George Kassianos, Jean-Marie Cohen, R. Čivljak, N. Davidovitch, Oana Falup Pecurariu, Filipe Froes, Andrei Galev, Inga Ivaskeviciene, Kadri Kõivumägi, Zuzana Kristufkova, Ernest Kuchar, Jan Kyncl, H. Maltezou, Miloš Marković, A. Nitsch-Osuch, Raul Ortiz de Lejarazu, Alessandro Rossi, Jörg Schelling, G. A. van Essen, D. Zavadska
{"title":"The influenza landscape and vaccination coverage in older adults during the SARS-Cov-2 pandemic: data from Several European Countries and Israel.","authors":"George Kassianos, Jean-Marie Cohen, R. Čivljak, N. Davidovitch, Oana Falup Pecurariu, Filipe Froes, Andrei Galev, Inga Ivaskeviciene, Kadri Kõivumägi, Zuzana Kristufkova, Ernest Kuchar, Jan Kyncl, H. Maltezou, Miloš Marković, A. Nitsch-Osuch, Raul Ortiz de Lejarazu, Alessandro Rossi, Jörg Schelling, G. A. van Essen, D. Zavadska","doi":"10.1080/17476348.2024.2340470","DOIUrl":"https://doi.org/10.1080/17476348.2024.2340470","url":null,"abstract":"INTRODUCTION\u0000The Raise Awareness of Influenza Strategies in Europe (RAISE) group gathered information about the healthcare burden of influenza (hospitalizations, intensive care unit [ICU] admissions, and excess deaths), surveillance systems, and the vaccine coverage rate (VCR) in older adults in 18 European countries and Israel.\u0000\u0000\u0000AREAS COVERED\u0000Published medical literature and official medical documentation on the influenza disease burden in the participating countries were reviewed from 2010/11 until the 2022/23 influenza seasons. Information on the framework for monitoring the disease burden and the provision for ensuring older adults had access to vaccination in their respective countries was provided. Data on influenza VCR in older adults were collected for the 2019/20 to 2022/23 influenza seasons. Data are reported descriptively.\u0000\u0000\u0000EXPERT OPINION\u0000Influenza presents a significant healthcare burden in older adults. Reporting outcomes across participating countries is heterogeneous, highlighting the need for standardized approaches. Although older adults receive free influenza vaccination, vaccine uptake is highly variable among countries. Moreover, hospitalization rates remain high even in countries reporting a high VCR. Increased awareness and education on the burden of disease and the broader use of improved influenza vaccines for older adults may help reduce the disease burden on this population.","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":"102 21","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140670188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Navneet Singh, Nadine Al-Naamani, Mary Beth Brown, Gary Marshall Long, Thenappan Thenappan, Soban Umar, Corey E Ventetuolo, Tim Lahm
{"title":"Extrapulmonary manifestations of pulmonary arterial hypertension.","authors":"Navneet Singh, Nadine Al-Naamani, Mary Beth Brown, Gary Marshall Long, Thenappan Thenappan, Soban Umar, Corey E Ventetuolo, Tim Lahm","doi":"10.1080/17476348.2024.2361037","DOIUrl":"10.1080/17476348.2024.2361037","url":null,"abstract":"<p><strong>Introduction: </strong>Extrapulmonary manifestations of pulmonary arterial hypertension (PAH) may play a critical pathobiological role and a deeper understanding will advance insight into mechanisms and novel therapeutic targets. This manuscript reviews our understanding of extrapulmonary manifestations of PAH.</p><p><strong>Areas covered: </strong>A group of experts was assembled and a complimentary PubMed search performed (October 2023 - March 2024). Inflammation is observed throughout the central nervous system and attempts at manipulation are an encouraging step toward novel therapeutics. Retinal vascular imaging holds promise as a noninvasive method of detecting early disease and monitoring treatment responses. PAH patients have gut flora alterations and dysbiosis likely plays a role in systemic inflammation. Despite inconsistent observations, the roles of obesity, insulin resistance and dysregulated metabolism may be illuminated by deep phenotyping of body composition. Skeletal muscle dysfunction is perpetuated by metabolic dysfunction, inflammation, and hypoperfusion, but exercise training shows benefit. Renal, hepatic, and bone marrow abnormalities are observed in PAH and may represent both end-organ damage and disease modifiers.</p><p><strong>Expert opinion: </strong>Insights into systemic manifestations of PAH will illuminate disease mechanisms and novel therapeutic targets. Additional study is needed to understand whether extrapulmonary manifestations are a cause or effect of PAH and how manipulation may affect outcomes.