Παρασκευή 27 Ιουλίου 2018

Relationship of Initial Pancreatic Enzyme Replacement Therapy Dose with Weight Gain in Infants with Cystic Fibrosis

Objective: To test the hypothesis of a positive relationship between initial dose of pancreatic enzyme replacement therapy (PERT) in infants with cystic fibrosis (CF) and optimal weight gain over the first two years of life. Methods: Using the CF Foundation Patient Registry, we identified 502 children born in 2010 and used multivariable models to compare as our primary analysis their 2-year changes in weight-for-age z score (WAZ) and as our secondary analysis weight-for-length percentile (W/L%) by initial PERT dose. We focused on initial dose without reference to subsequent changes in treatment in order to avoid confounding by indication (severity). Results: Initial PERT dose demonstrated a linear relationship to change in WAZ and W/L% at age 2 years. An initial dose of >1500 lipase units/kg/largest meal resulted in a higher likelihood of attaining WAZ at 2 years at or above the birth WAZ (adjusted odds ratio [aOR] 1.87, 95% confidence interval [CI]: 1.22–2.86) and at the top quartile for improvement over 2 years in WAZ (aOR 1.90, 95% CI: 1.19–3.05).. There was no correlation between initial PERT dose and weight at initial PERT encounter (p = 0.35). Findings were similar for W/L% and when the cohort was restricted to infants who began PERT in the first 3 months of life. Conclusion: Infants receiving higher initial PERT dose demonstrate better weight-related outcomes, as reflected by attainment of favorable changes in WAZ and W/L%, at age 2 years. This is an open access article distributed under the terms of the Creative Commons Attribution-Non Commercial-No Derivatives License 4.0 (CCBY-NC-ND), where it is permissible to download and share the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal. https://ift.tt/OBJ4xP Address correspondence and reprint requests to Michael Schechter, MD, MPH, Virginia Commonwealth University (VCU) Children's Hospital of Richmond at VCU, Department of Pediatrics Division of Pulmonary Medicine 1000 East Broad Street P.O. Box 980315 Richmond, Virginia 23298-0315 (e-mail: mschechter@vcu.edu). Received 1 October, 2017 Accepted 20 May, 2018 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). Clinical trial registration: Not applicable Conflicts of Interest and Source of Funding M.S. Schechter has no financial relationship with AbbVie Inc and received no funding for his work on this manuscript. He receives research support from the NIH, CF Foundation, Anthera, and Novartis; and has had consulting relationships with AbbVie, Vertex, Novartis, Gilead, Genentech, and Celtaxsys. S. Michel is a consultant to AbbVie Inc. She is also a technical advisor to MVW Nutritionals, makers of cystic fibrosis-specific vitamin products. M. Kapoor and R. Khurmi are employed by AbbVie Inc., and may own stock. S. Liu was an employee of AbbVie Inc. during the analysis. Her current affiliation is Astellas Inc., Northbrook, IL. B. Woun Seo was an employee of AbbVie Inc., during the analysis. His current affiliation is Rhythm Pharmaceuticals, Boston, MA. M. Haupt was an employee of AbbVie Inc. during the analysis. His current affiliation is ARIEL Precision Medicine, Pittsburgh, PA. Funding for this study was provided by AbbVie Inc. Author Contributions: M.S. Schechter contributed to designing and performing the study, analyzing and interpreting the data, and writing and critically revising the manuscript. S. Michel contributed to designing and performing the study, analyzing and interpreting the data, and critically revising the manuscript. S. Liu contributed to designing and performing the study, analyzing and interpreting the data, and writing and critically revising the manuscript. B.W. Seo contributed to designing the study, analyzing and interpreting the data, and critically revising the manuscript. M. Kapoor contributed to designing the study, analyzing and interpreting the data, and writing the manuscript. R Khurmi contributed to designing the study, analyzing and interpreting the data, and critically revising the manuscript. M. Haupt contributed to designing the study, analyzing and interpreting the data, and writing and critically revising the manuscript. All authors approved the manuscript for submission. © 2018 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,

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