Maintenance of nutritional status in patients with cystic fibrosis: New and emerging therapies

Drug Design, Development and Therapy

Volumn 6, Issue , 2012, Pages 151-161

  Kalnins, Daina ^a   Wilschanski, Michael ^b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

Author keywords

Cystic fibrosis; Fat soluble vitamins; Nutrition; Pancreatic enzymes

Indexed keywords

ALPHA TOCOPHEROL; APPETITE STIMULANT; GROWTH HORMONE; PANCRELIPASE; PLACEBO; RETINOL; VITAMIN D; VITAMIN K GROUP; VITAMIN;

ABDOMINAL PAIN; CYSTIC FIBROSIS; DIABETES MELLITUS; DRUG APPROVAL; DRUG EFFICACY; DRUG FORMULATION; ENZYME REPLACEMENT; HUMAN; LUNG TRANSPLANTATION; NUTRITIONAL PARAMETERS; NUTRITIONAL STATUS; OUTCOME ASSESSMENT; PANCREAS FUNCTION; PREGNANCY; REVIEW; TODDLER; VITAMIN SUPPLEMENTATION; AGE; METHODOLOGY; NUTRITIONAL REQUIREMENT; NUTRITIONAL SUPPORT; PATHOPHYSIOLOGY; PATIENT EDUCATION; SURVIVAL RATE;

AGE FACTORS; CYSTIC FIBROSIS; ENZYME REPLACEMENT THERAPY; HUMANS; NUTRITIONAL REQUIREMENTS; NUTRITIONAL STATUS; NUTRITIONAL SUPPORT; PATIENT EDUCATION AS TOPIC; SURVIVAL RATE; VITAMINS;

EID: 84867907884     PISSN: 11778881     EISSN: None     Source Type: Journal    
DOI: 10.2147/DDDT.S9258     Document Type: Review

References (78)

