共查询到20条相似文献，搜索用时 25 毫秒
1.
Matthew A. Tucker Reva Crandall Nichole Seigler Paula Rodriguez-Miguelez Kathleen T. McKie Caralee Forseen Jeffrey Thomas Ryan A. Harris 《Journal of cystic fibrosis》2017,16(6):752-758
Background
Responses to a single bout of exercise may provide critical information for maximizing improvements in pulmonary function following exercise training in cystic fibrosis (CF). We sought to determine if acute maximal exercise improves pulmonary function in patients with CF.Methods
Thirty-three patients with CF completed a comprehensive assessment of pulmonary function to determine forced vital capacity (FVC), forced expiratory volume in one second (FEV1), and lung clearance index (LCI) prior to and immediately following maximal aerobic exercise on a cycle ergometer.Results
Following exercise, FVC (? 0.08 ± 0.14 L) and FEV1 (? 0.06 ± 0.15 L) increased, while LCI decreased (?-0.71 ± 0.93) (all p < 0.05). Changes in FEV1 (%predicted) were associated with peak work (r = 0.40, p = 0.02) and peak pulmonary ventilation (r = 0.45, p = 0.01).Conclusions
A single bout of maximal exercise acutely improves pulmonary function in patients with CF and improvements may be related to peak work and peak pulmonary ventilation. 相似文献2.
Dominique Hubert Raphaël Chiron Boubou Camara Dominique Grenet Anne Prévotat Laurence Bassinet Stéphane Dominique Gilles Rault Julie Macey Isabelle Honoré Reem Kanaan Sylvie Leroy Nadine Desmazes Dufeu Pierre-Régis Burgel 《Journal of cystic fibrosis》2017,16(3):388-391
Objective
To investigate the short-term adverse events and effectiveness of lumacaftor/ivacaftor combination treatment in adults with cystic fibrosis (CF) and severe lung disease in a real life setting.Methods
A multicentre observational study investigated adverse events, treatment discontinuation, FEV1 and body mass index (BMI) one month and three months after lumacaftor/ivacaftor initiation in adults with CF and FEV1 below 40% predicted.Results
Respiratory adverse events (AEs) were reported by 27 of 53 subjects (51%) and 16 (30%) discontinued treatment. The mean absolute change in FEV1 was + 2.06% after one month of treatment (P = 0.086) and + 3.19% after 3 months (P = 0.009). BMI was unchanged.Conclusions
Treatment with lumacaftor/ivacaftor in patients with CF and severe lung disease was discontinued more frequently than reported in clinical trials, due to respiratory AEs. Nevertheless, the patients who continued treatment had an increase in lung function comparable to what was observed in pivotal trials. 相似文献3.
Barbara Bosch Diana Bilton Patrick Sosnay Karen S. Raraigh Denise Y.F. Mak Hiroshi Ishiguro Vincent Gulmans Muriel Thomas Harry Cuppens Margarida Amaral Kris De Boeck 《Journal of cystic fibrosis》2017,16(4):488-491
Background
The diagnosis of Cystic Fibrosis (CF) is by consensus based on the same parameters in all patients, yet the influence of ethnicity has only scarcely been studied. We aimed at elucidating the impact of Asian descent on the diagnosis of CF.Methods
We performed a retrospective analysis of the CFTR2 and UK CF databases for clinical phenotype, sweat chloride values and CFTR mutations and compared the diagnostic characteristics of Asian to non-Asian patients with CF.Results
Asian patients with CF do not have a worse clinical phenotype. The repeatedly reported lower FEV1 of Asian patients with CF is attributable to the influence of ethnicity on lung function in general. However, pancreatic sufficiency is more common in Asian patients with CF. The diagnosis of CF in people with Asian ancestry is heterogeneous as mean sweat chloride values are lower (92 ± 26 versus 99 ± 22 mmol/L in controls) and 14% have sweat chloride values below 60 mmol/L (versus 6% in non-Asians). Also, CFTR mutations differ from those in Caucasians: 55% of British Asian patients with CF do not have one mutation included in the routine newborn screening panel.Conclusions
Bringing together the largest cohort of patients with CF and Asian ethnicity, we demonstrate that Asian roots impact on all three CF diagnostic pillars. These findings have implications for clinical practice in the increasingly ethnically diverse Western population. 相似文献4.
