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Brain malformation

MedGen UID:
82742
Concept ID:
C0266449
Congenital Abnormality
Synonym: Brain malformations
SNOMED CT: Congenital brain anomaly (57148006); Congenital anomaly of brain (57148006); Deformity of brain (57148006)
 
Orphanet: ORPHA199633

Definition

Most brain malformations begin long before a baby is born. Something damages the developing nervous system or causes it to develop abnormally. Sometimes it''s a genetic problem. In other cases, exposure to certain medicines, infections, or radiation during pregnancy interferes with brain development. Parts of the brain may be missing, abnormally small or large, or not fully developed.CHAR(13) Treatment depends upon the problem. In many cases, treatment only helps with symptoms. It may include antiseizure medicines, shunts to drain fluid from the brain, and physical therapy.CHAR(13) There are head malformations that do not involve the brain. Craniofacial disorders are the result of abnormal growth of soft tissue and bones in the face and head. It''s common for new babies to have slightly uneven heads, but parents should watch the shape of their baby''s head for possible problems.CHAR(13) NIH: National Institute of Neurological Disorders and StrokeCHAR(13) [from MedlinePlus]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVBrain malformation

Professional guidelines

PubMed

Expert Panel on Pediatric Imaging, Radhakrishnan R, Shea LAG, Pruthi S, Silvera VM, Bosemani T, Desai NK, Gilbert DL, Glenn OA, Guimaraes CV, Ho ML, Lam HFS, Maheshwari M, Mirsky DM, Nadel HR, Partap S, Schooler GR, Udayasankar UK, Whitehead MT, Wright JN, Rigsby CK
J Am Coll Radiol 2022 Nov;19(11S):S240-S255. doi: 10.1016/j.jacr.2022.09.010. PMID: 36436955
Mercier S, Dubourg C, Belleguic M, Pasquier L, Loget P, Lucas J, Bendavid C, Odent S
Am J Med Genet C Semin Med Genet 2010 Feb 15;154C(1):191-6. doi: 10.1002/ajmg.c.30246. PMID: 20104616
Hahn JS, Plawner LL
Pediatr Neurol 2004 Aug;31(2):79-88. doi: 10.1016/j.pediatrneurol.2004.03.006. PMID: 15301825

Recent clinical studies

Etiology

Lai A, Soucy A, El Achkar CM, Barkovich AJ, Cao Y, DiStefano M, Evenson M, Guerrini R, Knight D, Lee YS, Mefford HC, Miller DT, Mirzaa G, Mochida G, Rodan LH, Patel M, Smith L, Spencer S, Walsh CA, Yang E, Yuskaitis CJ, Yu T, Poduri A; ClinGen Brain Malformation Variant Curation Expert Panel
Genet Med 2022 Nov;24(11):2240-2248. Epub 2022 Aug 23 doi: 10.1016/j.gim.2022.07.020. PMID: 35997716Free PMC Article
Fischer-Zirnsak B, Segebrecht L, Schubach M, Charles P, Alderman E, Brown K, Cadieux-Dion M, Cartwright T, Chen Y, Costin C, Fehr S, Fitzgerald KM, Fleming E, Foss K, Ha T, Hildebrand G, Horn D, Liu S, Marco EJ, McDonald M, McWalter K, Race S, Rush ET, Si Y, Saunders C, Slavotinek A, Stockler-Ipsiroglu S, Telegrafi A, Thiffault I, Torti E, Tsai AC, Wang X, Zafar M, Keren B, Kornak U, Boerkoel CF, Mirzaa G, Ehmke N
Am J Hum Genet 2019 Sep 5;105(3):631-639. Epub 2019 Jul 25 doi: 10.1016/j.ajhg.2019.07.002. PMID: 31353024Free PMC Article
Copp AJ, Adzick NS, Chitty LS, Fletcher JM, Holmbeck GN, Shaw GM
Nat Rev Dis Primers 2015 Apr 30;1:15007. doi: 10.1038/nrdp.2015.7. PMID: 27189655Free PMC Article
Jin Z, Luo R, Piao X
Prog Mol Biol Transl Sci 2009;89:1-13. Epub 2009 Oct 7 doi: 10.1016/S1877-1173(09)89001-7. PMID: 20374731
van Reeuwijk J, Brunner HG, van Bokhoven H
Clin Genet 2005 Apr;67(4):281-9. doi: 10.1111/j.1399-0004.2004.00368.x. PMID: 15733261

