy currently available for AATD is AAT augmentation therapy. The controversy surrounding AAT augmentation therapy concerns its efficiency, as protection of lung function decline is not demonstrated, despite the treatment’s proven significant effect on lung density change in the long term. In this review article, novel biomarkers of NE and PR3 activity and their use to assess the efficacy of AAT augmentation therapy are discussed. Furthermore, a series of seven synthetic NE and PR3 inhibitors that can be used to evaluate the specificity of the novel biomarkers, and with potential as new drugs, are discussed.

scholarly journals Cell therapy for lung disease

2017 ◽  
Vol 26 (144) ◽  
pp. 170044 ◽  
Author(s):  
Sabine Geiger ◽  
Daniela Hirsch ◽  
Felix G. Hermann
Keyword(s):  
Clinical Studies ◽  
Therapeutic Approach ◽  
Lung Diseases ◽  
Distress Syndrome ◽  
Treatment Options ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Cell Therapies ◽  
Promising Therapeutic Approach ◽  
Preclinical And Clinical Studies

Besides cancer and cardiovascular diseases, lung disorders are a leading cause of morbidity and death worldwide. For many disease conditions no effective and curative treatment options are available. Cell therapies offer a novel therapeutic approach due to their inherent anti-inflammatory and anti-fibrotic properties. Mesenchymal stem/stromal cells (MSC) are the most studied cell product. Numerous preclinical studies demonstrate an improvement of disease-associated parameters after MSC administration in several lung disorders, including chronic obstructive pulmonary disease, acute respiratory distress syndrome and idiopathic pulmonary fibrosis. Furthermore, results from clinical studies using MSCs for the treatment of various lung diseases indicate that MSC treatment in these patients is safe. In this review we summarise the results of preclinical and clinical studies that indicate that MSCs are a promising therapeutic approach for the treatment of lung diseases. Nevertheless, further investigations are required.

scholarly journals Implementation of a Targeted Screening Program to Detect Airflow Obstruction Suggestive of Chronic Obstructive Pulmonary Disease within a Presurgical Screening Clinic

2015 ◽  
Vol 22 (4) ◽  
pp. 209-214 ◽  
Author(s):  
Chantal Robitaille ◽  
Esther Dajczman ◽  
Andrew M Hirsch ◽  
David Small ◽  
Pierre Ernst ◽  
...  
Keyword(s):  
Chronic Obstructive Pulmonary Disease ◽  
Pulmonary Disease ◽  
Screening Program ◽  
Tertiary Care ◽  
Airflow Obstruction ◽  
University Hospital ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Targeted Screening ◽  
History Of

BACKGROUND: Targeted spirometry screening for chronic obstructive pulmonary disease (COPD) has been studied in primary care and community settings. Limitations regarding availability and quality of testing remain. A targeted spirometry screening program was implemented within a presurgical screening (PSS) clinic to detect undiagnosed airways disease and identify patients with COPD/asthma in need of treatment optimization.OBJECTIVE: The present quality assurance study evaluated airflow obstruction detection rates and examined characteristics of patients identified through the targeted screening program.METHODS: The targeted spirometry screening program was implemented within the PSS clinic of a tertiary care university hospital. Current or ex-smokers with respiratory symptoms and patients with a history of COPD or asthma underwent prebronchodilator spirometry. History of airways disease and smoking status were obtained during the PSS assessment and confirmed through chart reviews.RESULTS: After exclusions, the study sample included 449 current or ex-smokers. Abnormal spirometry results were found in 184 (41%) patients: 73 (16%) had mild, 93 (21%) had moderate and 18 (4%) had severe or very severe airflow obstruction. One hundred eighteen (26%) new cases of airflow obstruction suggestive of COPD were detected. One-half of these new cases had moderate or severe airflow obstruction. Only 34% of patients with abnormal spirometry results had reported a previous diagnosis of COPD. More than one-half of patients with abnormal spirometry results were current smokers.CONCLUSIONS: Undiagnosed airflow obstruction was detected in a significant number of smokers and ex-smokers through a targeted screening program within a PSS clinic. These patients can be referred for early intervention and secondary preventive strategies.

scholarly journals Household Food Insecurity, Lung Function, and COPD in US Adults

