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PubMed1981

Mucoid Escherichia coli in cystic fibrosis.

Patients with cystic fibrosis commonly harbor in their lungs strains of Pseudomonas aeruginosa that have a mucoid coating considered virtually pathognomonic for the disease. We found that strains of Escherichia coli with a morphologically similar mucoid coating were present in the respiratory tracts of eight (11.8 per cent) of 68 patients with cystic fibrosis whose sputum cultures yielded Esch. coli, as compared with none of 89 patients without cystic fibrosis who had Esch. coli in sputum. Mucoid strains of Esch. coli were also recovered from the stools of five (11.1 per cent) of 45 patients with cystic fibrosis, as compared with one (0.7 per cent) of 150 patients without cystic fibrosis. The mucoid substances purified from Esch. coli were biochemically and antigenically distinct from those of P. aeruginosa. We conclude that the respiratory tract in cystic fibrosis offers an environment conducive to the production of a mucoid coating not only by P. aeruginosa but by other gram-negative bacilli as well.

Bacteriological Techniques↗
PubMed2003

The Caregiver Quality of Life Cystic Fibrosis (CQOLCF) scale: modification and validation of an instrument to measure quality of life in cystic fibrosis family caregivers.

OBJECTIVE: Modify the Caregiver Quality of Life Index-Cancer (CQOLC) scale (Weitzner, Jacobsen et al. Qual Life Res 2000; 8: 55-63) as the Caregiver Quality of Life Cystic Fibrosis (CQOLCF) scale, validate it with cystic fibrosis (CF) family caregivers, and assess caregiver quality of life QOL with patient disease severity. METHODS: Following modifications, 100 family caregivers were administered the CQOLCF. Construct validity was assessed by the Medical Outcomes Study Short Form (SF-36) and the Beck Depression Inventory (BDI). Pulmonary function scores and hospitalizations assessed relationship between caregiver QOL and patient disease severity. RESULTS: Split-half reliability was 0.862 and internal consistency (Cronbach's alpha) was 0.909. As expected, there were relatively high correlations with the mental health (0.634) and emotional distress (-0.687); and low correlations with physical health (0.049). ANCOVA examined differences in the CQOLCF totals based on disease severity, controlling for age. Significant mean differences existed (days hospitalized F = 3.010, sig. = 0.022). DISCUSSION: The CQOLCF appears to be a valid, reliable, and internally consistent disease-specific scale with CF family caregivers. Future research recommendations include administering the CQOLCF to an increased study sample to explore item factor analysis.

Adolescent↗
PubMed1982

The incidence of anemia, hypoproteinemia, and edema in infants as presenting symptoms of cystic fibrosis: a retrospective survey of the frequency of this symptom complex in 130 patients with cystic fibrosis.

Among the total number of patients treated at the Danish National Center for cystic fibrosis during the period 1949-1980, 130 had been hospitalized during infancy. Seven of them (approximately 5%) had developed edema, hypoproteinemia, and anemia between their first and fifth month of life. All seven patients had survived for more than 1 year after the onset of the symptom complex. This finding differs markedly from observations in studies from abroad; the prognosis in children with these presenting symptoms of cystic fibrosis is generally stated as being poor. Most of the sweat electrolyte tests performed during the edema period were within the limits of normality but became elevated after the edema resolved.

Anemia↗
PubMed1997

Acceptance of home and clinic-based cystic fibrosis carrier education and testing by first, second, and third degree relatives of cystic fibrosis patients.

We contacted and offered free cystic fibrosis (CF) carrier education and testing to the first, second, and third degree relatives of individuals with CF followed at a large Southeastern US CF Clinic. Relatives were offered CF carrier education and testing either in their homes or in a genetic counseling clinic. Overall, of 514 relatives offered free CF carrier education and testing, 299 (58%) accepted. Significantly more (67%) of those offered education and testing in their homes accepted than those offered education and testing in a genetic counseling clinic (45%). Regression analyses identified several factors, including education, income, gender, perceived chance of being a carrier, and perceived chance of having a child who is a CF carrier, as predictors of acceptance of education and testing in both home and clinic sites. A smaller set of factors was identified that predicted acceptance of education and testing unique to each site. Within the limits of this study and its design, even when CF carrier testing is offered free of charge, including education and testing in the home, acceptance of education and testing, while higher than in general population samples, is not universal among at-risk relatives. Several factors which may have contributed to the observations reported in this study are discussed.