Diagnosis

Hung SC, Dahmoush H, Lee HJ, Chen HC, Guimaraes CV
Magn Reson Imaging Clin N Am 2024 Aug;32(3):395-412. Epub 2024 Apr 23 doi: 10.1016/j.mric.2024.03.002. PMID: 38944430
Nagaraj UD, Lawrence A, Vezina LG, Bulas DI, duPlessis AJ
Pediatr Radiol 2016 Jan;46(1):145-7. Epub 2015 Aug 11 doi: 10.1007/s00247-015-3440-7. PMID: 26260203
Copp AJ, Adzick NS, Chitty LS, Fletcher JM, Holmbeck GN, Shaw GM
Nat Rev Dis Primers 2015 Apr 30;1:15007. doi: 10.1038/nrdp.2015.7. PMID: 27189655Free PMC Article
James G, Shanmuganathan M, Harkness W
Childs Nerv Syst 2014 Sep;30(9):1617-9. Epub 2014 Mar 4 doi: 10.1007/s00381-014-2392-9. PMID: 24589886
Hilburger AC, Willis JK, Bouldin E, Henderson-Tilton A
Brain Dev 1993 May-Jun;15(3):234-6. doi: 10.1016/0387-7604(93)90072-g. PMID: 8214352

Therapy

Goel K, Phillips HW, Chen JS, Ngo J, Edmonds B, Ha PX, Wang A, Weil A, Russell BE, Salamon N, Nariai H, Fallah A
Epilepsia 2024 Jan;65(1):57-72. Epub 2023 Nov 11 doi: 10.1111/epi.17807. PMID: 37873610
Tabarki B, AlHashem A, Tlili-Graiess K
Pediatr Neurol 2018 Dec;89:73-74. Epub 2018 Aug 4 doi: 10.1016/j.pediatrneurol.2018.07.012. PMID: 30442439
Hackmann T, Skidmore DL, MacManus B
A A Case Rep 2017 Oct 15;9(8):244-247. doi: 10.1213/XAA.0000000000000577. PMID: 28604469
Copp AJ, Adzick NS, Chitty LS, Fletcher JM, Holmbeck GN, Shaw GM
Nat Rev Dis Primers 2015 Apr 30;1:15007. doi: 10.1038/nrdp.2015.7. PMID: 27189655Free PMC Article
Häberle J, Görg B, Toutain A, Rutsch F, Benoist JF, Gelot A, Suc AL, Koch HG, Schliess F, Häussinger D
J Inherit Metab Dis 2006 Apr-Jun;29(2-3):352-8. doi: 10.1007/s10545-006-0256-5. PMID: 16763901

Prognosis

Stephenson SEM, Costain G, Blok LER, Silk MA, Nguyen TB, Dong X, Alhuzaimi DE, Dowling JJ, Walker S, Amburgey K, Hayeems RZ, Rodan LH, Schwartz MA, Picker J, Lynch SA, Gupta A, Rasmussen KJ, Schimmenti LA, Klee EW, Niu Z, Agre KE, Chilton I, Chung WK, Revah-Politi A, Au PYB, Griffith C, Racobaldo M, Raas-Rothschild A, Ben Zeev B, Barel O, Moutton S, Morice-Picard F, Carmignac V, Cornaton J, Marle N, Devinsky O, Stimach C, Wechsler SB, Hainline BE, Sapp K, Willems M, Bruel AL, Dias KR, Evans CA, Roscioli T, Sachdev R, Temple SEL, Zhu Y, Baker JJ, Scheffer IE, Gardiner FJ, Schneider AL, Muir AM, Mefford HC, Crunk A, Heise EM, Millan F, Monaghan KG, Person R, Rhodes L, Richards S, Wentzensen IM, Cogné B, Isidor B, Nizon M, Vincent M, Besnard T, Piton A, Marcelis C, Kato K, Koyama N, Ogi T, Goh ES, Richmond C, Amor DJ, Boyce JO, Morgan AT, Hildebrand MS, Kaspi A, Bahlo M, Friðriksdóttir R, Katrínardóttir H, Sulem P, Stefánsson K, Björnsson HT, Mandelstam S, Morleo M, Mariani M; TUDP Study Group, Scala M, Accogli A, Torella A, Capra V, Wallis M, Jansen S, Weisfisz Q, de Haan H, Sadedin S; Broad Center for Mendelian Genomics, Lim SC, White SM, Ascher DB, Schenck A, Lockhart PJ, Christodoulou J, Tan TY
Am J Hum Genet 2022 Apr 7;109(4):601-617. doi: 10.1016/j.ajhg.2022.03.002. PMID: 35395208Free PMC Article
Hebebrand M, Hüffmeier U, Trollmann R, Hehr U, Uebe S, Ekici AB, Kraus C, Krumbiegel M, Reis A, Thiel CT, Popp B
Orphanet J Rare Dis 2019 Feb 11;14(1):38. doi: 10.1186/s13023-019-1020-x. PMID: 30744660Free PMC Article
James G, Shanmuganathan M, Harkness W
Childs Nerv Syst 2014 Sep;30(9):1617-9. Epub 2014 Mar 4 doi: 10.1007/s00381-014-2392-9. PMID: 24589886
Okamoto N, Yamagata T, Yada Y, Ichihashi K, Matsumoto N, Momoi MY, Mizuguchi T
Brain Dev 2014 Jun;36(6):523-7. Epub 2013 Jul 27 doi: 10.1016/j.braindev.2013.07.002. PMID: 23899771
Dubourg C, Bendavid C, Pasquier L, Henry C, Odent S, David V
Orphanet J Rare Dis 2007 Feb 2;2:8. doi: 10.1186/1750-1172-2-8. PMID: 17274816Free PMC Article

