Dietary practices in methylmalonic acidaemia: a European survey

A. Pinto; S. Evans; A. Daly; M.F. Almeida; M. Assoun; A. Belanger-Quintana; S.M. Bernabei; S. Bollhalder; D. Cassiman; H. Champion; H.D. Chan; K. Corthouts; J. Dalmau; F. de Boer; C. De Laet; A. de Meyer; A. Desloovere; A. Dianin; M. Dixon; K. Dokoupil; S. Dubois; F. Eyskens; A. Faria; I. Fasan; E. Favre; F. Feillet; A. Fekete; G. Gallo; C. Gingell; J. Gribben; K.K. Hansen; N. Ter Horst; C. Jankowski; R. Janssen-Regelink; I. Jones; C. Jouault; G.E. Kahrs; I. Kok; A. Kowalik; C. Laguerre; S. Le Verge; A. Liguori; R. Lilje; C. Maddalon; D. Mayr; U. Meyer; A. Micciche; U. Och; M. Robert; J.C. Rocha; Liesbeth van der Ploeg

doi:10.1515/jpem-2019-0277

Dietary practices in methylmalonic acidaemia: a European survey

A. Pinto^*, S. Evans, A. Daly, M.F. Almeida, M. Assoun, A. Belanger-Quintana, S.M. Bernabei, S. Bollhalder, D. Cassiman, H. Champion, H.D. Chan, K. Corthouts, J. Dalmau, F. de Boer, C. De Laet, A. de Meyer, A. Desloovere, A. Dianin, M. Dixon, K. DokoupilS. Dubois, F. Eyskens, A. Faria, I. Fasan, E. Favre, F. Feillet, A. Fekete, G. Gallo, C. Gingell, J. Gribben, K.K. Hansen, N. Ter Horst, C. Jankowski, R. Janssen-Regelink, I. Jones, C. Jouault, G.E. Kahrs, I. Kok, A. Kowalik, C. Laguerre, S. Le Verge, A. Liguori, R. Lilje, C. Maddalon, D. Mayr, U. Meyer, A. Micciche, U. Och, M. Robert, J.C. Rocha, Liesbeth van der Ploeg

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

Background: The dietary management of methylmalonic acidaemia (MMA) is a low-protein diet providing sufficient energy to avoid catabolism and to limit production of methylmalonic acid. The goal is to achieve normal growth, good nutritional status and the maintenance of metabolic stability.Aim: To describe the dietary management of patients with MMA across Europe.Methods: A cross-sectional questionnaire was sent to European colleagues managing inherited metabolic disorders (IMDs) (n = 53) with 27 questions about the nutritional management of organic acidaemias. Data were analysed by different age ranges (0-6 months; 7-12 months; 1-10 years; 11-16 years; >16 years).Results: Questionnaires were returned from 53 centres. Twenty-five centres cared for 80 patients with MMA vitamin B12 responsive (MMA112r) and 43 centres managed 215 patients with MMA vitamin B12 non-responsive (MMAB12nr). For MMAB12r patients, 44% of centres (n=11/25) prescribed natural protein below the World Health Organization/Food and Agriculture Organization/ United Nations University (WHO/FAO/UNU) 2007 safe levels of protein intake in at least one age range. Precursor-free amino acids (PFAA) were prescribed by 40% of centres (10/25) caring for 36% (29/80) of all the patients. For MMAB12nr patients, 72% of centres (n =31/43) prescribed natural protein below the safe levels of protein intake (WHO/FAO/UNU 2007) in at least one age range. PFAA were prescribed by 77% of centres (n= 33/43) managing 81% (n =174/215) of patients. In MMAB12nr patients, 90 (42%) required tube feeding: 25 via a nasogastric tube and 65 via a gastrostomy.Conclusions: A high percentage of centres used PFAA in MMA patients together with a protein prescription that provided less than the safe levels of natural protein intake. However, there was inconsistent practices across Europe. Long-term efficacy studies are needed to study patient outcome when using PFAA with different severities of natural protein restrictions in patients with MMA to guide future practice.

