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Cortical dysplasia, complex, with other brain malformations 10(CDCBM10)

MedGen UID:
1684859
Concept ID:
C5231458
Disease or Syndrome
Synonyms: CDCBM10; CORTICAL DYSPLASIA, COMPLEX, WITH OTHER BRAIN MALFORMATIONS 10
 
Gene (location): APC2 (19p13.3)
 
Monarch Initiative: MONDO:0032866
OMIM®: 618677

Definition

Complex cortical dysplasia with other brain malformations-10 (CDCBM10) is an autosomal recessive neurodevelopmental disorder characterized by severely impaired global development associated with abnormalities on brain imaging, including lissencephaly, cortical dysplasia, subcortical heterotopia, and paucity of white matter. The disorder results from defective neuronal migration during brain development. Affected individuals often develop seizures, are unable to walk, and do not acquire language (summary by Lee et al., 2019). For a discussion of genetic heterogeneity of CDCBM, see CDCBM1 (614039). [from OMIM]

Clinical features

From HPO
Intellectual disability, severe
MedGen UID:
48638
Concept ID:
C0036857
Mental or Behavioral Dysfunction
Severe mental retardation is defined as an intelligence quotient (IQ) in the range of 20-34.
Generalized-onset seizure
MedGen UID:
115963
Concept ID:
C0234533
Disease or Syndrome
A generalized-onset seizure is a type of seizure originating at some point within, and rapidly engaging, bilaterally distributed networks. The networks may include cortical and subcortical structures but not necessarily the entire cortex.
Lissencephaly
MedGen UID:
78604
Concept ID:
C0266463
Finding
A spectrum of malformations of cortical development caused by insufficient neuronal migration that subsumes the terms agyria, pachygyria and subcortical band heterotopia. See also neuropathological definitions for 2-, 3-, and 4-layered lissencephaly.
Cerebellar hypoplasia
MedGen UID:
120578
Concept ID:
C0266470
Congenital Abnormality
Cerebellar hypoplasia is a descriptive term implying a cerebellum with a reduced volume, but a normal shape and is stable over time.
Hypoplasia of the corpus callosum
MedGen UID:
138005
Concept ID:
C0344482
Congenital Abnormality
Underdevelopment of the corpus callosum.
Spastic tetraplegia
MedGen UID:
98433
Concept ID:
C0426970
Disease or Syndrome
Spastic paralysis affecting all four limbs.
Delayed speech and language development
MedGen UID:
105318
Concept ID:
C0454644
Finding
A degree of language development that is significantly below the norm for a child of a specified age.
Global developmental delay
MedGen UID:
107838
Concept ID:
C0557874
Finding
A delay in the achievement of motor or mental milestones in the domains of development of a child, including motor skills, speech and language, cognitive skills, and social and emotional skills. This term should only be used to describe children younger than five years of age.
Hypsarrhythmia
MedGen UID:
195766
Concept ID:
C0684276
Finding
Hypsarrhythmia is abnormal interictal high amplitude waves and a background of irregular spikes. There is continuous (during wakefulness), high-amplitude (>200 Hz), generalized polymorphic slowing with no organized background and multifocal spikes demonstrated by electroencephalography (EEG).
Delayed gross motor development
MedGen UID:
332508
Concept ID:
C1837658
Finding
A type of motor delay characterized by a delay in acquiring the ability to control the large muscles of the body for walking, running, sitting, and crawling.
Hypoplasia of the brainstem
MedGen UID:
334226
Concept ID:
C1842688
Finding
Underdevelopment of the brainstem.
Hypoplasia of the pons
MedGen UID:
341246
Concept ID:
C1848529
Finding
Underdevelopment of the pons.
Ventriculomegaly
MedGen UID:
480553
Concept ID:
C3278923
Finding
An increase in size of the ventricular system of the brain.
Infantile spasms
MedGen UID:
854616
Concept ID:
C3887898
Disease or Syndrome
Infantile spasms represent a subset of "epileptic spasms". Infantile Spasms are epileptic spasms starting in the first year of life (infancy).
Delayed early-childhood social milestone development
MedGen UID:
868509
Concept ID:
C4022906
Finding
A failure to meet one or more age-related milestones of social behavior.
Delayed fine motor development
MedGen UID:
869257
Concept ID:
C4023681
Finding
A type of motor delay characterized by a delay in acquiring the ability to control the fingers and hands.
Thick cerebral cortex
MedGen UID:
870522
Concept ID:
C4024970
Anatomical Abnormality
Myoclonic seizure
MedGen UID:
1385980
Concept ID:
C4317123
Sign or Symptom
A myoclonic seizure is a type of motor seizure characterized by sudden, brief (<100 ms) involuntary single or multiple contraction of muscles or muscle groups of variable topography (axial, proximal limb, distal). Myoclonus is less regularly repetitive and less sustained than is clonus.
Periventricular ribbonlike heterotopia
MedGen UID:
1698492
Concept ID:
C5139311
Disease or Syndrome
Heterotopia that forms a continuous wavy line along the ventricular wall.
Hypertonia
MedGen UID:
10132
Concept ID:
C0026826
Finding
A condition in which there is increased muscle tone so that arms or legs, for example, are stiff and difficult to move.
Hypotonia
MedGen UID:
10133
Concept ID:
C0026827
Finding
Hypotonia is an abnormally low muscle tone (the amount of tension or resistance to movement in a muscle). Even when relaxed, muscles have a continuous and passive partial contraction which provides some resistance to passive stretching. Hypotonia thus manifests as diminished resistance to passive stretching. Hypotonia is not the same as muscle weakness, although the two conditions can co-exist.

