共查询到20条相似文献，搜索用时 101 毫秒
1.
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2.
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.
Donald R. VanDevanter Nicole Mayer-Hamblett Michael Boyle 《Journal of cystic fibrosis》2017,16(4):496-498
Background
New CFTR modulators are in development that sponsors anticipate will be comparable or superior to approved modulators. Testing these agents for efficacy will require either placebo-controlled or active-comparator trials.Methods
We surveyed US CF physicians and their patients eligible to receive approved modulators or their families for willingness to participate in placebo-controlled modulator trials of varying duration.Results
Interest in placebo-controlled trials of short duration (2–4 weeks) was greatest, with few respondents, particularly among patient respondents, willing to consider 6 month studies. Patients/families with access to approved modulators were consistently less interested in placebo-controlled modulator trials of any duration.Conclusions
Sample size and interpretability advantages of placebo-controlled trials outweigh alternative active-comparator trials, but must consider physician and patient thresholds for forgoing treatment with approved modulators. Enrollment will be most feasible for short-duration trials and those conducted among populations without access to approved modulators. 相似文献5.
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6.
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7.
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8.
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9.
William Poncin Florian Singer Anne-Sophie Aubriot Patrick Lebecque 《Journal of cystic fibrosis》2017,16(2):258-266
Background
Comparability of multiple breath washout (MBW) systems has been little explored. We assessed agreement in lung clearance index (LCI) from two similar, commercial nitrogen MBW setups in patients with Cystic Fibrosis (CF) and controls.Methods
The EasyOne Pro (NDD) and Exhalyzer D (EM) were randomly applied in 85 adults (34 with CF) and 97 children (47 with CF and normal forced expiratory volume in one second). We assessed differences between setups in LCI, lung volumes and breathing pattern and diagnostic performance for detecting abnormal lung function.Results
Compared to NDD, EM measured higher LCI, functional residual capacity and cumulative expired volume while respiratory rate was lower. Mean difference (limits of agreement) in LCI was 1.30 (? 2.34 to 4.94). In CF, prevalence of abnormal LCI was greater in children and similar in adults using EM compared to NDD.Conclusions
Agreement of MBW outcomes between setups is poor and explained by nitrogen measurement techniques and breathing pattern. 相似文献10.
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11.
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12.
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13.
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14.
Angela C. Garinis Campbell P. Cross Priya Srikanth Kelly Carroll M. Patrick Feeney Douglas H. Keefe Lisa L. Hunter Daniel B. Putterman David M. Cohen Jeffrey A. Gold Peter S. Steyger 《Journal of cystic fibrosis》2017,16(3):401-409
Background
Aminoglycosides (AGs) and glycopeptides are antibiotics essential for treating life-threatening respiratory infections in patients with cystic fibrosis (CF). The goal of this study was to examine the effects of cumulative intravenous (IV)-AG (amikacin and/or tobramycin) and/or glycopeptide (vancomycin) dosing on hearing status in patients with CF.Methods
Hearing thresholds were measured from 0.25 to 16.0 kHz, in 81 participants with CF. Participants were categorized into two groups: normal hearing in both ears (≤ 25 dB HL for all frequency bands) or hearing loss (> 25 dB HL for any frequency band in either ear). Participants were also characterized into quartiles by their cumulative IV-AG (with or without vancomycin) exposure. Dosing was calculated using two strategies: (i) total number of lifetime doses, and (ii) total number of lifetime doses while accounting for the total doses per day. This was referred to as the “weighted” method.Results
Participants in the hearing loss group were significantly older than those in the normal-hearing group. After adjusting for gender and age at the time of hearing test, participants in the two highest-quartile exposure groups were almost 5 X more likely to have permanent sensorineural hearing loss than those in the two lowest-quartile exposure groups. There was a small group of CF patients who had normal hearing despite high exposure to IV-antibiotics.Conclusions
Cumulative IV-antibiotic dosing has a significant negative effect on hearing sensitivity in patients with CF, when controlling for age and gender effects. A trend for increasing odds of hearing loss was associated with increasing cumulative IV-antibiotic dosing. 相似文献15.
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16.
Thomas Radtke Helge Hebestreit Milo A. Puhan Susi Kriemler 《Journal of cystic fibrosis》2017,16(6):744-751
Background
We aimed to characterize the cardiopulmonary response during a 1-min sit-to-stand (STS) test and compare peak exercise cardiorespiratory variables to a maximal cardiopulmonary exercise test (CPET) in cystic fibrosis (CF). We further aimed to assess the validity of the STS power index (PowerSTS) as a measure of exercise capacity.Methods
Fifteen adult CF patients performed spirometry, CPET and the 1-min STS test with respiratory gas analysis.Results
Peak-exercise cardiorespiratory variables during the 1-min STS test correlated strongly (r = 0.69–0.98) with those measured during the CPET. Oxygen uptake, carbon dioxide production, heart rate, ventilation, and tidal volume at peak exercise were 24%, 26%, 9%, 10% and 21% lower in the 1-min STS test, while respiratory frequencies were 14% higher. PowerSTS showed strong to very strong correlations with CPET-derived absolute peak oxygen uptake and maximal workload.Conclusions
The 1-min STS test elicits a substantial but lower cardiorespiratory response compared to a maximal cycle ergometry CPET. While PowerSTS and STS repetitions are both valid outcome measures of functional capacity, STS repetitions are clinically more practical. 相似文献17.