Original language	English
Pages (from-to)	147-155
Number of pages	9
Journal	Journal of Pediatric Endocrinology & Metabolism
Volume	33
Issue number	1
DOIs	https://doi.org/10.1515/jpem-2019-0277
Publication status	Published - 1 Jan 2020

Keywords

acidurias
growth
management
methylmalonic acidaemia
natural protein
organic acidemias
precursor-free amino acids
propionic acidemia
protein-restricted diet
PROPIONIC ACIDEMIA
MANAGEMENT
ACIDURIAS
GROWTH
ORGANIC ACIDEMIAS

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10.1515/jpem-2019-0277

Cite this

Pinto, A., Evans, S., Daly, A., Almeida, M. F., Assoun, M., Belanger-Quintana, A., Bernabei, S. M., Bollhalder, S., Cassiman, D., Champion, H., Chan, H. D., Corthouts, K., Dalmau, J., de Boer, F., De Laet, C., de Meyer, A., Desloovere, A., Dianin, A., Dixon, M., ... van der Ploeg, L. (2020). Dietary practices in methylmalonic acidaemia: a European survey. Journal of Pediatric Endocrinology & Metabolism, 33(1), 147-155. https://doi.org/10.1515/jpem-2019-0277

@article{c4746619ba2147729021c6b8c5db9e89,

title = "Dietary practices in methylmalonic acidaemia: a European survey",

abstract = "Background: The dietary management of methylmalonic acidaemia (MMA) is a low-protein diet providing sufficient energy to avoid catabolism and to limit production of methylmalonic acid. The goal is to achieve normal growth, good nutritional status and the maintenance of metabolic stability.Aim: To describe the dietary management of patients with MMA across Europe.Methods: A cross-sectional questionnaire was sent to European colleagues managing inherited metabolic disorders (IMDs) (n = 53) with 27 questions about the nutritional management of organic acidaemias. Data were analysed by different age ranges (0-6 months; 7-12 months; 1-10 years; 11-16 years; >16 years).Results: Questionnaires were returned from 53 centres. Twenty-five centres cared for 80 patients with MMA vitamin B12 responsive (MMA112r) and 43 centres managed 215 patients with MMA vitamin B12 non-responsive (MMAB12nr). For MMAB12r patients, 44% of centres (n=11/25) prescribed natural protein below the World Health Organization/Food and Agriculture Organization/ United Nations University (WHO/FAO/UNU) 2007 safe levels of protein intake in at least one age range. Precursor-free amino acids (PFAA) were prescribed by 40% of centres (10/25) caring for 36% (29/80) of all the patients. For MMAB12nr patients, 72% of centres (n =31/43) prescribed natural protein below the safe levels of protein intake (WHO/FAO/UNU 2007) in at least one age range. PFAA were prescribed by 77% of centres (n= 33/43) managing 81% (n =174/215) of patients. In MMAB12nr patients, 90 (42%) required tube feeding: 25 via a nasogastric tube and 65 via a gastrostomy.Conclusions: A high percentage of centres used PFAA in MMA patients together with a protein prescription that provided less than the safe levels of natural protein intake. However, there was inconsistent practices across Europe. Long-term efficacy studies are needed to study patient outcome when using PFAA with different severities of natural protein restrictions in patients with MMA to guide future practice.",

keywords = "acidurias, growth, management, methylmalonic acidaemia, natural protein, organic acidemias, precursor-free amino acids, propionic acidemia, protein-restricted diet, PROPIONIC ACIDEMIA, MANAGEMENT, ACIDURIAS, GROWTH, ORGANIC ACIDEMIAS",