Professional guidelines

PubMed

Shen YW, Wang YY, Zhang MN, Xu Y, Lu Q, He W, Chen HM, Liu LY, Pang LY, Wang QH, Dun S, Li YF, Gao J, Han F, Zou LP
Seizure 2022 Apr;97:23-31. Epub 2022 Mar 8 doi: 10.1016/j.seizure.2022.03.003. PMID: 35286974
Bąbol-Pokora K, Bielska M, Bobeff K, Jatczak-Pawlik I, Borkowska J, Kotulska K, Jóźwiak S, Młynarski W, Trelińska J
Eur J Med Genet 2021 Oct;64(10):104309. Epub 2021 Aug 14 doi: 10.1016/j.ejmg.2021.104309. PMID: 34403804
Braun DA, Rao J, Mollet G, Schapiro D, Daugeron MC, Tan W, Gribouval O, Boyer O, Revy P, Jobst-Schwan T, Schmidt JM, Lawson JA, Schanze D, Ashraf S, Ullmann JFP, Hoogstraten CA, Boddaert N, Collinet B, Martin G, Liger D, Lovric S, Furlano M, Guerrera IC, Sanchez-Ferras O, Hu JF, Boschat AC, Sanquer S, Menten B, Vergult S, De Rocker N, Airik M, Hermle T, Shril S, Widmeier E, Gee HY, Choi WI, Sadowski CE, Pabst WL, Warejko JK, Daga A, Basta T, Matejas V, Scharmann K, Kienast SD, Behnam B, Beeson B, Begtrup A, Bruce M, Ch'ng GS, Lin SP, Chang JH, Chen CH, Cho MT, Gaffney PM, Gipson PE, Hsu CH, Kari JA, Ke YY, Kiraly-Borri C, Lai WM, Lemyre E, Littlejohn RO, Masri A, Moghtaderi M, Nakamura K, Ozaltin F, Praet M, Prasad C, Prytula A, Roeder ER, Rump P, Schnur RE, Shiihara T, Sinha MD, Soliman NA, Soulami K, Sweetser DA, Tsai WH, Tsai JD, Topaloglu R, Vester U, Viskochil DH, Vatanavicharn N, Waxler JL, Wierenga KJ, Wolf MTF, Wong SN, Leidel SA, Truglio G, Dedon PC, Poduri A, Mane S, Lifton RP, Bouchard M, Kannu P, Chitayat D, Magen D, Callewaert B, van Tilbeurgh H, Zenker M, Antignac C, Hildebrandt F
Nat Genet 2017 Oct;49(10):1529-1538. Epub 2017 Aug 14 doi: 10.1038/ng.3933. PMID: 28805828Free PMC Article

