Pub Date : 2023-03-01DOI: 10.1007/s41030-022-00208-6
Andrew Li, Pyng Lee
Despite the latest developments in therapeutic agents targeting airway endotypes, a significant proportion of patients with asthma and chronic obstructive pulmonary disease (COPD) remain symptomatic. Endoscopic therapies have a complementary role in the management of these airway diseases. The sustained efficacy of bronchial thermoplasty (BT) among patients with asthma over 10 years has been encouraging, as it has been shown to improve symptom control and reduce hospital admissions and exacerbations. Studies suggest that BT helps ameliorate airway inflammation and reduce airway smooth muscle thickness. While studies suggest that it is as effective as biologic agents, its role in the management of severe asthma has yet to be clearly defined and GINA 2022 still suggests limiting its use to patients with characteristics of the various populations studied. Conversely, bronchoscopic lung volume reduction has shown promise among patients with advanced COPD. Rigorous patient selection is important. Patients with minimal collateral ventilation (CV) and higher heterogeneity index have shown to benefit the most from endobronchial valve (EBV) therapy. For those with ongoing CV, endobronchial coils would be more appropriate. Both therapeutic modalities have demonstrated improved quality of life, effort tolerance, and lung function indices among appropriately selected patients. The emerging evidence suggests that endoscopic procedures among airway disease still have a substantial role to play despite the development of new therapeutic options.
{"title":"Which Endoscopic Procedure to Use and in What Patient? Valves, Coils, Foam, and Heat in COPD and Asthma.","authors":"Andrew Li, Pyng Lee","doi":"10.1007/s41030-022-00208-6","DOIUrl":"https://doi.org/10.1007/s41030-022-00208-6","url":null,"abstract":"<p><p>Despite the latest developments in therapeutic agents targeting airway endotypes, a significant proportion of patients with asthma and chronic obstructive pulmonary disease (COPD) remain symptomatic. Endoscopic therapies have a complementary role in the management of these airway diseases. The sustained efficacy of bronchial thermoplasty (BT) among patients with asthma over 10 years has been encouraging, as it has been shown to improve symptom control and reduce hospital admissions and exacerbations. Studies suggest that BT helps ameliorate airway inflammation and reduce airway smooth muscle thickness. While studies suggest that it is as effective as biologic agents, its role in the management of severe asthma has yet to be clearly defined and GINA 2022 still suggests limiting its use to patients with characteristics of the various populations studied. Conversely, bronchoscopic lung volume reduction has shown promise among patients with advanced COPD. Rigorous patient selection is important. Patients with minimal collateral ventilation (CV) and higher heterogeneity index have shown to benefit the most from endobronchial valve (EBV) therapy. For those with ongoing CV, endobronchial coils would be more appropriate. Both therapeutic modalities have demonstrated improved quality of life, effort tolerance, and lung function indices among appropriately selected patients. The emerging evidence suggests that endoscopic procedures among airway disease still have a substantial role to play despite the development of new therapeutic options.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"9 1","pages":"49-69"},"PeriodicalIF":3.0,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e6/54/41030_2022_Article_208.PMC9931990.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10737618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-03-01DOI: 10.1007/s41030-022-00207-7
Stephanie Lovinsky-Desir, Anna Volerman
Asthma is the most common chronic childhood condition and is a risk factor for severe respiratory viral infections. Thus, early during the coronavirus disease 2019 (COVID-19) pandemic there was concern that children with asthma would be at risk for severe COVID-19 illness and that asthma control could worsen as a result of the pandemic. This article seeks to summarize what was learned in the early stages of the pandemic about the impact of COVID-19 on children with asthma. We review evidence from several studies that demonstrated a significant decline in asthma morbidity in the first year of the pandemic. Additionally, we describe several potential mechanisms that may explain the reduced frequency in childhood asthma exacerbations as well as review lessons learned for future management of childhood asthma. While the COVID-19 pandemic initially brought uncertainty, it soon became clear that the pandemic had several positive effects for children with asthma. Now we can apply the lessons that were learned during the pandemic to re-examine asthma care practices as well as advocate for best approaches for asthma management.
