Introdução
O que você precisa saber de cara
Porencefalia é um distúrbio cefálico extremamente raro que envolve encefalomalácia. É um distúrbio neurológico do sistema nervoso central caracterizado por cistos ou cavidades dentro do hemisfério cerebral. A porencefalia foi denominada por Heschl em 1859 para descrever uma cavidade no cérebro humano. Derivada de raízes gregas, a palavra porencefalia significa 'buracos no cérebro'. Os cistos e cavidades são mais propensos a ser o resultado de uma causa destrutiva (encefaloclástica), mas também podem ser decorrentes de desenvolvimento anormal (malformativo), dano direto, inflamação ou hemorragia. Os cistos e cavidades causam uma ampla gama de sintomas fisiológicos, físicos e neurológicos.
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 38 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 92 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
7 genes identificados com associação a esta condição.
Transcriptional regulator which can act as both a transcriptional repressor and activator by binding a ATTA homeodomain core recognition sequence on these target genes. During forebrain development represses WNT1 expression allowing zona limitans intrathalamica formation and thereby ensuring proper anterio-posterior patterning of the diencephalon and formation of the rostral diencephalon. Acts as a direct upstream activator of SHH expression in the rostral diencephalon ventral midline and that i
Nucleus
Holoprosencephaly 2
A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE2 inheritance is autosomal dominant.
Required for centrosome duplication and formation and function of the mitotic spindle. Essential for the development of the cerebral cortex. May regulate the production of neurons by controlling the orientation of the mitotic spindle during division of cortical neuronal progenitors of the proliferative ventricular zone of the brain. Orientation of the division plane perpendicular to the layers of the cortex gives rise to two proliferative neuronal progenitors whereas parallel orientation of the
Cytoplasm, cytoskeletonCytoplasm, cytoskeleton, microtubule organizing center, centrosomeChromosome, centromere, kinetochoreCytoplasm, cytoskeleton, spindleCleavage furrowCytoplasmic vesicle membrane
Lissencephaly 4 with microcephaly
A neurodevelopmental disorder characterized by lissencephaly, severe brain atrophy, extreme microcephaly, and profound intellectual disability.
The C-terminal part of the sonic hedgehog protein precursor displays an autoproteolysis and a cholesterol transferase activity (By similarity). Both activities result in the cleavage of the full-length protein into two parts (ShhN and ShhC) followed by the covalent attachment of a cholesterol moiety to the C-terminal of the newly generated ShhN (By similarity). Both activities occur in the endoplasmic reticulum (By similarity). Once cleaved, ShhC is degraded in the endoplasmic reticulum (By simi
Endoplasmic reticulum membraneGolgi apparatus membraneSecretedCell membrane
Microphthalmia/Coloboma 5
A disorder of eye formation, ranging from small size of a single eye to complete bilateral absence of ocular tissues. Ocular abnormalities like opacities of the cornea and lens, scaring of the retina and choroid, and other abnormalities may also be present. Ocular colobomas are a set of malformations resulting from abnormal morphogenesis of the optic cup and stalk, and the fusion of the fetal fissure (optic fissure).
Transcription factor, which in cooperation with EMX1, acts to generate the boundary between the roof and archipallium in the developing brain. May function in combination with OTX1/2 to specify cell fates in the developing central nervous system. In the inner ear, it controls the distribution of GPR156 at hair cell boundaries, and regulates the organization of stereociliary bundles in opposite orientations across the line of polarity reversal (LPR)
NucleusCell projection, axon
Schizencephaly
Extremely rare human congenital disorder characterized by a full-thickness cleft within the cerebral hemispheres. These clefts are lined with gray matter and most commonly involve the parasylvian regions. Large portions of the cerebral hemispheres may be absent and replaced by cerebro-spinal fluid.
Type IV collagen is the major structural component of glomerular basement membranes (GBM), forming a 'chicken-wire' meshwork together with laminins, proteoglycans and entactin/nidogen Canstatin, a cleavage product corresponding to the collagen alpha 2(IV) NC1 domain, possesses both anti-angiogenic and anti-tumor cell activity. It inhibits proliferation and migration of endothelial cells, reduces mitochondrial membrane potential, and induces apoptosis. Specifically induces Fas-dependent apoptosis
Secreted, extracellular space, extracellular matrix, basement membrane
Brain small vessel disease 2
An autosomal dominant cerebrovascular disorder with variable manifestations reflecting the location and severity of the vascular defect. BSVD2 features include intracranial hemorrage, fluid-filled cysts or cavities within the cerebral hemispheres, delayed psychomotor development, hemiplegia, spasticity and seizures.
