Raras
Buscar doenças, sintomas, genes...
Infantile epileptic spasms syndrome
ORPHA:697160CID-10 · G40.4CID-11 · 8A62.0DOENÇA RARA

Síndrome de Lennox-Gastaut (SLG) ou encefalopatia epilética da infância é uma síndrome epilética pediátrica grave que constitui 1 a 4% das epilepsias da infância. SLG é comumente caracterizada por uma tríade de sintomas: epilepsia de vários padrões, retardo mental (RM) e complexos ponta-onda lentos no eletroencefalograma (EEG). Os padrões de epilepsia mais comuns relacionados à síndrome são tônica axial, atônica, e de ausência, mas convulsões parciais, mioclônicas e tônica-clônica generalizadas também são observadas.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Síndrome rara caracterizada por espasmos epilépticos na infância, frequentemente associada a regressão do desenvolvimento, anormalidades neurológicas e hipsarritmia. Pode apresentar morfologia cutânea alterada e mioclonias, com causas genéticas diversas.

Pesquisas ativas
1 ensaio
2 total registrados no ClinicalTrials.gov
Publicações científicas
223 artigos
Último publicado: 2026 Apr
Medicamentos
10 registrados
GANAXOLONE, CORTICOTROPIN, VIGABATRIN

Tem tratamento?

10 medicamentos registrados
Ver detalhes, fases e interações →
GANAXOLONECORTICOTROPINVIGABATRINPREDNISOLONECANNABIDIOLPYRIDOXINERADIPRODILCPP-115BLARCAMESINE HYDROCHLORIDEBLARCAMESINE
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: G40.4
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (2)
0202010694
Sequenciamento completo do exoma (WES)genetic_test
0301070040
Atendimento em reabilitação — doenças rarasrehabilitation
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

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

Partes do corpo afetadas

🧠
Neurológico
43 sintomas
😀
Face
7 sintomas
🫁
Pulmão
4 sintomas
📏
Crescimento
4 sintomas
💪
Músculos
4 sintomas
🦴
Ossos e articulações
3 sintomas

+ 43 sintomas em outras categorias

Características mais comuns

90%prev.
Espasmos infantis
Muito frequente (99-80%)
90%prev.
Mioclonias
Muito frequente (99-80%)
90%prev.
Regressão do desenvolvimento
Muito frequente (99-80%)
90%prev.
Hipsarritmia
Muito frequente (99-80%)
55%prev.
Morfologia anormal da pele
Frequente (79-30%)
55%prev.
Anormalidade do sistema nervoso
Frequente (79-30%)
118sintomas
Muito frequente (4)
Frequente (2)
Sem dados (112)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 118 características clínicas mais associadas, ordenadas por frequência.

Espasmos infantisInfantile spasms
Muito frequente (99-80%)90%
MiocloniasMyoclonus
Muito frequente (99-80%)90%
Regressão do desenvolvimentoDevelopmental regression
Muito frequente (99-80%)90%
HipsarritmiaHypsarrhythmia
Muito frequente (99-80%)90%
Morfologia anormal da peleAbnormal skin morphology
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico223PubMed
Últimos 10 anos200publicações
Pico202588 papers
Linha do tempo
2026Hoje · 2026🧪 1992Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

13 genes identificados com associação a esta condição.

Autosomal dominantAutosomal recessiveX-linked dominant
ARXHomeobox protein ARXDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor (PubMed:22194193, PubMed:31691806). Binds to specific sequence motif 5'-TAATTA-3' in regulatory elements of target genes, such as histone demethylase KDM5C (PubMed:22194193, PubMed:31691806). Positively modulates transcription of KDM5C (PubMed:31691806). Activates expression of KDM5C synergistically with histone lysine demethylase PHF8 and perhaps in competition with transcription regulator ZNF711; synergy may be related to enrichment of histone H3K4me3 in regulatory element

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Lissencephaly, X-linked 2

A classic type lissencephaly associated with abnormal genitalia. Patients have severe congenital or postnatal microcephaly, lissencephaly, agenesis of the corpus callosum, neonatal-onset intractable epilepsy, poor temperature regulation, chronic diarrhea, and ambiguous or underdeveloped genitalia.

OUTRAS DOENÇAS (9)
X-linked lissencephaly with abnormal genitaliaPartington syndromecorpus callosum agenesis-abnormal genitalia syndromedevelopmental and epileptic encephalopathy, 1
HGNC:18060UniProt:Q96QS3
SPTAN1Spectrin alpha chain, non-erythrocytic 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Fodrin, which seems to be involved in secretion, interacts with calmodulin in a calcium-dependent manner and is thus candidate for the calcium-dependent movement of the cytoskeleton at the membrane

LOCALIZAÇÃO

Cytoplasm, cytoskeletonCytoplasm, cell cortex

VIAS BIOLÓGICAS (10)
RAF/MAP kinase cascadeNCAM signaling for neurite out-growthCOPI-mediated anterograde transportInteraction between L1 and AnkyrinsCaspase-mediated cleavage of cytoskeletal proteins
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 5

A disorder characterized by seizures associated with hypsarrhythmia, profound intellectual disability with lack of visual attention and speech development, as well as spastic quadriplegia.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
345.3 TPM
Cerebelo
315.4 TPM
Nervo tibial
201.2 TPM
Brain Frontal Cortex BA9
191.6 TPM
Córtex cerebral
183.4 TPM
OUTRAS DOENÇAS (4)
neuronopathy, distal hereditary motor, autosomal dominant 11developmental and epileptic encephalopathy, 5developmental delay with or without epilepsyspastic paraplegia 91, autosomal dominant, with or without cerebellar ataxia
HGNC:11273UniProt:Q13813
CNPY3Protein canopy homolog 3Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Toll-like receptor (TLR)-specific co-chaperone for HSP90B1. Required for proper TLR folding, except that of TLR3, and hence controls TLR exit from the endoplasmic reticulum. Consequently, required for both innate and adaptive immune responses (By similarity)

LOCALIZAÇÃO

Endoplasmic reticulum

VIAS BIOLÓGICAS (1)
Trafficking and processing of endosomal TLR
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 60

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent. DEE60 is an autosomal recessive condition characterized by onset of seizures in the first months of life.

