Dataset Information

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective.

ABSTRACT:

Background

In phenylketonuria (PKU), modified casein glycomacropeptide supplements (CGMP-AA) are used as an alternative to the traditional phenylalanine (Phe)-free L-amino acid supplements (L-AA). However, studies focusing on the long-term nutritional status of CGMP-AA are lacking. This retrospective study evaluated the long-term impact of CGMP-AA over a mean of 29 months in 11 patients with a mean age at CGMP-AA onset of 28 years (range 15-43) [8 females; 2 hyperphenylalaninaemia (HPA), 3 mild PKU, 3 classical PKU and 3 late-diagnosed]. Outcome measures included metabolic control, anthropometry, body composition and biochemical parameters.

Results

CGMP-AA, providing 66% of protein equivalent intake from protein substitute, was associated with no significant change in blood Phe with CGMP-AA compared with baseline (562?±?289 µmol/L vs 628?±?317 µmol/L; p?=?0.065). In contrast, blood tyrosine significantly increased on CGMP-AA (52.0?±?19.2 ?mol/L vs 61.4?±?23.8 ?mol/L; p?=?0.027).

Conclusions

Biochemical nutritional markers remained unchanged which is an encouraging finding in adults with PKU, many of whom are unable to maintain full adherence with nutritionally fortified protein substitutes. Longitudinal, prospective studies with larger sample sizes are necessary to fully understand the metabolic impact of using CGMP-AA in PKU.

SUBMITTER: Pena MJ

PROVIDER: S-EPMC7881530 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature

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Publications

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective.

Pena Maria João MJ Pinto Alex A de Almeida Manuela Ferreira MF de Sousa Barbosa Catarina C Ramos Paula Cristina PC Rocha Sara S Guimas Arlindo A Ribeiro Rosa R Martins Esmeralda E Bandeira Anabela A Dias Cláudia Camila CC MacDonald Anita A Borges Nuno N Rocha Júlio César JC

Orphanet journal of rare diseases 20210213 1

<h4>Background</h4>In phenylketonuria (PKU), modified casein glycomacropeptide supplements (CGMP-AA) are used as an alternative to the traditional phenylalanine (Phe)-free L-amino acid supplements (L-AA). However, studies focusing on the long-term nutritional status of CGMP-AA are lacking. This retrospective study evaluated the long-term impact of CGMP-AA over a mean of 29 months in 11 patients with a mean age at CGMP-AA onset of 28 years (range 15-43) [8 females; 2 hyperphenylalaninaemia (HPA), ...[more]

PMID: 33581730

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Project description:To prevent cognitive impairment, phenylketonuria requires lifelong management of blood phenylalanine (Phe) concentration with a low-Phe diet. The diet restricts intake of Phe from natural proteins in combination with traditional amino acid medical foods (AA-MFs) or glycomacropeptide medical foods (GMP-MFs) that contain primarily intact protein and a small amount of Phe.We investigated the efficacy and safety of a low-Phe diet combined with GMP-MFs or AA-MFs providing the same quantity of protein equivalents in free-living subjects with phenylketonuria.This 2-stage, randomized crossover trial included 30 early-treated phenylketonuria subjects (aged 15-49 y), 20 with classical and 10 with variant phenylketonuria. Subjects consumed, in random order for 3 wk each, their usual low-Phe diet combined with AA-MFs or GMP-MFs. The treatments were separated by a 3-wk washout with AA-MFs. Fasting plasma amino acid profiles, blood Phe concentrations, food records, and neuropsychological tests were obtained.The frequency of medical food intake was higher with GMP-MFs than with AA-MFs. Subjects rated GMP-MFs as more acceptable than AA-MFs and noted improved gastrointestinal symptoms and less hunger with GMP-MFs. ANCOVA indicated no significant mean ± SE increase in plasma Phe (62 ± 40 ?mol/L, P = 0.136), despite a significant increase in Phe intake from GMP-MFs (88 ± 6 mg Phe/d, P = 0.026). AA-MFs decreased plasma Phe (-85 ± 40 ?mol/L, P = 0.044) with stable Phe intake. Blood concentrations of Phe across time were not significantly different (AA-MFs = 444 ± 34 ?mol/L, GMP-MFs = 497 ± 34 ?mol/L), suggesting similar Phe control. Results of the Behavior Rating Inventory of Executive Function were not significantly different.GMP-MFs provide a safe and acceptable option for the nutritional management of phenylketonuria. The greater acceptability and fewer side effects noted with GMP-MFs than with AA-MFs may enhance dietary adherence for individuals with phenylketonuria. This trial was registered at www.clinicaltrials.gov as NCT01428258.

Project description:In phenylketonuria, casein glycomacropeptide (CGMP) requires modification with the addition of some essential and semi essential amino acids to ensure suitability as a protein substitute. The optimal amount and ratio of additional amino acids is undefined. AIM:A longitudinal, parallel, controlled study over 12?months evaluating a CGMP (CGMP-AA2) formulation compared with phenylalanine-free L-amino acid supplements (L-AA) on blood Phe, Tyr, Phe:Tyr ratio, biochemical nutritional status and growth in children with PKU. The CGMP-AA2 contained 36?mg Phe per 20?g protein equivalent. METHODS:Children with PKU, with a median age of 9.2 y (5-16y) were divided into 2 groups: 29 were given CGMP-AA2, 19 remained on Phe-free L-AA. The CGMP-AA2 formula gradually replaced L-AA, providing blood Phe concentrations were maintained within target range. Median blood Phe, Tyr, Phe:Tyr ratio and anthropometry, were compared within and between the two groups at baseline, 26 and 52?weeks. Nutritional biochemistry was studied at baseline and 26?weeks only. RESULTS:At the end of 52?weeks only 48% of subjects were able to completely use CGMP-AA2 as their single source of protein substitute. At 52?weeks CGMP-AA2 provided a median of 75% (30-100) of the total protein substitute with the remainder being given as L-AA. Within the CGMP-AA2 group, blood Phe increased significantly between baseline and 52?weeks: [baseline to 26?weeks; baseline Phe 270??mol/L (170-430); 26?weeks, Phe 300??mol/L (125-485) p =?0.06; baseline to 52?weeks: baseline, Phe 270??mol/L (170-430), 52?weeks Phe 300??mol/L (200-490), p <?0.001)]. However, there were no differences between the CGMP-AA2 and L-AA group for Phe, Tyr, Phe:Tyr ratio or anthropometry at any of the three measured time points. Within the CGMP-AA2 group only weight (p =?0.0001) and BMI z scores (p?=?0.0001) increased significantly between baseline to 52?weeks. Whole blood and plasma selenium were significantly higher (whole blood selenium [p =?0.0002]; plasma selenium [p =?0.0007]) at 26?weeks in the CGMP-AA2 group compared L-AA. No differences were observed within the L-AA group for any of the nutritional markers. CONCLUSIONS:CGMP-AA increases blood Phe concentrations and so it can only be used partly to contribute to protein substitute in some children with PKU. CGMP-AA should be carefully introduced in children with PKU and close monitoring of blood Phe control is essential.

Dataset Information

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective.

Background

Results

Conclusions

Publications

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective.

Similar Datasets

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