Benefits of LYM-X-SORB, a Highly Absorbed Structured Lipid Compound, in Children with Cystic Fibrosis and Pancreatic Insufficiency with Varying Degrees of Malabsorption (P08-125-19)

Abstract Objectives The treatment of fat malabsorption and optimizing growth and nutritional status in patients with cystic fibrosis (CF) and pancreatic insufficiency (PI) is a challenge. A readily absorbable structured lipid (LYM-X-SORB™ [LXS]) improved fat absorption, growth, choline and essential fatty acid (EFA) status in children with CF and PI. Our objective in this secondary analysis is to determine if subjects with varying degrees of fat malabsorption show greater improvements with 3-month LXS treatment. Methods Subjects with CF and PI (5–17yrs) participated in a 12-month double-blind randomized placebo-controlled LXS trial with a 3-month interim visit. LXS and placebo had similar calorie (303 or 456 kcal/d) and fat content (11 or 18 g/d), and LXS had 7-fold greater choline; dose depended on age. CFA was assessed with 72-hour stool and 3-day weighed food records. Height, weight and BMI Z-scores were calculated. Plasma linoleic and α-linolenic acid were assessed. Secondary analyses were restricted to children with baseline CFA who completed 3-month treatment (n = 66, 10.5 ± 3.0 yrs, 40% female). Subjects were divided into two groups at baseline: those with lower CFA (≤87.8%, median) and higher CFA (above median). Results In those with lower baseline CFA, 3-month LXS treatment improved CFA significantly (8.7%, from 77.4 to 86.0%, P < 0.01), with a significant drop in stool fat loss (−6.6 g/24 hours) and no change in dietary fat intake. This was accompanied by significantly increased (P < 0.01) linoleic acid (434 nmol/L, 19% increase) and α-linolenic acid (25 nmol/L, 53% increase). Both weight and BMI Z scores increased ≥0.16 (P < 0.01). With placebo treatment, CFA did not change (72.5 to 71.1%), nor did EFA status, and growth status improved less (≤0.14, P < 0.05). For subjects with higher CFA at baseline, CFA did not change (92.1 to 90.1%) with either treatment, although EFA and growth status improved somewhat, with greater improvement evident in the LXS group. Conclusions Subjects with CF and PI at higher risk for fat malabsorption had a dramatic improvement in CFA with LXS treatment, accompanied by improved EFA and growth status. This suggests that LXS may help those with CF and other pancreatic diagnoses in need of optimizing nutritional status and avoiding unintentional weight loss. Funding Sources Supported by NIH.

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The role of nutrition and pancreatic enzyme replacement therapy in children with cystic fibrosis

World Nutrition Journal ◽

10.25220/wnj.v04.i2.0011 ◽

2021 ◽

Vol 4 (2) ◽

pp. 84-93

Author(s):

Muzal Kadim ◽

William Cheng

Keyword(s):

Cystic Fibrosis ◽

Nutritional Status ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pancreatic Insufficiency ◽

Pancreatic Enzyme ◽

Nutritional Intervention ◽

Enzyme Replacement ◽

Pancreatic Enzyme Replacement Therapy

Background Cystic fibrosis (CF) is an inherited genetic disorder with high mortality and morbidity. CF is strongly correlated with malnutrition due to higher energy losses, pancreatic insufficiency, chronic inflammation, higher resting energy expenditure, and feeding problems. Malnutrition in CF patients associated with worse survival. Thus, appropriate and prompt nutritional intervention should be addressed to reduced malnutrition in CF patients. Methods The literature search was performed on 9 August 2021 in four major databases such as MEDLINE, EBSCOhost, Cochrane Reviews, and Web of Sciences to find the role of nutrition and pancreatic enzyme replacement therapy in pediatrics population with cystic fibrosis. Recent findings In recent decades, early nutritional management and pancreatic enzyme replacement therapy (PERT) have been shown to improve CF patient’s outcomes. Nutrition should be given in higher calories compared to healthy individuals with close and regular nutritional status monitoring. High protein and fat diets are essential for CF patient’s overall survival. Adequate level of micronutrients should be ensured to avoid morbidity caused by micronutrients deficiency. Regular pancreatic insufficiency screening should be done annually in order to start PERT early. Further research focusing on body composition, growth chart, protein intake, and PERT are needed to further improve the management of CF patient. Conclusion Nutritional intervention and PERT play an important role in prolonging CF patient survival. Both treatments should be initiated early with nutritional status close monitoring and tailored to each individual. Collaboration with parents and children is critical to warrant that CF patients followed the dietary advice.

