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1.
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2.
E.P. Dellon J. Goggin E. Chen K. Sabadosa S.E. Hempstead A. Faro K. Homa 《Journal of cystic fibrosis》2018,17(3):416-421
Background
The goal of palliative care is to improve quality of life for people with serious illness. We aimed to create a cystic fibrosis (CF)-specific definition of palliative care.Methods
A working group of 36 CF care providers, researchers, palliative care providers, quality improvement experts, individuals with CF, and CF caregivers completed a series of questionnaires to rate the value of each of 22 attributes of palliative care, rank top attributes to construct definitions of palliative care, and then rate proposed definitions.Results
An average of 28 participants completed each of four questionnaires, with consistent distribution of stakeholder roles across questionnaires. Many identified overlaps in routine CF care and palliative care and highlighted the importance of a definition that feels relevant across the lifespan.Conclusion
Modified Delphi methodology was used to define palliative care in CF. The definition will be used as the foundation for development of CF-specific palliative care guidelines. 相似文献3.
Samuel Jessula Michiel Van Den Hof Dimas Mateos-Corral Jessica Mills Dafydd Davies Rodrigo LP Romao 《Journal of pediatric surgery》2018,53(11):2150-2154
Background/Purpose
To identify prenatal and perinatal predictors of surgery and describe surgical findings/outcomes of neonates with Meconium Ileus (MI) secondary to Cystic Fibrosis (CF).Methods
Potential risk factors (prenatal bowel echogenicity, CF genotype, birthweight, prematurity and sex) for MI and surgery were examined in a retrospective cohort of neonates with CF presenting to a tertiary center between 1997 and 2015. Following univariable analysis, predictors of MI and surgery were determined using multivariable logistic regression. For surgical patients, detailed operative findings and outcomes were examined.Results
MI was diagnosed in 26/120 (21.7%) neonates with CF and 19/26 (73.0%) required surgery. Prematurity was significantly associated with increased risk of MI and operative intervention (p-value 0.022 and p-value 0.016 respectively); lower birthweight was associated with operative intervention (p-value 0.039); genotype and echogenic bowel were associated with neither. Surgical data were available for 17/19 patients; median age at surgery was 2?days (IQR1–3), 4/17 had an atresia and 6/17 received an ostomy. Median NICU and hospital stays were 34.5 and 70?days while median time on TPN and time to ostomy reversal were 28.5 and 97?days, respectively.Conclusions
In patients with CF, prematurity and lower birthweight were identified as risk factors for meconium ileus and need for surgery. Specific genotypes and echogenic bowel were not predictors of either.Level of evidence
Level III. 相似文献4.
Katherine Keenan Annie Dupuis Katherine Griffin Carlo Castellani Elizabeth Tullis Tanja Gonska 《Journal of cystic fibrosis》2019,18(2):265-270
Background
The “mild” gene variant, p.Arg117His in cystic fibrosis (CF) results in highly variable phenotypes ranging from male infertility to severe lung disease. Due to current interest to include this group in CFTR-targeted therapies, this study aims to describe the disease spectrum.Methods
Retrospective study of Toronto CF and CFTR-related p.Arg117His patients. Longitudinally captured clinical data were compared between patients with 5T/7T-variants and those with a CF or CFTR-related diagnosis. Comparison was made between p.Arg117His adults and infants identified through CF newborn screening (NBS).Results
Twenty of fifty patients carried the 5T variant, all with a diagnosis of CF (p.Arg117His-5TCF), and 30/50 carried 7T, 7 diagnosed with CF (p.Arg117His-7TCF) and 23 with a CFTR-related disorder (p.Arg117His-7TCFTR). For those with chest HRCT results available, 75% p.Arg117His-5TCF, 33% p.Arg117His-7TCF and 27% p.Arg117His-7TCFTR patients had bronchiectasis. Further, 79% p.Arg117His-5T, 29% p.Arg117His-7TCF and 13% p.Arg117His-7TCFTR had abnormal lung function. Of those, 80% grew CF-related pathogens on respiratory culture. Interestingly, the mean maximum sweat chloride and the percentage of patients growing CF-related bacterial pathogens were identical in p.Arg117His-7?TCFTR adults and p.Arg117His infants.Conclusions
Generally, p.Arg117His-5T patients had more severe CF disease. However, a subset of p.Arg117His-7?T patients demonstrated equally severe disease, thus warranting clinical monitoring of all p.Arg117His patients including p.Arg117His infants identified via NBS. 相似文献5.
