Pancreatic Enzyme Replacement Therapy use in infants with Cystic Fibrosis Diagnosed by Newborn Screening

ABSTRACT Objectives: To describe pancreatic enzyme practices during the first year of life in infants with cystic fibrosis (CF) and evaluate associations between dosing and outcomes, including growth and gastrointestinal symptoms. Methods: We analyzed data from a subset of infants who were in a prospective cohort study conducted at 28 US CF centers. Anthropometric measurements and medications were recorded at each visit. Diaries with infant diet, pancreatic enzyme replacement therapy (PERT) dosing, stool frequency and consistency and pain were completed by a parent/guardian for three days prior to each visit. Results: 231 infants were enrolled in the main study; 205 of these met criteria for pancreatic insufficiency. PERT dose between birth and 6 months was on average 1882 LU/kg/meal (range: 492–3727) and was similar between 6 months and 12 months (mean: 1842 LU/kg/mean, range: 313–3612). PERT dose had a weak, negative association with weight z-score at 3 and 6 months (r = −0.16, 95% CI = −0.29, −0.02 and r = −0.18, 95% CI = −0.31, −0.04, respectively) but not at 12 months. There was not a clear relationship between PERT dosing and number of stools/day, stool consistency or pain. 144 infants (70%) were placed on acid suppression medication. Weight z-score mean was 0.37 higher in infants using proton pump inhibitors exclusively vs those using histamine-2 blockers exclusively (95% CI = −0.02, 0.76, p = 0.06). Conclusions: We did not observe that centers with a higher PERT dosing strategy yielded greater clinical benefit than dosing at the lower end of the recommended range. Address correspondence and reprint requests to Sonya L. Heltshe, Seattle Children's Research Institute, M/S CW805B, PO BOX 5371, Seattle WA 98145-5005 (e-mail: Sonya.Heltshe@seattlechildrens.org; http://ift.tt/1e5kjHX). Received 18 July, 2017 Accepted 9 October, 2017 See Appendix, Supplemental Digital Content 1, http://ift.tt/2ArRHqU for BONUS Study Investigators Supplemental digital content is available for this article. Direct URL citations appear in the printed text, and links to the digital files are provided in the HTML text of this article on the journal's Web site (www.jpgn.org). Funding/support: BONUS and its lead investigators were supported by CFFT BONUS11KO, NIH R01DK095738, NIH P30DK089507, NIH UL1TR000423, CFF LEUNG14GE0 and NIH P30 DK072482. Conflict of interest disclosures and Sources of Funding: Dr Gelfond has served as a consultant for Vertex, Abbvie and Chiesi Pharmaceuticals. Dr. Leung has served as a consultant for Vertex and receives research/grant support from Bristol Meyers Squibb, Gilead, Abbvie, and Roche pharmaceuticals outside of the submitted work. Dr. Ramsey discloses that over the past 3 years she has received grant support from the Cystic Fibrosis Foundation and the National Institutes of Health. She has been the principal investigator on contracts between Seattle Children's Hospital and the following companies: Aridis Pharmaceuticals, LLC, Celtaxsys, Kalobios, Flatley Discovery Labs, LLV, Vertex Pharmaceuticals Inc, Laurent Therapeutics, Inc, Nilvalis Therapeutics, Inc, and Synedgen, Inc. Dr Heubi has received personal fees from Alynlam Pharma and has financial interest in Asklepion Pharma LLC. No other disclosures are reported. Author Contributions: Design and Conduct of the Study: Gelfond, Heltshe, Borowitz, Leung, Heubi, Ramsey; Collection, management, analysis and interpretation of data: All authors; Preparation, review or approval of the manuscript: All authors; Statistical Analysis: Heltshe, Skalland, Kloster © 2017 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,

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