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Benefit of supplementation with LYM-X-SORBTM, an absorbable structured lipid, in improving fat absorption, essential fatty acids and growth status in children with CF and PI with varying degrees of fat malabsorption

Pancreatology ◽

10.1016/j.pan.2019.05.025 ◽

2019 ◽

Vol 19 ◽

pp. S13

Author(s):

Virginia Stallings ◽

Asim Maqbool ◽

Maria Mascarenhas ◽

Joan Schall

Keyword(s):

Fatty Acids ◽

Essential Fatty Acids ◽

Structured Lipid ◽

Fat Absorption ◽

Growth Status ◽

Fat Malabsorption

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Correction: Improved residual fat malabsorption and growth in children with cystic fibrosis treated with a novel oral structured lipid supplement: A randomized controlled trial

PLoS ONE ◽

10.1371/journal.pone.0239642 ◽

2020 ◽

Vol 15 (9) ◽

pp. e0239642

Author(s):

Virginia A. Stallings ◽

Alyssa M. Tindall ◽

Maria R. Mascarenhas ◽

Asim Maqbool ◽

Joan I. Schall

Keyword(s):

Cystic Fibrosis ◽

Randomized Controlled Trial ◽

Controlled Trial ◽

Structured Lipid ◽

Randomized Controlled ◽

Fat Malabsorption ◽

Lipid Supplement

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MON-LB680: Nutritional Status in Adults with Cystic Fibrosis and Pancreatic Insufficiency

Clinical Nutrition ◽

10.1016/s0261-5614(19)32135-1 ◽

2019 ◽

Vol 38 ◽

pp. S312

Author(s):

N. Sripalan ◽

I.E. Moen ◽

P.L. Finstad ◽

H.K. Brekke ◽

M. Ekornes ◽

...

Keyword(s):

Cystic Fibrosis ◽

Nutritional Status ◽

Pancreatic Insufficiency

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Liver cirrhosis and portal hypertension in cystic fibrosis

Srpski arhiv za celokupno lekarstvo ◽

10.2298/sarh1312764f ◽

2013 ◽

Vol 141 (11-12) ◽

pp. 764-769 ◽

Cited By ~ 1

Author(s):

Stojka Fustik

Keyword(s):

Cystic Fibrosis ◽

Liver Cirrhosis ◽

Portal Hypertension ◽

Liver Disease ◽

Nutritional Status ◽

Pancreatic Insufficiency ◽

Standard Deviation Score ◽

Liver Parenchyma ◽

Venous Flow ◽

Genetic Characteristics

Introduction. As the expected survival improves in individuals with the cystic fibrosis (CF), so they may be faced with a number of medical complications. Objective. The aim of this study was to analyze the prevalence of liver cirrhosis in our CF population as well as the clinical and genetic characteristics of these patients. Methods. All patients older than 2 years (n=96) were screened for liver disease. Liver cirrhosis was defined by ultrasonographic findings of distinct heterogeneity of liver parenchyma and nodular liver surface and/or by liver biopsy findings. Enlarged spleen, distended portal vein and abnormal portal venous flow indicated portal hypertension. Clinical and genotype data were analyzed. Results. Sixteen patients were found to have liver cirrhosis, three of them with portal hypertension. All patients had pancreatic insufficiency. Nutritional status expressed as standard deviation score (Z score) for weight, height, and body mass index was as follows: zW=-0.40?1.24, zH=-0.83?1.02, and BMI=20.1?2.3. CF patients with liver cirrhosis generally had mild-to-moderate lung disease, with average FVC and FEV1 values of 97.1?16.5% of predicted and 87.9?23.5% of predicted, respectively. Genetic analysis showed high frequency of F508del mutation in the group with cirrhosis (90.6%). Conclusion. The prevalence of liver cirrhosis in our CF population older than 2 years was 16.6%. Patients with pancreatic insufficiency and severe CFTR mutations, especially F508del, were exposed to higher risk of developing liver cirrhosis. Liver cirrhosis has no significant impact on the pulmonary function and the nutritional status, until the end-stage liver disease.

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CFTR knockdown stimulates lipid synthesis and transport in intestinal Caco-2/15 cells

AJP Gastrointestinal and Liver Physiology ◽

10.1152/ajpgi.00206.2009 ◽

2009 ◽

Vol 297 (6) ◽

pp. G1239-G1249 ◽

Cited By ~ 22

Author(s):

Geneviève Mailhot ◽

Zaava Ravid ◽

Soraya Barchi ◽

Alain Moreau ◽

Rémi Rabasa-Lhoret ◽

...

