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
Published:
2023-06-15
Online:
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 |
[1] | LUO Mingjing, YU Jiaming, WANG Xiaodong, ZHANG Xiaoling, YU Yue, ZHANG Yu, WEN Feiqiu, LIU Sixi. Clinical analysis of invasive fungal disease secondary to allogeneic hematopoietic stem cell transplantation in 424 children with thalassemia [J]. Journal of Clinical Pediatrics, 2025, 43(1): 21-28. |
[2] | LIU Dongxia, JIN Rong, LIN Rongjun. Risk factors analysis of severe refractory Mycoplasma pneumoniae pneumonia complicated with bronchitis obliterans in children [J]. Journal of Clinical Pediatrics, 2025, 43(1): 29-34. |
[3] | ZHONG Jinhong, WANG Can, CHEN Fang. Progress in the research of infantile fiberoptic bronchoscopy sedation [J]. Journal of Clinical Pediatrics, 2025, 43(1): 50-55. |
[4] | JIANG Weiqin, WANG Jing, CHENG Anna, CHEN Tingting, HUANG Yujuan. Predictors of recurrent febrile seizures during the same febrile illness in children with febrile seizures [J]. Journal of Clinical Pediatrics, 2025, 43(1): 8-13. |
[5] | QIU Xiu, WEI Dongmei, LIN Shanshan, XIA Huimin, ZHOU Wenhao. Principles and practice of the Born in Guangzhou Cohort Study [J]. Journal of Clinical Pediatrics, 2024, 42(9): 747-752. |
[6] | FAN Jianxia. The origins and development of the healthy life trajectory program: a cohort of community-family-mother-child multidimensional interventions for overweight and obesity in children [J]. Journal of Clinical Pediatrics, 2024, 42(9): 768-773. |
[7] | JIANG Tao, LI Shuangjie, TANG Lian, OUYANG Wenxian. Immunobiological properties of peripheral blood MAIT cells in children with chronic hepatitis B [J]. Journal of Clinical Pediatrics, 2024, 42(9): 787-790. |
[8] | ZHOU Jie, LIU Keqiang, WANG Jinling, WANG Ying. Megacystis-microcolon-intestinal hypoperistalsis syndrome caused by MYH11 elongating mutation : a case report and literatures review [J]. Journal of Clinical Pediatrics, 2024, 42(9): 798-804. |
[9] | CHU Sijia, TANG Jihong. Research progress of central nervous system injury associated with pediatric acute lymphoblastic leukemia and its treatment [J]. Journal of Clinical Pediatrics, 2024, 42(9): 811-816. |
[10] | DING Yaping, XIA Shanshan, ZHANG Chenmei. Interpretation of “2023 Children’s Renal Nutrition Working Group Clinical Practice Recommendations: Nutritional Management of Children with Acute Kidney Injury” [J]. Journal of Clinical Pediatrics, 2024, 42(8): 667-672. |
[11] | LI Yirong, LI Huiping, GAO Jingyu, XIAO Yuhua, CHEN Xiaomin, LU Yanling, ZHAO Nana, FENG Xiaoqin. Comparison of different doses of cytarabine for induction chemotherapy in children with acute myeloid leukemia in FLAG-IDA regimen [J]. Journal of Clinical Pediatrics, 2024, 42(8): 673-677. |
[12] | HUANG Bo, DONG Yanying, SONG Linlan. Clinical characteristics of 348 children with infectious mononucleosis [J]. Journal of Clinical Pediatrics, 2024, 42(8): 678-683. |
[13] | WANG Dan, SHAO Jingbo, LI Hong, ZHANG Na, ZHU Jiashi, FU Pan, WANG Zhen. Clinical analysis of 38 cases of hematological malignancies complicated with tumor lysis syndrome in children [J]. Journal of Clinical Pediatrics, 2024, 42(8): 684-690. |
[14] | MA Yan, WEI Xingjiao, BAI Hua, ZHANG Yan, TIAN Xinmin, Aqsa Ahmad, LIANG Lijun. Analysis of etiological composition and clinical features of stage 5 chronic kidney disease in children in a tertiary hospital in western China [J]. Journal of Clinical Pediatrics, 2024, 42(8): 697-703. |
[15] | WANG Ye, ZHANG Linlin, CHI Zuofei, SUN Ruowen, JIANG Zehui, XU Gang. A case of clinical report of T-lymphoblastic lymphoma secondary to acute promyelocytic leukemia in children [J]. Journal of Clinical Pediatrics, 2024, 42(8): 722-727. |
|