1. Corey M, McLaughlin FJ, Williams M, Levison H. A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto. J Clin Epidemiol. 1988;41(6):583-591.
2. Kerem E, Reisman J, Corey M, Canny GJ, Levison H. Prediction of mortality in patients with cystic fibrosis. N Engl J Med. 1992;326(18): 1187-1191.
3. Kraemer R, Rudeberg A, Hadorn B, Rossi E. Relative underweight in cystic fibrosis and its prognostic value. Acta Paediatr Scand. 1978; 67(1):33-37.
4. Borowitz D, Baker RD, Stallings V. Consensus report on nutrition for pediatric patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2002;35(3):246-259.
5. Cohen-Cymberknoh M, Shoseyov D, Kerem E. Managing cystic fibrosis: strategies that increase life expectancy and improve quality of life. Am J Respir Crit Care Med. 2011;183(11):1463-1471.
6. Lai HJ. Classifcation of nutritional status in cystic fibrosis. Curr Opin Pulm Med. 2006;12(6):422-427.
7. Pedreira CC, Robert RG, Dalton V, et al. Association of body composition and lung function in children with cystic fibrosis. Pediatr Pulmonol. 2005;39(3):276-280.
8. Quinton HB, O'Connor GT. Current issues in quality improvement in cystic fibrosis. Clin Chest Med. 2007;28(2):459-472.
9. Stallings VA, Stark LJ, Robinson KA, Feranchak A P, Quinton H. Evidence-based practice recommendations for nutrition-related management of children and adults with cystic fibrosis and pancreatic insufficiency: results of a systematic review. J Am Diet Assoc. 2008; 108(5):832-839.
10. Steinkamp G, Wiedemann B. Relationship between nutritional status and lung function in cystic fibrosis: cross sectional and longitudinal analyses from the German CF quality assurance (CFQA) project. Thorax. 2002;57(7):596-601.
11. Tiddens HA. Quality improvement in your CF centre: taking care of care. J Cyst Fibros. 2009;8 Suppl 1:S2-S5.
12. Sinaasappel M, Stern M, Littlewood J, et al. Nutrition in patients with cystic fibrosis: a European Consensus. J Cyst Fibros. 2002;1(2):51-75.
13. Borowitz D, Robinson KA, Rosenfeld M, et al. Cystic Fibrosis Foundation evidence-based guidelines for management of infants with cystic fibrosis. J Pediatr. 2009;155(6 Suppl):S73-S93.
14. Colombo C, Costantini D, Zazzeron L, et al. Benefits of breastfeeding in cystic fibrosis: a single-centre follow-up survey. Acta Paediatr. 2007; 96(8):1228-1232.
15. Ellis L, Kalnins D, Corey M, Brennan J, Pencharz P, Durie P. Do infants with cystic fibrosis need a protein hydrolysate formula? A prospective, randomized, comparative study. J Pediatr. 1998;132(2):270-276.
16. Pencharz PB, Durie PR. Pathogenesis of malnutrition in cystic fibrosis, and its treatment. Clin Nutr. 2000;19(6):387-394.
17. Efrati O, Mei-Zahav M, Rivlin J, et al. Long term nutritional rehabilitation by gastrostomy in Israeli patients with cystic fibrosis: clinical outcome in advanced pulmonary disease. J Pediatr Gastroenterol Nutr. 2006;42(2):222-228.
18. Walker SA, Gozal D. Pulmonary function correlates in the prediction of long-term weight gain in cystic fibrosis patients with gastrostomy tube feedings. J Pediatr Gastroenterol Nutr. 1998;27(1):53-56.
19. Williams SG, Ashworth F, McAlweenie A, Poole S, Hodson ME, Westaby D. Percutaneous endoscopic gastrostomy feeding in patients with cystic fibrosis. Gut. 1999;44(1):87-90.
20. Britton LJ, Thrasher S, Gutierrez H. Creating a culture of improvement: experience of a pediatric cystic fibrosis center. J Nurs Care Qual. 2008;23(2):115-120.
21. Feranchak A P, Sontag MK, Wagener JS, Hammond KB, Accurso FJ, Sokol RJ. Prospective, long-term study of fat-soluble vitamin status in children with cystic fibrosis identified by newborn screen. J Pediatr. 1999;135(5):601-610.
22. Hakim F, Kerem E, Rivlin J, et al. Vitamins A and E and pulmonary exacerbations in patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2007;45(3):347-353.
23. Drury D, Grey VL, Ferland G, Gundberg C, Lands LC. Efficacy of high dose phylloquinone in correcting vitamin K deficiency in cystic fibrosis. J Cyst Fibros. 2008;7(5):457-459.
24. Nicolaidou P, Stavrinadis I, Loukou I, et al. The effect of vitamin K supplementation on biochemical markers of bone formation in children and adolescents with cystic fibrosis. Eur J Pediatr. 2006;165(8):540-545.
25. Wilson DC, Rashid M, Durie PR, et al. Treatment of vitamin K deficiency in cystic fibrosis: Effectiveness of a daily fat-soluble vitamin combination. J Pediatr. 2001;138(6):851-855.
26. Beharry S, Ellis L, Corey M, Marcon M, Durie P. How useful is fecal pancreatic elastase 1 as a marker of exocrine pancreatic disease? J Pediatr. 2002;141(1):84-90.
27. Borowitz D, Baker SS, Duffy L, et al. Use of fecal elastase-1 to classify pancreatic status in patients with cystic fibrosis. J Pediatr. 2004;145(3):322-326.