{"title":"Applying Lessons from the COVID-19 Pandemic to Improve Pediatric Asthma Care.","authors":"Stephanie Lovinsky-Desir, Anna Volerman","doi":"10.1007/s41030-022-00207-7","DOIUrl":"https://doi.org/10.1007/s41030-022-00207-7","url":null,"abstract":"<p><p>Asthma is the most common chronic childhood condition and is a risk factor for severe respiratory viral infections. Thus, early during the coronavirus disease 2019 (COVID-19) pandemic there was concern that children with asthma would be at risk for severe COVID-19 illness and that asthma control could worsen as a result of the pandemic. This article seeks to summarize what was learned in the early stages of the pandemic about the impact of COVID-19 on children with asthma. We review evidence from several studies that demonstrated a significant decline in asthma morbidity in the first year of the pandemic. Additionally, we describe several potential mechanisms that may explain the reduced frequency in childhood asthma exacerbations as well as review lessons learned for future management of childhood asthma. While the COVID-19 pandemic initially brought uncertainty, it soon became clear that the pandemic had several positive effects for children with asthma. Now we can apply the lessons that were learned during the pandemic to re-examine asthma care practices as well as advocate for best approaches for asthma management.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"9 1","pages":"15-24"},"PeriodicalIF":3.0,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/00/3f/41030_2022_Article_207.PMC9707220.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9462220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-03-01DOI: 10.1007/s41030-022-00210-y
Graham F Hatfull
Non-tuberculous mycobacterium (NTM) infections are often clinically challenging, with lengthy antibiotic regimens that fail to resolve the infections with few good outcomes remaining. Mycobacteriophages-viruses that infect Mycobacterium hosts-show promise as therapeutic agents for NTM infections and have been used in 20 compassionate use cases. Favorable outcomes were observed in many but not all cases, although the phages show exceptional safety profiles and no evidence of phage resistance was observed, even when only a single phage was administered. Phage-specific antibodies are commonly present following intravenous administration and are often neutralizing for the phage in vitro. However, phage neutralization does not consistently correlate with poor treatment outcomes and may not be a therapeutic limitation in all patients, even when immunocompetent. Currently, the therapeutic potential of phages is substantially limited by the great variation in phage susceptibility and a relatively small repertoire of therapeutically useful phages. As many as 45% of clinical isolates can have a smooth colony morphotype, and phages that both efficiently infect and kill these strains have yet to be described. In contrast, ~ 75% of rough strains are susceptible to and killed by one or more phages and therapeutic options can be considered on a compassionate use basis. Although therapies must currently be personalized, elucidating the determinants of phage host specificity, expanding the useful phage repertoire, and identifying the key determinants of clinical outcomes will reveal their full therapeutic potential.
{"title":"Phage Therapy for Nontuberculous Mycobacteria: Challenges and Opportunities.","authors":"Graham F Hatfull","doi":"10.1007/s41030-022-00210-y","DOIUrl":"https://doi.org/10.1007/s41030-022-00210-y","url":null,"abstract":"<p><p>Non-tuberculous mycobacterium (NTM) infections are often clinically challenging, with lengthy antibiotic regimens that fail to resolve the infections with few good outcomes remaining. Mycobacteriophages-viruses that infect Mycobacterium hosts-show promise as therapeutic agents for NTM infections and have been used in 20 compassionate use cases. Favorable outcomes were observed in many but not all cases, although the phages show exceptional safety profiles and no evidence of phage resistance was observed, even when only a single phage was administered. Phage-specific antibodies are commonly present following intravenous administration and are often neutralizing for the phage in vitro. However, phage neutralization does not consistently correlate with poor treatment outcomes and may not be a therapeutic limitation in all patients, even when immunocompetent. Currently, the therapeutic potential of phages is substantially limited by the great variation in phage susceptibility and a relatively small repertoire of therapeutically useful phages. As many as 45% of clinical isolates can have a smooth colony morphotype, and phages that both efficiently infect and kill these strains have yet to be described. In contrast, ~ 75% of rough strains are susceptible to and killed by one or more phages and therapeutic options can be considered on a compassionate use basis. Although therapies must currently be personalized, elucidating the determinants of phage host specificity, expanding the useful phage repertoire, and identifying the key determinants of clinical outcomes will reveal their full therapeutic potential.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"9 1","pages":"91-107"},"PeriodicalIF":3.0,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/16/a4/41030_2022_Article_210.PMC9931961.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10740985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-12-01Epub Date: 2022-11-01DOI: 10.1007/s41030-022-00201-z
Lilla Tamási, Anna Bartha, Aranka Ferencz, Mihály Tímár, Mikko Vahteristo, Aino Takala, Veronika Müller
Introduction: For inhalation therapies to be effective, it is crucial that patients manage inhaler use correctly in their everyday life and achieve treatment compliance. We investigated the effectiveness of the salmeterol-fluticasone propionate Easyhaler® (SF EH) device-metered dry powder inhaler in a real-world setting in Hungary among adult patients with asthma, chronic obstructive pulmonary disease (COPD), or asthma-COPD overlap syndrome (ACO).