Recent clinical studies

Etiology

Calì E, Lin SJ, Rocca C, Sahin Y, Al Shamsi A, El Chehadeh S, Chaabouni M, Mankad K, Galanaki E, Efthymiou S, Sudhakar S, Athanasiou-Fragkouli A, Çelik T, Narlı N, Bianca S, Murphy D, De Carvalho Moreira FM; SYNaPS Study Group, Andrea Accogli, Petree C, Huang K, Monastiri K, Edizadeh M, Nardello R, Ognibene M, De Marco P, Ruggieri M, Zara F, Striano P, Şahin Y, Al-Gazali L, Abi Warde MT, Gerard B, Zifarelli G, Beetz C, Fortuna S, Soler M, Valente EM, Varshney G, Maroofian R, Salpietro V, Houlden H
Genet Med 2022 Oct;24(10):2194-2203. Epub 2022 Aug 24 doi: 10.1016/j.gim.2022.07.013. PMID: 36001086Free PMC Article
Islam MP
Semin Pediatr Neurol 2021 Apr;37:100875. Epub 2021 Feb 11 doi: 10.1016/j.spen.2021.100875. PMID: 33892851
Thiele EA, Bebin EM, Bhathal H, Jansen FE, Kotulska K, Lawson JA, O'Callaghan FJ, Wong M, Sahebkar F, Checketts D, Knappertz V; GWPCARE6 Study Group
JAMA Neurol 2021 Mar 1;78(3):285-292. doi: 10.1001/jamaneurol.2020.4607. PMID: 33346789Free PMC Article
Pecoraro A, Arehart E, Gallentine W, Radtke R, Smith E, Pizoli C, Kansagra S, Abdelnour E, McLendon R, Mikati MA
Epilepsy Behav 2017 Aug;73:137-141. Epub 2017 Jul 18 doi: 10.1016/j.yebeh.2017.05.011. PMID: 28633092
Sahin M, Henske EP, Manning BD, Ess KC, Bissler JJ, Klann E, Kwiatkowski DJ, Roberds SL, Silva AJ, Hillaire-Clarke CS, Young LR, Zervas M, Mamounas LA; Tuberous Sclerosis Complex Working Group to Update the Research Plan
Pediatr Neurol 2016 Jul;60:1-12. Epub 2016 Apr 2 doi: 10.1016/j.pediatrneurol.2016.03.015. PMID: 27267556Free PMC Article

Diagnosis

Islam MP
Semin Pediatr Neurol 2021 Apr;37:100875. Epub 2021 Feb 11 doi: 10.1016/j.spen.2021.100875. PMID: 33892851
Thiele EA, Bebin EM, Bhathal H, Jansen FE, Kotulska K, Lawson JA, O'Callaghan FJ, Wong M, Sahebkar F, Checketts D, Knappertz V; GWPCARE6 Study Group
JAMA Neurol 2021 Mar 1;78(3):285-292. doi: 10.1001/jamaneurol.2020.4607. PMID: 33346789Free PMC Article
Fahmy MD, Gupta A, Padilla RJ, Segura A, Brookes CD
Oral Surg Oral Med Oral Pathol Oral Radiol 2019 Aug;128(2):e92-e99. Epub 2019 Mar 16 doi: 10.1016/j.oooo.2019.03.008. PMID: 31078508
Derauf C, Kekatpure M, Neyzi N, Lester B, Kosofsky B
Semin Cell Dev Biol 2009 Jun;20(4):441-54. Epub 2009 Mar 13 doi: 10.1016/j.semcdb.2009.03.001. PMID: 19560049Free PMC Article
Moss J, DeCastro R, Patronas NJ, Taveira-DaSilva A
JAMA 2001 Oct 17;286(15):1879-81. doi: 10.1001/jama.286.15.1879. PMID: 11597290

Therapy

Leitner DF, Kanshin E, Askenazi M, Siu Y, Friedman D, Devore S, Jones D, Ueberheide B, Wisniewski T, Devinsky O
PLoS One 2022;17(5):e0268597. Epub 2022 May 19 doi: 10.1371/journal.pone.0268597. PMID: 35587487Free PMC Article
Thiele EA, Bebin EM, Bhathal H, Jansen FE, Kotulska K, Lawson JA, O'Callaghan FJ, Wong M, Sahebkar F, Checketts D, Knappertz V; GWPCARE6 Study Group
JAMA Neurol 2021 Mar 1;78(3):285-292. doi: 10.1001/jamaneurol.2020.4607. PMID: 33346789Free PMC Article
Overwater IE, Rietman AB, Mous SE, Bindels-de Heus K, Rizopoulos D, Ten Hoopen LW, van der Vaart T, Jansen FE, Elgersma Y, Moll HA, de Wit MY; ENCORE Expertise Centre for Neurodevelopmental Disorders
Neurology 2019 Jul 9;93(2):e200-e209. Epub 2019 Jun 19 doi: 10.1212/WNL.0000000000007749. PMID: 31217257
Sasongko TH, Ismail NF, Zabidi-Hussin Z
Cochrane Database Syst Rev 2016 Jul 13;7(7):CD011272. doi: 10.1002/14651858.CD011272.pub2. PMID: 27409709Free PMC Article
Moss J, DeCastro R, Patronas NJ, Taveira-DaSilva A
JAMA 2001 Oct 17;286(15):1879-81. doi: 10.1001/jama.286.15.1879. PMID: 11597290