Beta-galactosyltransferase that transfers beta-galactose to hydroxylysine residues of type I collagen (PubMed:19075007, PubMed:22216269, PubMed:27402836). By acting on collagen glycosylation, facilitates the formation of collagen triple helix (PubMed:27402836). Also involved in the biosynthesis of collagen type IV (PubMed:30412317)
Endoplasmic reticulum lumen
Brain small vessel disease 3
An autosomal recessive form of brain small vessel disease, a cerebrovascular disorder with variable manifestations reflecting the location and severity of the vascular defect. BSVD3 patients may have disease onset in utero or early infancy with subsequent global developmental delay, spasticity, and porencephaly on brain imaging. Other patients may have normal or mildly delayed development with sudden onset of intracranial hemorrhage causing acute neurologic deterioration.
Type IV collagen is the major structural component of glomerular basement membranes (GBM), forming a 'chicken-wire' meshwork together with laminins, proteoglycans and entactin/nidogen Arresten, comprising the C-terminal NC1 domain, inhibits angiogenesis and tumor formation. The C-terminal half is found to possess the anti-angiogenic activity. Specifically inhibits endothelial cell proliferation, migration and tube formation
Secreted, extracellular space, extracellular matrix, basement membrane
Hereditary angiopathy with nephropathy aneurysms and muscle cramps
The clinical renal manifestations include hematuria and bilateral large cysts. Histologic analysis revealed complex basement membrane defects in kidney and skin. The systemic angiopathy appears to affect both small vessels and large arteries.
Variantes genéticas (ClinVar)
944 variantes patogênicas registradas no ClinVar.
Vias biológicas (Reactome)
41 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Doença encefaloclástica
Selecione um estado ou use sua localização para ver resultados.
Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.
Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Pesquisa e ensaios clínicos
Nenhum ensaio clínico registrado para esta condição.
Publicações mais relevantes
Identification of Fowler syndrome (hydrocephaly-hydranencephaly proliferative vasculopathy) in a pregnancy following single euploid embryo transfer.
Genetic carrier screening is a useful tool for couples looking to understand the genetic risk profile of their offspring. However, this method is limited in identifying rare genetic variants. A couple undergoing single euploid embryo transfer (SEET) following planned embryo banking was followed in this study. The patient was a 39-year-old nulliparous female with negative expanded carrier screening (ECS) which included 400 autosomal recessive and X-linked conditions. Her partner was a 39-year-old male with no pertinent past medical history. Clinical pregnancy was achieved following SEET. Unfortunately, the fetus demonstrated a constellation of neurologic malformations at 28 weeks gestation and the couple opted for termination. Amniocentesis was conducted at the time of labor induction revealing unremarkable chromosome analysis and microarray testing. Whole genome sequencing (WGS) noted paternal and maternal variants of the fetal leukemia virus subgroup C cellular receptor family, member 2 protein (FLVCR2). The paternal variant was classified as pathogenic, and the maternal variant was classified as a variant of uncertain significance by the lab. The presence of balletic FLVCR2 variants in the setting of neurologic anomalies was consistent with Fowler syndrome, a rare autosomal recessive condition. This case highlights the inherent limitations of carrier screening in detecting rare genetic disease and marks the first documented case of Fowler syndrome following single euploid embryo transfer.
Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
Pyruvate dehydrogenase complex deficiency (PDCD) is a disorder of mitochondrial metabolism that is caused by pathogenic variants in multiple genes, including PDHA1. Typical neonatal brain imaging findings have been described, with a focus on malformative and encephaloclastic features. Fetal brain MRI in PDCD has not been comprehensively described. The aims of this study were (1) to further characterize the fetal brain MRI findings in PDCD using comprehensive fetal imaging and genetic testing and (2) to determine whether markers of diagnosis of PDCD could be identified on prenatal imaging. Fetuses with a diagnosis of PDCD related to a genetic etiology that had undergone fetal MRI were included. Fetuses were identified retrospectively from local databases of 4 fetal diagnostic clinics within tertiary pediatric health care centers. Electronic medical records were reviewed retrospectively: demographics, maternal and pregnancy history, fetal outcomes, and neonatal outcomes (if available) were reviewed and recorded. Fetal and neonatal imaging reports were reviewed; source fetal and neonatal brain MRI scans were reviewed by a single pediatric neuroradiologist (J.W.S.) for consistency. Genetic testing strategies and results including variant type, zygosity, inheritance pattern, and pathogenicity were recorded. Deidentified data were combined and reported descriptively. A total of 10 fetuses with a diagnosis of PDCD were included. 8 fetuses had corpus callosum dysgenesis, 6 had an abnormal gyration pattern, 10 had reduced brain volumes, and 9 had cystic lesions. 1 fetus had intraventricular hemorrhages. 1 fetus had a midbrain malformation with aqueductal stenosis and severe hydrocephalus. 6 fetuses imaged in the second trimester had cystic lesions involving the ganglionic eminences (GEs) while GE cysts were not present in the 4 fetuses imaged in the third trimester. Fetuses with PDCD have similar brain MRI findings to neonates described in the literature, although some of these findings are subtle early in pregnancy. Additional features, such as cystic lesions of the GEs, are noted in the second trimester in fetuses with PDCD. These may represent an early diagnostic marker of PDCD, although more data are needed to validate this association. Early diagnosis of PDCD using fetal MRI may inform genetic counseling, pregnancy decision making, and neonatal care planning.