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"189-205"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141156160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Preoperative frailty screening in elderly patients with non-small cell lung cancer surgery: an essential step for a good surgical outcome.","authors":"Radu Iacobescu, Sabina Antonela Antoniu, Alina Delia Popa, Mariana Pavel-Tanase, Teodora Alexa Stratulat","doi":"10.1080/17476348.2024.2349579","DOIUrl":"10.1080/17476348.2024.2349579","url":null,"abstract":"<p><strong>Introduction: </strong>Non-small cell lung cancer (NSCLC) is a disease commonly diagnosed in the elderly, often in advanced stages. However, elderly patients with lung cancer can benefit from surgery, provided that postoperative risks are assessed appropriately before surgery. Frailty is a measure of age-related impaired functional status and a predictor of mortality and morbidity. However, its importance as a preoperative marker is not well defined.</p><p><strong>Areas covered: </strong>This systematic review discusses the importance of preoperative frailty screening in elderly patients with NSCLC. A literature search was performed on the MEDLINE database in June 2023, and relevant studies on frailty or preoperative assessment of NSCLC which were published between 2000 and 2023 were retained and discussed in this review.</p><p><strong>Expert opinion: </strong>Among the types of existing methods used to assess frailty those on the geriatric assessment seem to be the most appropriate; however, they are unable to fully capture the 'surgical' frailty; thus, other instruments should be developed and validated in NSCLC.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"99-110"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140869510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Javier Sayas, Cristina Lalmolda, Marta Corral, Pablo Flórez, Ana Hernández-Voth, Jean Paul Janssens, Claudio Rabec, Bruno Langevin, Frédéric Lofaso, Annalisa Carlucci, Claudia Llontop, Joao Carlos Winck, Jesús González Bermejo, Manel Lujan
{"title":"Measurement of thoraco-abdominal synchrony using respiratory inductance plethysmography: technical aspects and a proposal to overcome its limitations.","authors":"Javier Sayas, Cristina Lalmolda, Marta Corral, Pablo Flórez, Ana Hernández-Voth, Jean Paul Janssens, Claudio Rabec, Bruno Langevin, Frédéric Lofaso, Annalisa Carlucci, Claudia Llontop, Joao Carlos Winck, Jesús González Bermejo, Manel Lujan","doi":"10.1080/17476348.2024.2363058","DOIUrl":"10.1080/17476348.2024.2363058","url":null,"abstract":"<p><strong>Background: </strong>Thoraco-abdominal asynchrony (TAA) is usually assessed by respiratory inductance plethysmography. The main parameter used for its assessment is the calculation of the phase angle based on Lissajous plots. However, there are some mathematical limitations to its use.</p><p><strong>Research design and methods: </strong>Sequences of five breaths were selected from a) normal subjects, b) COPD patients, both at rest and during exercise, and c) patients with obstructive apnea syndrome. Automated analysis was performed calculating phase angle, loop rotation (clockwise or counterclockwise), global phase delay and loop area. TAA severity was estimated quantitatively and in subgroups.</p><p><strong>Results: </strong>2290 cycles were analyzed (55% clockwise rotation). Phase angle ranged from -86.90 to + 88.4 degrees, while global phase delay ranged from -179.75 to + 178.54. Despite a good correlation with global phase delay (<i>p</i> < 0.01, ANOVA test), phase angle and loop area were not able to correctly classify breaths with severe deviation and paradoxical movements (p=ns, Bonferroni post hoc test).</p><p><strong>Conclusions: </strong>Global phase delay covers the whole spectrum of TAA situations in a single value. It may be a relevant parameter for diagnosis and follow-up of clinical conditions leading to TAA.</p><p><strong>Clinical trial registration: </strong>The trial from which the traces were obtained was registered at ClinicalTrials.gov ;(identifier: NCT04597606).</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"227-236"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141201572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Merna H Emam, Reham S Elezaby, Shady A Swidan, Rania M Hathout
