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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.
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5.
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6.
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7.
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8.
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9.
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.
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12.
Inhaled dry powder mannitol in children with cystic fibrosis: A randomised efficacy and safety trial
K De Boeck E Haarman J Hull LC Lands A Moeller A Munck J Riethmüller H Tiddens S Volpi J Leadbetter B Charlton A Malfroot 《Journal of cystic fibrosis》2017,16(3):380-387
Introduction
Inhaled mannitol has beneficial effects on lung function, mucociliary clearance, quality of life and sputum properties. This trial examined the efficacy of inhaled mannitol in children with cystic fibrosis (CF).Methods
The efficacy of inhaled mannitol in children with CF aged 6–17 years was assessed in a phase 2, randomised, placebo-controlled crossover study. Subjects were randomly assigned to mannitol 400 mg every 12 h or matching placebo for 8 weeks, followed by an 8 week washout and an 8 week period with the alternate treatment. The primary endpoint was the absolute change from baseline in ppFEV1 (percent predicted FEV1).Results
A total of 92 subjects were studied, with a mean age of 12 years and mean baseline ppFEV1 of 72.2%. During mannitol treatment ppFEV1 was 3.42% (p = 0.004) higher compared to placebo or a 4.97% (p = 0.005) relative difference; relative change from baseline FEF25-75 was 10.52% (p = 0.013). During mannitol treatment, acute post-treatment sputum weight was higher (p = 0.012). In pre-specified subgroups (rhDNase use, age, and disease severity), the treatment differences consistently favoured mannitol. The most common AEs were cough and pulmonary exacerbations. Pulmonary exacerbation AEs were approximately 30% lower in the mannitol group.Conclusions
In children with CF, inhaled mannitol was associated with significant improvements in lung function and sputum weight, irrespective of rhDNase use, age or disease severity. Inhaled mannitol was well tolerated and was associated with a reduced incidence of pulmonary exacerbation AEs. (Clinical Trials.Gov: NCT 01883531) 相似文献13.
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.
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16.
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17.
Scott H. Donaldson George M. Solomon Pamela L. Zeitlin Patrick A. Flume Alicia Casey Karen McCoy Edith T. Zemanick Arun Mandagere Janice M. Troha Steven A. Shoemaker James F. Chmiel Jennifer L. Taylor-Cousar 《Journal of cystic fibrosis》2017,16(3):371-379
Background
Cavosonstat (N91115), an orally bioavailable inhibitor of S-nitrosoglutathione reductase, promotes cystic fibrosis transmembrane conductance regulator (CFTR) maturation and plasma membrane stability, with a mechanism of action complementary to CFTR correctors and potentiators.Methods
A Phase I program evaluated pharmacokinetics, drug–drug interactions and safety of cavosonstat in healthy and cystic fibrosis (CF) subjects homozygous for F508del-CFTR. Exploratory outcomes included changes in sweat chloride in CF subjects.Results
Cavosonstat was rapidly absorbed and demonstrated linear and predictable pharmacokinetics. Exposure was unaffected by a high-fat meal or rifampin-mediated effects on drug metabolism and transport. Cavosonstat was well tolerated, with no dose-limiting toxicities or significant safety findings. At the highest dose, significant reductions from baseline in sweat chloride were observed (? 4.1 mmol/L; P = 0.032) at day 28.Conclusions
The favorable safety and clinical profile warrant further study of cavosonstat in CF.ClinicalTrials.gov Numbers: NCT02275936, NCT02013388, NCT02500667 相似文献18.
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19.
L. Nkam J. Lambert A. Latouche G. Bellis PR. Burgel M.N. Hocine 《Journal of cystic fibrosis》2017,16(6):702-708
Background
Therapeutic progress in patients with cystic fibrosis (CF) has resulted in improved prognosis over the past decades. We aim to reevaluate prognostic factors of CF and provide a prognostic score to predict the risk of death or lung transplantation (LT) within a 3-year period in adult patients.Methods
We developed a logistic model using data from the French CF Registry and combined the coefficients into a prognostic score. The discriminative abilities of the model and the prognostic score were assessed by c-statistic. The prognostic score was validated using a 10-fold cross-validation.Results
The risk of death or LT within 3 years was related to eight characteristics. The development and the validation provided excellent results for the prognostic score; the c-statistic was 0.91 and 0.90 respectively.Conclusion
The score developed to predict 3-year death or LT in adults with CF might be useful for clinicians to identify patients requiring specialized evaluation for LT. 相似文献20.
Talissa A. Altes Mac Johnson Meredith Fidler Martyn Botfield Nicholas J. Tustison Carlos Leiva-Salinas Eduard E. de Lange Deborah Froh John P. Mugler 《Journal of cystic fibrosis》2017,16(2):267-274