Feasibility study to inform the design of a randomised controlled trial to eradicate Pseudomonas aeruginosa infection in individuals with Cystic Fibrosis

There are controversies about the most effective treatment to eradicate first growth of Pseudomonas aeruginosa (P aeruginosa) from the lower airways of patients with cystic fibrosis (CF). UK guidelines recommend oral treatment, but some advocate intravenous (IV) treatment. The objective of this study was to assess the feasibility of conducting a randomised controlled trial comparing two treatment strategies to eradicate P aeruginosa in CF patients

Working together to deliver stratified medicine research effectively

Introduction or background: Stratified medicine is an important area of research across all clinical specialties, with far reaching impact in many spheres. Despite recently formulated global policy and research programmes, major challenges for delivering stratified medicine studies persist. Across the globe, clinical research infrastructures have been setup to facilitate high quality clinical research. Sources of data: This article reviews the literature and summarizes views collated from a workshop held by the UK Pharmacogenetics and Stratified Medicine Network and the NIHR Clinical Research Network in November 2016. Areas of agreement: Stratified medicine is an important area of clinical research and health policy, benefitting from substantial international, cross-sector investment and has the potential to transform patient care. However there are significant challenges to the delivery of stratified medicine studies. Areas of controversy: Complex methodology and lack of consistency of definition and agreement on key approaches to the design, regulation and delivery of research contribute to these challenges and would benefit from greater focus. Growing points: Effective partnership and development of consistent approaches to the key factors relating to stratified medicine research is required to help overcome these challenges. Areas timely for developing research: This paper examines the critical contribution clinical research networks can make to the delivery of national (and international) initiatives in the field of stratified medicine. Importantly, it examines the position of clinical research in stratified medicine at a time when pressures on the clinical and social services are mounting

Body composition of children with chronic and end-stage renal failure

AIM: Protein energy malnutrition is common in children with chronic renal failure (CRF) and may negatively impact on clinical outcome. Although the aetiology of malnutrition is multifactorial, descriptive information on body composition may guide nutritional interventions aimed at optimising nutritional status. METHODS: This prospective cohort study in children with CRF was conducted from April 1999 to November 2000. Patients were categorised according to their glomerular filtration rate (GFR) into CRF and end-stage renal failure (ESRF). Body composition was assessed based on anthropometry, total body potassium (TBK), total body protein (TBP) and dual X-ray absorptiometry (DEXA). RESULTS: Fifteen patients (10 male, 5 female; mean age: 13.4 +/- 4.3 years) were studied, including eight patients with CRF (mean GFR: 17.0 +/- 7.2 mL/min/1.73 m(2)) and seven patients with ESRF (mean GFR: 6.4 +/- 1.7 mL/min/1.73 m(2)). Patients in both groups (n = 15) had deficits in height and TBP (mean z-score height-for-age: -1.19 +/- 1.05, P < 0.01; mean z-score TBP: -0.71 +/- 0.71, P < 0.05). There were no significant differences in weight, height, fat-free mass, TBK and TBP between patients with CRF and ESRF. CONCLUSIONS: Linear growth impairment and decreased TBP are common in children with chronic and ESRF. TBK and DEXA may underestimate the degree of malnutrition in these patients