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Deficiency of phosphoglycerate kinase

MedGen UID:
146886
Concept ID:
C0684324
Disease or Syndrome
Synonym: Phosphoglycerate Kinase Deficiency
SNOMED CT: Deficiency of phosphoglycerate kinase (124335006)

Definition

Phosphoglycerate kinase deficiency is a genetic disorder that affects the body's ability to break down the simple sugar glucose, which is the primary energy source for most cells. Researchers have described two major forms of the condition. The most common form is sometimes called the hemolytic form. It is characterized by a condition known as chronic hemolytic anemia, in which red blood cells are broken down (undergo hemolysis) prematurely. Chronic hemolytic anemia can lead to unusually pale skin (pallor), yellowing of the eyes and skin (jaundice), fatigue, shortness of breath, and a rapid heart rate. Some people with the hemolytic form also have symptoms related to abnormal brain function, including intellectual disability, seizures, and stroke.

The other form of phosphoglycerate kinase deficiency is often called the myopathic form. It primarily affects muscles, causing progressive weakness, pain, and cramping, particularly with exercise. During exercise, muscle tissue can be broken down, releasing a protein called myoglobin. This protein is processed by the kidneys and released in the urine (myoglobinuria). If untreated, myoglobinuria can lead to kidney failure.

Most people with phosphoglycerate kinase deficiency have either the hemolytic form or the myopathic form. However, other combinations of signs and symptoms (such as muscle weakness with neurologic symptoms) have also been reported. [from MedlinePlus Genetics]

Professional guidelines

Recent clinical studies

Etiology

Fattizzo B, Giannotta JA, Cecchi N, Barcellini W
Orphanet J Rare Dis 2021 Oct 9;16(1):415. doi: 10.1186/s13023-021-02036-4. PMID: 34627331Free PMC Article
Echaniz-Laguna A, Nadjar Y, Béhin A, Biancalana V, Piraud M, Malfatti E, Laforêt P
J Inherit Metab Dis 2019 Sep;42(5):803-808. Epub 2019 Apr 8 doi: 10.1002/jimd.12087. PMID: 30887539
Zehavi Y, Mandel H, Eran A, Ravid S, Abu Rashid M, Jansen EEW, Wamelink MMC, Saada A, Shaag A, Elpeleg O, Spiegel R
Metab Brain Dis 2019 Apr;34(2):557-563. Epub 2019 Jan 12 doi: 10.1007/s11011-019-0384-x. PMID: 30637540
Sun J, Wang Y, Yang J, Du D, Li Z, Wei J, Yang A
Exp Mol Med 2012 Nov 30;44(11):674-83. doi: 10.3858/emm.2012.44.11.076. PMID: 22990115Free PMC Article
Berardo A, DiMauro S, Hirano M
Curr Neurol Neurosci Rep 2010 Mar;10(2):118-26. doi: 10.1007/s11910-010-0096-4. PMID: 20425236Free PMC Article

Diagnosis

Beecher G, Fleming MD, Liewluck T
Muscle Nerve 2022 Apr;65(4):374-390. Epub 2022 Jan 5 doi: 10.1002/mus.27474. PMID: 34985130
Di Lazzaro G, Magrinelli F, Estevez-Fraga C, Valente EM, Pisani A, Bhatia KP
Mov Disord 2021 Jul;36(7):1511-1525. Epub 2021 May 7 doi: 10.1002/mds.28565. PMID: 33960519
Tein I
Semin Perinatol 1999 Apr;23(2):125-51. doi: 10.1016/s0146-0005(99)80046-9. PMID: 10331465
Tein I
Semin Pediatr Neurol 1996 Jun;3(2):59-98. doi: 10.1016/s1071-9091(96)80038-6. PMID: 8795843
May A, Fitzsimons E
Baillieres Clin Haematol 1994 Dec;7(4):851-79. doi: 10.1016/s0950-3536(05)80128-3. PMID: 7881157