Nutrients ◽  
2021 ◽  
Vol 13 (6) ◽  
pp. 2098
Author(s):  
Francisca de Castro Mendes ◽  
Kirstie Ducharme-Smith ◽  
Gustavo Mora-Garcia ◽  
Saleh A. Alqahtani ◽  
Maria Stephany Ruiz-Diaz ◽  
...  
Keyword(s):  
Lung Function ◽  
Food Insecurity ◽  
Representative Sample ◽  
Airflow Obstruction ◽  
Forced Expiratory Volume ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
The Us ◽  
Nationally Representative ◽  
Lung Health

Increasing epidemiological evidence suggests that optimal diet quality helps to improve preservation of lung function and to reduce chronic obstructive pulmonary disease (COPD) risk, but no study has investigated the association of food insecurity (FI) and lung health in the general population. Using data from a representative sample of US adults who participated in the National Health and Nutrition Examination Survey (NHANES) 2007–2012 cycles, we investigated the association between FI with lung function and spirometrically defined COPD in 12,469 individuals aged ≥ 18 years of age. FI (high vs. low) was defined using the US Department of Agriculture’s Food Security Scale). Population-weighted adjusted regression models were used to investigate associations between FI, and forced expiratory volume in 1 s (FEV1), forced vital capacity (FVC), their ratio, and spirometrically defined restriction (FVC below the lower limit of normal) and airflow obstruction (COPD). The prevalence of household FI was 13.2%. High household FI was associated with lower FVC (adjusted β-coefficient −70.9 mL, 95% CI −116.6, −25.3), and with higher odds (OR) of spirometric restriction (1.02, 95% CI 1.00, 1.03). Stratified analyses showed similar effect sizes within specific ethnic groups. High FI was associated with worse lung health in a nationally representative sample of adults in the US.

scholarly journals Chronic obstructive pulmonary disease Assessment Test na avaliação de pacientes com doença pulmonar obstrutiva crônica em reabilitação pulmonar: há relação com nível de dispneia nas atividades de vida diária e com índice preditor de mortalidade? Estudo transversal

2013 ◽  
Vol 20 (4) ◽  
pp. 379-386
Author(s):  
Júlia Gianjoppe-Santos ◽  
Samantha Maria Nyssen ◽  
Bruna Varanda Pessoa ◽  
Renata Pedrolongo Basso-Vanelli ◽  
Mauricio Jamami ◽  
...  
Keyword(s):  
Daily Living ◽  
Research Council ◽  
Medical Research Council ◽  
Airflow Obstruction ◽  
Disease Assessment ◽  
Chronic Obstructive ◽  
Activity Of Daily Living ◽  
Obstructive Pulmonary Disease ◽  
Copd Assessment Test ◽  
Indice Bode

O objetivo do estudo foi investigar se há relação entre o impacto da Doença Pulmonar Obstrutiva Crônica (DPOC) no estado de saúde com o nível de dispneia nas atividades de vida diária (AVD) e o índice preditor de mortalidade em pacientes em reabilitação pulmonar (RP). Trata-se de um estudo transversal, em que foram avaliados 32 pacientes com DPOC moderada a muito grave (23 homens; 66,6±12,0 anos; VEF1: 40,6±15,6% previsto) por meio do COPD Assessment Test (CAT), Índice de Massa Corpórea (IMC), Teste de Caminhada de Seis Minutos (TC6), London Chest Activity of Daily Living Scale (LCADL), modified Medical Research Council (mMRC) e Índice BODE (Body mass index, airflow Obstruction, Dyspnea and Exercise capacity). Observaram-se correlações positivas moderadas do CAT com o questionário mMRC (r=0,35; p=0,048), a pontuação total da LCADL (r=0,60; p<0,001) e com a porcentagem da pontuação total da LCADL (r=0,57; p=0,001). Apenas a pontuação total da LCADL é capaz de predizer independentemente a pontuação do questionário CAT (p<0,05; r²=0,61). Não foram constatadas correlações significativas entre o CAT e o Índice BODE, IMC, TC6 e VEF1. Dessa forma, quanto maior o nível de dispneia nas AVD, maior o impacto da DPOC no estado de saúde do paciente, entretanto, o mesmo não ocorre em relação ao prognóstico de mortalidade nos pacientes com DPOC em RP.

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