OUTRAS DOENÇAS (1)
developmental and epileptic encephalopathy, 60
HGNC:11968UniProt:Q9BT09
GRIN2BGlutamate receptor ionotropic, NMDA 2BDisease-causing germline mutation(s) (gain of function) inAltamente restrito
FUNÇÃO

Component of N-methyl-D-aspartate (NMDA) receptors (NMDARs) that function as heterotetrameric, ligand-gated cation channels with high calcium permeability and voltage-dependent block by Mg(2+) (PubMed:24272827, PubMed:24863970, PubMed:26875626, PubMed:26919761, PubMed:27839871, PubMed:28095420, PubMed:28126851, PubMed:38538865, PubMed:8768735). Participates in synaptic plasticity for learning and memory formation by contributing to the long-term depression (LTD) of hippocampus membrane currents

LOCALIZAÇÃO

Cell membranePostsynaptic cell membraneCell projection, dendriteLate endosomeLysosomeCytoplasm, cytoskeleton

VIAS BIOLÓGICAS (10)
RAF/MAP kinase cascadeRas activation upon Ca2+ influx through NMDA receptorUnblocking of NMDA receptors, glutamate binding and activationLong-term potentiationNegative regulation of NMDA receptor-mediated neuronal transmission
MECANISMO DE DOENÇA

Intellectual developmental disorder, autosomal dominant 6, with or without seizures

A disorder characterized by significantly below average general intellectual functioning associated with impairments in adaptive behavior and manifested during the developmental period. MRD6 additional features may include seizures, hypotonia, abnormal movements, such as dystonia, and autistic features.

EXPRESSÃO TECIDUAL(Tecido-específico)
Brain Frontal Cortex BA9
6.9 TPM
Brain Nucleus accumbens basal ganglia
5.6 TPM
Córtex cerebral
5.1 TPM
Brain Caudate basal ganglia
4.0 TPM
Brain Anterior cingulate cortex BA24
3.8 TPM
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 27intellectual disability, autosomal dominant 6
HGNC:4586UniProt:Q13224
GUF1Translation factor GUF1, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Promotes mitochondrial protein synthesis. May act as a fidelity factor of the translation reaction, by catalyzing a one-codon backward translocation of tRNAs on improperly translocated ribosomes. Binds to mitochondrial ribosomes in a GTP-dependent manner

LOCALIZAÇÃO

Mitochondrion inner membrane

MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 40

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent. DEE40 inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
34.2 TPM
Fibroblastos
26.7 TPM
Músculo esquelético
18.4 TPM
Skin Sun Exposed Lower leg
17.4 TPM
Nervo tibial
16.9 TPM
OUTRAS DOENÇAS (1)
developmental and epileptic encephalopathy, 40
HGNC:25799UniProt:Q8N442
SCN2ASodium channel protein type 2 subunit alphaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Mediates the voltage-dependent sodium ion permeability of excitable membranes. Assuming opened or closed conformations in response to the voltage difference across the membrane, the protein forms a sodium-selective channel through which Na(+) ions may pass in accordance with their electrochemical gradient (PubMed:1325650, PubMed:17021166, PubMed:28256214, PubMed:29844171). Implicated in the regulation of hippocampal replay occurring within sharp wave ripples (SPW-R) important for memory (By simi

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (3)
Interaction between L1 and AnkyrinsPhase 0 - rapid depolarisationSensory perception of sweet, bitter, and umami (glutamate) taste
MECANISMO DE DOENÇA

Seizures, benign familial infantile, 3

A form of benign familial infantile epilepsy, a neurologic disorder characterized by afebrile seizures occurring in clusters during the first year of life, without neurologic sequelae. BFIS3 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
70.3 TPM
Cerebelo
65.2 TPM
Brain Frontal Cortex BA9
19.3 TPM
Córtex cerebral
11.5 TPM
Brain Nucleus accumbens basal ganglia
9.1 TPM
OUTRAS DOENÇAS (10)
seizures, benign familial infantile, 3episodic ataxia, type 9developmental and epileptic encephalopathy, 11complex neurodevelopmental disorder
HGNC:10588UniProt:Q99250
CDKL5Cyclin-dependent kinase-like 5Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Mediates phosphorylation of MECP2 (PubMed:15917271, PubMed:16935860). May regulate ciliogenesis (PubMed:29420175)

LOCALIZAÇÃO

NucleusCytoplasm, cytoskeleton, cilium basal bodyCytoplasm, cytoskeleton, microtubule organizing center, centrosome

OUTRAS DOENÇAS (5)
developmental and epileptic encephalopathy, 2X-linked retinoschisisCDKL5 disorderearly-infantile DEE
HGNC:11411UniProt:O76039
SIK1Serine/threonine-protein kinase SIK1Disease-causing germline mutation(s) inModerado
FUNÇÃO

Serine/threonine-protein kinase involved in various processes such as cell cycle regulation, gluconeogenesis and lipogenesis regulation, muscle growth and differentiation and tumor suppression. Phosphorylates HDAC4, HDAC5, PPME1, SREBF1, CRTC1/TORC1. Inhibits CREB activity by phosphorylating and inhibiting activity of TORCs, the CREB-specific coactivators, like CRTC2/TORC2 and CRTC3/TORC3 in response to cAMP signaling (PubMed:29211348). Acts as a tumor suppressor and plays a key role in p53/TP53

LOCALIZAÇÃO

CytoplasmNucleus

VIAS BIOLÓGICAS (1)
Phosphorylated BMAL1:CLOCK (ARNTL:CLOCK) activates expression of core clock genes
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 30

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent.

EXPRESSÃO TECIDUAL(Tecido-específico)
Skin Not Sun Exposed Suprapubic
14.4 TPM
Skin Sun Exposed Lower leg
10.9 TPM
Ovário
4.6 TPM
Fallopian Tube
4.2 TPM
Tecido adiposo
3.5 TPM
INTERAÇÕES PROTEICAS (5)
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 30early-infantile DEE
HGNC:11142UniProt:P57059
PHACTR1Phosphatase and actin regulator 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Binds actin monomers (G actin) and plays a role in multiple processes including the regulation of actin cytoskeleton dynamics, actin stress fibers formation, cell motility and survival, formation of tubules by endothelial cells, and regulation of PPP1CA activity (PubMed:21798305, PubMed:21939755). Involved in the regulation of cortical neuron migration and dendrite arborization (By similarity)

LOCALIZAÇÃO

CytoplasmSynapseNucleus

MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 70

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent. DEE70 is an autosomal dominant form with onset in first months of life and variable severity.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Nucleus accumbens basal ganglia
25.4 TPM
Brain Frontal Cortex BA9
21.7 TPM
Córtex cerebral
19.6 TPM
Linfócitos
19.0 TPM
Brain Anterior cingulate cortex BA24
15.7 TPM
OUTRAS DOENÇAS (1)
developmental and epileptic encephalopathy, 70
HGNC:20990UniProt:Q9C0D0
PLCB11-phosphatidylinositol 4,5-bisphosphate phosphodiesterase beta-1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Catalyzes the hydrolysis of 1-phosphatidylinositol 4,5-bisphosphate into diacylglycerol (DAG) and inositol 1,4,5-trisphosphate (IP3) and mediates intracellular signaling downstream of G protein-coupled receptors (PubMed:9188725). Regulates the function of the endothelial barrier