Methods: A prospective, open-label, multicenter, noninterventional, investigator-sponsored study was conducted in outpatient pneumonology centers. Eligible patients were aged ≥ 18 years with either a new diagnosis of asthma, COPD, or ACO, or whose disease was not controlled with preexisting medication. Data were collected at baseline and 12 + 4 weeks, including the asthma control test (ACT), COPD assessment test (CAT), spirometry parameters [including forced expiratory volume for 1 s (FEV1)], and physician- and patient-reported outcomes.
Results: Five hundred sixteen patients were recruited from 103 centers: 376 with asthma; 104 with COPD; and 36 with ACO. At week 12, there were significant improvements from baseline in both mean ACT score in patients with asthma (14.4 ± 4.2 versus 21.4 ± 2.8; P < 0.001) and mean CAT score in patients with COPD (24.0 ± 6.1 versus 16.0 ± 5.8; P < 0.001). Significant improvement was observed when the switch from the most frequently used previous inhalers was analyzed separately. Mean FEV1 improved from 76.0% ± 17.2 to 84.7% ± 16.1 (P < 0.001) and from 53.8% ± 15.0 to 59.9% ± 15.0 (P < 0.001) in patients with asthma or COPD, respectively. The study demonstrated improved physician-rated overall treatment compliance and patient preference for the SF EH over 3 months use compared with previous inhaler treatment, with patients effectively adopting the SF EH into everyday life.
Conclusions: Treatment with SF EH significantly improved patients' lung function parameters and disease control.
{"title":"Effectiveness and Treatment Compliance of Salmeterol-Fluticasone Easyhaler<sup>®</sup> Among Patients with Asthma, COPD, or Asthma-COPD Overlap Syndrome: Real-World Study Findings.","authors":"Lilla Tamási, Anna Bartha, Aranka Ferencz, Mihály Tímár, Mikko Vahteristo, Aino Takala, Veronika Müller","doi":"10.1007/s41030-022-00201-z","DOIUrl":"10.1007/s41030-022-00201-z","url":null,"abstract":"<p><strong>Introduction: </strong>For inhalation therapies to be effective, it is crucial that patients manage inhaler use correctly in their everyday life and achieve treatment compliance. We investigated the effectiveness of the salmeterol-fluticasone propionate Easyhaler<sup>®</sup> (SF EH) device-metered dry powder inhaler in a real-world setting in Hungary among adult patients with asthma, chronic obstructive pulmonary disease (COPD), or asthma-COPD overlap syndrome (ACO).</p><p><strong>Methods: </strong>A prospective, open-label, multicenter, noninterventional, investigator-sponsored study was conducted in outpatient pneumonology centers. Eligible patients were aged ≥ 18 years with either a new diagnosis of asthma, COPD, or ACO, or whose disease was not controlled with preexisting medication. Data were collected at baseline and 12 + 4 weeks, including the asthma control test (ACT), COPD assessment test (CAT), spirometry parameters [including forced expiratory volume for 1 s (FEV<sub>1</sub>)], and physician- and patient-reported outcomes.</p><p><strong>Results: </strong>Five hundred sixteen patients were recruited from 103 centers: 376 with asthma; 104 with COPD; and 36 with ACO. At week 12, there were significant improvements from baseline in both mean ACT score in patients with asthma (14.4 ± 4.2 versus 21.4 ± 2.8; P < 0.001) and mean CAT score in patients with COPD (24.0 ± 6.1 versus 16.0 ± 5.8; P < 0.001). Significant improvement was observed when the switch from the most frequently used previous inhalers was analyzed separately. Mean FEV<sub>1</sub> improved from 76.0% ± 17.2 to 84.7% ± 16.1 (P < 0.001) and from 53.8% ± 15.0 to 59.9% ± 15.0 (P < 0.001) in patients with asthma or COPD, respectively. The study demonstrated improved physician-rated overall treatment compliance and patient preference for the SF EH over 3 months use compared with previous inhaler treatment, with patients effectively adopting the SF EH into everyday life.</p><p><strong>Conclusions: </strong>Treatment with SF EH significantly improved patients' lung function parameters and disease control.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 4","pages":"369-384"},"PeriodicalIF":2.3,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/22/3d/41030_2022_Article_201.PMC9628494.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10736387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-12-01DOI: 10.1007/s41030-022-00203-x
Alan G Kaplan, James W Kim
Asthma is a chronic inflammatory disease involving multiple mediators and cytokines. While our current treatments have shown significant therapeutic benefits, there still appear to be some patients who, despite aggressive therapy, good adherence, and inhaler technique, continue to have exacerbations. Exacerbations lead to loss of lung function, exposure to systemic corticosteroids, effects on quality of life, and even mortality. There is a large number of glucagon-like peptide-1 (GLP-1) receptors in the lung even compared with other organs, and studies have shown evidence of reduced exacerbations in asthmatics treated with GLP-1 receptor agonists (GLP-1 RA). While weight loss may affect lung mechanics, evidence of inflammatory changes has been revealed that could explain this relationship. This article will review the data behind these conjectures and outline potential clinical utility and the need for future studies to truly understand the role of GLP-1 receptors in the lung.