Emily E. Barsky Luis M. Pereira Keri J. Sullivan Alanna Wong Alexander J. McAdam Gregory S. Sawicki Gregory P. Priebe Susan M. Goobie 《Journal of cystic fibrosis》2018,17(3):e25-e31
Background
Methicillin-resistant Staphylococcus aureus (MRSA) is a prevalent pathogen in patients with cystic fibrosis (CF) associated with increased morbidity. Ceftaroline fosamil is an intravenous (IV) cephalosporin with activity against MRSA. There are minimal data regarding dosing in the CF population. The objective of this study was to determine the pharmacokinetic and pharmacodynamic profile of IV ceftaroline in patients with CF.Methods
We conducted a single-center prospective study of children and young adults with CF receiving ceftaroline (15 mg/kg IV up to 600 mg every 8 h) as part of treatment for a CF pulmonary exacerbation between June 2016 and April 2017. Seven patients were enrolled for a total of 10 treatment courses. For each treatment course, up to 8 plasma samples were assayed for ceftaroline using ultra-high performance liquid chromatography with mass spectrometry. Maximum plasma concentration, systemic clearance, and elimination half-life were calculated. The area under the curve (AUC) above the minimum inhibitory concentration (MIC) and the percent time above the MIC (%fT > MIC) were determined for each subject using MICs of 0.5, 1, and 2 μg/mL and the measured MIC if available.Results
The mean (SD) age for the 7 patients was 20.3 (8.0) years. Mean (SD) maximum plasma concentration of ceftaroline was 22.7 (9.6) μg/mL, systemic clearance 7.9 (3.3) L/h, and half-life 1.1 (0.4) hours. Using a MIC of 1 μg/mL, accepted as the MIC 90 of MRSA isolates, AUC above MIC mean (SD) was 53.6 (19.5) μg·h/mL, mean (SD) %fT > MIC was 75.7 (10.4), and all subjects had > 60%fT > MIC.Conclusions
In this cohort of CF patients, mean ceftaroline half-life was 1.1 h, which is notably lower than the general population. The dosing regimen studied, which exceeds the recommended dosing in the non-CF population, was adequate to achieve > 60% time above the MIC in all patients. 相似文献6.
Although bronchoscopy remains the best definitive method for localization of the site of hemorrhage in patients who have massive hemopytsis, angiography remains an important adjunct to this localization. When combined with embolization of the bleeding bronchial artery, it is an effective method of therapy for the management of massive hemoptysis in patients with cystic fibrosis. 相似文献
7.
《International Journal of Obstetric Anesthesia》2014,23(1):75-79
Cystic fibrosis affects 1 in 1600–2500 live births and is inherited in an autosomal recessive manner. It primarily involves the respiratory, gastrointestinal and reproductive tracts, with impaired clearance of, and obstruction by, increasingly viscous secretions. Severe respiratory disease, diabetes and gastro-oesophageal reflux may result. Improvements in medical management and survival of cystic fibrosis patients means more are committing to pregnancies. Although guidance for anaesthesia in this patient group is available, management and outcome data associated with more severe cases are sparse. Patients with severe cystic fibrosis require multidisciplinary input and should be managed in a tertiary referral centre. Close monitoring of respiratory function and preoperative optimisation during pregnancy are mandatory. The risk of preterm labour and delivery is increased. Pregnancy and delivery can be managed successfully, even in patients with FEV1 <40% predicted. Neuraxial anaesthesia and analgesia should be the technique of choice for delivery. Postoperative care should be carried out in a critical care setting with the provision of postoperative ventilation if necessary. 相似文献
8.