Characteristic Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
Pyruvate dehydrogenase complex deficiency (PDCD) is a disorder of mitochondrial metabolism that is caused by pathogenic variants in multiple genes, including PDHA1. Typical neonatal brain imaging findings in PDCD have been described, with a focus on malformative features and chronic encephaloclastic changes. However, fetal brain MRI imaging in confirmed PDCD has not been comprehensively described. We sought to demonstrate the prenatal neurological and systemic manifestations of PDCD determined by comprehensive fetal imaging and genomic sequencing. All fetuses with a diagnosis of genetic PDCD who had undergone fetal MRI were included in the study. Medical records, imaging data, and genetic testing results were reviewed and reported descriptively. Ten patients with diagnosis of PDCD were included. Most patients had corpus callosum dysgenesis, abnormal gyration pattern, reduced brain volumes, and periventricular cystic lesions. One patient had associated intraventricular hemorrhages. One patient had a midbrain malformation with aqueductal stenosis and severe hydrocephalus. Fetuses imaged in the second trimester were found to have enlargement of the ganglionic eminences with cystic cavitations, while those imaged in the third trimester had germinolytic cysts. Fetuses with PDCD have similar brain MRI findings to neonates described in the literature, although some of these findings may be subtle early in pregnancy. Additional features, such as cystic cavitations of the ganglionic eminences, are noted in the second trimester in fetuses with PDCD, and these may represent a novel early diagnostic marker for PDCD. Using fetal MRI to identify these radiological hallmarks to inform prenatal diagnosis of PDCD may guide genetic counseling, pregnancy decision-making, and neonatal care planning.
Porencephaly and Psychosis: A Rare Case of Neurological and Psychiatric Intersection.
Porencephaly is an uncommon neurological condition characterized by cystic cavities or holes in the cerebral hemispheres of the brain filled with cerebrospinal fluid. There are two types of porencephaly: acquired porencephaly, also known as pseudo-porencephaly, and congenital porencephaly, also known as true porencephaly. Acquired porencephaly, also known as encephaloclastic porencephaly, typically results from late prenatal or perinatal vascular lesions caused by arterial ischemic stroke or venous thrombosis. Congenital porencephaly, or genetic porencephaly, arises from maldevelopment during early neuronal migration. Brain lesions associated with congenital porencephaly are thought to result from irregularities in cell migration during development and are often linked to additional brain deformities. Lesions resulting from degenerative disorders caused by vascular, viral, or traumatic events are classified under acquired porencephaly. Familial cases of porencephaly are believed to be caused by mutations in the COL4A1 gene, which lead to brain small-vessel disease with haemorrhage. Due to the variability in lesion size and location, porencephaly presents with a wide range of clinical symptoms. We report a case of a 41-year-old male who is diagnosed as a case of porencephaly with complaints of withdrawn behaviour, decreased interaction, suspiciousness, delusion of persecution and delusion of reference. These symptoms have started in the past two months. This case report contributes to the growing body of research suggesting a potential link between porencephaly and psychosis, despite the limited available data. Further investigation is required to validate this connection and explore the underlying mechanisms. Continued research into this potential association may help guide future psychosis diagnosis and treatment plans.
Are postnatal traumatic events an underestimated cause of porencephalic lesions in dogs and cats?