LOCALIZAÇÃO

Nucleus membraneCytoplasm

VIAS BIOLÓGICAS (8)
Synthesis of IP3 and IP4 in the cytosolPLC beta mediated eventsG alpha (q) signalling eventsG beta:gamma signalling through PLC betaCa2+ pathway
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 12

A form of epilepsy characterized by frequent tonic seizures or spasms beginning in infancy with a specific EEG finding of suppression-burst patterns, characterized by high-voltage bursts alternating with almost flat suppression phases. Patients may progress to West syndrome, which is characterized by tonic spasms with clustering, arrest of psychomotor development, and hypsarrhythmia on EEG.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Frontal Cortex BA9
16.7 TPM
Brain Caudate basal ganglia
10.1 TPM
Cólon sigmoide
8.6 TPM
Brain Putamen basal ganglia
8.1 TPM
Córtex cerebral
8.0 TPM
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 12malignant migrating partial seizures of infancy
HGNC:15917UniProt:Q9NQ66
WDR45WD repeat domain phosphoinositide-interacting protein 4Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Component of the autophagy machinery that controls the major intracellular degradation process by which cytoplasmic materials are packaged into autophagosomes and delivered to lysosomes for degradation (PubMed:23435086, PubMed:28561066). Binds phosphatidylinositol 3-phosphate (PtdIns3P) (PubMed:28561066). Activated by the STK11/AMPK signaling pathway upon starvation, WDR45 is involved in autophagosome assembly downstream of WIPI2, regulating the size of forming autophagosomes (PubMed:28561066).

LOCALIZAÇÃO

Preautophagosomal structureCytoplasm

VIAS BIOLÓGICAS (1)
Macroautophagy
MECANISMO DE DOENÇA

Neurodegeneration with brain iron accumulation 5

A neurodegenerative disorder associated with iron accumulation in the brain, primarily in the basal ganglia. NBIA5 is characterized by global developmental delay in early childhood that is essentially static, with slow motor and cognitive gains until adolescence or early adulthood. In young adulthood, affected individuals develop progressive dystonia, parkinsonism, extrapyramidal signs, and dementia resulting in severe disability.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Ubíquo)
Cervix Endocervix
73.7 TPM
Útero
67.5 TPM
Tireoide
67.1 TPM
Cervix Ectocervix
64.2 TPM
Fallopian Tube
58.6 TPM
OUTRAS DOENÇAS (1)
neurodegeneration with brain iron accumulation 5
HGNC:28912UniProt:Q9Y484
PIGAPhosphatidylinositol N-acetylglucosaminyltransferase subunit ADisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Catalytic subunit of the glycosylphosphatidylinositol-N-acetylglucosaminyltransferase (GPI-GnT) complex that catalyzes the transfer of N-acetylglucosamine from UDP-N-acetylglucosamine to phosphatidylinositol and participates in the first step of GPI biosynthesis

LOCALIZAÇÃO

Rough endoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
Synthesis of glycosylphosphatidylinositol (GPI)
MECANISMO DE DOENÇA

Paroxysmal nocturnal hemoglobinuria 1

A disorder characterized by hemolytic anemia with hemoglobinuria, thromboses in large vessels, and a deficiency in hematopoiesis. Red blood cell breakdown with release of hemoglobin into the urine is manifested most prominently by dark-colored urine in the morning.

EXPRESSÃO TECIDUAL(Ubíquo)
Pulmão
15.2 TPM
Skin Not Sun Exposed Suprapubic
11.5 TPM
Linfócitos
10.6 TPM
Tireoide
9.4 TPM
Skin Sun Exposed Lower leg
8.8 TPM
OUTRAS DOENÇAS (5)
ferro-cerebro-cutaneous syndromeparoxysmal nocturnal hemoglobinuria 1multiple congenital anomalies-hypotonia-seizures syndrome 2paroxysmal nocturnal hemoglobinuria
HGNC:8957UniProt:P37287
NTRK2BDNF/NT-3 growth factors receptorDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Receptor tyrosine kinase involved in the development and the maturation of the central and the peripheral nervous systems through regulation of neuron survival, proliferation, migration, differentiation, and synapse formation and plasticity (By similarity). Receptor for BDNF/brain-derived neurotrophic factor and NTF4/neurotrophin-4. Alternatively can also bind NTF3/neurotrophin-3 which is less efficient in activating the receptor but regulates neuron survival through NTRK2 (PubMed:15494731, PubM

LOCALIZAÇÃO

Cell membraneEndosome membraneEarly endosome membraneCell projection, axonCell projection, dendriteCytoplasm, perinuclear regionPostsynaptic density

VIAS BIOLÓGICAS (4)
NTF4 activates NTRK2 (TRKB) signalingBDNF activates NTRK2 (TRKB) signalingNTF3 activates NTRK2 (TRKB) signalingActivated NTRK2 signals through FYN
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 58

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent. DEE58 is an autosomal dominant condition characterized by onset of refractory seizures in the first days or months of life.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Anterior cingulate cortex BA24
79.5 TPM
Brain Frontal Cortex BA9
75.0 TPM
Córtex cerebral
74.2 TPM
Brain Caudate basal ganglia
73.0 TPM
Cérebro - Amígdala
73.0 TPM
OUTRAS DOENÇAS (5)
developmental and epileptic encephalopathy, 58obesity, hyperphagia, and developmental delaypilomyxoid astrocytomaearly-onset obesity-hyperphagia-severe developmental delay syndrome
HGNC:8032UniProt:Q16620

Medicamentos e terapias

GANAXOLONEAPPROVAL

Mecanismo: GABA-A receptor; anion channel positive allosteric modulator

CORTICOTROPINAPPROVAL

Mecanismo: Melanocortin receptor 2 agonist

VIGABATRINAPPROVAL

Mecanismo: Gamma-amino-N-butyrate transaminase inhibitor

PREDNISOLONEPHASE_3

Mecanismo: Glucocorticoid receptor agonist

CANNABIDIOLPHASE_3

Mecanismo: Cannabinoid CB1 receptor negative allosteric modulator

PYRIDOXINEPHASE_3
RADIPRODILPHASE_2

Mecanismo: Glutamate NMDA receptor; GRIN1/GRIN2B antagonist

CPP-115PHASE_1
BLARCAMESINE HYDROCHLORIDEPHASE_1
BLARCAMESINEPHASE_1
Ver mais no OpenTargets

Variantes genéticas (ClinVar)

816 variantes patogênicas registradas no ClinVar.

🧬 NTRK2: NM_006180.6(NTRK2):c.1202_1207del (p.Gly401_Thr402del) ()
🧬 NTRK2: NM_006180.6(NTRK2):c.952T>A (p.Phe318Ile) ()
🧬 NTRK2: NM_006180.6(NTRK2):c.5C>T (p.Ser2Leu) ()
🧬 NTRK2: NM_006180.6(NTRK2):c.2405T>C (p.Met802Thr) ()
🧬 NTRK2: NM_006180.6(NTRK2):c.1317dup (p.Ala440fs) ()
Ver todas no ClinVar

Vias biológicas (Reactome)

53 vias biológicas associadas aos genes desta condição.