Adolescent↗
PubMed1983

Serum pancreatic lipase activity in cystic fibrosis.

Patients with cystic fibrosis have been found to have abnormal serum concentrations of immunoreactive trypsin and abnormal activities of pancreatic isoamylase. A study was undertaken to discover whether activity of pancreatic lipase is also altered in cystic fibrosis. Serum from 23 patients with cystic fibrosis was assayed for immunoreactive trypsin and pancreatic lipase. Median serum pancreatic lipase activity was significantly lower in patients with cystic fibrosis than in controls, as was immunoreactive trypsin concentration (p less than 0.0001). Some patients had supranormal lipase concentrations but these were not always associated with absence of malabsorption. Serum pancreatic lipase activity is considerably changed in cystic fibrosis.

Adolescent↗
PubMed2005

Novel, mechanism-based therapies for cystic fibrosis.

PURPOSE OF REVIEW: Cystic fibrosis results from disruption of the biosynthesis or function of the cystic fibrosis transmembrane conductance regulator. Cystic fibrosis transmembrane conductance regulator plays a critical role in the regulation of epithelial ion transport. Restoration of cystic fibrosis transmembrane conductance regulator function should improve the cystic fibrosis phenotype. RECENT FINDINGS: Recent investigations affording a better understanding of the mechanism of dysfunction of mutant cystic fibrosis transmembrane conductance regulators, as well as the roles of cystic fibrosis transmembrane conductance regulator in regulating epithelial ion transport, have led to development of therapeutic strategies based on repair or bypass of mutant cystic fibrosis transmembrane conductance regulator dysfunction. The former strategy, coined 'protein repair therapy,' is aimed at improving or restoring the function of mutant cystic fibrosis transmembrane conductance regulators, whereas the latter approach aims to augment epithelial ion transport to compensate for the absent function mutant cystic fibrosis transmembrane conductance regulator. SUMMARY: Strategies to improve mutant cystic fibrosis transmembrane conductance regulator function or to bypass mutant cystic fibrosis transmembrane conductance regulator function hold great promise for development of novel therapies aimed at correcting the underlying pathophysiology of cystic fibrosis.

Animals↗
PubMed2005

Na+/H+ exchanger regulatory factor isoform 1 overexpression modulates cystic fibrosis transmembrane conductance regulator (CFTR) expression and activity in human airway 16HBE14o- cells and rescues DeltaF508 CFTR functional expression in cystic fibrosis cells.

There is evidence that cystic fibrosis transmembrane conductance regulator (CFTR) interacting proteins play critical roles in the proper expression and function of CFTR. The Na(+)/H(+) exchanger regulatory factor isoform 1 (NHERF1) was the first identified CFTR-binding protein. Here we further clarify the role of NHERF1 in the regulation of CFTR activity in two human bronchial epithelial cell lines: the normal, 16HBE14o-, and the homozygous DeltaF508 CFTR, CFBE41o-. Confocal analysis in polarized cell monolayers demonstrated that NHERF1 distribution was associated with the apical membrane in 16HBE14o- cells while being primarily cytoplasmic in CFBE41o- cells. Transfection of 16HBE14o- monolayers with vectors encoding for wild-type (wt) NHERF1 increased both apical CFTR expression and apical protein kinase A (PKA)-dependent CFTR-mediated chloride efflux, whereas transfection with NHERF1 mutated in the binding groove of the PDZ domains or truncated for the ERM domain inhibited both the apical CFTR expression and the CFTR-dependent chloride efflux. These data led us to hypothesize an important role for NHERF1 in regulating CFTR localization and stability on the apical membrane of 16HBE14o- cell monolayers. Importantly, wt NHERF1 overexpression in confluent DeltaF508 CFBE41o- and DeltaF508 CFT1-C2 cell monolayers induced both a significant redistribution of CFTR from the cytoplasm to the apical membrane and a PKA-dependent activation of CFTR-dependent chloride secretion.