Porencephaly is defined as a fluid-filled cavity of variable size in the brain cortex. It is regarded as a congenital condition and is typically considered a developmental or an encephaloclastic defect. Our hypothesis is that postnatal traumatic events in the first few months of life may represent a cause of canine and feline porencephaly that is more common than generally suspected. The aims of this study were to retrospectively investigate porencephaly in a large population of dogs and cats, detect MRI features that might be useful to differentiate postnatal acquired traumatic forms from congenital/perinatal porencephaly, and define the prevalence of seizure activity in porencephalic patients. This is a double-center, descriptive, retrospective case series. Databases were searched for cases within a 17-year time span that involve dogs and cats with an MRI-based diagnosis of cerebral cavitary lesions. Animals were included if a complete signalment and an exhaustive MRI of the brain were available. Besides the porencephalic lesions, MRIs of the head were reviewed to detect concomitant musculoskeletal abnormalities. Thirty-two cases involving nine cats and twenty-three dogs were selected. Of all the cases, 21.9% were aged six years or older at the time of diagnosis. All patients in which the neuroanatomical localization was available showed clinical signs of a prosencephalic disorder. Epileptic seizures were observed in 71.8% of cases. A single porencephalic cavity was found in 78.1% of cases. The most affected cerebral lobe was the parietal lobe (n = 20). The defects involved both the grey and white matter in 78.1% of cases. Twenty cases showed concomitant musculoskeletal abnormalities overlying the porencephalic cavities. Fourteen of twenty cases showed evidence of fractures, of which thirteen showed depression of the calvarium and twelve masticatory muscle abnormalities. Of these, seven of fourteen had a history consistent with a head trauma in the first period of life. The recognition of skull fractures and muscular abnormalities closely associated with the porencephalic cavity may support a diagnosis of a postnatal traumatic origin of porencephaly. Therefore, this study highlights the importance of evaluating musculoskeletal structures in the MRIs of the heads of porencephalic cases.
Publicações recentes
Magnetic Resonance and Computed Tomographic Findings in a Case of Encephaloclastic Porencephaly in a Sulawesi Crested Macaque.
Porencephaly and Psychosis: A Rare Case of Neurological and Psychiatric Intersection.
Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
Characteristic Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
Are postnatal traumatic events an underestimated cause of porencephalic lesions in dogs and cats?
📚 EuropePMCmostrando 13
Identification of Fowler syndrome (hydrocephaly-hydranencephaly proliferative vasculopathy) in a pregnancy following single euploid embryo transfer.
Journal of assisted reproduction and geneticsPorencephaly and Psychosis: A Rare Case of Neurological and Psychiatric Intersection.
CureusFetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
NeurologyCharacteristic Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
medRxiv : the preprint server for health sciencesAre postnatal traumatic events an underestimated cause of porencephalic lesions in dogs and cats?
Frontiers in veterinary scienceHypodipsic hypernatremia after long-standing polydipsia in a cat with suspect neonatal head trauma.
The Canadian veterinary journal = La revue veterinaire canadienneFetal brain small vessel disease 1 caused by a novel mutation in the COL4A1 gene.
Pediatric radiologyVariability of non-lethal Fowler syndrome phenotype associated with FLVCR2 variants.
Clinical geneticsExpanding the clinical spectrum of Fowler syndrome: Three siblings with survival into adulthood and systematic review of the literature.
Clinical geneticsDistinguishing Encephaloclastic Lesions Resulting From Primary or Secondary Pyruvate Dehydrogenase Deficiency From Other Neonatal or Infantile Cavitary Brain Lesions.
Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology SocietyProliferative vasculopathy and hydranencephaly-hydrocephaly syndrome or Fowler syndrome: Report of a family and insight into the disease's mechanism.
Molecular genetics & genomic medicinePhenotypic and Neuropathological Characterization of Fetal Pyruvate Dehydrogenase Deficiency.
Journal of neuropathology and experimental neurologyRecurrent encephaloclastic cyst induced by intraventricular topotecan.
Journal of the neurological sciencesAssociações
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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.
- Identification of Fowler syndrome (hydrocephaly-hydranencephaly proliferative vasculopathy) in a pregnancy following single euploid embryo transfer.
- Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
- Characteristic Fetal Brain MRI Abnormalities in Pyruvate Dehydrogenase Complex Deficiency.
- Porencephaly and Psychosis: A Rare Case of Neurological and Psychiatric Intersection.
- Are postnatal traumatic events an underestimated cause of porencephalic lesions in dogs and cats?
- Magnetic Resonance and Computed Tomographic Findings in a Case of Encephaloclastic Porencephaly in a Sulawesi Crested Macaque.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:269190(Orphanet)
- MONDO:0017103(MONDO)
- GARD:20987(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q55786814(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.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar