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Displasia cortical focal isolada tipo IIa
ORPHA:269001CID-10 · Q04.8CID-11 · LA05.51DOENÇA RARA
Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Esta é uma lista de códigos de doenças no banco de dados Online Mendelian Inheritance in Man (OMIM). São doenças que podem ser herdadas por meio de um mecanismo genético mendeliano. O OMIM é um dos bancos de dados abrigados no Centro Nacional de Informações sobre Biotecnologia dos Estados Unidos.

Publicações científicas
9 artigos
Último publicado: 2018 Jan

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Herança
Not applicable
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q04.8
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (5)
0202010503
Cariótipo — bandas G, Q ou Rgenetic_test
0202010600
Pesquisa de microdeleções/microduplicações por FISHlab_test
0202010694
Sequenciamento completo do exoma (WES)rehabilitation
0202010260
Dosagem de alfa-fetoproteína
0301070040
Atendimento em reabilitação — doenças raras
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Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa6desde 2020
Total histórico9PubMed
Últimos 10 anos6publicações
Pico20173 papers
Linha do tempo
20202020Hoje · 2026📈 2017Ano de pico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

2 genes identificados com associação a esta condição. Padrão de herança: Not applicable.

MTORSerine/threonine-protein kinase mTORDisease-causing somatic mutation(s) inAltamente restrito
FUNÇÃO

Serine/threonine protein kinase which is a central regulator of cellular metabolism, growth and survival in response to hormones, growth factors, nutrients, energy and stress signals (PubMed:12087098, PubMed:12150925, PubMed:12150926, PubMed:12231510, PubMed:12718876, PubMed:14651849, PubMed:15268862, PubMed:15467718, PubMed:15545625, PubMed:15718470, PubMed:18497260, PubMed:18762023, PubMed:18925875, PubMed:20516213, PubMed:20537536, PubMed:21659604, PubMed:23429703, PubMed:23429704, PubMed:257

LOCALIZAÇÃO

Lysosome membraneEndoplasmic reticulum membraneGolgi apparatus membraneCell membraneMitochondrion outer membraneCytoplasmNucleusNucleus, PML bodyMicrosome membraneCytoplasmic vesicle, phagosome

VIAS BIOLÓGICAS (10)
MacroautophagyEnergy dependent regulation of mTOR by LKB1-AMPKmTORC1-mediated signallingRegulation of PTEN gene transcriptionMTOR signalling
MECANISMO DE DOENÇA

Smith-Kingsmore syndrome

An autosomal dominant syndrome characterized by intellectual disability, macrocephaly, seizures, umbilical hernia, and facial dysmorphic features.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
35.5 TPM
Cerebelo
27.1 TPM
Cérebro - Hemisfério cerebelar
26.0 TPM
Útero
20.1 TPM
Nervo tibial
19.8 TPM
OUTRAS DOENÇAS (7)
isolated focal cortical dysplasia type IImacrocephaly-intellectual disability-neurodevelopmental disorder-small thorax syndromeobsolete cerebral malformationovergrowth syndrome and/or cerebral malformations due to abnormalities in MTOR pathway genes
HGNC:3942UniProt:P42345
TSC2TuberinDisease-causing somatic mutation(s) inAltamente restrito
FUNÇÃO

Catalytic component of the TSC-TBC complex, a multiprotein complex that acts as a negative regulator of the canonical mTORC1 complex, an evolutionarily conserved central nutrient sensor that stimulates anabolic reactions and macromolecule biosynthesis to promote cellular biomass generation and growth (PubMed:12172553, PubMed:12271141, PubMed:12842888, PubMed:12906785, PubMed:15340059, PubMed:22819219, PubMed:24529379, PubMed:28215400, PubMed:33436626, PubMed:35772404). Within the TSC-TBC complex

LOCALIZAÇÃO

Lysosome membraneCytoplasm, cytosol

VIAS BIOLÓGICAS (6)
Energy dependent regulation of mTOR by LKB1-AMPKTP53 Regulates Metabolic GenesInhibition of TSC complex formation by PKBTBC/RABGAPsAKT phosphorylates targets in the cytosol
MECANISMO DE DOENÇA

Tuberous sclerosis 2

An autosomal dominant multi-system disorder that affects especially the brain, kidneys, heart, and skin. It is characterized by hamartomas (benign overgrowths predominantly of a cell or tissue type that occurs normally in the organ) and hamartias (developmental abnormalities of tissue combination). Clinical manifestations include epilepsy, learning difficulties, behavioral problems, and skin lesions. Seizures can be intractable and premature death can occur from a variety of disease-associated causes.