Vijaylaxmi Grey Shawn R. Mohammed Argyrios A. Smountas Rasha Bahlool Larry C. Lands 《Journal of cystic fibrosis》2003,2(4):195-198
BACKGROUND: The lung disease of cystic fibrosis is associated with a chronic inflammatory reaction and an over abundance of oxidants relative to antioxidants. Glutathione functions as a major frontline defense against the build-up of oxidants in the lung. This increased demand for glutathione (GSH) in cystic fibrosis may be limiting if nutritional status is compromised. We sought to increase glutathione levels in stable patients with cystic fibrosis by supplementation with a whey-based protein. METHODS: Twenty-one patients who were in stable condition were randomly assigned to take a whey protein isolate (Immunocal, 10 g twice a day) or casein placebo for 3 months. Peripheral lymphocyte GSH was used as a marker of lung GSH. Values were compared with nutritional status and lung parameters. RESULTS: At baseline there were no significant differences in age, height, weight, percent ideal body weight or percent body fat. Lymphocyte GSH was similar in the two groups. After supplementation, we observed a 46.6% increase from baseline (P < 0.05) in the lymphocyte GSH levels in the supplemented group. No other changes were observed. CONCLUSION: The results show that dietary supplementation with a whey-based product can increase glutathione levels in cystic fibrosis. This nutritional approach may be useful in maintaining optimal levels of GSH and counteract the deleterious effects of oxidative stress in the lung in cystic fibrosis. 相似文献
9.
Background
Cystic fibrosis (CF, mucoviscidosis) is caused by mutations in the gene encoding CF transmembrane conductance regulator (CFTR), which is a chloride and bicarbonate channel necessary for fluid secretion and extracellular alkalization. For a long time, research concentrated on abnormal Cl- and Na+ transport, but neglected bicarbonate as a crucial factor in CF.Methods
The present short review reports early findings as well as recent insights into the role of CFTR for bicarbonate transport and its defects in CF.Results
The available data indicate impaired bicarbonate transport not only in pancreas, intestine, airways, and reproductive organs, but also in salivary glands, sweat duct and renal tubular epithelial cells. Defective bicarbonate transport is closely related to the impaired mucus properties and mucus blocking in secretory organs of CF patients, causing the life threatening lung disease.Conclusions
Apart from the devastating lung disease, abrogated bicarbonate transport also leads to many other organ dysfunctions, which are outlined in the present review. 相似文献10.
Rajeev Soni Catherine J. Dobbin Maree A. Milross Iven H. Young Peter P.T. Bye 《Journal of cystic fibrosis》2008,7(4):285-291
BACKGROUND: Studies using the multiple inert gas elimination technique (MIGET) to characterise the mechanisms of impaired gas exchange in CF, provide conflicting results on the importance of ventilation-perfusion (VA/Q) inequality over shunt. We hypothesise that the mechanisms of gas exchange abnormality have changed with changing CF management over the last two decades. METHODS: Detailed gas exchange was evaluated by MIGET with venous sampling in stable patients, age > 20 years, FEV1% predicted < or = 50. RESULTS: Fifteen (14 male) subjects were studied with a mean +/- SD age 28.1 +/- 8.4 years, FEV1% 32.6 +/- 10.3, TLC% 111.5 +/- 12.9, PaO2 9.3 +/- 1.3 kPa, (69.5 +/- 9.6 mm Hg), and PaCO2 6.2 +/- 0.7 kPa, (45.9 +/- 5.3 mm Hg). The predominant gas exchange abnormality was VA/Q inequality with a log SD of the distributions of perfusion 0.91 +/- 0.30 and of ventilation 0.60 +/- 0.14. Unimodal distributions were seen in nine subjects, a low VA/Q mode in five and one subject had a bimodal distribution, mean intrapulmonary shunt was negligible. CONCLUSIONS: Subjects had a lower FEV1% by comparison with previously published studies and demonstrated severe VA/Q inequality and negligible shunt. This suggests a low degree of complete obstruction of airways in adults with CF and severe stable pulmonary disease. The primary mechanism of hypoxaemia in CF subjects reaching adulthood today appears to have changed with modern management over the last two decades. 相似文献
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13.