Keyword(s):

Cystic Fibrosis ◽

Pancreatic Insufficiency ◽

Scavenger Receptor ◽

Lipid Synthesis ◽

Cftr Gene ◽

Nutritional Deficiencies ◽

Type I ◽

Protein Activity ◽

Fat Malabsorption ◽

The Impact

Cystic fibrosis transmembrane conductance regulator (CFTR) is a chloride channel highly expressed in epithelial cells of the gastrointestinal tract. Mutations in the CFTR gene cause cystic fibrosis (CF), a disease characterized by pancreatic insufficiency, fat malabsorption, and steatorrhea. Despite the administration of pancreatic enzymes to normalize malabsorption, CF patients still experienced lipid fecal loss, nutritional deficiencies, and abnormalities in serum lipid profile, suggesting the presence of intrinsic defects in the intestinal handling of nutrients. The objective of the present study was to assess the impact of CFTR gene knockdown on intracellular lipid metabolism of the intestinal Caco-2/15 cell line. Partial CFTR gene inactivation led to cellular lipid accretion of phospholipids, triglycerides, and cholesteryl esters. Likewise, secretion of these lipid fractions was significantly increased following CFTR gene manipulation. As expected from these findings, the output of triglyceride-rich lipoproteins showed the same increasing pattern. Investigation of the mechanisms underlying these changes revealed that CFTR knockdown resulted in raised levels of apolipoproteins in cells and media and microsomal transfer protein activity, two important factors for the efficient assembly and secretion of lipoproteins. Similarly, scrutiny of the enzymatic monoacylglycerol acyltransferase and diacylglycerol acyltransferase, which exhibit dynamic function in triacylglycerol resynthesis and chylomicron formation in enterocytes, revealed a significant augmentation in their activity. Conversely, cholesterol uptake mediated by Niemann-Pick C1 like 1, Scavenger Receptor Class B Type I, and ATP-binding cassette G8 remains unaffected by genetic modification of CFTR. Collectively, these results highlight the role played by CFTR in intestinal handling of lipids and may suggest that factors other than defective CFTR are responsible for the abnormal intracellular events leading to fat malabsorption in CF patients.

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Growth and Nutrition in Cystic Fibrosis

Seminars in Respiratory and Critical Care Medicine ◽

10.1055/s-0039-1696726 ◽

2019 ◽

Vol 40 (06) ◽

pp. 775-791 ◽

Cited By ~ 4

Author(s):

Jefferson N. Brownell ◽

Hillary Bashaw ◽

Virginia A. Stallings

Keyword(s):

Cystic Fibrosis ◽

Nutritional Status ◽

Treatment Guidelines ◽

Pubertal Development ◽

Pancreatic Insufficiency ◽

Adult Life ◽

Nutrition Support ◽

Vitamin Supplementation ◽

Enzyme Replacement ◽

Disease Outcomes

AbstractOptimal nutrition support has been integral in the management of cystic fibrosis (CF) since the disease was initially described. Nutritional status has a clear relationship with disease outcomes, and malnutrition in CF is typically a result of chronic negative energy balance secondary to malabsorption. As the mechanisms underlying the pathology of CF and its implications on nutrient absorption and energy expenditure have been elucidated, nutrition support has become increasingly sophisticated. Comprehensive nutrition monitoring and treatment guidelines from professional and advocacy organizations have unified the approach to nutrition optimization around the world. Newborn screening allows for early nutrition intervention and improvement in short- and long-term growth and other clinical outcomes. The nutrition support goal in CF care includes achieving optimal nutritional status to support growth and pubertal development in children, maintenance of optimal nutritional status in adult life, and optimizing fat soluble vitamin and essential fatty acid status. The mainstay of this approach is a high calorie, high-fat diet, exceeding age, and sex energy intake recommendations for healthy individuals. For patients with exocrine pancreatic insufficiency, enzyme replacement therapy is required to improve fat and calorie absorption. Enzyme dosing varies by age and dietary fat intake. Multiple potential impediments to absorption, including decreased motility, altered gut luminal bile salt and microbiota composition, and enteric inflammation must be considered. Fat soluble vitamin supplementation is required in patients with pancreatic insufficiency. In this report, nutrition support across the age and disease spectrum is discussed, with a focus on the relationships among nutritional status, growth, and disease outcomes.

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High attainment of optimal nutritional and growth status observed among Greek pediatric cystic fibrosis patients: results from the GreeCF study

Journal of Pediatric Endocrinology and Metabolism ◽

10.1515/jpem-2017-0013 ◽

2017 ◽

Vol 30 (11) ◽

Cited By ~ 3

Author(s):

Dimitrios Poulimeneas ◽

Argiri Petrocheilou ◽

Maria G. Grammatikopoulou ◽

Athanasios G. Kaditis ◽

Ioanna Loukou ◽

...

Keyword(s):

Cystic Fibrosis ◽

Nutritional Status ◽

Children And Adolescents ◽

Gene Mutation ◽

Total Sample ◽

Growth Status ◽

Published Research ◽

The Ideal

AbstractBackground:Pediatric cystic fibrosis (CF) patients suffer high rates of undernutrition, subject to several parameters. We aimed to assess growth and nutritional status of Greek children and adolescents with CF.Methods:Eighty-four patients (35 boys) formed the sample. Anthropometrics and FEVResults:In the total sample, 6.0% of the patients were underweight, 4.8% stunted, 8.3% wasted and 17.9% in nutritional failure, whereas 59.5% attained the ideal BMI for CF. FEVConclusions:The examined CF patients appear to be thriving. Unlike published research, the participants’ sex, gene mutation and acquisition of pathogens did not affect growth.

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