Positive parental attitudes to participating in research involving newborn screened infants with CF.

BACKGROUND: Information regarding recruitment of infants to research studies following the diagnosis of cystic fibrosis (CF) via newborn screening (NBS) is not currently available. This study aimed to assess parental attitudes and the feasibility of recruiting and retaining both NBS infants with CF and healthy control infants to a longitudinal, observational study. METHODS: All infants underwent pulmonary function tests (PFTs) at ~3 and ~12months of age. Infants with CF had additional combined chest high resolution computed tomography (HRCT), bronchoscopy and broncho-alveolar lavage (BAL) at ~12months of age. Parental attitude questionnaires (PAQs) were administered to all parents following the ~3month PFTs and to parents of infants with CF after completion of all tests at ~12months. RESULTS: 86% (92/107) of families whose infant had CF consented to participate, of whom 92% had PFTs at ~3months of age with 99% of these having PFTs at ~12months of age. Recruitment of healthy controls was feasible but more challenging; 29% of those contacted agreed to participate; 73% of these had PFTs at ~3months of age; of whom 83% had repeated PFTs at ~12months of age. Completed PAQs were received from 71% of parents, (both of CF and healthy infants) at ~3months and from 58% parents of infants with CF at ~12months. Responses from the PAQs were generally positive, 95% of parents indicated they would recommend participation in such studies to other families. Discrepancies between responses at 3 and 12months suggested that parental understanding of what the research entailed developed during the course of the study. CONCLUSIONS: The high recruitment and retention rates for newly diagnosed CF NBS infants to this observational study are encouraging. These findings will help inform future study design both in the field of CF and other conditions diagnosed by NBS.

Deaths in childhood from cystic fibrosis: 10-year analysis from two London specialist centres.

INTRODUCTION: Death in childhood from cystic fibrosis (CF) is now an uncommon event in the U.K. We wished to assess the circumstances surrounding deaths (and lung transplantation) in the modern era of CF care. METHODS: A retrospective review was carried out pooling data from two large paediatric specialist CF units in London for the 10-year period 2000-2009 inclusive. RESULTS: There were 11 deaths and eight children who had a lung transplant out of 1022 children cared for in this period. Median age of death was 14.2 years and transplant 13.0 years, with a female preponderance (82% deaths and 75% transplants). Apart from one child (forced expiratory volume in 1 s (FEV1) 69%), lung function indicated severe lung disease (median FEV1 33%, range 12%-69%). Values 5 years prior to death were not predictive (median FEV1 62%, range 32%-96%), and those 1 year prior were similar to the last recorded levels. Almost all (10/11) died in hospital and 5/11 (45%) were ventilated. Respiratory failure was the commonest mode of death (64%). Only four children (36%) were receiving palliative care, and in six cases (55%) care was withdrawn. CONCLUSIONS: The number of deaths in children with CF was small but often unpredictable, so active management was continued until late in the majority, reflected by the fact that almost all were in hospital, and more than half were ventilated. If death from respiratory failure is anticipated following a steady decline, palliative care should be instituted well in advance, with attention to appropriate end of life care.

Breath condensate pH in children with cystic fibrosis and asthma: a new noninvasive marker of airway inflammation?

STUDY OBJECTIVES: The noninvasive assessment and monitoring of airway inflammation could be important in respiratory disease. The pH of exhaled breath condensate (EBC) is a promising marker. Although pH has been measured in the EBC of adults with inflammatory airway diseases, no study has measured this in children. DESIGN: This study aimed to assess whether there is a change in pH in the EBC of children with cystic fibrosis (CF) and asthma, and to try to determine whether pH could be used as a marker of airway inflammation. Furthermore, the relationships among EBC pH, severity of disease, and oxidative stress were studied. PATIENTS AND METHODS: We studied 20 children with CF (mean [+/- SEM] age, 7 +/- 3 years), 20 children with asthma (mean age, 7 +/- 2 years), and 15 age-matched healthy children (mean age, 7 +/- 2 years). The pH of EBC was measured using a pH meter. MEASUREMENTS AND RESULTS: Lower pH values were observed in the EBC of children with CF and asthma compared to control subjects (mean pH, 7.23 +/- 0.03 and 7.42 +/- 0.01 vs 7.85 +/- 0.02, respectively). Furthermore, relationships among EBC pH, severity of asthma, and the presence of an infective exacerbation of CF was found. There was a negative correlation between exhaled pH and exhaled leukotriene B(4) concentrations (r = -0.5; p < 0.005). CONCLUSION: We conclude that the measurement of EBC pH may be useful in the evaluation of airway inflammation in children with asthma and CF.

Objective monitoring of cough in children with cystic fibrosis.

Increased cough frequency is a common symptom associated with infective pulmonary exacerbations of cystic fibrosis (CF), but subjective assessment of cough is very unreliable. The aims of this study were: 1) to validate a modification of our previously described ambulatory cough recording device (LR 100); 2) to determine how accurately children with CF assess levels of cough; and 3) to assess the change in cough in children with CF when treated with intravenous antibiotics for a respiratory exacerbation, and whether the children themselves were able accurately to perceive any change. Fourteen CF children (aged 13.6 +/- 2.6 years) were included in the study. All 14 children were simultaneously recorded with the LR 100 cough recorder and a conventional tape recorder during a chest physiotherapy session on the first or second day of admission for an infective exacerbation diagnosed by standard criteria. The difference between the two was an underestimate of 0.5 epoch/session by the tape recorder. Ten children were recorded on admission with the LR 100 cough recorder for a complete cycle (17 hr and 40 min), and we also assessed their day and night-time cough with cough scores and visual analogue scores (VAS). In 8 of these children, the same assessments were repeated on discharge. There was no significant correlation between any of the admission or discharge cough scores, daytime or night-time cough scores, and daytime or night-time VAS scores, with the actual number of coughs recorded on the LR 100 cough recorder. For the 8 children who had cough monitoring on admission and on discharge, there was no significant improvement in daytime or night-time cough scores or VAS on discharge, despite significant improvements in spirometry. There was also no significant improvement on daytime and night-time cough counts with the cough monitor on discharge, and no significant correlation with changes in lung function. There were weak correlations only between change in daytime VAS scores and change in forced expired volume in 1 sec (r = -0.794, P = 0.019) and forced vital capacity (r = -0.723, P = 0.04). In conclusion, we describe a reliable and well-tolerated method for obtaining cough counts objectively. The use of this objective method showed that CF children did not assess their cough frequency well. In addition, treatment of respiratory exacerbation improved neither subjective nor objective measures of cough in CF children.