28. Cohen JR, Schall JI, Ittenbach RF, Zemel BS, Stallings VA. Fecal elastase: pancreatic status verification and influence on nutritional status in children with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2005;40(4):438-444.
29. Dorlochter L, Aksnes L, Fluge G. Faecal elastase-1 and fat-soluble vitamin profiles in patients with cystic fibrosis in Western Norway. Eur J Nutr. 2002;41(4):148-152.
30. Walkowiak J, Nousia-Arvanitakis S, Cade A, et al. Fecal elastase-1 cut-off levels in the assessment of exocrine pancreatic function in cystic fibrosis. J Cyst Fibros. 2002;1(4):260-264.
31. Daftary A, Acton J, Heubi J, Amin R. Fecal elastase-1: utility in pancreatic function in cystic fibrosis. J Cyst Fibros. 2006;5(2):71-76.
32. Kalnins D, Durie PR, Pencharz P. Nutritional management of cystic fibrosis patients. Curr Opin Clin Nutr Metab Care. 2007;10(3):348-354.
33. Cade A, Walters M P, McGinley N, et al. Evaluation of fecal pancreatic elastase-1 as a measure of pancreatic exocrine function in children with cystic fibrosis. Pediatr Pulmonol. 2000;29(3):172-176.
34. Loser C, Mollgaard A, Folsch UR. Faecal elastase 1: a novel, highly sensitive, and specific tubeless pancreatic function test. Gut. 1996;39(4):580-586.
35. Borowitz D, Lin R, Baker SS. Comparison of monoclonal and polyclonal ELISAs for fecal elastase in patients with cystic fibrosis and pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2007;44(2):219-223.
36. Walkowiak J, Nousia-Arvanitakis S, Agguridaki C, et al. Longitudinal follow-up of exocrine pancreatic function in pancreatic sufficient cystic fibrosis patients using the fecal elastase-1 test. J Pediatr Gastroenterol Nutr. 2003;36(4):474-478.
37. Castellani C, Cuppens H, Macek M Jr, et al. Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice. J Cyst Fibros. 2008;7(3):179-196.
38. Wier HA, Kuhn RJ. Pancreatic enzyme supplementation. Curr Opin Pediatr. 2011;23(5):541-544.
39. Giuliano CA, Dehoorne-Smith ML, Kale-Pradhan PB. Pancreatic enzyme products: digesting the changes. Ann Pharmacother. 2011; 45(5):658-666.
40. Graff GR, Maguiness K, McNamara J, et al. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebocontrolled, two-period crossover, superiority study. Clin Ther. 2010;32(1):89-103.
41. Graff GR, McNamara J, Royall J, Caras S, Forssmann K. Safety and tolerability of a new formulation of pancrelipase delayed-release capsules (CREON) in children under seven years of age with exocrine pancreatic insufficiency due to cystic fibrosis: an open-label, multicentre, single-treatment-arm study. Clin Drug Investig. 2010;30(6): 351-364.
42. Trapnell BC, Maguiness K, Graff GR, Boyd D, Beckmann K, Caras S. Efficacy and safety of Creon 24,000 in subjects with exocrine pancreatic insufficiency due to cystic fibrosis. J Cyst Fibros. 2009;8(6):370-377.
43. Wooldridge JL, Heubi JE, Amaro-Galvez R, et al. EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency. J Cyst Fibros. 2009;8(6): 405-417.
44. Borowitz DS, Grand RJ, Durie PR. Use of pancreatic enzyme supplements for patients with cystic fibrosis in the context of fibrosing colonopathy. Consensus Committee. J Pediatr. 1995;127(5):681-684.
45. Colombo C, Fredella C, Russo MC, et al. Efficacy and tolerability of Creon for Children in infants and toddlers with pancreatic exocrine insufficiency caused by cystic fibrosis: an open-label, single-arm, multicenter study. Pancreas. 2009;38(6):693-699.
46. Kalnins D, Ellis L, Corey M, et al. Enteric-coated pancreatic enzyme with bicarbonate is equal to standard enteric-coated enzyme in treating malabsorption in cystic fibrosis. J Pediatr Gastroenterol Nutr. 2006;42(3):256-261.
47. Brady MS, Garson JL, Krug SK, et al. An enteric-coated high-buffered pancrelipase reduces steatorrhea in patients with cystic fibrosis: a prospective, randomized study. J Am Diet Assoc. 2006;106(8): 1181-1186.
48. Borowitz D, Konstan M, O'Rourke A, Cohen M, Hendeles L, Murray FT. Coefficients of fat and nitrogen absorption in healthy subjects and individuals with cystic fibrosis. Journal of Pediatric Pharmacology and Therapeutics. 2007;12:47-52.
49. Borowitz D, Stevens C, Brettman LR, Campion M, Chatfield B, Cipolli M. International phase III trial of liprotamase efficacy and safety in pancreatic-insufficient cystic fibrosis patients. J Cyst Fibros. 2011; 10(6):443-452.
50. Borowitz D, Stevens C, Brettma LR, Campion M, Wilschanski M, Thompson H. International open-label trial of liprotamase long-term safety and support of nutritional status in pancreatic-insufficient cystic fibrosis patients. J Pediatr Gastroenterol Nutr. August 26, 2011. [Epub ahead of print.]
51. Durie P, Kalnins D, Ellis L. Uses and abuses of enzyme therapy in cystic fibrosis. J R Soc Med. 1998;91 Suppl 34:2-13.
52. Butt AM, Ip W, Ellis L, et al. The fate of exogenous enzymes in patients with cystic fibrosis and pancreatic insufficiency (Abstr). J Pediatr Gastroenterol Nutr. 2001;33:391.