SLIT2 gene expression is stimulated by ISL1 Primary multipotent pancreatic progenitor cell produces trunk bipotent pancreatic progenitor cell Caspase-mediated cleavage of cytoskeletal proteins Nephrin family interactions NCAM signaling for neurite out-growth Interaction between L1 and Ankyrins RAF/MAP kinase cascade Neutrophil degranulation COPI-mediated anterograde transport RHOU GTPase cycle RHOV GTPase cycle Sensory processing of sound by inner hair cells of the cochlea Sensory processing of sound by outer hair cells of the cochlea Trafficking and processing of endosomal TLR EPHB-mediated forward signaling Unblocking of NMDA receptors, glutamate binding and activation Ras activation upon Ca2+ influx through NMDA receptor Neurexins and neuroligins Synaptic adhesion-like molecules MECP2 regulates neuronal receptors and channels Activated NTRK2 signals through FYN Assembly and cell surface presentation of NMDA receptors Negative regulation of NMDA receptor-mediated neuronal transmission Long-term potentiation Phase 0 - rapid depolarisation Sensory perception of sweet, bitter, and umami (glutamate) taste Transcriptional Regulation by MECP2 Loss of function of MECP2 in Rett syndrome Regulation of MECP2 expression and activity Phosphorylated BMAL1:CLOCK (ARNTL:CLOCK) activates expression of core clock genes PLC beta mediated events Synthesis of IP3 and IP4 in the cytosol Acetylcholine regulates insulin secretion Ca2+ pathway G alpha (q) signalling events G beta:gamma signalling through PLC beta Fatty Acids bound to GPR40 (FFAR1) regulate insulin secretion Presynaptic function of Kainate receptors Macroautophagy Synthesis of glycosylphosphatidylinositol (GPI) PIP3 activates AKT signaling NGF-independant TRKA activation Constitutive Signaling by Aberrant PI3K in Cancer PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling BDNF activates NTRK2 (TRKB) signaling NTF3 activates NTRK2 (TRKB) signaling NTF4 activates NTRK2 (TRKB) signaling Activated NTRK2 signals through RAS Activated NTRK2 signals through PLCG1 Activated NTRK2 signals through PI3K Activated NTRK2 signals through FRS2 and FRS3 NTRK2 activates RAC1 Activated NTRK2 signals through CDK5

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

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Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
3Fase 33
2Fase 22
1Fase 13
·Pré-clínico3
Medicamentos catalogadosEnsaios clínicos· 10 medicamentos · 1 ensaio
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Infantile epileptic spasms syndrome

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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.

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Publicações mais relevantes

🥉Melhor nível de evidência: Relato de caso
Timeline de publicações
223 papers (10 anos)
#1

Effectiveness, tolerability, and retention of the ketogenic diet for infantile epileptic spasms syndrome: a single-center cohort study.

Frontiers in neurology2026

This study aimed to investigate the effectiveness, tolerability, and retention of the ketogenic diet (KD) in patients with infantile epileptic spasms syndrome (IESS). In this single-center prospective cohort study, baseline data were collected from the Children's Hospital Affiliated to Shandong University. Follow-up assessments were conducted at 3, 6, and 12 months after initiating KD. Outcomes included seizure frequency, adverse reactions, and retention rates. Survival analysis was performed to examine the association between retention rates and follow-up duration. A total of 74 patients with IESS were admitted. The seizure response rates were 56.8% (42/74), 41.9% (31/74), and 25.7% (19/74) at 3, 6, and 12 months, respectively. The corresponding seizure-free rates were 13.5% (10/74), 13.5% (10/74), and 8.1% (8/74), respectively. Retention rates at 3, 6, and 12 months were 70.2% (52/74), 43.2% (32/74), and 25.7% (19/74), respectively. Survival analysis indicated that retention rates decreased over time, most markedly within the first 3 months. Responders to the KD exhibited significantly higher retention rates than non-responders throughout the 12 months (HR = 0.35, 95% CI: 0.19-0.64; p < 0.001). Adverse reactions were reported in 20.3% of patients, with gastrointestinal symptoms being the most common (16.2%), including constipation (6.8%), diarrhea (5.4%), and vomiting (4.0%). The KD demonstrated favorable effectiveness and an acceptable safety profile in patients with IESS, particularly in those who did not respond to first-line antiseizure medications (ASMs). The higher retention rates among responders supports its utility in children with IESS. Therefore, early initiation of the KD should be considered for IESS patients who do not respond to first-line ASMs.

#2

Deep learning-based real-time seizure detection and multi-seizure classification on pediatric EEG.

Frontiers in neurology2026

To develop a reliable and accurate seizure detection method using deep learning models capable of detecting and classifying multiple seizure types in real time. We retrospectively collected electroencephalography (EEG) recordings, which were acquired as part of routine diagnostic tests for patients aged 3 months to ≤18 years of age with childhood absence epilepsy, infantile epileptic spasms syndrome, other generalized epilepsy, and focal epilepsy, between January 2018 and December 2022 at Severance Children's Hospital. We used EEG recordings from both seizure and non-seizure patients, which were downsampled to 200 Hz for real-time seizure detection and multi-classification. Of the 199 patients (620 seizures), 49 (297 seizures) belonged to the childhood absence epilepsy group, 16 (200 seizures) to the infantile epileptic spasms syndrome group, 14 (76 seizures) to other generalized epilepsy group, 19 (47 seizures) to focal epilepsy group, and 101 to the normal group. The results showed the best overall performance of AUROC 0.98 and APROC of 0.73 with ResNet with Long-Short Term Network and a 12 s sliding window on real-time seizure detection task. Furthermore, ResNet50 without the frequency bands feature extractor showed the best overall weighted performance for multi-class seizure detection with 0.99 AUROC and 0.99 APPRC. Our approach proposes robust methods which include EEG preprocessing strategy with real-time detection/classification of multiple seizures, which helps monitor pediatric seizure. The result shows that real-time seizure detection can be effectively applied to real-world clinical datasets from a pediatric epilepsy unit with realistic performance and speed.

#3

Analysis of treatment outcome variations in infantile epileptic spasms syndrome.