author = "A. Pinto and S. Evans and A. Daly and M.F. Almeida and M. Assoun and A. Belanger-Quintana and S.M. Bernabei and S. Bollhalder and D. Cassiman and H. Champion and H.D. Chan and K. Corthouts and J. Dalmau and {de Boer}, F. and {De Laet}, C. and {de Meyer}, A. and A. Desloovere and A. Dianin and M. Dixon and K. Dokoupil and S. Dubois and F. Eyskens and A. Faria and I. Fasan and E. Favre and F. Feillet and A. Fekete and G. Gallo and C. Gingell and J. Gribben and K.K. Hansen and {Ter Horst}, N. and C. Jankowski and R. Janssen-Regelink and I. Jones and C. Jouault and G.E. Kahrs and I. Kok and A. Kowalik and C. Laguerre and {Le Verge}, S. and A. Liguori and R. Lilje and C. Maddalon and D. Mayr and U. Meyer and A. Micciche and U. Och and M. Robert and J.C. Rocha and {van der Ploeg}, Liesbeth",

note = "Funding Information: The authors would like to thank the following people for the assistance in data collection: Louise Robertson (University Hospitals Birmingham NHS Foundation Trust, UK); Sharan Lowry (Sheffield Children{\textquoteright}s Hospital, UK); Rychelle Winstone, Kate Stonstreet and Karen van Wyk (Evelina London Children{\textquoteright}s Hospital, Guy{\textquoteright}s and St Thomas{\textquoteright} NHS Foundation Trust, London, UK); Charlotte Ellerton and Rachel Carruthers (Charles Dent Metabolic Unit National Hospital for Neurology and Surgery, London, UK); Isabelle Nedellec (CHU Angers); Skadi Beblo (Hospital of Children{\textquoteright}s and Adolescents, University of Leipzig, Germany); Anne-Kathrin Neugebauer (Heinrich-Heine-University, Department of General Paediatrics, Neonatology and Paediatric Cardiology, Dusseldorf); Marianne Diels (Metabolic Center, University Hospitals Leuven and KU Leuven, Belgium); Sophie Defouny (H{\^o}pital Universitaire des Enfants, Reine fabiola, Bruxelles, Belgium); Esmeralda Martins and Anabela Bandeira (Unidade de Doen{\c c}as Metab{\'o}licas, Centro Hospitalar do Porto – CHP, Porto, Portugal); Elisa Le{\~a}o Teles and Esmeralda Rodrigues (Centro Hospitalar S{\~a}o Jo{\~a}o – Unidade de Doen{\c c}as Metab{\'o}licas, Porto, Portugal); Mercedes Martinez-Pardo (Unidad de Enfermedades Metabolicas, Servicio de Pediatria, Hospital Ramon y Cajal Madrid, Spain); Jolanta Sykut-Cegielska and Joanna Taybert (Institute of Mother and Child, Poland) and Camilla Caroe and Ann Roskjaer (National University Hospital, Copenhagen, Denmark). We thank the Association “La Vita e{\textquoteright} un Dono” for supporting the fellowship of Giorgia Gallo. We also acknowledge the regional centre for Newborn Screening, Diagnosis and Treatment of Inherited Metabolic Diseases and ministerial endorsement for the participation in the European network for inherited metabolic disorders metabERN. Author contributions: All authors participated in data collection, critical revision of the paper and final approval of the version to be published. Alex Pinto was involved in data quality assessment, data analysis and development of the manuscript. Anita MacDonald was involved in questionnaire development, interpretation of data and writing of the manuscript. Research funding: None declared. Employment or leadership: None declared. Honorarium: None declared. Competing interests: The funding organisation(s) played no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication. Conflict of interest: Alex Pinto received an educational grant from Cambrooke Therapeutics and grants from Vitaflo, Nutricia, Merck Serono, Biomarin and Mevalia to attend scientific meetings. Sharon Evans is a research dietitian funded by Nutricia; financial support from Nutricia and Vitaflo to attend study days and conferences. Anne Daly undertook evaluation work for the nutritional companies – Vitaflo Ltd, Nutricia Ltd and Metax. Anita MacDonald received research funding and honoraria from Nutricia, Vitaflo International and Merck Serono. She is a member of the European Nutrition Expert Panel (Biomarin), member of Sapropterin Advisory Board (Biomarin), member of the Advisory Board entitled ELEMENT (Danone-Nutricia) and member of an Advisory Board for Arla and Applied Pharma Research. Allyson Terry received research honoraria and congress travel allowances from Nutricia and Vitaflo International. Liesbeth van der Ploeg received financial support from Nutricia and Vitaflo to attend conferences. Annemiek van Wegberg received several grants from Nutricia and Vitaflo to visit conferences and received honoraria as a speaker from Excemed and Biomarin. Agnieszka Kowalik received financial support from Nutricia and Vitaflo to attend conferences. Gudrun Elise Kahrs received support from Vitaflo and SHS/Nutricia to attend meetings. Corrie Timmer received financial support from Nutricia and Vitaflo to attend conferences. Manuela Ferreira Almeida received grants from Glutamine, Nutricia, Merck Serono, Biomarin, Orphan and Lifediet to attend congress and for education. J{\'u}lio C{\'e}sar Rocha is member of the European Nutrition Expert Panel (Biomarin), member of an Advisory Board for Applied Pharma Research and Nutricia and received fees from Merck Serono, Vitaflo, Nutricia and Cambrooke. Amaya B{\'e}langer-Quintana, Martine Robert, Esther van Dam, Carina Heidenborg and Margreet van Rijn are members of the European Nutrition Expert Panel (Biomarin). Fiona White received honoraria from Alexion, Nutricia Metabolics and Vitaflo as well as educational and travel grants from Nutricia Metabolics and Vitaflo. Marjorie Dixon received research honoraria and congress travel allowances from Nutricia and Vitaflo International and is a member of the Nutricia Advisory Board. Katharina Dokoupil is a member of the European Nutrition Expert Panel (Biomarin) and member of an Advisory Board (Nestl{\'e}). She received honoraria and symposia travel allowances from Nutricia and Vitaflo. Alice Dianin received financial support from Mevalia and DMF to attend conferences. Carmen Rohde received financial support from MetaX, Nutricia and Vitaflo to attend conferences and honoraria from MetaX and Nutricia. Ulrike Och received honoraria from Biomarin, Dr. Sch{\"a}r medical nutrition, Vitaflo and Nutricia as well as support to attend meetings. Publisher Copyright: {\textcopyright} 2019 Walter de Gruyter GmbH, Berlin/Boston.",

