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Sustitutos proteicos para niños y adultos con fenilcetonuria

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Referencias

Referencias de los estudios incluidos en esta revisión

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Prince 1997 {published data only}

Prince AP, McMurry MP, Buist NRM. Treatment products and approaches for phenylketonuria: improved palatability and flexibility demonstrate safety, efficacy and acceptance in US clinical trials. Journal of Inherited Metabolic Disease 1997;20(4):486‐98.

Referencias de los estudios excluidos de esta revisión

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Acosta 1994 {published data only}

Acosta PB, Yannicelli S. Protein intake affects phenylalanine requirements and growth of infants with phenylketonuria. Acta Paediatrica 1994;407:66‐7.

Baumgartner 2004 {published data only}

Baumgartner C, Bohm C, Baumgartner D, Marini G, Weinberger K, Olgemoller B, et al. Supervised machine learning techniques for the classification of metabolic disorders in newborns. Bioinformatics 2004;20(17):2985‐96.

Clemens 1991 {published data only}

Clemens PC, Heddrich‐Ellerbrok M, Wachtel V, Link RM. Plasma amino acids in adolescents and adults with phenylketonuria on three different levels of protein intake. Acta Paediatrica Scandinavica 1991;80(5):577‐80.

Kalkanoglu 2005 {published data only}

Kalkanoglu HS, Ahring KK, Sertkaya D, Moller LB, Romstad A, Mikkelsen I, et al. Behavioural effects of phenylalanine‐free amino acid tablet supplementation in intellectually disabled adults with untreated phenylketonuria. Acta Paediatrica 2005;94(9):1218‐22.

MacDonald 2003a {published data only}

MacDonald A, Ferguson C, Rylance G, Morris AA, Asplin D, Hall SK, et al. Are tablets a practical source of protein substitute in phenylketonuria?. Archives of Disease in Childhood 2003;88(4):327‐9.

MacDonald 2005 {published data only}

MacDonald A, Lilburn M, Daly A, Hall SK, Hendriksz C, Chakrapani A, et al. Does ready to drink protein substitute improve compliance in PKU [abstract]. Society for the Study of Inborn Errors of Metabolism 42nd Annual Symposium; 2005 Sep 6‐9; Paris, France. 2005:41.

Rose 2005 {published data only}

Rose HJ, White F, Macdonald A, Rutherford PJ, Favre E. Fat intakes of children with PKU on low phenylalanine diets. Journal of Human Nutrition and Dietetics 2005;18(5):395‐400.

SHS 2001 {published data only}

SHS, Royal Hospital for Sick Children (Glasgow), Alder Hey Children's Hospital (Liverpool), Hospital for Sick Children Great Ormond Street (London), Royal Victoria Hospital (Belfast), The Children's Hospital (Sheffield). A multicentre study to evaluate and compare the nutritional adequacy of XP Analog and Lofenelac in the dietary management of infants with phenylketonuria(PKU). XP Analog and XP Analog LCP ‐ a compilation of abstracts2001.

Referencias de los estudios en espera de evaluación

Ir a:

Cleary 2003 {published and unpublished data}

Cleary MA. Large neutral amino acids and phenylketonuria [abstract]. SHS Inborn Errors Review Series. 2003; Vol. Number 13.

MacDonald 2003b {published data only}

MacDonald A, Rylance G, Davies P, Asplin D, Hall SK, Booth IW. Administration of protein substitute and quality of control in phenylketonuria: A randomized study. Journal of Inherited Metabolic Disease 2003;26(4):319‐26.

Marsden 2005 {published data only}

Marsden D, Mulkern R, Young‐Poussaint T, Rohr F, Waisbren S. Large neutral amino acid treatment in adult patients with PKU ‐ a pilot study [abstract]. Society for the Study of Inborn Errors of Metabolism 42nd Annual Symposium; 2005 Sep 6‐9; Paris, France. 2005:22.

MacDonald 2004 {published data only}

MacDonald A, Daly A, Chakrapani A, Rylance G, Asplin D, Hall SK, et al. What is the ideal dose of protein substitute in PKU. SHS Inborn Error Review Series 2004, (14):23.