EXPRESSÃO TECIDUAL(Ubíquo)
Cerebelo
133.2 TPM
Cérebro - Hemisfério cerebelar
103.9 TPM
Pituitária
94.3 TPM
Tireoide
88.0 TPM
Testículo
86.3 TPM
OUTRAS DOENÇAS (9)
lymphangioleiomyomatosistuberous sclerosis 2isolated focal cortical dysplasia type IItuberous sclerosis
HGNC:12363UniProt:P49815

Variantes genéticas (ClinVar)

3,069 variantes patogênicas registradas no ClinVar.

🧬 MTOR: NM_004958.4(MTOR):c.1643T>G (p.Met548Arg) ()
🧬 MTOR: NM_004958.4(MTOR):c.3583G>A (p.Val1195Met) ()
🧬 MTOR: NM_004958.4(MTOR):c.7228C>T (p.His2410Tyr) ()
🧬 MTOR: GRCh37/hg19 1p36.32-36.22(chr1:4995984-11364920)x1 ()
🧬 MTOR: NM_004958.4(MTOR):c.3674A>C (p.Glu1225Ala) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 1,406 variantes classificadas pelo ClinVar.

914
492
Patogênica (65.0%)
VUS (35.0%)
VARIANTES MAIS SIGNIFICATIVAS
TSC1: NM_000368.5(TSC1):c.631G>T (p.Glu211Ter) [Pathogenic]
TSC2: NM_000548.5(TSC2):c.4618_4619del (p.Tyr1540fs) [Pathogenic]
TSC2: NM_000548.5(TSC2):c.1445_1448del (p.Glu482fs) [Likely pathogenic]
TSC2: NC_000016.9:g.(?_2131598)_(2138613_?)del [Pathogenic]
MIR1225: NC_000016.9:g.(?_2120457)_(2185690_?)del [Pathogenic]

Diagnóstico

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Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

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Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Displasia cortical focal isolada tipo IIa

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Publicações mais relevantes

Timeline de publicações
4 papers (10 anos)
#1

Oligodendrocyte lineage and myelination are compromised in the gray matter of focal cortical dysplasia type IIa.

Epilepsia2020 Jan

Focal cortical dysplasias (FCDs) are local malformations of the human neocortex and a leading cause of medically intractable epilepsy. FCDs are characterized by local architectural disturbances of the neocortex and often by a blurred gray-white matter boundary indicating abnormal white matter myelination. We have recently shown that myelination is also compromised in the gray matter of dysplastic areas, since transcripts encoding factors for oligodendrocyte differentiation and myelination are downregulated and myelin fibers appear fractured and disorganized. Here, we characterized the gray matter-associated myelination pathology in detail by in situ hybridization, immunohistochemistry, and electron microscopy with markers for myelin, mature oligodendrocytes, and oligodendrocyte precursor cells in tissue sections of FCD IIa and control cortices. In addition, we isolated oligodendrocyte precursor cells from resected dysplastic tissue and performed proliferation assays. We show that the proportion of myelinated gray matter is similar in the dysplastic cortex to that in controls and myelinated fibers extend up to layer III. On the ultrastructural level, however, we found that the myelin sheaths of layer V axons are thinner in dysplastic specimens than in controls. In addition, the density of oligodendrocyte precursor cells and of mature oligodendrocytes was reduced. Finally, we show for the first time that oligodendrocyte precursor cells isolated from resected dysplastic cortex have a reduced proliferation capacity in comparison to controls. These results indicate that proliferation and differentiation of oligodendrocyte precursor cells and the formation of myelin sheaths are compromised in FCD and might contribute to the epileptogenicity of this cortical malformation.

#2

Histopathology of 3 Tesla MRI-negative extratemporal focal epilepsies.

Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia2018 Apr

Information about the histopathology in 3 Tesla MRI negative extratemporal epilepsies is relatively limited. Most common histopathological findings in earlier (mixed 1.5 or 3 Tesla) MRI-negative series are focal cortical dysplasia (FCD), gliosis or normal findings. These series mostly use the older Palmini criteria for classification and grading. We focus on histopathology of only 3 Tesla MRI-negative extratemporal epilepsies according to the current ILAE criteria and investigate potential correlation to seizure outcome 1 year postoperatively. Sixteen substrates of 3 Tesla MRI-negative extratemporal epilepsies were examined in two steps. Standard stains and immunohistochemical reactions and Palmini criteria were used prospectively during the initial examination. Retrospectively, all specimens were re-examined and re-evaluated. Phospho-6 and calretinin stains and ILAE criteria were used during the review examination. Initial examination revealed 5 FCDs Palmini 1b, two 1a, five 2a and 4 cases of gliosis. The review examination according to ILAE criteria revealed 6 FCDs type IIa, 2 FCDs Ib and 7 mild malformations of cortical development (mMCD) type II. None of our cases was labelled as isolated gliosis after the review examination. The incidence of FCD, after the review examination per ILAE criteria, was reduced to 56%; versus 75% per Palmini. In "true" 3 Tesla MRI-negative extratemporal epilepsies, incidence of FCD may be lower than in earlier MRI-negative series that included weaker MRI-field. Furthermore, consistent review examination may confirm the diagnosis of mMCD type II as substrate in cases diagnosed as "gliosis" or "normal" in the past.

#3

Surgical outcome and predictive factors of epilepsy surgery in pediatric isolated focal cortical dysplasia.

Epilepsy research2018 Jan

Focal cortical dysplasia (FCD) is a common cause of medically intractable epilepsy in children. Epilepsy surgery has been a valuable treatment option to achieve seizure freedom in these intractable epilepsy patients. We aimed to present long-term surgical outcome, in relation to pathological severity, and to assess predictive factors of epilepsy surgery in pediatric isolated FCD. We retrospectively analyzed the data of 58 children and adolescents, with FCD International League Against Epilepsy (ILAE) task force classification types I and II, who underwent resective epilepsy surgery and were followed for at least 2 years after surgery. The mean age at epilepsy onset was 4.3 years (0-14.2 years), and mean age at epilepsy surgery was 9.4 years (0.4-17.5 years). The mean duration of postoperative follow-up was 5.1±2.6 years (2-12.4 years). Of 58 patients, 62% of patients achieved Engel class I at 2 years postoperatively, 58% at 5 years postoperatively, and 53% at the last follow up. Forty eight percent of our cohort successfully discontinued antiepileptic medication. Of 30 patients with seizure recurrence, 83% of seizures recurred within 2 years after surgery. We observed that FCD type IIb was significantly associated with a better surgical outcome. At fifth postoperative year, 88% of FCD IIb patients were seizure free compared with 21% of type I and 57% of type IIa patients (P=0.043). By multivariate analysis, lesion on MRI (P=0.02) and complete resection (P<0.01) were the most important predictive factors for a seizure-free outcome. Epilepsy surgery is highly effective; more than half of medically intractable epilepsy patients achieved seizure freedom after surgery. In addition, we found significant difference in surgical outcomes according to the ILAE task force classification. Lesion on MRI and complete resection were the most important predictive factors for favorable seizure outcome in isolated FCD patients.

#4

Putting the new ILAE classification of focal cortical dysplasia into practice in western China.

Seizure2017 Oct

To re-examine drug-resistant epilepsy cases using the revised 2011 ILAE classification of focal cortical dysplasia (FCD). Patients with drug-resistant epilepsy who have undergone epilepsy surgery in West China Hospital between July 2012 and Jun 2014 were included. Clinical histories, pathological diagnoses, and surgical outcomes were reviewed. A questionnaire was developed to investigate the clinical practice of the new classification. A short-term training program on FCD was carried out to improve pathological diagnosis accuracy. 260 consecutive cases (177 male and 83 female) were included. Pathological diagnosis was changed in 70 cases (26.9%) after re-examination. The five most common pathological types were hippocampal sclerosis (19.2%, 50/260), brain tumors (17.7%, 46/260), vascular malformations (16.2%, 42/260), glial scars (11.2%, 29/260) and FCD (10.0%, 26/260). The most common subtype of isolated FCD was FCD IIb (53.8%, 14/26), followed by FCD IIa (42.3%, 11/26) and FCD Ib (3.8%, 1/26). In addition, forty-five cases were diagnosed as associated FCD type III (17.3%, 45/260). Half of patients with FCD achieved Engel class I at two-year follow-up. Questionnaire investigation suggested most participant pathologists lack sufficient knowledge on the new classification. The diagnostic sensitivity for different FCD subtypes was significantly improved by two to six folds after short-term training. FCD is an important etiology of drug-resistant epilepsy in western China. It is essential to provide continuing trainings to improve diagnostic precision of FCD in developing countries.