{"title":"Nanofiberous facemasks as protectives against pandemic respiratory viruses.","authors":"Merna H Emam, Reham S Elezaby, Shady A Swidan, Rania M Hathout","doi":"10.1080/17476348.2024.2356601","DOIUrl":"10.1080/17476348.2024.2356601","url":null,"abstract":"<p><strong>Introduction: </strong>Wearing protective face masks and respirators has been a necessity to reduce the transmission rate of respiratory viruses since the outbreak of the coronavirus (COVID-19) disease. Nevertheless, the outbreak has revealed the need to develop efficient air filter materials and innovative anti-microbial protectives. Nanofibrous facemasks, either loaded with antiviral nanoparticles or not, are very promising personal protective equipment (PPE) against pandemic respiratory viruses.</p><p><strong>Areas covered: </strong>In this review, multiple types of face masks and respirators are discussed as well as filtration mechanisms of particulates. In this regard, the limitations of traditional face masks were summarized and the advancement of nanotechnology in developing nanofibrous masks and air filters was discussed. Different methods of preparing nanofibers were explained. The various approaches used for enhancing nanofibrous face masks were covered.</p><p><strong>Expert opinion: </strong>Although wearing conventional face masks can limit viral infection spread to some extent, the world is in great need for more protective face masks. Nanofibers can block viral particles efficiently and can be incorporated into face masks in order to enhance their filtration efficiency. Also, we believe that other modifications such as addition of antiviral nanoparticles can significantly increase the protection power of facemasks.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"127-143"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961257","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Andrés Calvache-Mateo, Gregory Reychler, Alejandro Heredia-Ciuró, Javier Martín-Núñez, Araceli Ortiz-Rubio, Alba Navas-Otero, Marie Carmen Valenza
{"title":"Respiratory training effects in Long COVID-19 patients: a systematic review and meta-analysis.","authors":"Andrés Calvache-Mateo, Gregory Reychler, Alejandro Heredia-Ciuró, Javier Martín-Núñez, Araceli Ortiz-Rubio, Alba Navas-Otero, Marie Carmen Valenza","doi":"10.1080/17476348.2024.2358933","DOIUrl":"10.1080/17476348.2024.2358933","url":null,"abstract":"<p><strong>Introduction: </strong>To date, it is unknown whether respiratory training interventions can benefit Long COVID-19 patients. The main objective was to analyze the effects of respiratory training on patients with Long COVID-19, concretely on respiratory muscle strength, lung function, dyspnea, and functional capacity.</p><p><strong>Methods: </strong>We performed a systematic review following PRISMA statement using PubMed, Scopus, and PEDro (last search November 2023). The risk of bias was assessed using the Cochrane tool. We included randomized controlled trials testing the effect of respiratory training interventions in Long COVID-19 patients versus no intervention, control, or placebo intervention. The data was pooled, and a meta-analysis was complete.</p><p><strong>Results: </strong>We selected 7 studies, which included 572 patients. Meta-analysis results show significant differences in favor of respiratory training in respiratory muscle strength (MD = 13.71; 95% CI = 5.41; 22; <i>p</i> = 0.001), dyspnea (SDM = 1.39; 95% CI = 0.33; 2.46; <i>p</i> = 0.01) and functional capacity (SDM = 0.90; 95% CI = 0.37; 1.43; <i>p</i> = 0.0009), but not in lung function (MD = 0.28; 95%CI = -0.27; 0.83; <i>p</i> = 0.32).</p><p><strong>Conclusion: </strong>The results of this systematic review with meta-analysis suggest that respiratory training improves respiratory muscle strength and functional capacity in Long COVID-19 patients, as well as dyspnea if combined with therapeutic exercise. However, respiratory training does not improve lung function in these patients.</p><p><strong>Review registration prospero identifier: </strong>CRD42022371820.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"207-217"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141156142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Micheál Mac Aogáin, Pei Yee Tiew, Tavleen Kaur Jaggi, Jayanth Kumar Narayana, Shivani Singh, Philip M Hansbro, Leopoldo N Segal, Sanjay H Chotirmall
