Journal of Clinical Pediatrics ›› 2023, Vol. 41 ›› Issue (6): 411-416.doi: 10.12372/jcp.2022.23e0263
• Expert Review • Previous Articles Next Articles
XU Wei
Received:
2023-04-04
Online:
2023-06-15
Published:
2023-06-12
XU Wei. Carbohydrate metabolic emergencies in inborn errors of metabolism[J].Journal of Clinical Pediatrics, 2023, 41(6): 411-416.
[1] |
Saudubray JM, Garcia-Cazorla À. Inborn errors of metabolism overview: pathophysiology, manifestations, evaluation, and management[J]. Pediatr Clin North Am, 2018, 65(2): 179-208.
doi: 10.1016/j.pcl.2017.11.002 |
[2] |
Romão A, Simon PEA, Góes JEC, et al. Initial clinical presentation in cases of inborn errors of metabolism in a reference children's hospital: still a diagnostic challenge[J]. Rev Paul Pediatr, 2017, 35(3): 258-264.
doi: 10.1590/1984-0462/;2017;35;3;00012 |
[3] | Stone WL, Basit H, Adil A. Glycogen storage disease[M]. StatPearls. Treasure Island (FL): StatPearls Publishing Copyright © 2023, StatPearls Publishing LLC, 2023. |
[4] | Lak R, Yazdizadeh B, Davari M, et al. Newborn screening for galactosaemia[J]. Cochrane Database Syst Rev, 2017, 12(12): CD012272. |
[5] |
Succoio M, Sacchettini R, Rossi A, et al. Galactosemia: biochemistry, molecular genetics, newborn screening, and Treatment[J]. Biomolecules, 2022, 12(7): 968.
doi: 10.3390/biom12070968 |
[6] |
Oldfors A, DiMauro S. New insights in the field of muscle glycogenoses[J]. Curr Opin Neurol, 2013, 26(5): 544-553.
doi: 10.1097/WCO.0b013e328364dbdc pmid: 23995275 |
[7] |
Charles J, Pollack A, Miller G. Cardiomyopathy[J]. Aust Fam Physician, 2014, 43(5): 253.
pmid: 24791762 |
[8] |
Vasilescu C, Ojala TH, Brilhante V, et al. Genetic basis of severe childhood-onset cardiomyopathies[J]. J Am Coll Cardiol, 2018, 72(19): 2324-2338.
doi: S0735-1097(18)38436-5 pmid: 30384889 |
[9] | Tarnopolsky MA. Metabolic myopathies[J]. Continuum (Minneapolis, Minn), 2022, 28(6): 1752-1777. |
[10] |
Berardo A, DiMauro S, Hirano M. A diagnostic algorithm for metabolic myopathies[J]. Curr Neurol Neurosci Rep, 2010, 10(2): 118-126.
doi: 10.1007/s11910-010-0096-4 |
[11] | Haller RG, Vissing J. Spontaneous “second wind” and glucose-induced second "second wind" in McArdle disease: oxidative mechanisms[J]. Arch Neurol, 2002, 59(9): 1395-1402. |
[12] |
Haller RG, Vissing J. No spontaneous second wind in muscle phosphofructokinase deficiency[J]. Neurology, 2004, 62(1): 82-86.
pmid: 14718702 |
[13] |
Haller RG, Lewis SF. Glucose-induced exertional fatigue in muscle phosphofructokinase deficiency[J]. N Engl J Med, 1991, 324(6): 364-369.
doi: 10.1056/NEJM199102073240603 |
[14] |
Finsterer J. An update on diagnosis and therapy of metabolic myopathies[J]. Expert Rev Neurother, 2018, 18(12): 933-943.
doi: 10.1080/14737175.2018.1550360 pmid: 30479175 |
[15] |
Lilleker JB, Keh YS, Roncaroli F, et al. Metabolic myopathies: a practical approach[J]. Pract Neurol, 2018, 18(1): 14-26.
doi: 10.1136/practneurol-2017-001708 pmid: 29223996 |
[16] |
Sharma S, Prasad AN. Inborn errors of metabolism and epilepsy: current understanding, diagnosis, and treatment approaches[J]. Int J Mol Sci, 2017, 18(7):1384.
doi: 10.3390/ijms18071384 |
[17] |
Calvo M, Artuch R, Macià E, et al. Diagnostic approach to inborn errors of metabolism in an emergency unit[J]. Pediatr Emerg Care, 2000, 16(6): 405-408.
doi: 10.1097/00006565-200012000-00006 pmid: 11138882 |
[18] |
Gold NB, Kritzer A, Weiner DL, et al. Emergency laboratory evaluations for patients with inborn errors of metabolism[J]. Pediatr Emerg Care, 2021, 37(12): e1154-e1159.