year = "2020",

month = jan,

day = "1",

doi = "10.1515/jpem-2019-0277",

language = "English",

volume = "33",

pages = "147--155",

journal = "Journal of Pediatric Endocrinology & Metabolism",

issn = "0334-018X",

publisher = "De Gruyter",

number = "1",

}

Pinto, A, Evans, S, Daly, A, Almeida, MF, Assoun, M, Belanger-Quintana, A, Bernabei, SM, Bollhalder, S, Cassiman, D, Champion, H, Chan, HD, Corthouts, K, Dalmau, J, de Boer, F, De Laet, C, de Meyer, A, Desloovere, A, Dianin, A, Dixon, M, Dokoupil, K, Dubois, S, Eyskens, F, Faria, A, Fasan, I, Favre, E, Feillet, F, Fekete, A, Gallo, G, Gingell, C, Gribben, J, Hansen, KK, Ter Horst, N, Jankowski, C, Janssen-Regelink, R, Jones, I, Jouault, C, Kahrs, GE, Kok, I, Kowalik, A, Laguerre, C, Le Verge, S, Liguori, A, Lilje, R, Maddalon, C, Mayr, D, Meyer, U, Micciche, A, Och, U, Robert, M, Rocha, JC & van der Ploeg, L 2020, 'Dietary practices in methylmalonic acidaemia: a European survey', Journal of Pediatric Endocrinology & Metabolism, vol. 33, no. 1, pp. 147-155. https://doi.org/10.1515/jpem-2019-0277

TY - JOUR

T1 - Dietary practices in methylmalonic acidaemia: a European survey

AU - Pinto, A.