Frontiers in neurology2026

To explore the key factors influencing outcomes in children with infantile epileptic spasms syndrome (IESS) and to elucidate their interrelationships to provide insights for optimizing clinical practice. This is a retrospective, single-center design study, included children diagnosed with IESS at West China Second Hospital of Sichuan University from April 2019 to April 2024. Descriptive analyses were performed to evaluate genetic and non-genetic etiological subgroups, categorized as structural and unknown causes. Genetic testing results were compared across groups. Pearson correlation and logistic regression analyses were employed to examine differences in treatment efficacy and identify associated risk factors. In this study, 128 children diagnosed with IESS were enrolled and evenly divided into gene-positive and gene-negative groups. The gene-positive group exhibited earlier seizure onset, with a higher prevalence of hypotonia and developmental regression compared to the gene-negative group. Within the gene-negative group, children were further categorized into structural abnormality and unknown causes subgroups, among which EEG hypsarrhythmia was more frequently observed in the structural abnormality subgroup. The gene-positive group showed significantly poorer responses to ACTH, vigabatrin, and other ASMs. ACTH combined with vigabatrin therapy improved outcomes in some of the children. The non-ACTH treatment group demonstrated superior EEG improvement outcomes when compared with the ACTH-treated group (p = 0.028). The overall therapeutic response rate was satisfactory, with 75% in the gene-positive group and 100% in the gene-negative group. In the gene-negative group, frequent seizures and developmental regression emerged as significant risk factors for poor treatment response. The prognosis for IESS remains challenging, with treatment responses closely tied to etiology. Children with genetic etiologies demonstrate poorer responses to ACTH and other ASMs. However, ACTH combination with vigabatrin may improve treatment outcomes in some cases. Our findings suggest that ACTH treatment may not exert a substantial influence on long-term EEG outcomes in children with IESS.

#4

Long-term outcome in children with infantile epileptic spasms syndrome: a multicenter retrospective study in Korea.

Clinical and experimental pediatrics2026 Feb 19

Infantile epileptic spasms syndrome (IESS) is a severe form of infantile epilepsy with a high lifetime morbidity burden. We aimed to assess the long-term epilepsy and neurodevelopmental outcomes based on how children with IESS have been managed over the past few decades. This retrospective multicenter study included children diagnosed with IESS between 1994 and 2021 with a minimum follow-up period of 2 years. Data on demographics, clinical features, medical history, diagnostic evaluations, and treatments used to control spasms were collected. Epilepsy and neurodevelopmental outcomes were assessed at final follow-up. A total of 378 infants with IESS were included. The mean age at onset of spasms was 7.3 (range, 1-24) months and mean follow-up duration was 7.9 (range, 2-28) years. Etiologies were identified in 65.1% of cases, with acquired structural etiologies being the most prevalent (29.9%). Among the genetic and genetic-structural etiologies, tuberous sclerosis complex (n=35), Down syndrome (n=8), Miller-Dieker syndrome (n=3), and 15q duplication syndrome (n=3) were the most common. Vigabatrin was prescribed to 93.9% of the patients, suggesting that it was the mainstay of treatment. At the last follow-up, 77.8% of the children remained on antiseizure medications and 29.1% had drug-resistant epilepsy. Approximately 90% had intellectual disabilities, and half of the eligible individuals had received special education. The IESS imposes a substantial burden on affected children and their families and often leads to chronic epilepsy and impaired cognitive function. Consensus diagnostic and treatment guidelines tailored to the Korean clinical practice are necessary to ensure early diagnosis and timely treatment.

#5

Does First-Line Treatment Impact Outcomes in Trisomy 21-Associated Infantile Epileptic Spasms Syndrome? A Multicenter North American Analysis.

Pediatric neurology2026 Apr

This study aimed to evaluate electroclinical remission and long-term outcomes in children with infantile epileptic spasms syndrome (IESS) associated with trisomy 21 (T21). We hypothesized that initial treatment with hormone therapy would result in higher remission rates compared to treatment with vigabatrin. A retrospective study of T21 and IESS patients was conducted across six North American tertiary pediatric centers. A total of 114 children with IESS were identified. Electroclinical remission without relapse occurred in 31.5% (17/54) of patients receiving hormone therapy as first-line treatment compared with 16.7% (6/36) treated with vigabatrin monotherapy (P = 0.114). Median time to remission was shorter with hormone therapy (41 days) than with vigabatrin (142 days; P < 0.001). Median age at last follow-up was 33 months (IQR 18-83) with a median follow-up duration of 25 months (IQR 11-74). At last follow-up, ongoing epilepsy was present in 30.7% (35/114) and autism spectrum disorder (ASD) in 25.4% (29/114), with similar rates across first-line treatment groups (ongoing epilepsy: hormonal 28.6% vs vigabatrin 30.6%; ASD: 21.4% vs 33.3%; all P > 0.05). Children with T21-associated IESS were approximately two times more likely to achieve electroclinical remission with hormone therapy as first treatment compared to vigabatrin, although this difference did not reach statistical significance. The median time to remission was significantly shorter in children who received hormone therapy as their first treatment compared to those treated with vigabatrin. The initial treatment did not impact long-term clinical outcomes, such as ongoing epilepsy or ASD.

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📚 EuropePMC150 artigos no totalmostrando 196

2026

KCNJ11-associated infantile epileptic spasms syndrome and neonatal diabetes.

Epileptic disorders : international epilepsy journal with videotape
2026

Rethinking Corticosteroid Therapy in Pediatric Neurology.

Journal of inflammation research
2026

Prediction of treatment response in infantile epileptic spasms syndrome using EEG phase-amplitude coupling.

Epilepsia open
2026

Effectiveness, tolerability, and retention of the ketogenic diet for infantile epileptic spasms syndrome: a single-center cohort study.

Frontiers in neurology
2026

Deep learning-based real-time seizure detection and multi-seizure classification on pediatric EEG.

Frontiers in neurology
2026

Case report: A systematic approach for the forensic evaluation of child abuse: lessons from a complex case.

Frontiers in psychiatry
2026

Efficacy and tolerability of early assessment of epileptic spasms to guide sequential treatment in children with infantile epileptic spasms syndrome:A nested case-control Study.

Seizure
2026

Analysis of treatment outcome variations in infantile epileptic spasms syndrome.

Frontiers in neurology
2026

Etiological Analysis and Classification of 108 Patients with Infantile Epileptic Spasms Syndrome Based on the 2017 International League Against Epilepsy Classification.

Noro psikiyatri arsivi
2026

Landscaping evidence on first-line therapies for infantile epileptic spasms syndrome: An umbrella review.

Epilepsy research
2026

Long-term outcome in children with infantile epileptic spasms syndrome: a multicenter retrospective study in Korea.

Clinical and experimental pediatrics
2026

A case of maple syrup urine disease with infantile epileptic spasms syndrome: Challenges in a resource-limited setting.

Pediatrics international : official journal of the Japan Pediatric Society
2026

Does First-Line Treatment Impact Outcomes in Trisomy 21-Associated Infantile Epileptic Spasms Syndrome? A Multicenter North American Analysis.

Pediatric neurology
2026

Spindle density relates to cognitive outcomes in infantile epileptic spasms syndrome with unknown etiology: A retrospective cohort study.