53. Kalivianakis M, Minich DM, Bijleveld CM, et al. Fat malabsorption in cystic fibrosis patients receiving enzyme replacement therapy is due to impaired intestinal uptake of long-chain fatty acids. Am J Clin Nutr. 1999;69(1):127-134.
54. Laiho KM, Gavin J, Murphy JL, Connett GJ, Wootton SA. Maldigestion and malabsorption of 13C labelled tripalmitin in gastrostomy-fed patients with cystic fibrosis. Clin Nutr. 2004;23(3):347-353.
55. Bower TR, Pringle KC, Soper RT. Sodium defcit causing decreased weight gain and metabolic acidosis in infants with ileostomy. J Pediatr Surg. 1988;23(6):567-572.
56. Bianchi ML, Romano G, Saraifoger S, Costantini D, Limonta C, Colombo C. BMD and body composition in children and young patients affected by cystic fibrosis. J Bone Miner Res. 2006;21(3):388-396.
57. Buntain HM, Greer RM, Schluter PJ, et al. Bone mineral density in Australian children, adolescents and adults with cystic fibrosis: a controlled cross sectional study. Thorax. 2004;59(2):149-155.
58. Gronowitz E, Garemo M, Lindblad A, Mellstrom D, Strandvik B. Decreased bone mineral density in normal-growing patients with cystic fibrosis. Acta Paediatr. 2003;92(6):688-693.
59. Aris RM, Merkel PA, Bachrach LK, et al. Guide to bone health and disease in cystic fibrosis. J Clin Endocrinol Metab. 2005;90(3):1888-1896.
60. Moran A, Brunzell C, Cohen RC, et al. Clinical care guidelines for cystic fibrosis-related diabetes: a position statement of the American Diabetes Association and a clinical practice guideline of the Cystic Fibrosis Foundation, endorsed by the Pediatric Endocrine Society. Diabetes Care. 2010;33(12):2697-2708.
61. Cheung MS, Bridges NA, Prasad SA, et al. Growth in children with cystic fibrosis-related diabetes. Pediatr Pulmonol. 2009;44(12): 1223-1225.
62. Hameed S, Morton JR, Field PI, et al. Once daily insulin detemir in cystic fibrosis with insulin deficiency. Arch Dis Child. April 14, 2011. [Epub ahead of print.]
63. Kolouskova S, Zemkova D, Bartosova J, et al. Low-dose insulin therapy in patients with cystic fibrosis and early-stage insulinopenia prevents deterioration of lung function: a 3-year prospective study. J Pediatr Endocrinol Metab. 2011;24(7-8):449-454.
64. Phung OJ, Coleman CI, Baker EL, et al. Recombinant human growth hormone in the treatment of patients with cystic fibrosis. Pediatrics. 2010;126(5):e1211-e1226.
65. Stalvey MS, Anbar RD, Konstan M W, et al. A multi-center controlled trial of growth hormone treatment in children with cystic fibrosis. Pediatr Pulmonol. September 8, 2011. [Epub ahead of print.]
66. Chinuck RS, Fortnum H, Baldwin DR. Appetite stimulants in cystic fibrosis: a systematic review. J Hum Nutr Diet. 2007;20(6):526-537.
67. Edenborough F P, Borgo G, Knoop C, et al. Guidelines for the management of pregnancy in women with cystic fibrosis. J Cyst Fibros. 2008; 7 Suppl 1:S2-S32.
68. Goss CH, Rubenfeld GD, Otto K, Aitken ML. The effect of pregnancy on survival in women with cystic fibrosis. Chest. 2003;124(4): 1460-1468.
69. Schwebel C, Pin I, Barnoud D, et al. Prevalence and consequences of nutritional depletion in lung transplant candidates. Eur Respir J. 2000;16(6):1050-1055.
70. Aris RM, Neuringer I P, Weiner MA, Egan TM, Ontjes D. Severe osteoporosis before and after lung transplantation. Chest. 1996;109(5): 1176-1183.
71. Daniels MW, Wilson DM, Paguntalan HG, Hoffman AR, Bachrach LK. Bone mineral density in pediatric transplant recipients. Transplantation. 2003;76(4):673-678.
72. McPartland KJ, Pomposelli JJ. Update on immunosuppressive drugs used in solid-organ transplantation and their nutrition implications. Nutr Clin Pract. 2007;22(5):467-473.
73. Ollech JE, Kramer MR, Peled N, et al. Post-transplant diabetes mellitus in lung transplant recipients: incidence and risk factors. Eur J Cardiothorac Surg. 2008;33(5):844-848.
74. Stephenson A, Brotherwood M, Robert R, et al. Increased vitamin A and E levels in adult cystic fibrosis patients after lung transplantation. Transplantation. 2005;79(5):613-615.
75. Ho T, Gupta S, Brotherwood M, et al. Increased serum vitamin A and E levels after lung transplantation. Transplantation. 2011;92(5):601-606.
76. Stiebellehner L, Quittan M, End A, et al. Aerobic endurance training program improves exercise performance in lung transplant recipients. Chest. 1998;113(4):906-912.
77. Houwen RH, van der Doef H P, Sermet I, et al. Defining DIOS and constipation in cystic fibrosis with a multicentre study on the incidence, characteristics, and treatment of DIOS. J Pediatr Gastroenterol Nutr. 2010;50(1):38-42.
78. Colombo C, Ellemunter H, Houwen R, Munck A, Taylor C, Wilschanski M. Guidelines for the diagnosis and management of distal intestinal obstruction syndrome in cystic fibrosis patients. J Cyst Fibros. 2011; 10 Suppl 2:S24-S28.