A pinboard by
Gemma Zeybel

PhD, MRes and BSc Hons


CF nutrition research continues to improve nutrition status, reduce symptoms and improve prognosis

In 10 seconds - In the 1930's, a tell tale sign of cystic fibrosis was the failure to gain weight, accompanied by steatorrhoea and sputum production. Today, we know this is due to deficient CFTR expression in the cells expressing the CFTR gene. The pancreatic ducts and airway epithelia express the CFTR.

Why do people with cystic fibrosis fail to gain weight The pancreatic ducts become blocked with mucus, leading to insufficient pancreatic enzyme secretion. Food cannot digest efficiently in the absence of pancreatic enzymes. Dietary fat is a source of energy but it is passed in the stool.

Sputum production in the airways requires nitrogen, leading to a deficiency in amino acids. Sputum blocks the airways resulting in symptoms of chronic cough and heavy breathing. In turn, this increases energy expenditure. Also, bacteria and fungi populate in the airways resulting in a chronic lung infection. which commonly leads to a decreased appetite.

A dietitian will help a person with cystic fibrosis to overcome malnutrition. Weight gain has been shown to increase lung function and associated increased survival. The dietetic community advises patients to increase fat and energy in their diet. Pancreatic enzyme replacement therapy helps the digestion of fat.

Patients should opt for food high in fat to meet their increased energy requirements. Fat is dense in energy and has 9 calories per gram. Fortifying meals increases the energy content without increasing the quantity. This is done by adding high fat items to meals such as butter, cheese, oil and cream. It is better to eat food rather than oral nutrition supplements. They provide high energy, fat and protein in each small serving. Oral nutrition supplements are prescribed when patients can't increase weight with food alone. Pancreatic enzyme replacement therapy aims to maximise the digestion of food, and vitamin A,D, E and K from a vitamin supplement. The dose is individual and dependant on fat intake. For example, one capsule taken before a high fat snack and two capsules prior to a high fat meal. Protein promotes growth and development, thus a high protein diet is advised. Amino acids are present in abundance in sputum, so it is required to replace the losses.

This board focuses on emerging evidence in nutrition and dietetics for patients with cystic fibrosis.


Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor.

Abstract: The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability.To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation.Patients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).Studies included 213 patients (aged ≤ 20 years, n = 105; aged > 20 years, n = 108). In patients ≤20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p < 0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p < 0.0001). In patients >20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight.Nutritional status improved following treatment with ivacaftor for 48 weeks.

Pub.: 08 Aug '15, Pinned: 24 Apr '17

The Vitamin D for Enhancing the Immune System in Cystic Fibrosis (DISC) trial: Rationale and design of a multi-center, double-blind, placebo-controlled trial of high dose bolus administration of vitamin D3 during acute pulmonary exacerbation of cystic fibrosis

Abstract: Vitamin D deficiency is highly prevalent in children and adults with cystic fibrosis (CF). Recent studies have found an association between vitamin D status and risk of pulmonary exacerbations in children and adults with CF. The ongoing Vitamin D for enhancing the Immune System in Cystic fibrosis (DISC) study is a multi-center, double-blind, randomized, placebo-controlled trial will test the hypothesis of whether high dose vitamin D given as a single oral bolus of 250,000 IU to adults with CF during a pulmonary exacerbation followed by a maintenance dose of vitamin D will improve time to next pulmonary exacerbation and re-hospitalization, improve survival and lung function compared to placebo and reduce the rates of pulmonary exacerbation. Subjects will be randomized 1:1 at each clinical site to vitamin D or placebo within 72 h of hospital admission for pulmonary exacerbation. Clinical follow-up visits will occur at 1, 2, 3, and 7 days, and 1, 3, 6 and 12 months after randomization. Blood and sputum will be collected and determination of clinical outcomes will be assessed at each visit. The primary endpoint will be the time to next pulmonary exacerbation requiring antibiotics, re-hospitalization or death. The secondary endpoints will include lung function assessed by forced expiratory volume in 1 s (FEV1), blood markers of inflammatory cytokines, anti-microbial peptide expression by peripheral blood mononuclear cells and circulating concentrations in blood. Other exploratory endpoints will examine the phenotype of neutrophils and monocyte/macrophages in sputum. Nutritional status will be assessed by 3 day food records and food frequency questionnaire.

Pub.: 09 Mar '17, Pinned: 23 Apr '17

ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis.