Jeffrey S. Wagener Michael J. Williams Stefanie J. Millar Wayne J. Morgan David J. Pasta Michael W. Konstan 《Journal of cystic fibrosis》2018,17(4):496-502
Background
Patients with cystic fibrosis (CF) who experience acute declines in percent predicted FEV1 (ppFEV1 decreased ≥10% relative to baseline) are often not treated with antibiotics for pulmonary exacerbations (PEx), whereas other patients are treated even when they have not experienced a decline in lung function.Methods
We analyzed 2 patient cohorts using 3?years of Epidemiologic Study of CF data. Cohort 1 (12,837 patients) experienced a ≥10% acute decline in ppFEV1 (n?=?22,898) and Cohort 2 (10,416 patients) had a clinician-diagnosed PEx (n?=?20,731).Results
70.7% of ≥10% decline events were treated with antibiotics; with intravenous antibiotics used 67.1% of the time. 32.0% of clinician-diagnosed PEx declined <10%; with intravenous antibiotics used 36.9% of the time.Conclusions
A clinician's decision to diagnose a PEx and treat with antibiotics often is not defined by measured lung function: a ≥10% FEV1 decline is not considered an absolute indication of a PEx and the lack of a decline does not contraindicate a PEx. Clinicians appear to use the history of prior PEx plus other variables as factors for diagnosing PEx. 相似文献14.
A.S. Barbas M.J. Dib D.P. Al-Adra N. Goldaracena G. Sapisochin T.K. Waddell S. Keshavjee N. Selzner C. Chaparro M.S. Cattral 《Journal of cystic fibrosis》2018,17(1):e1-e4
Cystic fibrosis (CF) affects multiple organs including the lung, liver, and pancreas. Lung transplant, liver transplant, and combined lung-liver transplant have become well-established therapies for CF patients with end-stage organ failure. Thus far, however, there has been limited experience with pancreas transplantation in CF. In this report, we detail the clinical history, transplant procedure, and post-operative recovery of a patient who underwent combined lung-liver-pancreas transplant for advanced CF. 相似文献
15.
Gregory S. Sawicki Deborah E. Sellers Kimberly McGuffie Walter Robinson 《Journal of cystic fibrosis》2007,6(6):411-416
BACKGROUND: The informational needs of the growing population of adults with cystic fibrosis (CF) have not been previously assessed. METHODS: Adults with CF enrolled in the Project on Adult Care in CF (PAC-CF) completed a survey including 22 items in which information topics were rated on the importance of receiving more information and the satisfaction with sources of information. Unmet needs were defined as those topics rated with both high importance and low satisfaction with information sources. RESULTS: The median age of the 233 respondents was 34 years, median FEV(1) was 68% predicted, and 59% were female. The information topics with the highest mean importance ratings were on CF treatments and managing infection. The percentage of respondents rating an individual information need as unmet ranged from 2-32%. Information on "ways to deal with decreased energy, "new CF therapies," and "ways to deal with the unpredictability of the future" were reported as unmet by almost one-third of respondents. For all but four of the information topics assessed, clinical and socio-demographic factors were not significantly associated with increased likelihood of reporting unmet informational needs. CONCLUSIONS: Adults with CF rated information on treatment topics as most important. In contrast, patients were more likely to report information needs on disease self-management and future planning as unmet. Clinical and socio-demographic patient characteristics were not systematically associated with unmet informational needs. Clinicians caring for adult CF patients should not solely rely socio-demographic factors or markers of disease severity as signals for providing comprehensive information on disease self-management and future planning. 相似文献
16.
Katarzyna Walicka-Serzysko Monika Peckova Jacquelien J. Noordhoek Dorota Sands Pavel Drevinek 《Journal of cystic fibrosis》2018,17(4):475-477
Background
The European cystic fibrosis (CF) Society Standards of Care were set to facilitate the delivery of high-quality care throughout Europe. However, their implementation may be difficult for less economically advantaged countries. This survey was performed to explore the gap in the knowledge of the level of CF care in Eastern Europe.Methods
Questionnaires were sent online to one CF professional and one CF patient representative for every Eastern European country.Results
Although most respondents indicated the presence of CF centres, disparities in their framework among individual countries and between them and the European CF Standards of Care became apparent. A minority of countries achieved CF centre recognition by the government (6 of 16), provided CF care for adults (6 countries) and had a multidisciplinary team with all team members represented (2 countries). Patients were significantly more critical in the evaluation of various aspects of CF care than physicians, especially in the Balkan region.Conclusions
The survey results indicate that the organization and level of CF care across Eastern Europe is largely variable and lacks some of its fundamental attributes in several countries. 相似文献17.
