The effects of casein glycomacropeptide on general health status in children with PKU: A randomized crossover trial.

Autor: Pinto A; Birmingham Women's and Children's Hospital, Birmingham, UK; Plymouth Institute of Health and Care Research, Faculty of Health, University of Plymouth, UK. Electronic address: alex.pinto@nhs.net., Daly A; Birmingham Women's and Children's Hospital, Birmingham, UK., Newby C; Bristol Royal Hospital for Children, Upper Maudlin St, Bristol, UK., Robotham A; Bristol Royal Hospital for Children, Upper Maudlin St, Bristol, UK., Heales S; UCL Great Ormond Street Institute of Child Health, London, UK., Eaton S; UCL Great Ormond Street Institute of Child Health, London, UK., Aitkenhead H; Department of Chemical Pathology, Great Ormond Street Hospital NHS Foundation Trust, London, UK., Gilmour K; Cell Therapy and Immunology, NIHR GOSH BRC, Camelia Botnar Laboratories, Great Ormond Street Hospital for Children, London, UK., Jackson R; Department of Health Data Science, University of Liverpool, Liverpool, UK., Ashmore C; Birmingham Women's and Children's Hospital, Birmingham, UK., Evans S; Birmingham Women's and Children's Hospital, Birmingham, UK., Rocha JC; Nutrition and Metabolism, NOVA Medical School (NMS), Faculdade de Ciências Médicas (FCM), Universidade Nova de Lisboa, Lisboa, Portugal,; CINTESIS@RISE, Nutrition and Metabolism, NOVA Medical School (NMS), Faculdade de Ciências Médicas, (FCM), Universidade NOVA de Lisboa, Campo Mártires da Pátria, Lisboa, Portugal; Reference Center of Inherited Metabolic Diseases, Centro Hospitalar Universitario de Lisboa Central, Lisboa, Portugal; Comprehensive Health Research Centre (CHRC), NOVA Medical School|Faculdade de Ciências Médicas, NMS|FCM, Universidade Nova de Lisboa, 1169-056 Lisboa, Portugal., Ilgaz F; Department of Nutrition and Dietetics, Faculty of Health Sciences, Hacettepe University, Ankara, Turkey., Hickson M; Plymouth Institute of Health and Care Research, Faculty of Health, University of Plymouth, UK., MacDonald A; Birmingham Women's and Children's Hospital, Birmingham, UK.
Jazyk: angličtina
Zdroj: Molecular genetics and metabolism [Mol Genet Metab] 2024 Dec; Vol. 143 (4), pp. 108607. Date of Electronic Publication: 2024 Nov 04.
DOI: 10.1016/j.ymgme.2024.108607
Abstrakt: In PKU, it is suggested that casein glycomacropeptide based protein substitute (GMP) may have physiological advantage when satiety, oxidative stress, renal function and inflammation are considered. Its prebiotic properties may also help gastrointestinal (GI) tolerance. In children with PKU, a randomized/crossover trial comparing phenylalanine-free amino acids (AA) vs GMP as the single source of protein substitute for 12-weeks in each arm was conducted. There was a 4-week wash out period with AA in-between. At baseline and end of each intervention, blood and fecal samples were taken to monitor gut health, oxidative stress, renal function, inflammatory markers and plasma amino acids. Satiety and Pediatric Quality of Life (PedsQL) GI symptoms questionnaires were completed. Usual weekly blood spots for phenylalanine and tyrosine were done. Twelve patients (8 males; aged 4-9y) with PKU participated. GMP improved the following GI symptoms: stomach pain (p = 0.003), heartburn and reflux (p = 0.041) wind and bloating (p = 0.018). With GMP, there was also a trend for less constipation (p = 0.068), discomfort with eating (p = 0.065) and nausea and vomiting (p = 0.087). There were no changes on stool gut health markers (IgA, short chain fatty acids and fecal calprotectin). There were no statistically significant differences for renal, oxidative stress, inflammatory and gut health markers or measures of satiety except for adiponectin (p = 0.028) and total antioxidant capacity (p = 0.049), although the latter was possibly without clinical significance. Mean dried blood spot phenylalanine (Phe) was 114 μmol/L higher with GMP vs AA (p < 0.001). There was no difference in tyrosine levels. In conclusion, GI symptoms statistically significantly improved with GMP versus AA. The Phe content of GMP may present challenges when it is used as the only protein substitute in children with classical PKU with low Phe tolerance.
Competing Interests: Declaration of competing interest A.P. received an educational grant from Cambrooke Therapeutics and Biomarin and grants from Vitaflo International, Nutricia, Biomarin, Mevalia, Galen, PIAM, and Applied Pharma Research to attend scientific meetings. This project is also part of A.P.'s PhD, which is funded by Vitaflo International®. A.D. received research funding from Vitaflo International and financial support from Nutricia, Mevalia, and Vitaflo International to attend study days and conferences. C.N. received honoraria from Nutricia and Vitaflo International to attend study days and conferences. J.C.R. was a member of the European Nutritionist Expert Panel (Biomarin) and the Advisory Board for AppliedPharma Research, Vitaflo, Synlogic, Biomarin, and Nutricia and received honoraria as a speaker from APR, Merck Serono, Biomarin, Nutricia, Vitaflo, Cambrooke, PIAM, and Life diet. S.E. (Sharon Evans) received research funding and financial support to attend study days and conferences from both Nutricia and Vitaflo International. C.A. received honoraria from Nutricia and Vitaflo International to attend study days and conferences. A.M. received research funding and honoraria from Arla, Applied Pharma research, Cambrookes, Galen, Nutricia, PTC Pharmaceuticals, Vitaflo International, Metax, PIAM, and Biomarin. S.E. (Simon Eaton) received research funding and financial support to attend study days and conferences from both Nutricia and Vitaflo International and has also received research funding and is co-inventor on patents with Vitaflo international (all unrelated to patients with PKU). S.H. is a consultant for Vitaflo and has also received research funding from said company.
(Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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