Binding Sites↗
PubMed1995

Improved results of lung transplantation for patients with cystic fibrosis.

Patients with cystic fibrosis pose particular challenges for lung transplant surgeons. Earlier reports from North American centers suggested that patients with cystic fibrosis were at greater risk for heart-lung or isolated lung transplantation than other patients with end-stage pulmonary disease. During a 3 1/2 year period, 44 patients with end-stage lung disease resulting from cystic fibrosis underwent double lung transplantation at this institution. During the same interval, 18 patients with cystic fibrosis died while waiting for lung transplantation. The ages of the recipients ranged from 8 to 45 years, and mean forced expiratory volume in 1 second was 21% predicted. Seven patients had Pseudomonas cepacia bacteria before transplantation. Bilateral sequential implantation with omentopexy was used in all patients. There were no operative deaths, although two patients required urgent retransplantation because of graft failure. Cardiopulmonary bypass was necessary in six procedures in five patients and was associated with an increased blood transfusion requirement, longer postoperative ventilation, and longer hospital stay. Actuarial survival was 85% at 1 year and 67% at 2 years. Infection was the most common cause of death within 6 months of transplantation (Pseudomonas cepacia pneumonia was the cause of death in two patients), and bronchiolitis obliterans was the most common cause of death after 6 months. Actuarial freedom from development of clinically significant bronchiolitis obliterans was 59% at 2 years. Results of pulmonary function tests improved substantially in survivors, with forced expiratory volume in 1 second averaging 78% predicted 2 years after transplantation. Double lung transplantation can be accomplished with acceptable morbidity and mortality in patients with cystic fibrosis.

Actuarial Analysis↗
PubMed1992

Levels and molecular forms of immunoreactive trypsin and chymotrypsin in amniotic fluids from normal and cystic fibrosis fetus: evidence for a lack of activation of proteolytic zymogens in cystic fibrosis fetus.

We previously suggested that an activation defect of pancreatic proteolytic zymogens in newborns suffering from cystic fibrosis (CF) might contribute (by an adaptative-like process) to the significant increase of the serum trypsin level observed in the disease at birth. To give support to this hypothesis we studied two pancreatic enzymes: trypsin 1 (IRT) and chymotrypsin A (IRChT) by noncompetitive enzyme immunoassays in amniotic fluids taken at 17-18 weeks of pregnancy. In normal fluids (102), the levels of the two enzymes were widely dispersed between 5 and 100 micrograms/L. A similar pattern was observed for the fluids with a 1 in 4 risk of CF with a normal outcome (24). In contrast, the levels of pancreatic enzymes in the fluids with affected fetus (40) were always below 45 micrograms/L for IRT and 55 micrograms/L for IRChT and most of them were under 20 micrograms/L for both enzymes. The molecular forms of IRT and IRChT in amniotic fluids were studied by gel filtration. In amniotic fluids with affected fetus, a major form of IRT was eluted in a position consistent with the elution of proteins around 25 kDa and two peaks of IRChT were eluted at 75 kDa and 25 kDa. These patterns are similar to those observed in normal serum when zymogens are present and are quite different from the patterns obtained by gel filtration of amniotic fluids with normal outcome.(ABSTRACT TRUNCATED AT 250 WORDS)

Amniocentesis↗
PubMed1984

Effects of alpha-tocopherol on platelet membrane function in cystic fibrosis.