doi: 10.1097/PEC.0000000000001936 |
[19] |
Lord K, Radcliffe J, Gallagher PR, et al. High risk of diabetes and neurobehavioral deficits in individuals with surgically treated hyperinsulinism[J]. J Clin Endocrinol Metab, 2015, 100(11): 4133-4139.
doi: 10.1210/jc.2015-2539 pmid: 26327482 |
[20] |
Winchester B, Bali D, Bodamer OA, et al. Methods for a prompt and reliable laboratory diagnosis of pompe disease: report from an international consensus meeting[J]. Mol Genet Metab, 2008, 93(3): 275-281.
doi: 10.1016/j.ymgme.2007.09.006 pmid: 18078773 |
[21] |
Squires JE, McKiernan PJ, Squires RH. Acute liver dysfunction criteria in critically ill children: The PODIUM consensus conference[J]. Pediatrics, 2022, 149(1 Suppl 1): s59-s65.
doi: 10.1542/peds.2021-052888I pmid: 34970684 |
[22] | Lopez MD. Textbook of pediatric emergency medicine[M]. 5th edition. Lippincott Williams & Wilkins, 2005. |
[23] |
Wappner RS. Biochemical diagnosis of genetic diseases[J]. Pediatr Ann, 1993, 22(5): 282-292.
pmid: 8510995 |
[24] |
Cossu M, Pintus R, Zaffanello M, et al. Metabolomic studies in inborn errors of metabolism: last years and future perspectives[J]. Metabolites, 2023, 13(3):447.
doi: 10.3390/metabo13030447 |
[25] |
Squires JE, Alonso EM, Ibrahim SH, et al. North american society for pediatric gastroenterology, hepatology, and nutrition position paper on the diagnosis and management of pediatric acute liver failure[J]. J Pediatr Gastroenterol Nutr, 2022, 74(1): 138-158.
doi: 10.1097/MPG.0000000000003268 |
[26] |
Hamed A, Hamed A, Lambert K. Branched-chain amino acids for people with hepatic encephalopathy[J]. Am Fam Physician, 2020, 101(1): Online. doi: 10.1002/14651858.
doi: 10.1002/14651858 |
[27] |
Jain V, Dhawan A. Extracorporeal liver support systems in paediatric liver failure[J]. J Pediatr Gastroenterol Nutr, 2017, 64(6): 855-863.
doi: 10.1097/MPG.0000000000001500 |
[28] |
Squires JE, Rudnick DA, Hardison RM, et al. Liver transplant listing in pediatric acute liver failure: practices and participant characteristics[J]. Hepatology (Baltimore, Md), 2018, 68(6): 2338-2347.
doi: 10.1002/hep.30116 |
[29] |
Ridel KR, Leslie ND, Gilbert DL. An updated review of the long-term neurological effects of galactosemia[J]. Pediatr Neurol, 2005, 33(3): 153-161.
pmid: 16087312 |
[30] | Chien YH, Tsai WH, Chang CL, et al. Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset pompe disease: evidence from real-world experiences[J]. Mol Genet Metab Rep, 2020, 23: 100591. |
[31] |
Poelman E, van den Dorpel JJA, Hoogeveen-Westerveld M, et al. Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long-term clinical outcome of classic infantile Pompe patients[J]. J Inherit Metab Dis, 2020, 43(6): 1243-1253.
doi: 10.1002/jimd.v43.6 |
[32] |
Manwaring V, Prunty H, Bainbridge K, et al. Urine analysis of glucose tetrasaccharide by HPLC; a useful marker for the investigation of patients with Pompe and other glycogen storage diseases[J]. J Inherit Metab Dis, 2012, 35(2): 311-316.
doi: 10.1007/s10545-011-9360-2 pmid: 21687968 |
[33] |
Nicolino M, Byrne B, Wraith JE, et al. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced pompe disease[J]. Genet Med, 2009, 11(3): 210-219.
doi: 10.1097/GIM.0b013e31819d0996 pmid: 19287243 |
[34] |
Burton BK, Kronn DF, Hwu WL, et al. The initial evaluation of patients after positive newborn screening: recommended algorithms leading to a confirmed diagnosis of pompe disease[J]. Pediatrics, 2017, 140(Suppl 1): s14-s23.
doi: 10.1542/peds.2016-0280D |
[35] |
Kishnani PS, Sun B, Koeberl DD. Gene therapy for glycogen storage diseases[J]. Hum Mol Genet, 2019, 28(R1): r31-r41.
doi: 10.1093/hmg/ddz133 |
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