Ammani Prasad

Who needs chest physiotherapy? Moving from anecdote to evidence

“One case which I had was that of a newborn child with acquired atelectasis, which required my presence, or that of my assistant, for 24 hours. In such cases there is nothing, I believe, as efficacious as flagellation. I usually tell the attendant to take a rubber band and flip the soles of the feet whenever the child begins to tire of breathing.” Dr Sanders. In: Disturbances of respiration in the newborn. Transactions of the American Pediatric Society 1903; 15 :47. At the beginning of this century, paediatrics was an art. Skills were learnt from a mentor, picking up tips and anecdotes while standing at your master’s side. Now, as practitioners of child health in the final years of this same century, life has changed. Anecdote and word of mouth have lost credibility and are replaced by scientific scrutiny and the rigour of evidence from carefully controlled and sufficiently powerful trials. We do our best to find the truth, but in many areas of care there remains a dearth of sufficient evidence. Often in the closets of our own practices, we continue to do what our teachers taught us and what, over time, we believe works. The central function of chest physiotherapy in paediatric respiratory disease is to assist in the removal of tracheobronchial secretions. The intention is to remove airway obstruction, reduce airway resistance, enhance gas exchange, and reduce the work of breathing. In the acute situation, recovery should be hastened and in the child with a chronic respiratory disorder, the progression of the lung disease is hopefully delayed. Chest physiotherapy can improve a patient’s respiratory status and expedite recovery. But in certain situations it may be a useless intervention or even harmful—perhaps by increasing bronchospasm, inducing pulmonary hypertension, repositioning a foreign body, or destabilising a sick infant. What …

Global Lung Function Initiative equations improve interpretation of FEV1 decline among patients with cystic fibrosis

The prognosis for individuals with cystic fibrosis has improved markedly over the last 50 years, with most countries now reporting that 50% of the population reach at least 35 years of age [1–3]. Despite this improvement, the average age at death is still in the late 20s and varies enormously between cystic fibrosis centres and countries [4]. To further improve the trajectory of lung disease, it is critical to identify patients that would benefit most from early intervention. Adolescent FEV1 decline in cystic fibrosis may be an artefact of the spirometry reference equations used http://ow.ly/J0com

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.

Cystic fibrosis newborn screening: outcome of infants with normal sweat tests

Newborn babies positively screened for cystic fibrosis (CF) (high serum immunoreactive trypsin (IRT) with DNA analysis) are referred for a diagnostic sweat test, which may be normal (sweat chloride <30 mmol/L). Unless two gene mutations are identified during Newborn screening (NBS), the babies are discharged from follow-up. We wished to check that none had subsequently developed symptoms suggestive of CF. We retrospectively reviewed patient notes and contacted general practitioners of all babies with a negative sweat test, conducted in one of the four paediatric specialist CF centres in London, over the first 6 years of screening in South East England. Of 511 babies referred, 95 (19%) had a normal sweat test. Five (5%) had CF diagnosed genetically, two of them on extended genome sequencing after clinical suspicion. Eleven (12%) were designated as CF screen positive inconclusive diagnosis (CFSPID); one of the five CF children was originally designated as CFSPID. Seventy-nine (83%) were assumed to be false-positive cases and discharged; follow-up data were available for 51/79 (65%); 32/51 (63%) had no health issues, 19/51 (37%) had other significant non-CF pathology. These results are reassuring in that within the limitations of those lost to follow-up, CF symptoms have not emerged in the discharged children. The high non-CF morbidity in these children may relate to known causes of high IRT at birth. Clinicians need to be aware that a child can have CF despite a normal sweat test following NBS, and if symptoms suggest the diagnosis, further testing, including extended genome sequencing, is required.

WS21.1 Recruitment and retention of newborn screened (NBS) infants to an observational study with invasive outcome measures

Objectives: 1. Assess feasibility of recruiting and retaining a. NBS infants with CF to an invasive observational study involving pulmonary function tests (PFTs), CT and bronchoscopy, b. healthy controls (HC) to a longitudinal study of PFTs. 2. Investigate parental attitudes to participating in such a study. Methods: PFTs were undertaken at 3 mths and 1 yr in both CF and healthy infants between Jan 2010-Dec 2011. CT and bronchoscopy was also undertaken in those with CF at 1 yr. Parental Attitude Questionnaires (PAQ) were administered to all parents at the 3mth PFT. A further questionnaire was completed anonymously by parents of CF infants following the 1 yr assessments. Results: Of 107 eligible CF infants, 79% (84) attended the 3 mth PFT. By Dec 2011, 72 were at least 1 yr of age, of whom 90% (65) completed the 1 yr assessments. Of the 270 HC invited to participate, 21% (56) had PFTs at 3mth. By Dec 2011, 46 were at least 1 yr of age, of whom 78% (36) completed PFTs. Responses from the PAQ indicated that most parents of infants with CF chose to be involved in the study to help their own and other infants in the future. Parents of HC cited altruistic reasons. Most (95%) parents of CF infants would recommend involvement in research to other parents. Conclusions: Recruitment and retention of infants with CF was excellent, providing encouraging data for future interventional trials. While recruitment of HC was more difficult, retention was similar. Parental attitudes were very positive, with the majority of parents willing to be involved in such studies for the good of their own and other children.