{"title":"Asthma Exacerbations and Glucagon-Like Peptide-1 Receptor Agonists: a Review of the Current Evidence.","authors":"Alan G Kaplan, James W Kim","doi":"10.1007/s41030-022-00203-x","DOIUrl":"https://doi.org/10.1007/s41030-022-00203-x","url":null,"abstract":"<p><p>Asthma is a chronic inflammatory disease involving multiple mediators and cytokines. While our current treatments have shown significant therapeutic benefits, there still appear to be some patients who, despite aggressive therapy, good adherence, and inhaler technique, continue to have exacerbations. Exacerbations lead to loss of lung function, exposure to systemic corticosteroids, effects on quality of life, and even mortality. There is a large number of glucagon-like peptide-1 (GLP-1) receptors in the lung even compared with other organs, and studies have shown evidence of reduced exacerbations in asthmatics treated with GLP-1 receptor agonists (GLP-1 RA). While weight loss may affect lung mechanics, evidence of inflammatory changes has been revealed that could explain this relationship. This article will review the data behind these conjectures and outline potential clinical utility and the need for future studies to truly understand the role of GLP-1 receptors in the lung.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 4","pages":"343-358"},"PeriodicalIF":3.0,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/83/d5/41030_2022_Article_203.PMC9727043.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10373160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-12-01DOI: 10.1007/s41030-022-00200-0
Takako Nagai, Hiroshi Uei, Kazuyoshi Nakanishi
Introduction: Aspiration pneumonia is the predominant form of pneumonia in the elderly. Low oral intake levels and malnutrition have been reported to be associated with increased mortality and loss of function in aspiration pneumonia. However, the relationship between start of feeding and readmission, which is associated with malnutrition and low oral intake levels, has not been reported. The purpose of this study was to clarify the relationship between start of feeding and functional prognosis in aspiration pneumonia.
Methods: Patients' basic information, comorbidities, severity of pneumonia, swallowing function, time from admission to the start of feeding, geriatric nutritional risk index (GNRI), readmission, and Barthel index (BI) were evaluated in 160 patients. The patients were divided into two groups-a readmission group and a non-readmission group-and statistical verification was performed.
Results: The readmission group was 62 cases (38.8%). Univariate analysis showed that the time from admission to the start of feeding was significantly longer in the readmission group (p < 0.001). Age was significantly higher and nutrition parameters were lower in the readmission group (p = 0.001, 0.006). Furthermore, according to logistic regression analysis, readmission was associated with age (odds ratio, 1.063; p = 0.007; 95% confidence interval (CI) 1.017-1.111) and time from admission to the start of feeding (odds ratio 1.080; p < 0.001; 95% CI 1.025-1.137).
Conclusion: The time from admission to the start of feeding was significantly longer in the readmitted patients. A comprehensive intervention with multidisciplinary collaboration should be performed from the early stage of hospitalization.
Trial registration: This study is registered in the UMIN-Clinical Trials Registry (UMIN-CTR). UMIN-CTR meets the criteria of the International Committee of Medical Journal Editors (ICMJE). (Registration number: 000047141).