{"title":"Targeting respiratory microbiomes in COPD and bronchiectasis.","authors":"Micheál Mac Aogáin, Pei Yee Tiew, Tavleen Kaur Jaggi, Jayanth Kumar Narayana, Shivani Singh, Philip M Hansbro, Leopoldo N Segal, Sanjay H Chotirmall","doi":"10.1080/17476348.2024.2355155","DOIUrl":"10.1080/17476348.2024.2355155","url":null,"abstract":"<p><strong>Introduction: </strong>This review summarizes our current understanding of the respiratory microbiome in COPD and Bronchiectasis. We explore the interplay between microbial communities, host immune responses, disease pathology, and treatment outcomes.</p><p><strong>Areas covered: </strong>We detail the dynamics of the airway microbiome, its influence on chronic respiratory diseases, and analytical challenges. Relevant articles from PubMed and Medline (January 2010-March 2024) were retrieved and summarized. We examine clinical correlations of the microbiome in COPD and bronchiectasis, assessing how current therapies impact upon it. The potential of emerging immunotherapies, antiinflammatories and antimicrobial strategies is discussed, with focus on the pivotal role of commensal taxa in maintaining respiratory health and the promising avenue of microbiome remodeling for disease management.</p><p><strong>Expert opinion: </strong>Given the heterogeneity in microbiome composition and its pivotal role in disease development and progression, a shift toward microbiome-directed therapeutics is appealing. This transition, from traditional 'pathogencentric' diagnostic and treatment modalities to those acknowledging the microbiome, can be enabled by evolving crossdisciplinary platforms which have the potential to accelerate microbiome-based interventions into routine clinical practice. Bridging the gap between comprehensive microbiome analysis and clinical application, however, remains challenging, necessitating continued innovation in research, diagnostics, trials, and therapeutic development pipelines.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"111-125"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140924129","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Raíssa S Freire, Camila M S S Barros, Jefferson Valente, Cássia da Luz Goulart, Anna G R Santos, Fernando H Fonseca, Sabrina T Saenz, Andiana S Dias, Maria G A Rodrigues, Bernardo Maia Silva, Eduardo Fernandes, Nadia Cubas-Vega, Vanderson Sampaio, Mariana Simão, Djane Baía-da-Silva, Richard Severin, Guilherme Peixoto Tinoco Arêas, Roberta Lins Gonçalves, Renata Gonçalves Mendes, Flor E Martinez-Espinosa, Fernando Val
{"title":"Prone positioning in awake patients without ventilatory support does not alter major clinical outcomes in severe COVID-19: results from a retrospective observational cohort study, systematic review and meta-analysis.","authors":"Raíssa S Freire, Camila M S S Barros, Jefferson Valente, Cássia da Luz Goulart, Anna G R Santos, Fernando H Fonseca, Sabrina T Saenz, Andiana S Dias, Maria G A Rodrigues, Bernardo Maia Silva, Eduardo Fernandes, Nadia Cubas-Vega, Vanderson Sampaio, Mariana Simão, Djane Baía-da-Silva, Richard Severin, Guilherme Peixoto Tinoco Arêas, Roberta Lins Gonçalves, Renata Gonçalves Mendes, Flor E Martinez-Espinosa, Fernando Val","doi":"10.1080/17476348.2024.2350587","DOIUrl":"10.1080/17476348.2024.2350587","url":null,"abstract":"<p><strong>Objectives: </strong>During the Coronavirus disease (COVID-19) pandemic, clinicians recommended awake-prone positioning (APP) to avoid the worst outcomes. The objectives of this study were to investigate if APP reduces intubation, death rates, and hospital length of stay (HLOS) in acute COVID-19.</p><p><strong>Methods: </strong>We performed a retrospective cohort with non-mechanically ventilated patients hospitalized in a reference center in Manaus, Brazil, 2020. Participants were stratified into APP and awake-not-prone positioning (ANPP) groups. Also, we conducted a systematic review and performed a meta-analysis to understand if this intervention had different outcomes in resource-limited settings (PROSPERO CRD42023422452).</p><p><strong>Results: </strong>A total of 115 participants were allocated into the groups. There was no statistical difference between both groups regarding time to intubation (HR: 0.861; 95CI: 0.474-1.1562; p=0.622) and time to death (HR: 1.666; 95CI: 0.939-2.951; p=0.081). APP was not significantly associated with reduced HLOS. A total of 86 articles were included in the systematic review, of which 76 (88,3%) show similar findings after APP. Also, low/middle, and high-income countries were similar regarding such outcomes.</p><p><strong>Conclusion: </strong>APP in COVID-19 does not present clinical improvement that affects mortality, intubation rate and HLOS. The lack of a prone position protocol, obtained through a controlled study, is necessary. After 3 years, APP benefits are still inconclusive.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"219-226"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140875028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ramsy Abdelghani, Mohamed Omballi, David Abia-Trujillo, Ernesto Casillas, Regina Villalobos, Faraz Badar, Sandeep Bansal, Fayez Kheir
