ECFS best practice guidelines: the 2018 revision.

Developments in managing CF continue to drive dramatic improvements in survival. As newborn screening rolls-out across Europe, CF centres are increasingly caring for cohorts of patients who have minimal lung disease on diagnosis. With the introduction of mutation-specific therapies and the prospect of truly personalised medicine, patients have the potential to enjoy good quality of life in adulthood with ever-increasing life expectancy. The landmark Standards of Care published in 2005 set out what high quality CF care is and how it can be delivered throughout Europe. This underwent a fundamental re-write in 2014, resulting in three documents; center framework, quality management and best practice guidelines. This document is a revision of the latter, updating standards for best practice in key aspects of CF care, in the context of a fast-moving and dynamic field. In continuing to give a broad overview of the standards expected for newborn screening, diagnosis, preventative treatment of lung disease, nutrition, complications, transplant/end of life care and psychological support, this consensus on best practice is expected to prove useful to clinical teams both in countries where CF care is developing and those with established CF centres. The document is an ECFS product and endorsed by the CF Network in ERN LUNG and CF Europe.

A cross-sectional study of bone mineral density in children and adolescents attending a Cystic Fibrosis Centre.

BACKGROUND: Low bone mineral density is common in adults with cystic fibrosis. Children with good lung function compared to controls matched for body size have normal bone mineralisation. There are few data in large unselected populations of children. METHODS: All children between five and 16 years were invited to take part. Disease severity was assessed. Bone mineral measurements using a GE-Lunar Prodigy densitometer were expressed as age and gender matched Z-scores. Bone mineral apparent density for L2-L4 was estimated and data from UK Caucasian children used to create age and gender specific reference ranges for predicted values. Z-scores were calculated. Total body analysis utilised the Molgaard method. Blood was sampled for measurement of 25-hydroxyvitamin D, and parathyroid hormone levels. RESULTS: 107 children entered the study. 18 and 10 children had low areal and apparent bone mineral density respectively. Short, narrow bones were common. Fifteen children reported 22 fractures, 20 with associated trauma. The best predictors of bone status were ZBMI and percent predicted FEV(1). CONCLUSIONS: Bone mineral density corrected for body size was normal in over 90% of children. These results are similar to previously reported results in small studies of children with well preserved respiratory function.

Nutritional intake and status in children with cystic fibrosis: does age matter?

OBJECTIVES: To determine whether nutritional intake and status vary with age in children with cystic fibrosis (CF). METHODS: Case-control study examining differences in nutritional parameters and intakes in 58 children with CF recruited from a regional centre (2000-2001) and 45 controls. Participants were divided into age groups of 5-8 years, 9-12 years and 13-16 years. Weight, height, body mass index and standard deviation scores were recorded. A 4-day food diary (51 CF, 31 controls) was calculated for macronutrients and micronutrients. RESULTS: Energy intakes (%EAR) increased with age (112%, 115% and 116%, respectively) and were significantly higher in children with CF than controls. Lower weight and growth trends were observed in children ages 5 to 8 years (NS). Weight gain and growth was normal in children with CF ages 9 to 12 years but declined at 13 to 16 years (weight z score -0.85 vs 0.68 P = 0.003, height z score -0.54 vs 0.53 P = 0.002, body mass index z score -0.72 vs 0.41 P = 0.03). Lung function was the most significant predictor of nutritional status at 9 to 12 years (r2 = 0.37, P = 0.006) and 13 to 16 years (r2 = 0.31, P = 0.01), but was not significant in children ages 5 to 8 years. CONCLUSION: Energy intakes increased with age in children with CF and exceeded that of healthy peers in all age groups. Weight gain and growth equaled that of healthy peers at 9 to 12 years but was suboptimal at 5 to 8 years and dramatically declines at 13 to 16 years. Energy intakes were unable to meet the clinical demands of children in these age groups. Both remain vulnerable and require greater nutritional targeting.

Diagnosis and treatment of intestinal malabsorption in cystic fibrosis.

Intestinal malabsorption is severe and of early onset in virtually all people who have cystic fibrosis. The main cause is deficiency of pancreatic enzymes. Bicarbonate deficiency, abnormal bile salts, mucosal transport problems, motility differences, and anatomical structural changes are other contributory factors. Effective treatment should allow a normal to high-fat diet to be taken, control symptoms, correct malabsorption, and achieve a normal nutritional state and growth. Appropriate pancreatic enzyme replacement therapy will achieve normal or near-normal absorption in most people with cystic fibrosis. Early identification and treatment of intestinal malabsorption is critical to achieving optimal nutritional status. The occurrence of fibrosing colonopathy in a few patients on very high doses of those enzymes which have the copolymer Eudragit L30 D55 in their covering resulted in guidelines in the UK to avoid doses equivalent to more than 10,000 IU lipase per kg per day, and also to avoid preparations containing this copolymer in children and adolescents. For patients not responding to 10,000 IU lipase per kg per day review of adherence to treatment, change of enzyme preparation, variation in time of administration, and reduction in gastric acid may improve absorption. The importance of early investigation to exclude other gastrointestinal disorders as a cause of the patient's symptoms, rather than merely increasing the dose of enzymes, is stressed. With modern pancreatic enzymes in doses up to or only slightly in excess of 10,000 IU lipase per kg per day, adequate control of gastrointestinal symptoms and absorption can be achieved, and a normal nutritional state and growth rate maintained in most people with cystic fibrosis.

Vitamin K status among children with cystic fibrosis and its relationship to bone mineral density and bone turnover.

OBJECTIVE: The aim of this study was to assess vitamin K status in an unselected population of children with cystic fibrosis (CF) and to investigate any vitamin K effect on bone turnover and bone mineral status. METHODS: Children > or =5 years of age who were attending the CF unit were invited to enter the study. Fasting blood samples were analyzed for levels of vitamin K1 and prothrombin produced in vitamin K absence; total, undercarboxylated, and carboxylated osteocalcin (OC); and bone-specific alkaline phosphatase and procollagen I carboxy-terminal propeptide (bone formation markers). Levels of N-telopeptide and free pyridinoline and deoxypyridinoline (bone breakdown products) were measured in urine samples. Bone mineral density and bone mineral content were measured at the lumbar spine and for the total body with a GE Lunar Prodigy densitometer. Statistical analyses were performed with Minitab version 9.1. RESULTS: One hundred six children entered the study. Sixty-five of 93 children (70%) from whom blood samples were obtained showed suboptimal vitamin K status, on the basis of low serum vitamin K1 levels, increased prothrombin produced in vitamin K absence levels, or both abnormalities. Vitamin K1 levels showed a significant negative correlation with undercarboxylated OC levels but showed no significant correlation with any marker of bone turnover or measurement of bone mineral status. Undercarboxylated OC levels were correlated significantly with bone turnover markers, which themselves showed a significant negative correlation with measurements of bone mineral density and content. There were no significant correlations between carboxylated or undercarboxylated OC levels and bone density measurements. CONCLUSIONS: Vitamin K1 deficiency is common among children with CF, and routine supplements should be considered. Through its role in the carboxylation of OC, vitamin K deficiency may be associated with an uncoupling of the balance between bone resorption and bone formation. A cause-effect relationship between vitamin K deficiency and low bone mass has not been proved.