{"title":"Relationship Between Start of Feeding and Functional Outcome in Aspiration Pneumonia: A Retrospective Cohort Study.","authors":"Takako Nagai, Hiroshi Uei, Kazuyoshi Nakanishi","doi":"10.1007/s41030-022-00200-0","DOIUrl":"https://doi.org/10.1007/s41030-022-00200-0","url":null,"abstract":"<p><strong>Introduction: </strong>Aspiration pneumonia is the predominant form of pneumonia in the elderly. Low oral intake levels and malnutrition have been reported to be associated with increased mortality and loss of function in aspiration pneumonia. However, the relationship between start of feeding and readmission, which is associated with malnutrition and low oral intake levels, has not been reported. The purpose of this study was to clarify the relationship between start of feeding and functional prognosis in aspiration pneumonia.</p><p><strong>Methods: </strong>Patients' basic information, comorbidities, severity of pneumonia, swallowing function, time from admission to the start of feeding, geriatric nutritional risk index (GNRI), readmission, and Barthel index (BI) were evaluated in 160 patients. The patients were divided into two groups-a readmission group and a non-readmission group-and statistical verification was performed.</p><p><strong>Results: </strong>The readmission group was 62 cases (38.8%). Univariate analysis showed that the time from admission to the start of feeding was significantly longer in the readmission group (p < 0.001). Age was significantly higher and nutrition parameters were lower in the readmission group (p = 0.001, 0.006). Furthermore, according to logistic regression analysis, readmission was associated with age (odds ratio, 1.063; p = 0.007; 95% confidence interval (CI) 1.017-1.111) and time from admission to the start of feeding (odds ratio 1.080; p < 0.001; 95% CI 1.025-1.137).</p><p><strong>Conclusion: </strong>The time from admission to the start of feeding was significantly longer in the readmitted patients. A comprehensive intervention with multidisciplinary collaboration should be performed from the early stage of hospitalization.</p><p><strong>Trial registration: </strong>This study is registered in the UMIN-Clinical Trials Registry (UMIN-CTR). UMIN-CTR meets the criteria of the International Committee of Medical Journal Editors (ICMJE). (Registration number: 000047141).</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 4","pages":"359-368"},"PeriodicalIF":3.0,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/2d/2f/41030_2022_Article_200.PMC9727170.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10367503","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-12-01DOI: 10.1007/s41030-022-00202-y
Gregory S Sawicki, Michael W Konstan, Edward F McKone, Richard B Moss, Barry Lubarsky, Ellison Suthoff, Stefanie J Millar, David J Pasta, Nicole Mayer-Hamblett, Christopher H Goss, Wayne J Morgan, Margaret E Duncan, Yoojung Yang
Introduction: Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Approximately 5% of people with CF have residual function (RF) CFTR mutations that result in partially retained CFTR activity. Published literature on disease trajectory among those with RF mutations is limited. In this retrospective study, we characterized lung function decline across different age groups in CFTR modulator-untreated people with CF heterozygous for F508del and an RF mutation (F/RF).
Methods: Rate of decline in percent predicted forced expiratory volume in 1 s (ppFEV1) was analyzed using data from the US CF Foundation Patient Registry (2006-2014) in F/RF (all), F/RF (excluding R117H), and F508del homozygous (F/F) cohorts. Annual rates of ppFEV1 decline were estimated over 2-year periods based on calendar year. Subgroup analyses by age [6-12 (children), 13-17 (adolescents), 18-24 (young adults), and ≥ 25 years (adults)] were performed.
Results: The estimated annualized rate of ppFEV1 decline was - 0.70 percentage points per year (95% CI -1.09, -0.30) in the F/RF (all) cohort (N = 1242) versus -1.91 percentage points per year (95% CI -2.01, -1.80) in the F/F cohort (N = 11,916) [difference, 1.29 percentage points per year (95% CI 0.88, 1.70); P < 0.001]. In the F/RF (all) cohort, all age groups demonstrated lung function decline ranging from -0.30 to -1.38. In the F/RF (excluding R117H) cohort, the rate of decline was -1.05 percentage points per year (95% CI -1.51, -0.60) [difference versus F/F cohort, 0.95 percentage points per year (95% CI 0.48, 1.41; P < 0.001); not statistically significant in children and young adults].
Conclusion: Progressive lung function decline was observed in people with F/RF genotypes across all assessed age groups, reinforcing the importance of early intervention and clinical monitoring to preserve lung function in all people with CF.