#5

A distinct clinicopathological variant of focal cortical dysplasia IIId characterized by loss of layer 4 in the occipital lobe in 12 children with remote hypoxic-ischemic injury.

Epilepsia2017 Oct

In 2011, the International League Against Epilepsy (ILAE) proposed a consensus classification system of focal cortical dysplasia (FCD) to distinguish clinicopathological subtypes, for example, "isolated" FCD type Ia-c and IIa-b, versus "associated" FCD type IIIa-d. The histopathological differentiation of FCD type I and III variants remains, however, a challenging issue in everyday practice. We present a unique histopathological pattern in patients with difficult-to-diagnose FCD, which highlights this dilemma, but also helps to refine the current ILAE classification scheme of FCD. We present a retrospective series of 11 male and one female patient with early onset pharmacoresistant epilepsy of the posterior quadrant (mean age at seizure onset = 4.6 years). All surgical specimens were reviewed. Clinical histories were retrieved and extracted from archival patient files. Microscopic inspection revealed abnormalities in cortical architecture with complete loss of layer 4 in all surgical samples of the occipital lobe, as confirmed by semiquantitative measurements (p < 0.01). Clinical history reported early transient hypoxic condition in nine patients (75%). Magnetic resonance imaging (MRI) revealed abnormal signals in the occipital lobe in all patients, and signal changes suggestive of subcortical encephalomalacia were found in seven patients. Surgical treatment achieved favorable seizure control (Engel class I and II) in seven patients with an available follow-up period of 6.1 years. Prominent disorganization of cortical layering and lack of any other microscopically visible principle lesion in the surgical specimen would result in this neuropathological pattern hitherto being classified as FCD ILAE type Ib. However, perinatal hypoxia with distinctive MRI changes suggested primarily a hypoxemic lesion and acquired pathomechanism of neuronal cell loss in the occipital lobe of our patient series. We propose, therefore, classifying this distinctive clinicopathological pattern as a separate variant of FCD ILAE type IIId.

Publicações recentes

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Doenças relacionadas

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Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Oligodendrocyte lineage and myelination are compromised in the gray matter of focal cortical dysplasia type IIa.
    Epilepsia· 2020· PMID 31872870mais citado
  2. Histopathology of 3&#x202f;Tesla MRI-negative extratemporal focal epilepsies.
    Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia· 2018· PMID 29422363mais citado
  3. Surgical outcome and predictive factors of epilepsy surgery in pediatric isolated focal cortical dysplasia.
    Epilepsy research· 2018· PMID 29197666mais citado
  4. Putting the new ILAE classification of focal cortical dysplasia into practice in western China.
    Seizure· 2017· PMID 28843774mais citado
  5. A distinct clinicopathological variant of focal cortical dysplasia IIId characterized by loss of layer 4 in the occipital lobe in 12 children with remote hypoxic-ischemic injury.
    Epilepsia· 2017· PMID 28833053mais citado
  6. Childhood-Onset Epileptic Encephalopathy Associated With Isolated Focal Cortical Dysplasia and a Novel TSC1 Germline Mutation.
    Clin EEG Neurosci· 2018· PMID 28762286recente
  7. Outcome of surgery for temporal lobe epilepsy in adults - A cohort study.
    Int J Surg· 2016· PMID 25979111recente
  8. Cytochrome c oxidase deficit is associated with the seizure onset zone in young patients with focal cortical dysplasia Type II.
    Metab Brain Dis· 2015· PMID 25957585recente
  9. Acute neuroinflammation in a clinically relevant focal cortical ischemic stroke model in rat: longitudinal positron emission tomography and immunofluorescent tracking.
    Brain Struct Funct· 2016· PMID 25601153recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:269001(Orphanet)
  2. MONDO:0017101(MONDO)
  3. GARD:17270(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q55786812(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

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Compêndio · Raras BR

Displasia cortical focal isolada tipo IIa

ORPHA:269001 · MONDO:0017101
Prevalência
Unknown
Herança
Not applicable
CID-10
Q04.8 · Outras malformações congênitas especificadas do encéfalo
CID-11
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C5679770
EuropePMC
Wikidata
Papers 10a
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