Acosta 2001

Acosta PB, Yanicelli S. Protocol 1‐Phenylketonuria (PKU). Ross metabolic formula system, nutrition support protocols. 4th Edition. Columbus, Ohio: Ross Products Division, Abbott Laboratories, 2001:1‐32.

Allen 1996

Allen JR, Baur LA, Waters DL, Humphries IR, Allen BJ, Roberts DC, et al. Body protein in prepubertal children with phenylketonuria. European Journal of Clinical Nutrition 1996;50(3):178‐86.

Bickel 1953

Bickel H, Gerrard J, Hickmans EM. Influence of phenylalanine intake on phenylketonuria. Lancet 1953;265(6790):812‐3.

Bickel 1954

Bickel H, Gerrard J, Hickmans EM. The influence of phenylalanine intake on the chemistry and behaviour of a phenylketonuric child. Acta Paediatrica 1954;43:64‐77.

DOH 1991

Department of Health. Dietary Reference Values of Food Energy and Nutrients for the United Kingdom (Report on Health and Social Subjects No.41). London: The Stationery office, 1991.

Gropper 1993

Gropper SS, Gropper DM, Acosta PB. Plasma amino acid response to ingestion of L‐amino acids and whole protein. Journal of Paediatric Gastroenterology and Nutrition 1993;16(2):143‐50.

Higgins 2003

Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta‐analyses. BMJ 2003;327(7414):557‐60.

Hudson 1970

Hudson FP, Mordaunt VL, Leahy I. Evaluation of treatment begun in first three months of life in 184 cases of phenylketonuria. Archives of Disease in Childhood 1970;45(239):5‐12.

Jüni 2001

Jüni P, Altman DG, Egger M. Systematic reviews in health care: Assessing the quality of controlled clinical trials. BMJ 2001;323(7303):42‐6.

MacDonald 1997

MacDonald A, Rylance G, Asplin D, Harris G, Booth IW. Abnormal feeding patterns in PKU. Journal of Human Nutrition and Dietetics 1997;10:163‐70.

MacDonald 1999

MacDonald A. Diet and Phenylketonuria, Birmingham University. PhD thesis1999.

MacDonald 2001

MacDonald A. Phenylketonuria. In: Shaw V, Lawson M editor(s). Clinical Paediatric Dietetics. 2nd Edition. Oxford: Blackwell Science, 2001:235‐67. [ISBN 0‐632‐05241‐4]

MRC 1993

Medical Research Council Working Party on Phenylketonuria. Recommendations on the dietary management of phenylketonuria. Archives of Disease in Childhood 1993;68(3):426‐7.

Murphy 1959

Murphy D. Phenylketonuria from early infancy. Irish Journal of Medical Science 1959;405:425‐7.

NIH 2000

NIH. Phenylketonuria (PKU): Screening and Management. NIH Consensus Statement 2000 October 16‐18; Vol. 17, issue 3:1‐33.

Pietz 1999

Pietz J, Kreis R, Rupp A, Mayatepek E, Rating D, Boesch C, Bremer HJ. Large neutral amino acids block phenylalanine transport into brain tissue in patients with phenylketonuria. Journal of Clinical Investigation 1999;103(8):1169‐78.

Scriver 1995

Scriver CR, Kaufman S, Woo SLC. The hyperphenylalaninaemias. In: Scriver CR, Beaudet AL, Sly WS, Valle D editor(s). The Metabolic and Molecular Bases of Inherited Disease. 7th Edition. New York: McGraw‐Hill, 1995:1015‐76.

Smith 1974

Smith I, Woolf DH. Natural history of phenylketonuria and influence of early treatment. Lancet 1974;2(7880):540‐4.

WHO 1985

WHO (World Health Organization). Energy and Protein Requirements. Report of a joint FAO/WHO/UNU Expert Consultation. Geneva: World Health Organization, 1985; Vol. Technical report series 724.

Woolf 1955

Woolf LI, Griffiths R, Moncrieff A. Treatment of Phenylketonuria with a diet low in phenylalanine. British Medical Journal 1955;1:57‐64.

Characteristics of studies

Characteristics of included studies [ordered by study ID]

Ir a:

Prince 1997

Methods

Randomised controlled parallel trial. Neither participant nor investigator was blinded.

Participants

28 participants, with moderate to severe phenylketonuria, aged 4 ‐ 10 years. All were prescribed full treatment consisting of natural protein restriction and supplementation with a protein substitute.