{"title":"Imaging modalities during navigational bronchoscopy.","authors":"Ramsy Abdelghani, Mohamed Omballi, David Abia-Trujillo, Ernesto Casillas, Regina Villalobos, Faraz Badar, Sandeep Bansal, Fayez Kheir","doi":"10.1080/17476348.2024.2359601","DOIUrl":"10.1080/17476348.2024.2359601","url":null,"abstract":"<p><strong>Introduction: </strong>Lung nodules are commonly encountered in clinical practice. Technological advances in navigational bronchoscopy and imaging modalities have led to paradigm shift from nodule screening or follow-up to early lung cancer detection. This is due to improved nodule localization and biopsy confirmation with combined modalities of navigational platforms and imaging tools. To conduct this article, relevant literature was reviewed via PubMed from January 2014 until January 2024.</p><p><strong>Areas covered: </strong>This article highlights the literature on different imaging modalities combined with commonly used navigational platforms for diagnosis of peripheral lung nodules. Current limitations and future perspectives of imaging modalities will be discussed.</p><p><strong>Expert opinion: </strong>The development of navigational platforms improved localization of targets. However, published diagnostic yield remains lower compared to percutaneous-guided biopsy. The discordance between the actual location of lung nodule during the procedure and preprocedural CT chest is the main factor impacting accurate biopsies. The utilization of advanced imaging tools with navigation-based bronchoscopy has been shown to assist with localizing targets in real-time and improving biopsy success. However, it is important for interventional bronchoscopists to understand the strengths and limitations of these advanced imaging technologies.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"175-188"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141094494","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Unmet challenges in cystic fibrosis treatment with modulators.","authors":"Federica Corrao, Mairead Kelly-Aubert, Isabelle Sermet-Gaudelus, Michaela Semeraro","doi":"10.1080/17476348.2024.2357210","DOIUrl":"10.1080/17476348.2024.2357210","url":null,"abstract":"<p><strong>Introduction: </strong>'Highly effective' modulator therapies (HEMTs) have radically changed the Cystic Fibrosis (CF) therapeutic landscape.</p><p><strong>Areas covered: </strong>A comprehensive search strategy was undertaken to assess impact of HEMT in life of pwCF, treatment challenges in specific populations such as very young children, and current knowledge gaps.</p><p><strong>Expert opinion: </strong>HEMTs are prescribed for pwCF with definite genotypes. The heterogeneity of variants complicates treatment possibilities and around 10% of pwCF worldwide remains ineligible. Genotype-specific treatments are prompting theratyping and personalized medicine strategies. Improvement in lung function and quality of life increase survival rates, shifting CF from a pediatric to an adult disease. This implies new studies addressing long-term efficacy, side effects, emergence of adult co-morbidities and possible drug-drug interactions. More sensitive and predictive biomarkers for both efficacy and toxicity are warranted. As HEMTs cross the placenta and are found in breast milk, studies addressing the potential consequences of treatment during pregnancy and breastfeeding are urgently needed. Finally, although the treatment and expected outcomes of CF have improved dramatically in high- and middle-income countries, lack of access in low-income countries to these life-changing medicines highlights inequity of care worldwide.</p>","PeriodicalId":94007,"journal":{"name":"Expert review of respiratory medicine","volume":" ","pages":"145-157"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}