Barry J Plant Damian G Downey Joe A Eustace Cedric Gunaratnam Charles S Haworth Andrew M Jones Edward F McKone Daniel G Peckham R. Ian Ketchell Diana Bilton 《Journal of cystic fibrosis》2017,16(6):695-701
Background
Studies are required that evaluate real-world outcomes of inhaled aztreonam lysine in patients with cystic fibrosis (CF).Methods
Our treatment-evaluator tool assessed the effectiveness of inhaled aztreonam in routine practice in 117 CF patients across four time periods (6–12 (P2) and 0–6 months (P1) pre-initiation, and 0–6 (T1) and 6–12 months (T2) post-initiation). Outcomes were: changes in %-predicted forced expiratory volume in 1 s (FEV1), body-mass index (BMI), hospitalisation days and intravenous antibiotic usage.Results
Median FEV1% predicted for each 6-month period was 38.9%, 34.6%, 37.1% and 36.5%; median change was ? 2.0% between P2 and P1, increasing to + 0.6% (p < 0.001) between P1 and T1. Annualised hospital bed-days was reduced (p = 0.05) post-initiation, as was intravenous antibiotics days (p = 0.001). BMI increased over 6 months post-initiation (p ≤ 0.001).Conclusions
In patients with CF in routine practice, inhaled aztreonam lysine is associated with improved lung function and weight, and reduced hospitalisation and intravenous antibiotic use. 相似文献5.
K.A. Waters A. Lowe P. Cooper S. Vella Hiran Selvadurai 《Journal of cystic fibrosis》2017,16(2):250-257
Background
In Cystic Fibrosis (CF), early detection and treatment of respiratory disease is considered the standard for respiratory care. Overnight polysomnography (PSG) may help identify respiratory deterioration in young patients with CF.Methods
A prospective cohort study of 46 patients with CF, aged 8–12 years, from a specialist clinic in a tertiary paediatric hospital. Daytime pulmonary function, shuttle test exercise testing and overnight PSG were studied.Results
Of 81 children aged 8–12 years, 46 (57%) agreed to participate. FEV1 (% predicted, mean 74.6%) was normal in 23 (50%), mildly abnormal in 12 (26.1%), moderately abnormal in 10 (21.7%) and severely abnormal in 1 (2.2%). Amongst sleep study parameters, FEV1 (% predicted) showed significant correlation with the respiratory rate (RR) in slow wave sleep (SWS), CO2 change in REM, baseline SaO2, and the arousal index (h? 1). Backward, stepwise linear regression modelling for FEV1 (% predicted) included the entire group with a wide spectrum of clinical severity. From sleep, variables remaining in the multivariate model for FEV1 (F = 16.81, p < 0.001) were the RR in SWS (min? 1) and the CO2 change in REM (p = 0.003, and 0.014, respectively). When daytime tests were included, the variables remaining were RR in SWS and SD score for BMI (BMIsds) (F = 18.70, p < 0.001).Conclusions
Respiratory abnormalities on overnight sleep studies included elevated respiratory rates during SWS and mild CO2 retention in REM sleep, and these incorporated into a model correlating with FEV1 (% predicted). Thus, mild mechanical impairment of ventilation is evident on overnight sleep studies in children with cystic fibrosis although the significance of this finding will require further investigation. 相似文献6.