Interventions

Two‐phase study (phase 1 study length: 2 years; phase 2 study length: 3 years). Only phase 1 was an RCT. This investigates use of a protein substitute with a 50% lower concentration of protein (product contained 10 g protein equivalent to 400 kcal/100 g dry powder) than the control product (PhenylFree, Mead‐Johnson Co., Evansville, IN, USA).

Outcomes

Phase 1:
mean serum concentrations of essential amino acids;
mean amino acid intakes;
mean serum protein concentrations;
height and weight centiles.

Notes

One participant changed groups and was excluded from the final analysis.

Risk of bias

Bias

Authors' judgement

Support for judgement

Allocation concealment (selection bias)

Low risk

A ‐ Adequate

RCT: randomised controlled trial

Characteristics of excluded studies [ordered by study ID]

Ir a:

Study

Reason for exclusion

Acosta 1994

Not an RCT.
Ascertained by communication with current head of metabolic products who contacted the author (her predecessor in post).
Infants fed Analog XP were studied first and Phenex study begun after this.

Baumgartner 2004

Intervention not eligible for inclusion.

Clemens 1991

Not an RCT.

Kalkanoglu 2005

Group of participants not eligible for inclusion.

MacDonald 2003a

Intervention not eligible for inclusion. The trial did not set out to compare two different doses of protein substitute intake, this studied a group of 20 participants with PKU and studied the effects of giving participants at least 40% of their daily protein substitute requirements as tablets for at least 12 weeks. There was better compliance with protein substitute as tablets, therefore, in this trial, different intakes of protein substitute were only measured as outcomes.

MacDonald 2005

Intervention not eligible for inclusion.

Rose 2005

Intervention not eligible for inclusion.

SHS 2001

Not an RCT (ascertained by communication with co‐ordinator of original study at SHS).

RCT: randomised controlled trial

Characteristics of ongoing studies [ordered by study ID]

Ir a:

MacDonald 2004

Trial name or title

What is the ideal dose of protein substitute in PKU?

Methods

Participants

25 children with phenylketonuria.

Interventions

2 doses of protein substitute were compared. Protocol A: 2 g protein/kg/day
Protocol B: 1.2 g protein/kg/day.

Outcomes

Plasma phenylalanine levels.

Starting date

Contact information

Notes

Author completing genotyping before full publication.

Table 1. Characteristics of studies awaiting assessment

Study ID

Methods

Participants

Interventions

Outcomes

Notes

Cleary 2003

RCT.

Investigating 5 adults with PKU on a normal diet using MRS of brain. Entrants had classical PKU diagnosed and treated in the newborn period, at the time of study aged between 22‐37 years.

Treatment group were given a mixture of large neutral amino acids at a dose of 0.81 g amino acids per kg or a similar tasting placebo .This was given over 24 hours in 5 divided doses. MRS was performed at 5 intervals during the 24‐hour period. Participants crossed over after 1 week.

This study has not yet been published in full as author of abstract does not have ownership of the data. Author has agreed to attempt to pursue publication after having been approached by the review authors.

MacDonald 2003

RCT. Cross‐over design.

16 children with PKU.

Initially only 13 participants were entered into the trial and randomised into 3 groups. Intervention was by administering protein substitute at different times and in different doses. Protocol A: protein substitute was administered in 3 equal doses over 10 hours during the day; Protocol B: protein substitute was administered in 3 equal divided doses over a 14‐hour period; Protocol C: protein substitute was administered in 4 equal divided doses over 14‐hour period. Because of interim analysis results, a further 3 participants were entered into the trial. At this stage a new group was introduced ‐ Protocol D and these 3 participants were entered into protocol A, B and D. Protocol D: protein substitute administered in 6 equal divided doses over a 24‐hour period. As 3 participants were introduced after the initial randomisation phase their results cannot be included in the review. At the present time only information from group C can be used as the extra participants did not enter this group.

Individual patient data have been sought from the author. This would enable the 3 extra participants to be identified within groups A and B and D and excluded from the analysis.

Footnotes ‐ MRS: magnetic resonance spectroscopy; PKU: phenylketonuria; RCT: randomised controlled trial

Figuras y tablas -
Table 1. Characteristics of studies awaiting assessment
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