Clinical prediction guides

Stephenson SEM, Costain G, Blok LER, Silk MA, Nguyen TB, Dong X, Alhuzaimi DE, Dowling JJ, Walker S, Amburgey K, Hayeems RZ, Rodan LH, Schwartz MA, Picker J, Lynch SA, Gupta A, Rasmussen KJ, Schimmenti LA, Klee EW, Niu Z, Agre KE, Chilton I, Chung WK, Revah-Politi A, Au PYB, Griffith C, Racobaldo M, Raas-Rothschild A, Ben Zeev B, Barel O, Moutton S, Morice-Picard F, Carmignac V, Cornaton J, Marle N, Devinsky O, Stimach C, Wechsler SB, Hainline BE, Sapp K, Willems M, Bruel AL, Dias KR, Evans CA, Roscioli T, Sachdev R, Temple SEL, Zhu Y, Baker JJ, Scheffer IE, Gardiner FJ, Schneider AL, Muir AM, Mefford HC, Crunk A, Heise EM, Millan F, Monaghan KG, Person R, Rhodes L, Richards S, Wentzensen IM, Cogné B, Isidor B, Nizon M, Vincent M, Besnard T, Piton A, Marcelis C, Kato K, Koyama N, Ogi T, Goh ES, Richmond C, Amor DJ, Boyce JO, Morgan AT, Hildebrand MS, Kaspi A, Bahlo M, Friðriksdóttir R, Katrínardóttir H, Sulem P, Stefánsson K, Björnsson HT, Mandelstam S, Morleo M, Mariani M; TUDP Study Group, Scala M, Accogli A, Torella A, Capra V, Wallis M, Jansen S, Weisfisz Q, de Haan H, Sadedin S; Broad Center for Mendelian Genomics, Lim SC, White SM, Ascher DB, Schenck A, Lockhart PJ, Christodoulou J, Tan TY
Am J Hum Genet 2022 Apr 7;109(4):601-617. doi: 10.1016/j.ajhg.2022.03.002. PMID: 35395208Free PMC Article
Hebebrand M, Hüffmeier U, Trollmann R, Hehr U, Uebe S, Ekici AB, Kraus C, Krumbiegel M, Reis A, Thiel CT, Popp B
Orphanet J Rare Dis 2019 Feb 11;14(1):38. doi: 10.1186/s13023-019-1020-x. PMID: 30744660Free PMC Article
Dobyns WB, Aldinger KA, Ishak GE, Mirzaa GM, Timms AE, Grout ME, Dremmen MHG, Schot R, Vandervore L, van Slegtenhorst MA, Wilke M, Kasteleijn E, Lee AS, Barry BJ, Chao KR, Szczałuba K, Kobori J, Hanson-Kahn A, Bernstein JA, Carr L, D'Arco F, Miyana K, Okazaki T, Saito Y, Sasaki M, Das S, Wheeler MM, Bamshad MJ, Nickerson DA; University of Washington Center for Mendelian Genomics; Center for Mendelian Genomics at the Broad Institute of MIT and Harvard, Engle EC, Verheijen FW, Doherty D, Mancini GMS
Am J Hum Genet 2018 Dec 6;103(6):1009-1021. Epub 2018 Nov 21 doi: 10.1016/j.ajhg.2018.10.019. PMID: 30471716Free PMC Article
Jin Z, Luo R, Piao X
Prog Mol Biol Transl Sci 2009;89:1-13. Epub 2009 Oct 7 doi: 10.1016/S1877-1173(09)89001-7. PMID: 20374731
Dubourg C, Bendavid C, Pasquier L, Henry C, Odent S, David V
Orphanet J Rare Dis 2007 Feb 2;2:8. doi: 10.1186/1750-1172-2-8. PMID: 17274816Free PMC Article

Recent systematic reviews

Jiménez-Murillo D, Castro-Ospina AE, Duque-Muñoz L, Martínez-Vargas JD, Suárez-Revelo JX, Vélez-Arango JM, de la Iglesia-Vayá M
Sensors (Basel) 2023 Aug 10;23(16) doi: 10.3390/s23167072. PMID: 37631608Free PMC Article
Siffredi V, Anderson V, Leventer RJ, Spencer-Smith MM
Dev Neuropsychol 2013;38(1):36-57. doi: 10.1080/87565641.2012.721421. PMID: 23311314

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