Patients with cystic fibrosis and their parents were reported to have abnormal platelet aggregation responses to prostaglandin E1. To determine whether this is a property of the platelets, we studied the adenosine 3':5'-cyclic monophosphate (cAMP) response of washed platelets to prostaglandin E1. The cAMP response to prostaglandin E1 was the same in platelets from obligate heterozygotes for cystic fibrosis and from those of healthy controls. Patients with cystic fibrosis who had deficient vitamin E levels (plasma alpha-tocopherol, less than 500 micrograms/dl) had significantly (p less than 0.01) reduced platelet cAMP response to prostaglandin E1 compared with patients who had sufficient vitamin E, and supplementation with water-miscible vitamin E in these patients resulted in significant increases in plasma alpha-tocopherol levels (p less than 0.01) and in cAMP response to prostaglandin E1 (p less than 0.05). Plasma alpha-tocopherol levels correlated significantly with platelet cAMP response to prostaglandin E1 in patients with cystic fibrosis (r = 0.58, p less than 0.05). However, plasma alpha-tocopherol level was unrelated to the lymphocyte and granulocyte cAMP response to prostaglandin E1 or to the platelet cAMP response to alpha 2-adrenergic stimulation. Our data suggest that patients with cystic fibrosis have no inherited defect in platelet cAMP response to prostaglandin E1. In patients who have sufficient vitamin E, cAMP responses to prostaglandin E1 are normal in all the formed elements of the blood.

Adolescent↗
PubMed1996

Genotype-phenotype relationships in a cohort of adult cystic fibrosis patients.

In cystic fibrosis (CF), relationships between genotype and phenotype have been shown for pancreatic status but not for pulmonary disease. One hundred and ten adult CF patients were classified according to the expected effect of their mutations on cystic fibrosis transmembrane conductance regulator (CFTR) protein: Group 1 (n=48) included deltaF508 homozygotes; Group 2 (n=26), patients with two "severe" mutations and no expected CFTR production; Group 3 (n=17), patients with expected partly functional CFTR corresponding to at least one "mild" mutation; Group 4 (n=19), patients with no mutation identified or only one identified "severe" mutation. As compared to Groups 1 and 2: patients from Groups 3 and 4 had higher arterial oxygen tension (Pa,O2) (9.5+/-1.9 and 9.9+/-1.5 vs 8.8+/-1.5 and 8.3+/-1.7 kPa, respectively p<0.02); and a slower decline in their pulmonary function, estimated by the mean annual loss in forced vital capacity (FVC) (1.2+/-1.0 and 1.5+/-1.1 vs 2.0+/-0.9 and 2.2+/-1.0%, respectively; p<0.01) and in forced expiratory volume in one second (FEV1) (1.7+/-1.1 and 1.9+/-1.3 vs 2.6+/-1.0 and 2.8+/-1.0%, respectively; p<0.005). They had fewer episodes of colonization of the airways by Pseudomonas aeruginosa, and colonization occurred at a more advanced age (median age 25 and 19 vs 15 and 17 yrs, respectively; p<0.01) and required fewer intravenous antibiotic courses (p<0.01). Pancreatic insufficiency was less frequent in Groups 3 (23%) and 4 (63%) than in Groups 1 (100%) and 2 (96%). This study suggests that the phenotype of adult cystic fibrosis patients, including the severity of the lung disease, is related to the severity of the cystic fibrosis transmembrane conductance regulator mutations.

Adolescent↗
PubMed1999

Allergic bronchopulmonary aspergillosis in cystic fibrosis: reported prevalence, regional distribution, and patient characteristics. Scientific Advisory Group, Investigators, and Coordinators of the Epidemiologic Study of Cystic Fibrosis.