Abstract: Malnutrition is both a frequent feature and a comorbidity of cystic fibrosis (CF), with nutritional status strongly associated with pulmonary function and survival. Nutritional management is therefore standard of care in CF patients. ESPEN, ESPGHAN and ECFS recommended guidelines to cover nutritional management of patients with CF.The guidelines were developed by an international multidisciplinary working group in accordance with officially accepted standards. The GRADE system was used for determining grades of evidence and strength of recommendation. Statements were discussed, submitted to Delphi rounds, reviewed by ESPGHAN and ECFS and accepted in an online survey among ESPEN members.The Working Group recommends that initiation of nutritional management should begin as early as possible after diagnosis, with subsequent regular follow up and patient/family education. Exclusive breast feeding is recommended but if not possible a regular formula is to be used. Energy intake should be adapted to achieve normal weight and height for age. When indicated, pancreatic enzyme and fat soluble vitamin treatment should be introduced early and monitored regularly. Pancreatic sufficient patients should have an annual assessment including fecal pancreatic elastase measurement. Sodium supplementation is recommended and a urinary sodium:creatinine ratio should be measured, corresponding to the fractional excretion of sodium. If iron deficiency is suspected, the underlying inflammation should be addressed. Glucose tolerance testing should be introduced at 10 years of age. Bone mineral density examination should be performed from age 8-10 years. Oral nutritional supplements followed by polymeric enteral tube feeding are recommended when growth or nutritional status is impaired. Zinc supplementation may be considered according to the clinical situation. Further studies are required before essential fatty acids, anti-osteoporotic agents, growth hormone, appetite stimulants and probiotics can be recommended.Nutritional care and support should be an integral part of management of CF. Obtaining a normal growth pattern in children and maintaining an adequate nutritional status in adults are major goals of multidisciplinary cystic fibrosis centers.

Pub.: 14 Apr '16, Pinned: 22 Apr '17

[Liver disease, gastrointestinal complications, nutritional management and feeding disorders in pediatric cystic fibrosis].

Abstract: In cystic fibrosis (CF), approximately 5-8% of the patients develop multilobular cirrhosis during the first decade of life. Annual screening (clinical examination, liver biochemistry, ultrasonography) is recommended in order to identify early signs of liver involvement, initiate ursodeoxycholic acid therapy and detect complications (portal hypertension and liver failure). Management should focus on nutrition and prevention of variceal bleeding. The gut may also be involved in children with CF. Gastroesophageal reflux is frequent, although often neglected and should be investigated by pH monitoring and impedancemetry, if available. Acute pancreatitis occurs in patients with persistent exocrine pancreatic activity. Intussusception, appendicular mucocele, distal intestinal occlusion syndrome, small bowel bacterial overgrowth and Clostridium difficile colitis should be considered in case of abdominal pain. Preventive nutritional support should be started as soon as possible after diagnosis of CF. Attainment of normal growth is one of the main goals and can be achieved with hypercaloric and salt supplemented food. Pancreatic enzyme replacement therapy should be started as soon as exocrine pancreatic insufficiency is confirmed and ingested immediately prior to meals with intake of fat-soluble vitamins. Curative nutritional interventions are more likely to be effective in the early stages of pulmonary disease. Feeding disorders, related to the physiopathology and the psychologic aspects of the disease are frequent. Repeated corporeal aggressions, associated with inappropriate medical and parental pressure, may increase the child's refusal of food. The multidisciplinary team should guide parents in order to avoid all intrusive feeding practices and promote pleasant mealtimes.

Pub.: 25 Feb '17, Pinned: 22 Apr '17

Prevalence of malnutrition and obesity among cystic fibrosis patients.

Abstract: Optimal nutritional status (NS) in cystic fibrosis (CF) is associated with better lung function and increased overall survival. This study estimated the prevalence of malnutrition and obesity among CF patients in a tertiary center.In a cross-sectional study of 68 CF patients (33 female; 37 children/adolescents) weight, height, body composition, respiratory function (% of the predicted forced expiratory volume in 1 s; FEV1%pred ) and serum lipids were measured; body mass index (BMI), BMI standard deviation score (BMI-SDS) and BMI percentiles were calculated; Pseudomonas colonization, pancreatic insufficiency, diabetes mellitus (CFDM), liver disease (CFLD) and genotype were recorded; NS was classified according to the 2005 Cystic Fibrosis Foundation (CFF) criteria. Frequency distributions and associations between anthropometric and clinical parameters (univariate/multivariate) were calculated.Mean age (±SD) was 19.81 ± 8.98 years. Regarding NS: 22.1% were malnourished, 13.2% overweight/obese and 29.4% had optimal NS. Pancreatic function (PF), Pseudomonas colonization, CFDM, CFLD and genotype differed significantly among the three groups. FEV1%pred was significantly higher among overweight/obese patients and correlated positively with anthropometric characteristics as well as serum cholesterol and negatively with age. BMI-SDS was associated with PF, FEV1%pred and CFDM. Among overweight/obese patients 89.9% had adequate PF and 66.7% carried mutations other than F508del. No patient had any traits of metabolic syndrome.Despite appropriate management only one-third of the present patients had optimal NS. One-fourth were malnourished and a significant percentage were overweight/obese. The latter were mostly carriers of mutations other than F508del and had better pulmonary function. CF patients require intensive monitoring for both malnutrition and overweight/obesity.