Epilepsia
2026

Automatic recognition of epileptic spasm via large-scale visual AI model.

Seizure
2026

Clinical features of Infantile Epileptic Spasms Syndrome: a systematic review.

Orphanet journal of rare diseases
2026

Targeting CRHR1 Signaling in Experimental Infantile Epileptic Spasms Syndrome: Evidence for Route-Dependent Efficacy.

Children (Basel, Switzerland)
2025

IESS-FusionNet: Physiologically Inspired EEG-EMG Fusion with Linear Recurrent Attention for Infantile Epileptic Spasms Syndrome Detection.

Bioengineering (Basel, Switzerland)
2026

The global research hotspots and future trends of infantile epileptic spasms syndrome: A bibliometric analysis of trends and themes.

Brain &amp; development
2026

Etiology of Infantile Epileptic Spasms Syndrome and Clinical Response With Vigabatrin as the First Treatment.

Pediatric neurology
2026

Cerebral Cavernous Malformations Presenting With Epileptic Spasms in Children.

Pediatric neurology
2025

Prediction of Long-term Prognosis in Infantile Epileptic Spasms Syndrome of Unknown Etiology based on Hypsarrhythmia by Self-Attention Autoencoder.

IEEE journal of biomedical and health informatics
2026

Infantile epileptic spasms syndrome: Mechanisms and therapeutic approaches.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
2026

Distinctive genetic architecture of infantile epileptic spasms syndrome compared to self-limited infantile epilepsy by trios whole-exome sequencing.

Epilepsia open
2025

The acid-base balance homeostasis reflected by urine pH in children with infantile epileptic spasm syndrome may be associated with response to adrenocorticotropic hormone treatment: a retrospective cohort study.

Translational pediatrics
2025

Genetic Etiology of Infantile Spasms in Peruvian Children: A Multicenter Study.

Journal of child neurology
2025

The Enigma of West Syndrome: A Case of Infantile Spasms Without Genetic Clues.

Case reports in pediatrics
2026

Prevalence and incidence of infantile epileptic spasms syndrome in India: A systematic review and meta-analysis.

Epilepsia open
2025

Infantile epileptic spasms syndrome associated with a PTEN variant.

Acta neurologica Belgica
2026

EEG functional connectivity as a marker of evolution from infantile epileptic spasms syndrome to Lennox-Gastaut syndrome.

Epilepsia
2026

Long-term administration of EQU-001 (ivermectin) suppresses spasms in a rat model of infantile epileptic spasms syndrome.

Epilepsy research
2025

Cardiac safety of low-dose ACTH therapy in infantile spasms: Evidence from electrocardiography and advanced echocardiography.

Seizure
2025

[Genetic and clinical characteristics in epilepsy patients with ATP6V1A gene variants].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2025

"Time to Feed the Baby": Should There Be a Paradigm Change in the Treatment of Infantile Epileptic Spasms Syndrome?

Epilepsy currents
2025

Infantile Spasms in Inborn Errors of Metabolism: Diagnostic and Therapeutic Considerations.

Journal of child neurology
2026

Altered Neuronal Networks in Infantile Epileptic Spasms Syndrome: Investigation of Cross-Channel Interactions and Relapse.

IEEE transactions on neural systems and rehabilitation engineering : a publication of the IEEE Engineering in Medicine and Biology Society
2025

Genotypic and phenotypic analysis of epilepsy associated with NPRL2/NPRL3 genes.

Seizure
2025

Understanding the neurobiology of infantile epileptic spasms syndrome (IESS): A comprehensive review.

Seizure
2026

Treatment Practices for Infantile Epileptic Spasms Syndrome: Consensus and Variation in Major Pediatric Epilepsy Centers.

Pediatric neurology
2025

Ketogenic diet for infantile epileptic spasms.

Epilepsia open
2025

One Size Does Not Fit All: Novel Individualized Use of Dexamethasone in Infantile Epileptic Spasms Syndrome.

Neurology and therapy
2025

Severe nonketotic hyperglycinaemia due to a synonymous variant.

Molecular genetics and metabolism reports
2025

Efficacy and safety of vigabatrin as preventive therapy for children with tuberous sclerosis complex: A systematic review and meta-analysis.

Seizure
2025

Novel PAK1 variants related to a variable phenotypic spectrum ranging from mild developmental delay to infantile epileptic spasms syndrome.

Seizure
2025

Synchronized Hypsarrhythmia as a Potential EEG Signature in Genetic Infantile Epileptic Spasms Syndrome: Is there a Gene Network Association?

Indian journal of pediatrics
2025

A Proof-of-Concept Study on Effectiveness of Nitrazepam in Resistant Infantile Epileptic Spasms Syndrome (NitRIS Trial).

Pediatric neurology
2025

Serum matrix metallopeptidase-9 levels in patients with infantile epileptic spasms syndrome before and after the initiation of vigabatrin therapy.

Brain &amp; development
2025

Genetics of infantile epileptic spasms syndrome in China.

Developmental medicine and child neurology
2025

Consanguinity and treatment strategy determine seizure outcome and mortality in infantile epileptic spasms syndrome in Azerbaijan.

Seizure
2025

[Clinical and genetic characteristics analysis of 18 children with infantile epileptic spasms syndrome associated with mitochondrial gene variants].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2025

Epileptic drop attacks: More than just atonic seizures.

Epilepsia open
2025

Distinct T Cell Dysregulation Reflects Disease Severity and Progression in Infantile Epileptic Spasms Syndrome and Lennox-Gastaut Syndrome.

Immune network
2025

Dose optimization of NMDA for rat model of infantile spasms: Approach using EEG, behavior (Seizure) and histopathology.

Behavioural brain research
2025

EEG Scoring Systems in Developmental Epileptic Encephalopathies With and Without Epileptic Spasms: A Comparative Analysis of Reliability and Diagnostic Clarity.

Pediatric neurology
2026

Genetic etiologies of the infantile epileptic spasms syndrome: What do we know so far?

Developmental medicine and child neurology
2025

A Case of Infantile Epileptic Spasms Syndrome with the SPTBN1 Mutation and Review of βII-Spectrin Variants.

Genes
2025

Rapid Electroclinical Evolution in HECW2-Related Developmental and Epileptic Encephalopathy: Report of a Likely Splicing Variant With Familial Transmission.

International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience
2025

Gut Microbiota Composition and Modulation in Developmental and Epileptic Encephalopathies.

The European journal of neuroscience
2025

Upward eye deviation as a precursor to epileptic spasms: A case successfully treated with early corpus callosotomy without adrenocorticotropic hormone therapy.

Epilepsy &amp; behavior reports
2025

An interpretable machine learning approach for predicting drug-resistant epilepsy in children with tuberous sclerosis complex.