Moya Vandeleur Lisa M. Walter David S. Armstrong Philip Robinson Gillian M. Nixon Rosemary S.C. Horne 《Journal of cystic fibrosis》2017,16(6):719-726
Background
Sleep disturbance is common in children with cystic fibrosis (CF) however there are limited studies investigating the causes for poor sleep quality. In a cross sectional observational study we aimed to evaluate the clinical correlates of sleep disturbance in this population.Methods
Children with CF (7–18 years) free from pulmonary exacerbation completed medical review, overnight oximetry, the OSA-18 and 14 days of actigraphy recordings with a sleep diary.Results
In addition to FEV1 < 80% and low baseline SpO2, CF-related diabetes, PEG feeding and co-morbid behaviour disorder were associated with lower objective sleep quantity. Paternal smoking and a family member with a mood disorder were also associated with sleep disturbance. The use of electronic devices before bedtime was associated with lower sleep quantity and quality. FEV1, nocturnal cough, age and a behaviour disorder predicted sleep duration. FEV1, nocturnal cough, SpO2 nadir and asthma predicted sleep efficiency. Conversely, sleep efficiency independently predicted FEV1.Conclusions
Reduced sleep quality in children with CF is related to lung health and co-morbidities. However, family characteristics and poor sleep hygiene in the child were also associated with sleep disturbance. Optimal management of CF would seem to be the primary intervention to alleviate children's sleep disturbance, however our data raises additional targets for attempts to improve sleep. 相似文献7.
Natalie E. West Valeria V. Beckett Raksha Jain Don B. Sanders Jerry A. Nick Sonya L. Heltshe Elliott C. Dasenbrook Donald R. VanDevanter George M. Solomon Christopher H. Goss Patrick A. Flume 《Journal of cystic fibrosis》2017,16(5):600-606
Background
Pulmonary Exacerbations (PEx) are associated with increased morbidity and mortality in individuals with CF. PEx management practices vary widely, and optimization through interventional trials could potentially improve outcomes. The object of this analysis was to evaluate current physician treatment practices and patient outcomes for PEx.Methods
The Standardized Treatment of Pulmonary Exacerbations (STOP) observational study enrolled 220 participants ≥ 12 years old admitted to the hospital for PEx at 11 U.S. CF centers. Spirometry and daily symptom scores were collected during the study. Physicians were surveyed on treatment goals and their management practices were observed. Treatment outcomes were compared to stated goals.Results
The mean (SD) duration of IV antibiotic treatment was 15.9 (6.0) days. Those individuals with more severe lung disease (< 50% FEV1) were treated nearly two days longer than those with > 50% FEV1. Physician-reported FEV1 improvement goals were 10% (95% CI: 5%, 14%) lower for patients with 6-month baseline FEV1 ≤ 50% predicted compared with those with 6-month baseline FEV1 > 50% predicted. There were clinically and statistically significant improvements in symptoms from the start of IV antibiotic treatment to the end of IV antibiotic treatment and 28 days after the start of treatment. The mean absolute increase in FEV1 from admission was 9% predicted at end of IV antibiotic treatment, and 7% predicted at day 28. Only 39% fully recovered lost lung function, and only 65% recovered at least 90% of lost lung function. Treatment was deemed successful by 84% of clinicians, although 6-month baseline FEV1 was only recovered in 39% of PEx.Conclusions
In this prospective observational study of PEx, treatment regimens and durations showed substantial variation. A significant proportion of patients did not reach physician's treatment goals, yet treatment was deemed successful. 相似文献8.
D.R. VanDevanter S.L. Heltshe J. Spahr V.V. Beckett C.L. Daines E.C. Dasenbrook R.L. Gibson R. Jain D.B. Sanders C.H. Goss P.A. Flume 《Journal of cystic fibrosis》2017,16(5):607-615
Background
Given the variability in pulmonary exacerbation (PEx) management within and between Cystic Fibrosis (CF) Care Centers, it is possible that some approaches may be superior to others. A challenge with comparing different PEx management approaches is lack of a community consensus with respect to treatment-response metrics. In this analysis, we assess the feasibility of using different response metrics in prospective randomized studies comparing PEx treatment protocols.Methods
Response parameters were compiled from the recent STOP (Standardized Treatment of PEx) feasibility study. Pulmonary function responses (recovery of best prior 6-month and 12-month FEV1% predicted and absolute and relative FEV1% predicted improvement from treatment initiation) and sign and symptom recovery from treatment initiation (measured by the Chronic Respiratory Infection Symptom Score [CRISS]) were studied as categorical and continuous variables. The proportion of patients retreated within 30 days after the end of initial treatment was studied as a categorical variable. Sample sizes required to adequately power prospective 1:1 randomized superiority and non-inferiority studies employing candidate endpoints were explored.Results
The most sensitive endpoint was mean change in CRISS from treatment initiation, followed by mean absolute FEV1% predicted change from initiation, with the two responses only modestly correlated (R2 = .157; P < 0.0001). Recovery of previous best FEV1 was a problematic endpoint due to missing data and a substantial proportion of patients beginning PEx treatment with FEV1 exceeding their previous best measures (12.1% > 12-month best, 19.6% > 6-month best). Although mean outcome measures deteriorated approximately 2-weeks post-treatment follow-up, the effect was non-uniform: 62.7% of patients experienced an FEV1 worsening versus 49.0% who experienced a CRISS worsening.Conclusions
Results from randomized prospective superiority and non-inferiority studies employing mean CRISS and FEV1 change from treatment initiation should prove compelling to the community. They will need to be large, but appear feasible. 相似文献9.