Valerie Waters Yvonne Yau Trevor Beaudoin Jillian Wettlaufer Sean Kevin Tom Nancy McDonald Leena Rizvi Michelle Klingel Felix Ratjen Elizabeth Tullis 《Journal of cystic fibrosis》2017,16(4):492-495
There is no effective chronic suppressive therapy Burkholderia cepacia complex infection in cystic fibrosis (CF) patients. This was a pilot, open-label clinical trial of tobramycin inhalation powder (TIP) delivered via Podhaler twice daily for 28 days in adults and children with CF and chronic B. cepacia complex infection in Toronto, Canada. A total of 10 subjects (4 pediatric, 6 adult patients) were treated. There was a mean drop of 1.4 log (CFU/ml) in sputum bacterial density (p = 0.01) and sputum IL-8 levels decreased significantly after 28 days of TIP (p = 0.04). The mean relative change in FEV1 (L) from Day 0 to Day 28 of TIP administration was a 4.6% increase but this was not statistically significant. The majority of patients (70%) had no or mild adverse events. 相似文献
18.
《Journal of cystic fibrosis》2022,21(5):811-820
BackgroundClub cell secretory protein (CC16) exerts anti-inflammatory functions in lung disease. We sought to determine the relation of serum CC16 deficits and genetic variants that control serum CC16 to lung function among children with cystic fibrosis (CF).MethodsWe used longitudinal data from CF children (EPIC Study) with no positive cultures for Pseudomonas aeruginosa prior to enrollment. Circulating levels of CC16 and an inflammatory score (generated from CRP, SAA, calprotectin, G-CSF) were compared between participants with the lowest and highest FEV1 levels in adolescence (LLF and HLF groups, respectively; N = 130-per-group). Single nucleotide variants (SNVs) in the SCGB1A1, EHF-APIP loci were tested for association with circulating CC16 and with decline of FEV1 and FEV1/FVC% predicted levels between ages 7–16 using mixed models.ResultsCompared with the HLF group, the LLF group had lower levels of CC16 (geometric means: 8.2 vs 6.5 ng/ml, respectively; p = 0.0002) and higher levels of the normalized inflammatory score (-0.21 vs 0.21, p = 0.0007). Participants in the lowest CC16 and highest inflammation tertile had the highest odds for having LLF (p<0.0001 for comparison with participants in the highest CC16 and lowest inflammation tertile). Among seven SNVs associated with circulating CC16, the top SNV rs3741240 was associated with decline of FEV1/FVC and, marginally, FEV1 (p = 0.003 and 0.025, respectively; N = 611 participants, 20,801 lung function observations).ConclusionsSerum CC16 deficits are strongly associated with severity of CF lung disease and their effects are additive with systemic inflammation. The rs3741240 A allele is associated with low circulating CC16 and, possibly, accelerated lung function decline in CF. 相似文献
19.
In patients with cystic fibrosis, most treatments addressing the underlying basic defect are mutation or mutation class specific. These treatments are disease modifying if they lower the year to year change in lung function. We therefore calculated the current loss of lung function, measured by year to year change in forced expired volume in 1 s in 11,417 patients included in the European Cystic Fibrosis Society Patient Registry. Whereas patients with at least one mutation of class IV or V have on average a lower year to year change, we did not find a difference between patients with a stop codon mutation, homozygous for F508del or at least one class III mutation. These data are useful background information to discuss the impact of different disease modifying treatments. 相似文献
20.
Malena Cohen-Cymberknoh Tzlil Tanny Oded Breuer Hannah Blau Huda Mussaffi Diana Kadosh Silvia Gartner Alma Salinas Lea Bentur Vered Nir Michal Gur Joel Reiter David Shoseyov Eitan Kerem Itai Berger 《Journal of cystic fibrosis》2018,17(2):281-285