AU - Evans, S.

AU - Daly, A.

AU - Almeida, M.F.

AU - Assoun, M.

AU - Belanger-Quintana, A.

AU - Bernabei, S.M.

AU - Bollhalder, S.

AU - Cassiman, D.

AU - Champion, H.

AU - Chan, H.D.

AU - Corthouts, K.

AU - Dalmau, J.

AU - de Boer, F.

AU - De Laet, C.

AU - de Meyer, A.

AU - Desloovere, A.

AU - Dianin, A.

AU - Dixon, M.

AU - Dokoupil, K.

AU - Dubois, S.

AU - Eyskens, F.

AU - Faria, A.

AU - Fasan, I.

AU - Favre, E.

AU - Feillet, F.

AU - Fekete, A.

AU - Gallo, G.

AU - Gingell, C.

AU - Gribben, J.

AU - Hansen, K.K.

AU - Ter Horst, N.

AU - Jankowski, C.

AU - Janssen-Regelink, R.

AU - Jones, I.

AU - Jouault, C.

AU - Kahrs, G.E.

AU - Kok, I.

AU - Kowalik, A.

AU - Laguerre, C.

AU - Le Verge, S.

AU - Liguori, A.

AU - Lilje, R.

AU - Maddalon, C.

AU - Mayr, D.

AU - Meyer, U.

AU - Micciche, A.

AU - Och, U.

AU - Robert, M.

AU - Rocha, J.C.