Frontiers in neurology
2025

A model-based cost-effectiveness estimates of hormonal therapy in Indian children with infantile epileptic spasms syndrome.

Epilepsia open
2025

The Clinical and Genetic Landscape of a French Multicenter Cohort of 2563 Epilepsy Patients Referred for Genetic Diagnosis.

European journal of neurology
2025

Hippocampal proteomic signatures of the ketogenic diet in a model of infantile epileptic spasms syndrome.

Biochimica et biophysica acta. Molecular basis of disease
2025

Alpha-ERSP as a prognostic biomarker in infantile epileptic spasms syndrome: Insights from quantitative EEG analysis.

Seizure
2026

Vigabatrin Versus Adrenocorticotropic Hormone After High-Dose Prednisolone Failure for the Treatment of Epileptic Spasms.

Journal of child neurology
2026

Beyond Myoclonus-Seizures, Epilepsy Syndromes and Atypical Electroencephalographic (EEG) Findings in Children With Subacute Sclerosing Panencephalitis.

Journal of child neurology
2025

TUBA1A-related tubulinopathy associated with the infantile epileptic spasms syndrome and atypical absence seizures.

Epileptic disorders : international epilepsy journal with videotape
2025

AFG2A-related encephalopathy: Effectiveness of ketogenic diet in epilepsy and mitochondrial dynamics modulation.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2025

Brain tumours as an unrecognized etiology of infantile epileptic spasms syndrome (IESS): The role of resective epilepsy surgery.

Epilepsy &amp; behavior reports
2025

Efficacy and tolerability of magnesium sulfate in children with infantile epileptic spasms syndrome: A systematic review and meta-analysis.

Epilepsy research
2025

Infantile epileptic spasms syndrome as a new phenotype in TOP2B deficiency caused by a de novo variant: a case report and literature review.

Frontiers in pediatrics
2025

Role of the Ketogenic Diet Therapy and ACTH as Second Treatments in Drug-Resistant Infantile Epileptic Spasms Syndrome.

Nutrients
2026

Electroencephalogram (EEG) Spike Metrics Discriminate Impending Epileptic Spasms From Other Seizures in Children With Tuberous Sclerosis Complex: A Pilot Study.

Journal of child neurology
2025

A Novel Gene (UFSP2) Associated with Infantile Epileptic Spasms Syndrome.

Indian journal of pediatrics
2025

Safety, Efficacy, and Tolerability of Ketogenic Diet Versus Adrenocorticotropic Hormone in Infantile Epileptic Spasms Syndrome: A Randomized Controlled Trial.

Pediatric neurology
2025

New variants and genotype-phenotype correlation of PPP3CA-related developmental and epileptic encephalopathy.

Frontiers in neuroscience
2025

Comparative effectiveness of second-line treatments for epileptic spasms.

Epilepsia
2025

Diffusion MRI biomarkers for predicting treatment outcomes in infantile epileptic spasms syndrome with non-lesional MRI.

Seizure
2025

Endocrinological study of low-dose adrenocorticotropic hormone therapy without tapering in infantile epileptic spasms syndrome.

Brain &amp; development
2025

A novel pathogenic variant causing POU3F3-related neurodevelopmental disorder in a child presenting with infantile epileptic spasms syndrome: Expanding the epileptic phenotype.

Seizure
2025

Association Between Scalp High-Frequency Oscillations and Burden of Amplitudes and Epileptiform Discharges (BASED) Scores in Infantile Epileptic Spasms Syndrome.

Biomolecules
2025

Association of serum insulin-like growth factor-1 and adrenocorticotropic hormone therapeutic response in patients with infantile epileptic spasms syndrome.

Frontiers in pharmacology
2025

Genetic etiologies with a large NGS panel in a monocentric cohort of 1000 patients with pediatric onset epilepsies.

Epilepsia open
2025

New insights into epileptic spasm generation and treatment from the TTX animal model.

Epilepsia open
2025

Predictive model for initial response to first-line treatment in children with infantile epileptic spasms syndrome.

Italian journal of pediatrics
2024

Surgical outcomes of drug-refractory infantile epileptic spasms syndrome and related prognostic factors: a retrospective study.

Acta epileptologica
2024

SPOUT1 variants associated with autosomal-recessive developmental and epileptic encephalopathy.

Acta epileptologica
2025

Infantile Epileptic Spasms Syndrome Due to Neonatal Hypoglycemic Brain Injury: A Retrospective Audit.

Indian pediatrics
2025

COVID-19 infection and vaccination in children with Dravet syndrome or infantile epileptic spasms syndrome: An internet survey in Japan.

Brain &amp; development
2025

Clinical Features and Predictors of Unfavorable Outcomes in Infantile Epileptic Spasms Syndrome: Results from a Multiancestry Cohort.

Journal of child neurology
2025

Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms.

Annals of clinical and translational neurology
2025

Aetiopathogenesis of infantile epileptic spasms syndrome and mechanisms of action of adACTH/corticosteroids in children: A scoping review.

Developmental medicine and child neurology
2025

Identification of topological alterations using microstate dynamics in patients with infantile epileptic spasms syndrome.

Scientific reports
2025

Relationship between the time course of Burden of Amplitudes and Epileptiform Discharges scores and relapse in children with infantile epileptic spasms syndrome.

Epilepsia
2026

Modeling epilepsy by loss-of-function of the CUG-binding protein Elav-like family member 2 in zebrafish with multi-omics analysis.

Chinese medical journal
2025

Factors Influencing the Response of Patients with Infantile Epileptic Spasms Syndrome to ACTH as Repeated First-Line Therapy.

Neurology and therapy
2025

Evidence of thalamocortical network activation during epileptic spasms: A thalamic stereotactic EEG study.

Epilepsia
2024

Contribution of Cross-Phase-Amplitude Coupling to Relapse in Infantile Epileptic Spasms Syndrome.

Annual International Conference of the IEEE Engineering in Medicine and Biology Society. IEEE Engineering in Medicine and Biology Society. Annual International Conference
2024

Long-short term memory autoencoder using delta with beta bands of EEG enables highly accurate prediction of seizure outcome in Infantile Epileptic Spasms Syndrome of unknown etiology.

Annual International Conference of the IEEE Engineering in Medicine and Biology Society. IEEE Engineering in Medicine and Biology Society. Annual International Conference
2025

Aetiopathogenesis of infantile epileptic spasms syndrome and mechanisms of action of adrenocorticotrophin hormone/corticosteroids in children: A scoping review.

Developmental medicine and child neurology
2025

Treatment efficacy for infantile epileptic spasms syndrome in children with trisomy 21.

Frontiers in pediatrics
2025

A Multicenter Training and Interrater Reliability Study of the BASED Score for Infantile Epileptic Spasms Syndrome.