简介:囊性纤维化(CF)是一种常染色体隐性遗传病,由CF跨膜传导调节因子(CFTR)基因突变引起。大约5%的CF患者有残留功能(RF) CFTR突变,导致CFTR活性部分保留。已发表的关于RF突变患者疾病轨迹的文献是有限的。在这项回顾性研究中,我们研究了CFTR调节剂未经治疗的CF杂合F508del和RF突变(F/RF)的不同年龄组肺功能下降。方法:使用美国CF基金会患者登记处(2006-2014)F/RF(全部)、F/RF(不包括R117H)和F508del纯合子(F/F)队列的数据,分析1 s内预测用力呼气量百分比下降率(ppFEV1)。ppFEV1的年递减率是基于日历年估算的2年期间。按年龄进行亚组分析[6-12岁(儿童)、13-17岁(青少年)、18-24岁(青年)和≥25岁(成人)]。结果:F/RF(所有)队列(N = 1242)中ppFEV1的估计年化下降率为- 0.70个百分点/年(95% CI -1.09, -0.30),而F/F队列(N = 11,916)中ppFEV1的估计年化下降率为-1.91个百分点/年(95% CI -2.01, -1.80)[差异为1.29个百分点/年(95% CI 0.88, 1.70);结论:在所有被评估的年龄组中,F/RF基因型患者的肺功能均出现进行性下降,这加强了早期干预和临床监测对所有CF患者肺功能保护的重要性。
{"title":"Rate of Lung Function Decline in People with Cystic Fibrosis Having a Residual Function Gene Mutation.","authors":"Gregory S Sawicki, Michael W Konstan, Edward F McKone, Richard B Moss, Barry Lubarsky, Ellison Suthoff, Stefanie J Millar, David J Pasta, Nicole Mayer-Hamblett, Christopher H Goss, Wayne J Morgan, Margaret E Duncan, Yoojung Yang","doi":"10.1007/s41030-022-00202-y","DOIUrl":"https://doi.org/10.1007/s41030-022-00202-y","url":null,"abstract":"<p><strong>Introduction: </strong>Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Approximately 5% of people with CF have residual function (RF) CFTR mutations that result in partially retained CFTR activity. Published literature on disease trajectory among those with RF mutations is limited. In this retrospective study, we characterized lung function decline across different age groups in CFTR modulator-untreated people with CF heterozygous for F508del and an RF mutation (F/RF).</p><p><strong>Methods: </strong>Rate of decline in percent predicted forced expiratory volume in 1 s (ppFEV<sub>1</sub>) was analyzed using data from the US CF Foundation Patient Registry (2006-2014) in F/RF (all), F/RF (excluding R117H), and F508del homozygous (F/F) cohorts. Annual rates of ppFEV<sub>1</sub> decline were estimated over 2-year periods based on calendar year. Subgroup analyses by age [6-12 (children), 13-17 (adolescents), 18-24 (young adults), and ≥ 25 years (adults)] were performed.</p><p><strong>Results: </strong>The estimated annualized rate of ppFEV<sub>1</sub> decline was - 0.70 percentage points per year (95% CI -1.09, -0.30) in the F/RF (all) cohort (N = 1242) versus -1.91 percentage points per year (95% CI -2.01, -1.80) in the F/F cohort (N = 11,916) [difference, 1.29 percentage points per year (95% CI 0.88, 1.70); P < 0.001]. In the F/RF (all) cohort, all age groups demonstrated lung function decline ranging from -0.30 to -1.38. In the F/RF (excluding R117H) cohort, the rate of decline was -1.05 percentage points per year (95% CI -1.51, -0.60) [difference versus F/F cohort, 0.95 percentage points per year (95% CI 0.48, 1.41; P < 0.001); not statistically significant in children and young adults].</p><p><strong>Conclusion: </strong>Progressive lung function decline was observed in people with F/RF genotypes across all assessed age groups, reinforcing the importance of early intervention and clinical monitoring to preserve lung function in all people with CF.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 4","pages":"385-395"},"PeriodicalIF":3.0,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a1/f1/41030_2022_Article_202.PMC9727051.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9276818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-09-01DOI: 10.1007/s41030-022-00195-8
Benjamin Pippard, Malvika Bhatnagar, Lisa McNeill, Mhairi Donnelly, Katie Frew, Avinash Aujayeb
Hepatic hydrothorax (HH) represents a distinct clinical entity within the broader classification of pleural effusion that is associated with significant morbidity and mortality. The median survival of patients with cirrhosis who develop HH is 8-12 months. The diagnosis is typically made in the context of advanced liver disease and ascites, in the absence of underlying cardio-pulmonary pathology. A multi-disciplinary approach to management, involving respiratory physicians, hepatologists, and palliative care specialists is crucial to ensuring optimal patient-centered care. However, the majority of accepted therapeutic options are based on expert opinion rather than large, adequately powered randomized controlled trials. In this narrative review, we discuss the epidemiology, pathophysiology, clinical characteristics, and management of HH, highlighting the use of salt restriction and diuretic therapy, porto-systemic shunts, and liver transplantation. We include specific sections focusing on the role of pleural interventions and palliative care, respectively.