Alison DaCosta Cameron L. Jordan Olivia Giddings Feng-Chang Lin Peter Gilligan Charles R. Esther 《Journal of cystic fibrosis》2017,16(4):483-487
Background
Mycobacterium abscessus infection is associated with declining lung function in cystic fibrosis (CF), but there is little evidence on clinical efficacy to guide treatment.Methods
Retrospective review of 37 CF patients treated for M. abscessus respiratory infection at a single center from 2006 to 2014. Outcomes included change in FEV1 at 30, 60, 90, 180, and 365 days after treatment and clearance of M. abscessus from sputum cultures.Results
Lung function was significantly improved after 30 and 60 days of treatment, but not at later time points. Gains were inversely related to starting lung function. Antibiotic choices did not influence outcomes except for greater clearance with clarithromycin.Conclusions
Treatment of M. abscessus resulted in short term improvement in lung function that is inversely related to pre-treatment FEV1. 相似文献10.
George M. Solomon Bo Liu Isabelle Sermet-Gaudelus Isabelle Fajac Michael Wilschanski Francois Vermeulen Steven M. Rowe 《Journal of cystic fibrosis》2017,16(5):573-578
Background
Nasal Potential Difference (NPD) is a biomarker of CFTR activity used to diagnose CF and monitor experimental therapies. Limited studies have been performed to assess agreement between expert readers of NPD interpretation using a scoring algorithm.Methods
We developed a standardized scoring algorithm for “interpretability” and “confidence” for PD (potential difference) measures, and sought to determine the degree of agreement on NPD parameters between trained readers.Results
There was excellent agreement for interpretability between NPD readers for CF and fair agreement for normal tracings but slight agreement of interpretability in indeterminate tracings. Amongst interpretable tracings, excellent correlation of mean scores for Ringer's Baseline PD, Δamiloride, and ΔCl-free + Isoproterenol was observed. There was slight agreement regarding confidence of the interpretable PD tracings, resulting in divergence of the Ringers and Δamiloride, and ΔCl-free + Isoproterenol PDs between “high” and “low” confidence CF tracings.Conclusion
A multi-reader process with adjudication is important for scoring NPDs for diagnosis and in monitoring of CF clinical trials. 相似文献11.
Don B. Sanders George M. Solomon Valeria V. Beckett Natalie E. West Cori L. Daines Sonya L. Heltshe Donald R. VanDevanter Jonathan E. Spahr Ronald L. Gibson Jerry A. Nick Bruce C. Marshall Patrick A. Flume Christopher H. Goss 《Journal of cystic fibrosis》2017,16(5):592-599
Background
The Standardized Treatment of Pulmonary Exacerbations (STOP) program has the intent of defining best practices in the treatment of pulmonary exacerbations (PEx) in patients with cystic fibrosis (CF). The objective of this analysis was to describe the clinical presentations of patients admitted for intravenous (IV) antibiotics and enrolled in a prospective observational PEx study as well as to understand physician treatment goals at the start of the intervention.Methods
We enrolled adolescents and adults admitted to the hospital for a PEx treated with IV antibiotics. We recorded patient and PEx characteristics at the time of enrollment. We surveyed treating physicians on treatment goals as well as their willingness to enroll patients in various study designs. Additional demographic and clinical data were obtained from the CF Foundation Patient Registry.Results
Of 220 patients enrolled, 56% were female, 19% were adolescents, and 71% were infected with P. aeruginosa. The mean (SD) FEV1 at enrollment was 51.1 (21.6)% predicted. Most patients (85%) experienced symptoms for ≥ 7 days before admission, 43% had received IV antibiotics within the previous 6 months, and 48% received oral and/or inhaled antibiotics prior to IV antibiotic initiation. Forty percent had ≥ 10% FEV1 decrease from their best value recorded in the previous 6 months, but for 20% of patients, their enrollment FEV1 was their best FEV1 recorded within the previous 6 months. Physicians reported that their primary treatment objectives were lung function recovery (53%) and improvement of symptoms (47%) of PEx. Most physicians stated they would enroll patients in studies involving 10-day (72%) or 14-day (87%), but not 7-day (29%), treatment regimens.Conclusions
Based on the results of this study, prospective studies are feasible and physician willingness for interventional studies of PEx exists. Results of this observational study will help design future PEx trials. 相似文献12.