Prognosis

Jin L, Li Y, Luo S, Peng Q, Zhai QX, Zhai JX, Gao LD, Guo JJ, Song W, Yi YH, He N, Chen YJ
Seizure 2023 Oct;111:172-177. Epub 2023 Aug 18 doi: 10.1016/j.seizure.2023.08.008. PMID: 37657306
Huang Q, Zhou J, Wang X, Li T, Wang M, Wang J, Teng P, Qi X, Zhu M, Luan G, Zhai F
Seizure 2021 May;88:45-52. Epub 2021 Mar 25 doi: 10.1016/j.seizure.2021.03.022. PMID: 33812307
Thiele EA, Bebin EM, Bhathal H, Jansen FE, Kotulska K, Lawson JA, O'Callaghan FJ, Wong M, Sahebkar F, Checketts D, Knappertz V; GWPCARE6 Study Group
JAMA Neurol 2021 Mar 1;78(3):285-292. doi: 10.1001/jamaneurol.2020.4607. PMID: 33346789Free PMC Article
Rosengren T, Nanhoe S, de Almeida LGD, Schönewolf-Greulich B, Larsen LJ, Hey CAB, Dunø M, Ek J, Risom L, Nellist M, Møller LB
Sci Rep 2020 Jun 18;10(1):9909. doi: 10.1038/s41598-020-66588-4. PMID: 32555378Free PMC Article
Derauf C, Kekatpure M, Neyzi N, Lester B, Kosofsky B
Semin Cell Dev Biol 2009 Jun;20(4):441-54. Epub 2009 Mar 13 doi: 10.1016/j.semcdb.2009.03.001. PMID: 19560049Free PMC Article

Clinical prediction guides

Jin L, Li Y, Luo S, Peng Q, Zhai QX, Zhai JX, Gao LD, Guo JJ, Song W, Yi YH, He N, Chen YJ
Seizure 2023 Oct;111:172-177. Epub 2023 Aug 18 doi: 10.1016/j.seizure.2023.08.008. PMID: 37657306
Pecoraro A, Arehart E, Gallentine W, Radtke R, Smith E, Pizoli C, Kansagra S, Abdelnour E, McLendon R, Mikati MA
Epilepsy Behav 2017 Aug;73:137-141. Epub 2017 Jul 18 doi: 10.1016/j.yebeh.2017.05.011. PMID: 28633092
Derauf C, Kekatpure M, Neyzi N, Lester B, Kosofsky B
Semin Cell Dev Biol 2009 Jun;20(4):441-54. Epub 2009 Mar 13 doi: 10.1016/j.semcdb.2009.03.001. PMID: 19560049Free PMC Article
Moss J, DeCastro R, Patronas NJ, Taveira-DaSilva A
JAMA 2001 Oct 17;286(15):1879-81. doi: 10.1001/jama.286.15.1879. PMID: 11597290
Striano S, Nocerino C, Striano P, Boccella P, Meo R, Bilo L, Cirillo S
Neurol Sci 2000 Jun;21(3):151-5. doi: 10.1007/s100720070090. PMID: 11076003

Recent systematic reviews

Romero VI, Arias-Almeida B, Aguiar SA
BMC Genomics 2022 Dec 22;23(1):849. doi: 10.1186/s12864-022-09071-w. PMID: 36550402Free PMC Article
Sasongko TH, Ismail NF, Zabidi-Hussin Z
Cochrane Database Syst Rev 2016 Jul 13;7(7):CD011272. doi: 10.1002/14651858.CD011272.pub2. PMID: 27409709Free PMC Article

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