Therapy

Siddique AHH, Kale PP
Rev Neurol (Paris) 2024 Oct;180(8):736-753. Epub 2023 Nov 30 doi: 10.1016/j.neurol.2023.08.011. PMID: 38040547
Han XY, Wang YN, Dou DQ
Chin J Nat Med 2018 Dec;16(12):936-945. doi: 10.1016/S1875-5364(18)30135-3. PMID: 30595218
Baba S, Kobayashi A, Yokoyama H, Moriyama K, Kashimada A, Oyama J, Owada A, Oyama S, Morio T, Takagi M
Brain Dev 2018 Feb;40(2):150-154. Epub 2017 Aug 8 doi: 10.1016/j.braindev.2017.07.009. PMID: 28801086
Lach B, Cupler EJ
J Child Neurol 2008 Jun;23(6):614-9. Epub 2008 Feb 15 doi: 10.1177/0883073807312370. PMID: 18281624
May A, Fitzsimons E
Baillieres Clin Haematol 1994 Dec;7(4):851-79. doi: 10.1016/s0950-3536(05)80128-3. PMID: 7881157

Prognosis

Ma Q, Zeng Q, Wang K, Qian M, Li J, Wang H, Zhang H, Jiang J, Chen Z, Huang W
Int J Mol Sci 2024 Jan 15;25(2) doi: 10.3390/ijms25021057. PMID: 38256128Free PMC Article
Yun HJ, Li M, Guo D, Jeon SM, Park SH, Lim JS, Lee SB, Liu R, Du L, Kim SH, Shin TH, Eyun SI, Park YY, Lu Z, Lee JH
J Exp Clin Cancer Res 2023 Dec 15;42(1):340. doi: 10.1186/s13046-023-02927-3. PMID: 38098117Free PMC Article
Ding MJ, Fang HR, Zhang JK, Shi JH, Yu X, Wen PH, Wang ZH, Cao SL, Zhang Y, Shi XY, Zhang HP, He YT, Yan B, Tang HW, Guo DF, Gao J, Liu Z, Zhang L, Zhang SJ, Zhang XJ, Guo WZ
Hepatology 2022 Jul;76(1):94-111. Epub 2022 Jan 23 doi: 10.1002/hep.32226. PMID: 34735734Free PMC Article
Fattizzo B, Giannotta JA, Cecchi N, Barcellini W
Orphanet J Rare Dis 2021 Oct 9;16(1):415. doi: 10.1186/s13023-021-02036-4. PMID: 34627331Free PMC Article
Qian X, Li X, Cai Q, Zhang C, Yu Q, Jiang Y, Lee JH, Hawke D, Wang Y, Xia Y, Zheng Y, Jiang BH, Liu DX, Jiang T, Lu Z
Mol Cell 2017 Mar 2;65(5):917-931.e6. Epub 2017 Feb 23 doi: 10.1016/j.molcel.2017.01.027. PMID: 28238651Free PMC Article

Clinical prediction guides

Shimizu J, Kasai T, Yoshida H, Huynh AM, Nakao-Azuma Y, Shinomoto M, Tokuda T, Mizuno T, Yamaguchi M
Neurochem Int 2020 Oct;139:104816. Epub 2020 Aug 3 doi: 10.1016/j.neuint.2020.104816. PMID: 32758590
Guin D, Gelman H, Wang Y, Gruebele M
PLoS One 2019;14(9):e0222990. Epub 2019 Sep 26 doi: 10.1371/journal.pone.0222990. PMID: 31557226Free PMC Article
Echaniz-Laguna A, Nadjar Y, Béhin A, Biancalana V, Piraud M, Malfatti E, Laforêt P
J Inherit Metab Dis 2019 Sep;42(5):803-808. Epub 2019 Apr 8 doi: 10.1002/jimd.12087. PMID: 30887539
Chiarelli LR, Morera SM, Bianchi P, Fermo E, Zanella A, Galizzi A, Valentini G
PLoS One 2012;7(2):e32065. Epub 2012 Feb 14 doi: 10.1371/journal.pone.0032065. PMID: 22348148Free PMC Article
DiMauro S, Spiegel R
Acta Myol 2011 Oct;30(2):96-102. PMID: 22106711Free PMC Article

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