AU - van der Ploeg, Liesbeth

N1 - Funding Information: The authors would like to thank the following people for the assistance in data collection: Louise Robertson (University Hospitals Birmingham NHS Foundation Trust, UK); Sharan Lowry (Sheffield Children’s Hospital, UK); Rychelle Winstone, Kate Stonstreet and Karen van Wyk (Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust, London, UK); Charlotte Ellerton and Rachel Carruthers (Charles Dent Metabolic Unit National Hospital for Neurology and Surgery, London, UK); Isabelle Nedellec (CHU Angers); Skadi Beblo (Hospital of Children’s and Adolescents, University of Leipzig, Germany); Anne-Kathrin Neugebauer (Heinrich-Heine-University, Department of General Paediatrics, Neonatology and Paediatric Cardiology, Dusseldorf); Marianne Diels (Metabolic Center, University Hospitals Leuven and KU Leuven, Belgium); Sophie Defouny (Hôpital Universitaire des Enfants, Reine fabiola, Bruxelles, Belgium); Esmeralda Martins and Anabela Bandeira (Unidade de Doenças Metabólicas, Centro Hospitalar do Porto – CHP, Porto, Portugal); Elisa Leão Teles and Esmeralda Rodrigues (Centro Hospitalar São João – Unidade de Doenças Metabólicas, Porto, Portugal); Mercedes Martinez-Pardo (Unidad de Enfermedades Metabolicas, Servicio de Pediatria, Hospital Ramon y Cajal Madrid, Spain); Jolanta Sykut-Cegielska and Joanna Taybert (Institute of Mother and Child, Poland) and Camilla Caroe and Ann Roskjaer (National University Hospital, Copenhagen, Denmark). We thank the Association “La Vita e’ un Dono” for supporting the fellowship of Giorgia Gallo. We also acknowledge the regional centre for Newborn Screening, Diagnosis and Treatment of Inherited Metabolic Diseases and ministerial endorsement for the participation in the European network for inherited metabolic disorders metabERN. Author contributions: All authors participated in data collection, critical revision of the paper and final approval of the version to be published. Alex Pinto was involved in data quality assessment, data analysis and development of the manuscript. Anita MacDonald was involved in questionnaire development, interpretation of data and writing of the manuscript. Research funding: None declared. Employment or leadership: None declared. Honorarium: None declared. Competing interests: The funding organisation(s) played no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication. Conflict of interest: Alex Pinto received an educational grant from Cambrooke Therapeutics and grants from Vitaflo, Nutricia, Merck Serono, Biomarin and Mevalia to attend scientific meetings. Sharon Evans is a research dietitian funded by Nutricia; financial support from Nutricia and Vitaflo to attend study days and conferences. Anne Daly undertook evaluation work for the nutritional companies – Vitaflo Ltd, Nutricia Ltd and Metax. Anita MacDonald received research funding and honoraria from Nutricia, Vitaflo International and Merck Serono. She is a member of the European Nutrition Expert Panel (Biomarin), member of Sapropterin Advisory Board (Biomarin), member of the Advisory Board entitled ELEMENT (Danone-Nutricia) and member of an Advisory Board for Arla and Applied Pharma Research. Allyson Terry received research honoraria and congress travel allowances from Nutricia and Vitaflo International. Liesbeth van der Ploeg received financial support from Nutricia and Vitaflo to attend conferences. Annemiek van Wegberg received several grants from Nutricia and Vitaflo to visit conferences and received honoraria as a speaker from Excemed and Biomarin. Agnieszka Kowalik received financial support from Nutricia and Vitaflo to attend conferences. Gudrun Elise Kahrs received support from Vitaflo and SHS/Nutricia to attend meetings. Corrie Timmer received financial support from Nutricia and Vitaflo to attend conferences. Manuela Ferreira Almeida received grants from Glutamine, Nutricia, Merck Serono, Biomarin, Orphan and Lifediet to attend congress and for education. Júlio César Rocha is member of the European Nutrition Expert Panel (Biomarin), member of an Advisory Board for Applied Pharma Research and Nutricia and received fees from Merck Serono, Vitaflo, Nutricia and Cambrooke. Amaya Bélanger-Quintana, Martine Robert, Esther van Dam, Carina Heidenborg and Margreet van Rijn are members of the European Nutrition Expert Panel (Biomarin). Fiona White received honoraria from Alexion, Nutricia Metabolics and Vitaflo as well as educational and travel grants from Nutricia Metabolics and Vitaflo. Marjorie Dixon received research honoraria and congress travel allowances from Nutricia and Vitaflo International and is a member of the Nutricia Advisory Board. Katharina Dokoupil is a member of the European Nutrition Expert Panel (Biomarin) and member of an Advisory Board (Nestlé). She received honoraria and symposia travel allowances from Nutricia and Vitaflo. Alice Dianin received financial support from Mevalia and DMF to attend conferences. Carmen Rohde received financial support from MetaX, Nutricia and Vitaflo to attend conferences and honoraria from MetaX and Nutricia. Ulrike Och received honoraria from Biomarin, Dr. Schär medical nutrition, Vitaflo and Nutricia as well as support to attend meetings. Publisher Copyright: © 2019 Walter de Gruyter GmbH, Berlin/Boston.