OBJECTIVES: Using the large database from the Epidemiologic Study of Cystic Fibrosis (ESCF), the objectives of this study were to (1) estimate the reported prevalence of allergic bronchopulmonary aspergillosis (ABPA) in patients with cystic fibrosis (CF); (2) compare reported prevalence rates across geographic regions; (3) compare reported prevalence rates between patient subgroups based on demographic and disease characteristics; and (4) describe the ABPA group with regard to their sex, age, and disease severity. STUDY DESIGN: All patients > or = 5 years of age enrolled in ESCF between December 1993 and May 1996 were eligible. Criteria for the diagnosis of ABPA were defined by the ESCF guidelines. Prevalence rates for ABPA were calculated, and potential risk factors for the diagnosis of ABPA were analyzed, including sex, age, pulmonary function, diagnosis of asthma, presence of wheeze, and positive respiratory culture for Pseudomonas. RESULTS: There were 14,210 eligible patients enrolled in ESCF during this period, and ABPA was diagnosed in 281 patients (2%). Regional prevalence varied from 0.9% in the Southwest to 4.0% in the West. Increased prevalence rates occurred in female patients, the adolescent age group, and subjects with lower lung function, wheeze, asthma, and positive Pseudomonas cultures. Although most ABPA patients had evidence of airway obstruction, 10% had an FEV1 of > 100% of predicted. The rates of wheeze (17%) and asthma (30%) were lower than expected in the ABPA group. CONCLUSIONS: This observational study found a reported prevalence rate of ABPA of 2% of CF patients in a large database. This rate was lower than the 5 to 15% rate reported in smaller studies, suggesting that ABPA is underdiagnosed in the CF population. There was wide regional variation in reported prevalence rates, which is unexplained at this time. The characteristics of the patients with ABPA and the epidemiologic risk factors for diagnosis of ABPA were described. Simplified diagnostic criteria were adapted for ESCF with the intent of increasing awareness of ABPA among the participants in this study.

Adolescent↗
PubMed2000

Oral steroids for cystic fibrosis.

BACKGROUND: In cystic fibrosis, airway obstruction and recurrent respiratory infection leads to inflammation and eventually long term lung damage, (bronchiectasis), respiratory failure and death. Inflammation occurs early in the disease process, hence the rationale for the use of anti-inflammatory agents such as oral steroids. OBJECTIVES: To assess the effectiveness of oral steroids in management of respiratory complications cystic fibrosis with particular regard to lung function and occurrence of adverse events. We aimed to to examine short term use for a respiratory exacerbation separately (up to 30 days) from long term anti-inflammatory use (greater than 30 days). SEARCH STRATEGY: We searched The Cochrane Cystic Fibrosis and Genetic Disorders Group specialist trials register. Date of the most recent search of the Group's specialised register: November 1999. SELECTION CRITERIA: All randomised or pseudorandomised trials comparing oral corticosteroids given for a period of five to 30 days for treatment of an exacerbation or for more than 30 days used long term, with placebo or no additional therapy in patients with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two reviewers independently assessed trial eligibility and quality. MAIN RESULTS: Three trials were identified studying a total of 354 patients. Two of these were long term trials with four year follow up whilst one had follow up to 12 weeks only. There was a lack of data on our predefined outcomes with common outcomes examined at different time-points and also variations in the presentation of common outcomes. A meta-analysis was not possible. Oral corticosteroids at a prednisolone equivalent dose of 1 mg/kg alternate days appear to slow the progression of lung disease in cystic fibrosis. At 24 months from commencement, 70.4% patients treated with 1mg/kg prednisolone on alternate days had an increase in per cent predicted forced vital capacity (FVC) compared to 41.6% patients treated with placebo. The mean absolute change in per cent predicted forced expiratory volume at one second (FEV1) 48 months from commencement was -2% in the 1 mg/kg alternate days prednisolone group but -6% in the placebo group. In the long term, this benefit needs to be weighed against the occurrence of adverse events. Linear growth retardation was observed as early as six months from start of treatment in the 2 mg/kg alternate days prednisolone group and from 24 months of treatment in the 1 mg/kg alternate days prednisolone group. Occurrence of adverse events, particularly glucose abnormalities, cataracts and growth retardation resulted in early termination of one of the four year studies ( approximately approximately Eigen 1995 approximately approximately ), with the group taking 2 mg/kg prednisolone on alternate days being stopped first but followed by the 1 mg/kg alternate days. REVIEWER'S CONCLUSIONS: Oral corticosteroids at a prednisolone equivalent dose of 1-2 mg/kg alternate days appear to slow the progression of lung disease in CF but this benefit needs to be weighed against the occurrence of adverse events, in particular, development of cataracts and effect on linear growth. A risk/benefit analysis of low-dose alternate days corticosteroids would be important and the role of short term use of oral steroids should be more fully evaluated.

Anti-Inflammatory Agents↗