{"title":"Hepatic Hydrothorax: A Narrative Review.","authors":"Benjamin Pippard, Malvika Bhatnagar, Lisa McNeill, Mhairi Donnelly, Katie Frew, Avinash Aujayeb","doi":"10.1007/s41030-022-00195-8","DOIUrl":"https://doi.org/10.1007/s41030-022-00195-8","url":null,"abstract":"<p><p>Hepatic hydrothorax (HH) represents a distinct clinical entity within the broader classification of pleural effusion that is associated with significant morbidity and mortality. The median survival of patients with cirrhosis who develop HH is 8-12 months. The diagnosis is typically made in the context of advanced liver disease and ascites, in the absence of underlying cardio-pulmonary pathology. A multi-disciplinary approach to management, involving respiratory physicians, hepatologists, and palliative care specialists is crucial to ensuring optimal patient-centered care. However, the majority of accepted therapeutic options are based on expert opinion rather than large, adequately powered randomized controlled trials. In this narrative review, we discuss the epidemiology, pathophysiology, clinical characteristics, and management of HH, highlighting the use of salt restriction and diuretic therapy, porto-systemic shunts, and liver transplantation. We include specific sections focusing on the role of pleural interventions and palliative care, respectively.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 3","pages":"241-254"},"PeriodicalIF":3.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f1/5b/41030_2022_Article_195.PMC9458779.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10390825","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-06-01Epub Date: 2022-04-25DOI: 10.1007/s41030-022-00189-6
Benjamin Wu, David Mannino, George Mu, Marjorie Stiegler, Michael Bogart
Introduction: Single-inhaler fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) triple therapy was approved by the United States Food and Drug Administration in 2017 as a maintenance therapy for chronic obstructive pulmonary disease (COPD). Patient characteristics and treatment patterns prior to initiating FF/UMEC/VI are currently unknown. This study assessed patient characteristics, exacerbation, and medication history in patients with COPD before the initiation of FF/UMEC/VI or multiple-inhaler triple therapy (MITT).
Methods: This was a retrospective study using the Optum Clinformatics® Data Mart. Patients who initiated FF/UMEC/VI triple therapy or MITT (consisting of a long-acting muscarinic antagonist [LAMA], long-acting β2-agonist [LABA], and inhaled corticosteroid [ICS]) between October 2017 and September 2018, were enrolled in commercial or Medicare Advantage Prescription Drug plans, were aged > 40 years, and had a COPD diagnosis were eligible. Patient characteristics, comorbidities, COPD medication use, exacerbations, and eosinophil counts were assessed in the 12-month baseline period prior to initiation of FF/UMEC/VI triple therapy or MITT.
Results: The study population included 3933 FF/UMEC/VI users and 18,244 MITT users. Mean (standard deviation) patient age was 72.2 (8.6) years in FF/UMEC/VI users and 70.7 (9.7) years in MITT users. Prior to initiating triple therapy, the majority of FF/UMEC/VI (89.1%) and MITT (93.8%) users experienced a moderate or severe exacerbation or used a COPD maintenance therapy during the baseline period. In addition, 41.2% of FF/UMEC/VI users received overlapping ICS/LAMA/LABA, 20.3% received ICS/LABA, and 9.7% received LAMA/LABA.
Conclusion: In this population of COPD patients, triple therapy was frequently initiated after previous maintenance medication use or an exacerbation, in line with treatment guideline recommendations.
导言:单吸入剂糠酸氟替卡松/优甲乐胺/维兰特罗(FF/UMEC/VI)三联疗法于2017年获得美国食品和药物管理局批准,作为慢性阻塞性肺疾病(COPD)的维持疗法。目前尚不清楚患者特征和开始使用 FF/UMEC/VI 之前的治疗模式。本研究评估了慢性阻塞性肺病患者在开始使用 FF/UMEC/VI 或多吸入器三联疗法(MITT)前的患者特征、病情加重和用药史:这是一项使用 Optum Clinformatics® Data Mart 进行的回顾性研究。在2017年10月至2018年9月期间开始接受FF/UMEC/VI三联疗法或MITT(由长效毒蕈碱类拮抗剂[LAMA]、长效β2-受体激动剂[LABA]和吸入性皮质类固醇[ICS]组成)的患者,参加了商业或医疗保险优势处方药计划,年龄大于40岁,并确诊为慢性阻塞性肺病,均符合条件。在开始 FF/UMEC/VI 三联疗法或 MITT 之前的 12 个月基线期内,对患者特征、合并症、慢性阻塞性肺病药物使用、病情加重和嗜酸性粒细胞计数进行了评估:研究对象包括3933名FF/UMEC/VI使用者和18244名MITT使用者。FF/UMEC/VI使用者的平均年龄(标准差)为72.2(8.6)岁,MITT使用者的平均年龄为70.7(9.7)岁。在开始使用三联疗法之前,大多数 FF/UMEC/VI 用户(89.1%)和 MITT 用户(93.8%)在基线期间经历过中度或重度病情加重或使用过慢性阻塞性肺病维持疗法。此外,41.2%的FF/UMEC/VI用户接受了ICS/LAMA/LABA重叠治疗,20.3%接受了ICS/LABA治疗,9.7%接受了LAMA/LABA治疗:在这部分慢性阻塞性肺病患者中,根据治疗指南的建议,三联疗法通常是在之前使用过维持性药物或病情加重后开始使用的。