PY - 2020/1/1

Y1 - 2020/1/1

N2 - Background: The dietary management of methylmalonic acidaemia (MMA) is a low-protein diet providing sufficient energy to avoid catabolism and to limit production of methylmalonic acid. The goal is to achieve normal growth, good nutritional status and the maintenance of metabolic stability.Aim: To describe the dietary management of patients with MMA across Europe.Methods: A cross-sectional questionnaire was sent to European colleagues managing inherited metabolic disorders (IMDs) (n = 53) with 27 questions about the nutritional management of organic acidaemias. Data were analysed by different age ranges (0-6 months; 7-12 months; 1-10 years; 11-16 years; >16 years).Results: Questionnaires were returned from 53 centres. Twenty-five centres cared for 80 patients with MMA vitamin B12 responsive (MMA112r) and 43 centres managed 215 patients with MMA vitamin B12 non-responsive (MMAB12nr). For MMAB12r patients, 44% of centres (n=11/25) prescribed natural protein below the World Health Organization/Food and Agriculture Organization/ United Nations University (WHO/FAO/UNU) 2007 safe levels of protein intake in at least one age range. Precursor-free amino acids (PFAA) were prescribed by 40% of centres (10/25) caring for 36% (29/80) of all the patients. For MMAB12nr patients, 72% of centres (n =31/43) prescribed natural protein below the safe levels of protein intake (WHO/FAO/UNU 2007) in at least one age range. PFAA were prescribed by 77% of centres (n= 33/43) managing 81% (n =174/215) of patients. In MMAB12nr patients, 90 (42%) required tube feeding: 25 via a nasogastric tube and 65 via a gastrostomy.Conclusions: A high percentage of centres used PFAA in MMA patients together with a protein prescription that provided less than the safe levels of natural protein intake. However, there was inconsistent practices across Europe. Long-term efficacy studies are needed to study patient outcome when using PFAA with different severities of natural protein restrictions in patients with MMA to guide future practice.

AB - Background: The dietary management of methylmalonic acidaemia (MMA) is a low-protein diet providing sufficient energy to avoid catabolism and to limit production of methylmalonic acid. The goal is to achieve normal growth, good nutritional status and the maintenance of metabolic stability.Aim: To describe the dietary management of patients with MMA across Europe.Methods: A cross-sectional questionnaire was sent to European colleagues managing inherited metabolic disorders (IMDs) (n = 53) with 27 questions about the nutritional management of organic acidaemias. Data were analysed by different age ranges (0-6 months; 7-12 months; 1-10 years; 11-16 years; >16 years).Results: Questionnaires were returned from 53 centres. Twenty-five centres cared for 80 patients with MMA vitamin B12 responsive (MMA112r) and 43 centres managed 215 patients with MMA vitamin B12 non-responsive (MMAB12nr). For MMAB12r patients, 44% of centres (n=11/25) prescribed natural protein below the World Health Organization/Food and Agriculture Organization/ United Nations University (WHO/FAO/UNU) 2007 safe levels of protein intake in at least one age range. Precursor-free amino acids (PFAA) were prescribed by 40% of centres (10/25) caring for 36% (29/80) of all the patients. For MMAB12nr patients, 72% of centres (n =31/43) prescribed natural protein below the safe levels of protein intake (WHO/FAO/UNU 2007) in at least one age range. PFAA were prescribed by 77% of centres (n= 33/43) managing 81% (n =174/215) of patients. In MMAB12nr patients, 90 (42%) required tube feeding: 25 via a nasogastric tube and 65 via a gastrostomy.Conclusions: A high percentage of centres used PFAA in MMA patients together with a protein prescription that provided less than the safe levels of natural protein intake. However, there was inconsistent practices across Europe. Long-term efficacy studies are needed to study patient outcome when using PFAA with different severities of natural protein restrictions in patients with MMA to guide future practice.

KW - acidurias

KW - growth

KW - management

KW - methylmalonic acidaemia

KW - natural protein

KW - organic acidemias

KW - precursor-free amino acids

KW - propionic acidemia

KW - protein-restricted diet

KW - PROPIONIC ACIDEMIA

KW - MANAGEMENT

KW - ACIDURIAS

KW - GROWTH

KW - ORGANIC ACIDEMIAS

U2 - 10.1515/jpem-2019-0277

DO - 10.1515/jpem-2019-0277

M3 - Article

C2 - 31846426

SN - 0334-018X

VL - 33

SP - 147

EP - 155

JO - Journal of Pediatric Endocrinology & Metabolism

JF - Journal of Pediatric Endocrinology & Metabolism

IS - 1

ER -