Background
There are few tools to quantify the impact of cough in cystic fibrosis (CF). The psychometric properties of the Leicester Cough Questionnaire (LCQ) and Respiratory Symptoms in CF (ReS-CF) tool were investigated in adults with CF.Methods
Validity and reliability were assessed in clinically stable participants who completed the questionnaires twice, along with the Cystic Fibrosis Questionnaire – Revised (CFQ-R). Responsiveness was assessed by change in questionnaires following treatment for an acute respiratory exacerbation.Results
Correlations between the LCQ and CFQ-R respiratory domain were moderate (n = 59, rs = 0.78, p < 0.001). Correlations between ReS-CF and CFQ-R respiratory domain were fair (rs = ? 0.50, p < 0.001). The LCQ total score was repeatable (ICC 0.92, 95%CI 0.87–0.96, n = 50). In those reporting improvement in symptoms following treatment (n = 36), LCQ total score had a mean change of 4.6 (SD 3.7) and effect size of 1.2.Conclusions
The LCQ and ReS-CF appear to be valid, reliable and responsive in CF.Trial Registration: www.anzctr.org.au: ACTRN12615000262505 相似文献13.
Millie Garg Steven T. Leach Michael J. Coffey Tamarah Katz Roxanne Strachan Tamara Pang Bronwen Needham Kei Lui Fathalla Ali Andrew S. Day Laura Appleton Vesal Moeeni Adam Jaffe Chee Y. Ooi 《Journal of cystic fibrosis》2017,16(5):631-636
Background
Fecal calprotectin may be used as a non-invasive method to assess the effect of novel therapies on the gut in cystic fibrosis (CF).Method
Stools from CF patients and healthy controls (HC) (0–10 years old) were prospectively collected for evaluation of temporal trends.Results
130 CF samples (64 subjects) and 114 HC samples (101 subjects) were collected. Overall, fecal calprotectin levels were different in CF patients and HC from 0 to 10 years (P = 0.0002). Fecal calprotectin in CF was significantly lower than HC from 0 to 1 years (P = 0.03) and demonstrated an upward trajectory until 4 years. From > 4 to 10 years calprotectin was consistently higher in CF patients compared with HC (P = 0.007).Conclusions
Fecal calprotectin levels in children with CF and HC were age-dependent and had distinct trajectories. Careful interpretation of calprotectin is required if used in drug trials for CF, particularly in children less than 4 years old. 相似文献14.
Nina Dickerhof Rufus Turner Irada Khalilova Emmanuelle Fantino Peter D Sly Anthony J Kettle 《Journal of cystic fibrosis》2017,16(2):214-221
Background
In cystic fibrosis (CF) there is an urgent need for earlier diagnosis of pulmonary infections and inflammation using blood- and urine-based biomarkers.Methods
Using mass spectrometry, oxidation products of glutathione and uric acid were measured in matched samples of bronchoalveolar lavage (BAL), serum and urine from 36 infants and children with CF, and related to markers of neutrophilic inflammation and infection in BAL.Results
Oxidation products of glutathione (glutathione sulfonamide, GSA) and uric acid (allantoin), were elevated in BAL of children with pulmonary infections with Pseudomonas aeruginosa (PsA) compared to those without (p < 0.05) and correlated with other markers of neutrophilic inflammation. Serum GSA was significantly elevated in children with PsA infections (p < 0.01). Urinary GSA correlated with pulmonary GSA (r = 0.42, p < 0.05) and markers of neutrophilic inflammation.Conclusions
This proof-of-concept study demonstrates that urinary GSA but not allantoin shows promise as a non-invasive marker of neutrophilic inflammation in early CF lung disease. 相似文献15.
