{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T00:16:32Z","timestamp":1791332192735,"version":"4.3.1"},"reference-count":12,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2011,7,1]],"date-time":"2011-07-01T00:00:00Z","timestamp":1309478400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. pediatr. gastroenterol. nutr."],"published-print":{"date-parts":[[2011,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background:<\/jats:title><jats:p>Pancreatic enzyme replacement therapy (PERT) improves nutritional status and growth in patients with cystic fibrosis (CF) with pancreatic insufficiency (PI). The current recommendation for infants and young children, who are not able to swallow the whole capsule, is to open the capsule and mix the beads in a spoon with some applesauce; however, the efficacy and safety data of this approach are currently lacking. The aim of this study was to assess the efficacy, palatability (ease of swallowing), and safety of 4 dose levels of pancrelipase microtablets (Pancrease MT) in infants and young children with CF\u2010related PI.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients and Methods:<\/jats:title><jats:p>This study was a phase II randomized, investigator\u2010blinded, parallel\u2010group pilot study in DNA\u2010proven infants with CF and PI. The study design included a run\u2010in period (days 1\u20135) and an experimental period (days 6\u201311). Pancrelipase microtablets (2\u2010mm, enteric coated) were provided orally. Sixteen subjects, 6 to 30 months of age, were provided 500 U lipase\/kg\/meal for 5 days (baseline period). Subsequently, subjects were randomly assigned to 1 of 4 treatment groups (each n = 4), receiving 500, 1000, 1500, or 2000 U (Ph. EUR) of lipase\/kg\/meal, respectively, for 5 days (experimental period). The primary endpoint was medication efficacy assessed by the 72\u2010hour fecal fat excretion, expressed as coefficient of fecal fat absorption (CFA), and <jats:sup>13<\/jats:sup>C mixed triglyceride breath test. Secondary endpoints were safety and palatability.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p>Overall compliance, defined as used study medication, was 89% to 99% for the entire study. None of the 4 dose regimens significantly influenced the CFA, relative to the baseline period (median range 83%\u201393%). During the run\u2010in period the median cumulative % <jats:sup>13<\/jats:sup>C was 11 (range \u22128 to 59). After randomization the median cumulative % <jats:sup>13<\/jats:sup>C was 18 (range 14\u201323) in the 500\u2010U, 14 (range \u22121 to 17) in the 1000\u2010U, 10 (range 10\u201327) in the 1500\u2010U, and 3 (range 1\u201349) in the 2000\u2010U groups. Palatability was scored fair to good by the parents in each of the treatment groups. Gastrointestinal symptoms were reported in some patients, including common adverse events reported in clinical trials involving pancreatic enzyme therapy. No serious or other adverse events were reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion:<\/jats:title><jats:p>Treatment with Pancrease MT at a dosage of 500 U lipase\/kg\/meal resulted in a CFA of approximately 89% in pediatric subjects ages 6 to 30 months with PI resulting from CF. Pancrease MT doses were well tolerated and mean palatability was scored as fair to good. Present results do not indicate that a dosage higher than 500 U (Ph. EUR) lipase\/kg\/meal increases the coefficient of fat absorption in a cohort of infants 6 to 30 months of age.<\/jats:p><\/jats:sec>","DOI":"10.1097\/mpg.0b013e31820e208e","type":"journal-article","created":{"date-parts":[[2011,6,21]],"date-time":"2011-06-21T02:48:18Z","timestamp":1308624498000},"page":"61-64","source":"Crossref","is-referenced-by-count":52,"title":["Treatment of Infants and Toddlers With Cystic Fibrosis\u2013related Pancreatic Insufficiency and Fat Malabsorption With Pancrelipase MT"],"prefix":"10.1002","volume":"53","author":[{"given":"Els","family":"Van de Vijver","sequence":"first","affiliation":[{"name":"Department of Pediatrics University Hospital Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristine","family":"Desager","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University Hospital Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew E","family":"Mulberg","sequence":"additional","affiliation":[{"name":"Johnson &amp; Johnson Pharmaceutical Research &amp; Development Titusville NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sofie","family":"Staelens","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology Queen Paola Children's Hospital and University Hospital Antwerp Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henkjan J","family":"Verkade","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center for Liver Digestive and Metabolic Diseases Pediatric Gastroenterology Groningen University Institute for Drug Exploration University Hospital Groningen Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank AJA","family":"Bodewes","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Center for Liver Digestive and Metabolic Diseases Pediatric Gastroenterology Groningen University Institute for Drug Exploration University Hospital Groningen Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Malfroot","sequence":"additional","affiliation":[{"name":"Department of Pediatric Respiratory Medicine Infectious Diseases and Cystic Fibrosis Clinic University Hospital UZ Brussels VUB Brussels Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruno","family":"Hauser","sequence":"additional","affiliation":[{"name":"Department of Pediatric Respiratory Medicine Infectious Diseases and Cystic Fibrosis Clinic University Hospital UZ Brussels VUB Brussels Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maarten","family":"Sinaasappel","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology Erasmus Medical Centre Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefanie","family":"Van Biervliet","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology University Hospital Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Behm","sequence":"additional","affiliation":[{"name":"Johnson &amp; Johnson Pharmaceutical Research &amp; Development Titusville NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Pelckmans","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology University Hospital Antwerp Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dirk","family":"Callens","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology University Hospital Antwerp Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gigi","family":"Veereman\u2010Wauters","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology Queen Paola Children's Hospital and University Hospital Antwerp Antwerp Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2011,7]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/j.1536-4801.2002.tb07816.x"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1569-1993(02)00032-2"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(95)70153-2"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199705013361803"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2008.07.003"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.20286"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2362.1997.1310678.x"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinbiochem.2008.08.087"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1136\/gut.43.2008.S13"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/0016-5085(89)91632-6"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/j.1536-4801.2001.tb07377.x"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(99)70166-6"}],"container-title":["Journal of Pediatric Gastroenterology and Nutrition"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1097\/MPG.0b013e31820e208e","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,18]],"date-time":"2024-01-18T09:07:53Z","timestamp":1705568873000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1097\/MPG.0b013e31820e208e"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,7]]},"references-count":12,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2011,7]]}},"alternative-id":["10.1097\/MPG.0b013e31820e208e"],"URL":"https:\/\/doi.org\/10.1097\/mpg.0b013e31820e208e","archive":["Portico"],"relation":{},"ISSN":["0277-2116","1536-4801"],"issn-type":[{"value":"0277-2116","type":"print"},{"value":"1536-4801","type":"electronic"}],"subject":[],"published":{"date-parts":[[2011,7]]}}}