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Glycomacropeptide: long-term use and impact on blood phenylalanine, growth and nutritional status in children with PKU

Daly, A., Evans, S., Chahal, S., Santra, S., Pinto, A., Jackson, R., Gingell, C., Rocha, J., Van Spronsen, F. J. & MacDonald, A., 15-Feb-2019, In : Orphanet journal of rare diseases. 14, 12 p., 44.

Research output: Contribution to journalArticleAcademicpeer-review

  • A. Daly
  • S. Evans
  • S. Chahal
  • S. Santra
  • A. Pinto
  • R. Jackson
  • C. Gingell
  • J. Rocha
  • F. J. Van Spronsen
  • A. MacDonald

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.AimA longitudinal, parallel, controlled study over 12months 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 36mg Phe per 20g protein equivalent.MethodsChildren 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 52weeks. Nutritional biochemistry was studied at baseline and 26weeks only.ResultsAt the end of 52weeks only 48% of subjects were able to completely use CGMP-AA2 as their single source of protein substitute. At 52weeks 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 52weeks: [baseline to 26weeks; baseline Phe 270mol/L (170-430); 26weeks, Phe 300mol/L (125-485) p=0.06; baseline to 52weeks: baseline, Phe 270mol/L (170-430), 52weeks Phe 300mol/L (200-490), p

Original languageEnglish
Article number44
Number of pages12
JournalOrphanet journal of rare diseases
Volume14
Publication statusPublished - 15-Feb-2019

    Keywords

  • Glycomacropeptide, Phenylalanine, Large neutral amino acids, Protein substitute, Phenylketonuria, NEUTRAL AMINO-ACIDS, SUPPORTS GROWTH, MURINE MODEL, PHENYLKETONURIA, PROTEIN, TRANSPORT, MANAGEMENT, INSULIN, BRAIN, SECRETION

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