Siddhartha G. Kapnadak Gregory A. Kicska Kathleen J. Ramos Desiree A. Marshall Tamara Y. Carroll Sudhakar N. Pipavath Michael S. Mulligan Christopher H. Goss Moira L. Aitken 《Journal of cystic fibrosis》2017,16(6):735-743
Background
Tracheal diverticula (TD) are rare anomalies that may harbor infected secretions, posing potential risk to patients with lung disease. In an end-stage cystic fibrosis (CF) cohort, we describe the characteristics and associated post-lung transplant (LTx) outcomes of TD.Methods
Pre-transplant computed tomography (CT)'s were reviewed in CF patients undergoing LTx. TD were characterized radiographically and on autopsy when available. Pre-transplant clinical variables and post-transplant outcomes were compared by TD status.Results
Of 93 patients, 35 (37.6%) had TD. 58% of TD had fat-stranding, and post-mortem TD examinations revealed histology carrying intense submucosal inflammation, and purulent contents that cultured identical species to sputum. There was no difference in post-LTx survival [HR 1.77 (0.82–3.82), p = 0.147], bacterial re-colonization, or rejection in patients with TD compared to those without. Patients with TD were more likely to die from infection, but the result was not statistically significant [HR 2.02 (0.62–6.63), p = 0.245].Conclusions
We found a high prevalence of TD in end-stage CF, where diverticula may represent a large-airway bacterial reservoir. TD were not associated with differences in post-LTx outcomes, but given the infectious concerns further investigation is necessary. 相似文献18.
Erin M. Lowery William Adams Shellee A. Grim Nina M. Clark Leah Edwards Jennifer E. Layden 《Journal of cystic fibrosis》2017,16(6):727-734
Background
Post-transplant lymphoproliferative disease (PTLD) is an important cause of morbidity and mortality following lung transplantation. Recipients with cystic fibrosis (CF) may have an increased risk of PTLD although the literature is limited to single center cohorts. Our primary aim is to examine PTLD in an adult lung transplant population by utilizing the International Society for Heart and Lung Transplantation Registry.Methods
We studied 30,598 adult recipients of lung transplants performed between 1999 and 2011. The primary outcome was development of and time to PTLD. In addition to indication for transplant, other predictors examined included Epstein–Barr virus (EBV) and cytomegalovirus (CMV) serostatus, gender, and age. Outcomes were assessed with univariable and multivariable Cox proportional hazard models to obtain hazard ratios (HR).Results
17% of the cohort had a diagnosis of CF. PTLD developed in 2% of CF recipients compared to 1% for non-CF recipients (p < 0.001). Compared to non-CF recipients, CF recipients had higher prevalence of EBV and CMV seronegativity and higher prevalences of high risk EBV and CMV mismatch (D +/R ?). There is a significant association between CF and the development of PTLD [HR 1.66 (95% CI 1.30–2.12)]. Stratified multivariable analysis controlling for age revealed EBV negative non-CF recipients have an almost 2 fold increased risk of developing PTLD, whereas EBV negative CF recipients had an almost 6.5 fold increased risk.Conclusions
CF recipients have a higher risk for PTLD compared to non-CF recipients. Further studies are needed to account for additional risk factors and management in this population post-transplant. 相似文献19.
Sonya L. Heltshe Emily M. Godfrey Tatiana Josephy Moira L. Aitken Jennifer L. Taylor-Cousar 《Journal of cystic fibrosis》2017,16(6):687-694
Background
Little is known about how new therapies that partially correct the basic cystic fibrosis (CF) defect (ivacaftor and lumacaftor) might alter hormonal contraceptive effectiveness, impact pregnancy outcomes, or affect pregnancy timing. Examination of pregnancy rates among CF women during periods of CFTR modulator therapy initiation will provide foundation for further research in this area.Methods
The Cystic Fibrosis Foundation Patient Registry was used to examine pregnancy rates and outcomes by genotype class before, during, and after the introduction of CFTR modulator therapies between 2005 and 2014.Results
Among women with CF, ages 15–44 years, there was a slight downward trend in annual pregnancy rates from 2005 to 2014 (2% reduction per year, p = 0.041). Among women with G551D, pregnancy rates during phase 3 ivacaftor trial years was 14.4/1000 women-years compared to 34.0/1000 prior to the trial period (relative risk [RR] = 0.65; 95% CI = 0.43–0.96; p = 0.011) and 38.4/1000 after drug approval in June 2012 (RR = 1.52 post-approval compared to trial period; 95% CI = 1.26, 1.83; p < 0.001). Pregnancy outcomes did not significantly change between 2005 and 2014 for any genotype class.Conclusion
Evidence of significantly increased numbers of pregnancies among women taking approved CFTR modulators is important because of the unknown risk to pregnancy and fetal outcomes. Increases may be temporary following pregnancy prevention during controlled clinical trials, or from altered perceptions about maternal survival with new approved treatments. As more women with CF become eligible to receive modulators, the CF community must study their effect on contraceptive efficacy and safety during pregnancy. With increased health and survival due to modulation, family planning topics will become more common in CF. 相似文献20.
L. Nkam J. Lambert A. Latouche G. Bellis PR. Burgel M.N. Hocine 《Journal of cystic fibrosis》2017,16(6):702-708