Pub.: 06 Sep '13, Pinned: 23 Apr '17

Dietary intake and nutritional status in a Scandinavian adult cystic fibrosis-population compared with recommendations.

Abstract: Malnutrition is a well-known complication in cystic fibrosis (CF). There is good evidence that maintaining a normal body-weight correlates well with improved survival in CF. Energy intake in excess of 120% of the estimated average requirement (EAR) has been advised since 1980s.To investigate the nutritional intake and status in the adult Scandinavian CF-population.A cross-sectional multi-centre study was used to investigate the nutritional status of 456 adult CF-patients (2003 2006). Height and weight were measured and body mass index (BMI) and z-scores were calculated. Pulmonary function was examined by dynamic spirometry. A 7-day pre-coded food record (FR) obtained energy and nutrient intake data in 180 patients.The mean energy intake was 114 (SD 30.0)% of EAR and thus significantly lower than the target of 120% EAR (p< 0.001) for patients with pancreatic insufficiency (PI) (n=136). Mean BMI was 22.0 (SD 2.9), the prevalence of BMI <18 was 13% and the prevalence of BMI ≥25 was 15% (n=136). Mean BMI was 20.8 (SD 2.4) in PI-patients with FEV(1) <70% and 23.2% (SD 3.0), in PI-patients with FEV(1) ≥70%, mean difference 2.4, (95% CI: 1.5, 3.3) (p<0.001), but there was no difference in energy intake. BMI ≥18.5 and a reported energy intake <120% were revealed in 54% of the PI-patients.The energy intake did not reach the recommended 120% EAR, but the prevalence of underweight was lower than reported in other studies. The recommendation may exceed the requirement for a number of CF-patients. The nutritional status must still be closely monitored and nutritional advice and intervention should be individualised and adjusted to actual needs.

Pub.: 24 Nov '11, Pinned: 22 Apr '17

Improving nutritional status in a pediatric cystic fibrosis center.

Abstract: The nutritional status of patients with cystic fibrosis (CF) is strongly associated with pulmonary function, respiratory status and survival. Malnutrition could result from a discrepancy between energy needs and food intake while malabsorption results from pancreatic insufficiency which occurs in 85% of people with CF.A quality improvement (QI) project was designed to improve the nutritional status of patients with CF with low Body Mass Index (BMI) between 3 and 19 years of age. An algorithm was developed which included clinic-based assessments of patients' nutritional status and periodic assessment by a dietitian, social worker and/or psychologist during the project. Gastrostomy tube placement and feeding was offered as a last resort to improve caloric intake.173 patients seen during January-June, 2010, were included in this project. They were classified into four BMI groups and data were collected quarterly through June, 2012. The project target population (BMI percentile ≤ 24) had a median BMI percentile at the start of the project of 11.8. At the end of the project median BMI percentile was 22 (46% improvement).Improving nutrition and BMI for patients with CF is achievable. There must be a motivated, multi-disciplinary team that includes patients and families. A patient-specific combination of interventions must be used. These interventions could be quite basic for patients with BMI percentile ≥ 25, yet more elaborate for patients with BMI percentile <25. Clinic-based algorithms such as ours can successfully improve the BMI percentile in patients with CF.

Pub.: 13 Nov '14, Pinned: 22 Apr '17

[Growth and pulmonary function in Chilean children and adolescents with cystic fibrosis].