{"title":"Patient and Clinical Demographics of New Users to Single-Inhaler Triple Therapy in Patients with Chronic Obstructive Pulmonary Disease.","authors":"Benjamin Wu, David Mannino, George Mu, Marjorie Stiegler, Michael Bogart","doi":"10.1007/s41030-022-00189-6","DOIUrl":"10.1007/s41030-022-00189-6","url":null,"abstract":"<p><strong>Introduction: </strong>Single-inhaler fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) triple therapy was approved by the United States Food and Drug Administration in 2017 as a maintenance therapy for chronic obstructive pulmonary disease (COPD). Patient characteristics and treatment patterns prior to initiating FF/UMEC/VI are currently unknown. This study assessed patient characteristics, exacerbation, and medication history in patients with COPD before the initiation of FF/UMEC/VI or multiple-inhaler triple therapy (MITT).</p><p><strong>Methods: </strong>This was a retrospective study using the Optum Clinformatics<sup>®</sup> Data Mart. Patients who initiated FF/UMEC/VI triple therapy or MITT (consisting of a long-acting muscarinic antagonist [LAMA], long-acting β2-agonist [LABA], and inhaled corticosteroid [ICS]) between October 2017 and September 2018, were enrolled in commercial or Medicare Advantage Prescription Drug plans, were aged > 40 years, and had a COPD diagnosis were eligible. Patient characteristics, comorbidities, COPD medication use, exacerbations, and eosinophil counts were assessed in the 12-month baseline period prior to initiation of FF/UMEC/VI triple therapy or MITT.</p><p><strong>Results: </strong>The study population included 3933 FF/UMEC/VI users and 18,244 MITT users. Mean (standard deviation) patient age was 72.2 (8.6) years in FF/UMEC/VI users and 70.7 (9.7) years in MITT users. Prior to initiating triple therapy, the majority of FF/UMEC/VI (89.1%) and MITT (93.8%) users experienced a moderate or severe exacerbation or used a COPD maintenance therapy during the baseline period. In addition, 41.2% of FF/UMEC/VI users received overlapping ICS/LAMA/LABA, 20.3% received ICS/LABA, and 9.7% received LAMA/LABA.</p><p><strong>Conclusion: </strong>In this population of COPD patients, triple therapy was frequently initiated after previous maintenance medication use or an exacerbation, in line with treatment guideline recommendations.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 1","pages":"195-208"},"PeriodicalIF":2.3,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9098773/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47827349","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-06-01Epub Date: 2022-02-11DOI: 10.1007/s41030-022-00184-x
Mariam Louis, Peter Staiano, Lavender Micalo, Nauman Chaudary
Cystic fibrosis (CF) is due to a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR), which leads to unusual water and chloride secretion across epithelial surfaces. The lungs are responsible for most morbidity, though other organs are frequently affected. Sleep abnormalities have long been recognized in CF. Abnormal ventilation and oxygenation, sinus disease, deconditioning due to muscle weakness and recurrent infections, and inflammation have been thought to play a role in sleep disorders in CF. However, there is evidence that CFTR gene dysregulation can affect circadian rhythms in CF. Early recognition and treatment of circadian rhythms may improve outcomes in CF.
{"title":"Cystic Fibrosis and Sleep Circadian Rhythms.","authors":"Mariam Louis, Peter Staiano, Lavender Micalo, Nauman Chaudary","doi":"10.1007/s41030-022-00184-x","DOIUrl":"https://doi.org/10.1007/s41030-022-00184-x","url":null,"abstract":"<p><p>Cystic fibrosis (CF) is due to a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR), which leads to unusual water and chloride secretion across epithelial surfaces. The lungs are responsible for most morbidity, though other organs are frequently affected. Sleep abnormalities have long been recognized in CF. Abnormal ventilation and oxygenation, sinus disease, deconditioning due to muscle weakness and recurrent infections, and inflammation have been thought to play a role in sleep disorders in CF. However, there is evidence that CFTR gene dysregulation can affect circadian rhythms in CF. Early recognition and treatment of circadian rhythms may improve outcomes in CF.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"8 2","pages":"139-147"},"PeriodicalIF":3.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a7/54/41030_2022_Article_184.PMC9098776.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39911912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}