16.
Danielle Usatin Elizabeth H. Yen Catherine McDonald Fadi Asfour John Pohl Jacob Robson 《Journal of cystic fibrosis》2017,16(4):503-509
Background
Early childhood growth status has been used to predict long-term clinical outcomes in Cystic Fibrosis (CF) patients. Adulthood CF outcomes based on early weight-for-length (WFL) measurements, using either World Health Organization (WHO) or Centers for Disease Control (CDC) scales, have not been compared.Methods
Cystic Fibrosis Foundation registry patients were studied (n = 3014). Participants were categorized at age two years as WFL < 50th percentile on both WHO and CDC scales, ≥ 50th percentile on WHO but not CDC, or ≥ 50th percentile on both. Pulmonary function and overall survival were assessed at age 18 years.Results
Stepwise gains in pulmonary function and lung transplant-free survival were noted across the three increasing WFL categories.Conclusions
Children with CF who achieve higher WFL at age two years have improved pulmonary and survival outcomes into adulthood. CF providers should continue to utilize current early growth recommendations, with goal WFL ≥ 50th percentile on CDC growth charts before age two. 相似文献17.
Rhonda Szczesniak Sonya L. Heltshe Sanja Stanojevic Nicole Mayer-Hamblett 《Journal of cystic fibrosis》2017,16(3):318-326
Background
Forced expiratory volume in 1 s (FEV1) is an established marker of cystic fibrosis (CF) disease progression that is used to capture clinical course and evaluate therapeutic efficacy. The research community has established FEV1 surveillance data through a variety of observational data sources such as patient registries, and there is a growing pipeline of new CF therapies demonstrated to be efficacious in clinical trials by establishing improvements in FEV1.Results
In this review, we summarize from a statistical perspective the clinical relevance of FEV1 based on its association with morbidity and mortality in CF, its role in epidemiologic studies of disease progression and comparative effectiveness, and its utility in clinical trials. In addition, we identify opportunities to advance epidemiologic research and the clinical development pipeline through further statistical considerations.Conclusions
Our understanding of CF disease course, therapeutics, and clinical care has evolved immensely in the past decades, in large part due to the thoughtful application of rigorous research methods and meaningful clinical endpoints such as FEV1. A continued commitment to conduct research that minimizes the potential for bias, maximizes the limited patient population, and harmonizes approaches to FEV1 analysis while maintaining clinical relevance, will facilitate further opportunities to advance CF care. 相似文献18.
19.
C. Lehoux Dubois V. Boudreau F. Tremblay A. Lavoie Y. Berthiaume R. Rabasa-Lhoret A. Coriati 《Journal of cystic fibrosis》2017,16(3):418-424
Background
Diabetes is common in cystic fibrosis (CF). Glucose can be detected in the airway when the blood glucose is elevated, which favours bacterial growth. We investigated the relationship between dysglycemia and lung pathogens in CF.Methods
Cross-sectional and prospective analysis of CF patients (N = 260) who underwent a 2 h-oral glucose tolerance test. Clinical data was collected.Results
Stenotrophomonas maltophilia (S. maltophilia) was the sole bacteria increased in dysglycemic (AGT: 20.2%, CFRD: 21.6%) patients compared to normotolerants (NGT: 8.7%). S. maltophilia positive patients with dysglycemia had more pulmonary exacerbation events compared to NGTs (1.22 vs 0.63, P = 0.003). The interaction between S. maltophilia colonisation and glucose tolerance status significantly increases the risk of lower lung function (P = 0.003). Its growth was not affected by the evolution of the glucose tolerance after three years follow-up.Conclusion
Prevalence of S. maltophilia was higher in dysglycemic patients, supporting the idea that S. maltophilia is a marker of disease severity in CF. 相似文献20.