Abstract: Nutrition influences morbidity and mortality in patients with cystic fibrosis (CF), affecting their lung function.To characterize the nutritional status of a group of CF patients and to analyze its evolution and relationship to lung function.A retrospective cohort of CF children and adolescents attended in our institution for 15 years, was analyzed. Age and form of presentation, mutation, weight and stature (measured annually at least), microbial colonization and forced expiratory volume in the first second (FEV(1)) were registered.We gathered information about 33 patients, 64% males, diagnosed at 23.8 ± 45.6 months old (range 1-216), 85% had a genetic study (10 children had one or more Df508 alleles) and 94% had pancreatic insufficiency. In their last visit they were 13.0 ± 5.8 years old, their body mass index z-score (BMIz) was -0.25 ± 1.2 and their FEV(1) was 80.4 ± 28.6%. According to BMI: 73% were eutrophic, 18% undernourished and 9% were overweight. According to weight/ for height index (WH), the figures for eutrophy, undernutrition and overweight were 70, 6 and 24%, respectively. Only 12% had short stature. Those with P. aeruginosa infection had lower BMI. There was a positive correlation between FEV(1) and BMIz (+0.46, p = 0.02), but not with WH. During follow-up, there was a gradual deterioration of weight, height, and BMIz after 10-12 years of age and an overall gradual FEV(1) decrease.The prevalence of malnutrition in these patients with CF is high; undernutrition is higher if defined by BMIz and unlike WH, correlates to lung function. Nutritional deterioration starts before adolescence.

Pub.: 05 Jan '12, Pinned: 22 Apr '17

Pancreatic enzyme replacement therapy for people with cystic fibrosis.

Abstract: Most people with cystic fibrosis (80% to 90%) need pancreatic enzyme replacement therapy to prevent malnutrition. Enzyme preparations need to be taken whenever food is taken, and the dose needs to be adjusted according to the food consumed. A systematic review on the efficacy and safety of pancreatic enzyme replacement therapy is needed to guide clinical practice, as there is variability between centres with respect to assessment of pancreatic function, time of commencing treatment, dose and choice of supplements. This is an updated version of a published review.To evaluate the efficacy and safety of pancreatic enzyme replacement therapy in children and adults with cystic fibrosis and to compare the efficacy and safety of different formulations of this therapy and their appropriateness in different age groups. Also, to compare the effects of pancreatic enzyme replacement therapy in cystic fibrosis according to different diagnostic subgroups (e.g. different ages at introduction of therapy and different categories of pancreatic function).We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. Most recent search: 15 July 2016.We also searched an ongoing trials website and the websites of the pharmaceutical companies who manufacture pancreatic enzyme replacements for any additional trials. Most recent search: 22 July 2016.Randomised and quasi-randomised controlled trials in people of any age, with cystic fibrosis and receiving pancreatic enzyme replacement therapy, at any dosage and in any formulation, for a period of not less than four weeks, compared to placebo or other pancreatic enzyme replacement therapy preparations.Two authors independently assessed trials and extracted outcome data. They also assessed the risk of bias of the trials included in the review.One parallel trial and 12 cross-over trials of children and adults with cystic fibrosis were included in the review. The number of participants in each trial varied between 14 and 129 with a total of 512 participants included in the review. All the included trials were for a duration of four weeks. The included trials had mostly an unclear risk of bias from the randomisation process as the details of this were not given; they also mostly had a high risk of attrition bias and reporting bias.We could not combine data from all the trials as they compared different formulations. Findings from individual studies provided insufficient evidence to determine the size and precision of the effects of different formulations. Ten studies reported information on the review's primary outcome (nutritional status); however, we were only able to combine data from two small cross-over studies (n = 41). The estimated gain in body weight was imprecise, 0.32 kg (95% confidence interval -0.03 to 0.67; P = 0.07). Combined data from the same studies gave statistically significant results favouring enteric-coated microspheres over enteric-coated tablets for our secondary outcomes stool frequency, mean difference -0.58 (95% confidence interval -0.85 to -0.30; P < 0.0001); proportion of days with abdominal pain, mean difference -7.96% (95% confidence interval -12.97 to -2.94; P = 0.002); and fecal fat excretion, mean difference -11.79 g (95% confidence interval -17.42 to -6.15; P < 0.0001). Data from another single small cross-over study also favoured enteric-coated microspheres over non-enteric-coated tablets with adjuvant cimetidine in terms of stool frequency, mean difference -0.70 (95% confidence interval -0.90 to -0.50; P < 0.00001).There is limited evidence of benefit from enteric-coated microspheres when compared to non-enteric coated pancreatic enzyme preparations up to one month. In the only comparison where we could combine any data, the fact that these were cross-over studies is likely to underestimate the level of inconsistency between the results of the studies due to over-inflation of confidence intervals from the individual studies.There is no evidence on the long-term effectiveness and risks associated with pancreatic enzyme replacement therapy. There is also no evidence on the relative dosages of enzymes needed for people with different levels of severity of pancreatic insufficiency, optimum time to start treatment and variations based on differences in meals and meal sizes. There is a need for a properly designed study that can answer these questions.

Pub.: 24 Nov '16, Pinned: 22 Apr '17