Journal of clinical neurophysiology : official publication of the American Electroencephalographic Society
2025

Disparities in the utilization of genetic testing for non-acquired infantile epileptic spasms syndrome in a single healthcare center in North Carolina.

Epilepsia open
2025

Evaluating the effectiveness of medical therapy in infantile epileptic spasms syndrome due to surgically-remediable lesions.

Epilepsia
2025

Infantile Epileptic Spasms Syndrome: Unveiling clinical and genetic variability in a case series from Argentina.

Seizure
2025

The genetic landscape and classification of infantile epileptic spasms syndrome requiring surgery due to suspected focal brain malformations.

Brain communications
2025

Integrated analysis of proteomics and metabolomics in infantile epileptic spasms syndrome.

Scientific reports
2025

Adrenocorticotropic Hormone versus Prednisolone for Infantile Epileptic Spasms Syndrome: A Systematic Review and Economic Evaluation.

Neuropediatrics
2024

Infantile epileptic spasms syndrome: an etiologic study of 361 patients with infantile epileptic spasms syndrome.

Frontiers in pediatrics
2025

Favorable response to ketogenic diet therapy in a patient with DYNC1H1-related epilepsy.

Epilepsy &amp; behavior reports
2025

Ventricular tachycardia induced by adrenocorticotropic hormone therapy in infantile epileptic spasms syndrome: A case report.

Seizure
2024

Outcomes following resective and disconnective strategies in the treatment of epileptic spasms: a systematic review of the literature and individual patient data meta-analysis.

Frontiers in neurology
2025

Short-Term Effectiveness and Tolerability of Vigabatrin Therapy in Infantile Epileptic Spasms Syndrome.

Indian journal of pediatrics
2025

Quantitative analysis of 123I-iomazenil single-photon emission computed tomography findings from patients with infantile epileptic spasm syndrome.

Brain &amp; development
2025

Elementary school enrollment after ACTH therapy for patients with infantile epileptic spasms syndrome.

Seizure
2025

Abnormalities in brain magnetic resonance imaging associated with vigabatrin therapy in an infant with infantile epileptic spasms syndrome.

Clinical toxicology (Philadelphia, Pa.)
2024

A Multicenter Retrospective Observational Cohort Study of Seizure Freedom before Lennox-Gastaut Syndrome (the "Gap"). Opportunities for Prevention.

medRxiv : the preprint server for health sciences
2025

Ketogenic diet therapy for the treatment of pediatric epilepsy.

Epileptic disorders : international epilepsy journal with videotape
2025

Evaluating the patient needs and tolerability of Clobazam liquid formulation (Likozam® 1 mg/mL): A French patient and care-givers' centered survey.

Epilepsy &amp; behavior : E&amp;B
2025

Infantile epileptic spasms syndrome: When spasms come out of the blue.

Epilepsy &amp; behavior : E&amp;B
2024

Optimal lead time for treatment of infantile epileptic spasms syndrome-a secondary data analysis.

Translational pediatrics
2025

Diagnostic yields of genetic testing and related benefits in infantile epileptic spasms syndrome: A systematic review and meta-analysis.

Seizure
2024

Corpus callosotomy for refractory epileptic spasms: Systematic review and meta-analysis.

Seizure
2025

Symptomatic vigabatrin-associated MRI toxicity is associated with simultaneous hormonal therapy among patients with infantile spasms.

Epilepsia open
2024

Mean global field power is reduced in infantile epileptic spasms syndrome after response to vigabatrin.

Frontiers in neurology
2025

IGF-1 impacts neocortical interneuron connectivity in epileptic spasm generation and resolution.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
2024

First-choice hormonal therapies for children with infantile epileptic spasms syndrome in South Asia: A network meta-analysis of randomized controlled trials.

Epilepsia open
2025

Two familial cases of infantile epileptic spasms syndrome associated with UDP-glucose-6-dehydrogenase deficiency.

Epileptic disorders : international epilepsy journal with videotape
2024

Characteristics of overnight video-EEG monitoring in infantile epileptic spasms syndrome at 2-week follow-up.

Epilepsia
2024

Impact of Bacille Calmette-Guérin vaccination on the therapeutic schedule of infantile epileptic spasms syndrome: A 25-year Japanese single-center survey.

Vaccine: X
2024

Vitamin B12 responsive developmental and epileptic encephalopathy due to a novel mutation in the FUT2 gene: a case report.

BMC pediatrics
2024

Serum matrix metallopeptidase-9 levels in infantile epileptic spasms syndrome of unknown etiology.

Epilepsy research
2024

Factors influencing efficacy and relapse of adrenocorticotropic hormone in infantile epileptic spasms syndrome.

Epilepsy &amp; behavior : E&amp;B
2024

Predictive modeling based on functional connectivity of interictal scalp EEG for infantile epileptic spasms syndrome.

Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology
2024

Characterization of the Epileptogenic Phenotype and Response to Antiseizure Medications in Lissencephaly Patients.

Neuropediatrics
2024

Epilepsy surgery in developmental and epileptic encephalopathies.

Epilepsy &amp; behavior : E&amp;B
2024

Adrenocorticotropic hormone therapy alters Q-albumin ratios in patients with infantile epileptic spasms syndrome of unknown etiology.

Journal of the neurological sciences
2024

Mitigating treatment lag for infantile epileptic spasms syndrome in low- and middle-income countries: Key recommendations from the South Asia allied IESS Research Group.

Epilepsia
2024

Genotypic and phenotypic analysis of Korean patients with tuberous sclerosis complex.

Neurogenetics
2024

Have epilepsy outcomes changed for children with tuberous sclerosis complex in Queensland, Australia?

Epilepsia
2024

Clinical features and genotype-phenotype correlations in epilepsy patients with de novo DYNC1H1 variants.

Epilepsia
2024

A retrospective study of the yield of next-generation sequencing in the diagnosis of developmental and epileptic encephalopathies and epileptic encephalopathies in 0-12 years aged children at a single tertiary care hospital in South India.

Epileptic disorders : international epilepsy journal with videotape
2024

Electroclinical Features of Infantile Epileptic Spasms Syndrome.

Annals of Indian Academy of Neurology
2024

The Utility of Genetic Testing in Infantile Epileptic Spasms Syndrome: A Step-Based Approach in the Next-Generation Sequencing Era.

Pediatric neurology
2024

Infantile Epileptic Spasms Syndrome Complicated by Leigh Syndrome and Leigh-Like Syndrome: A Retrospective, Nationwide, Multicenter Case Series.

Pediatric neurology
2024

Risk of autism spectrum disorder in children with infantile epileptic spasms syndrome: a retrospective study in a single center in Brazil.

Jornal de pediatria
2024

Identification and treatment of surgically-remediable causes of infantile epileptic spasms syndrome.

Expert review of neurotherapeutics
2024

Early Response, Long-Term Seizure Outcome, and Very-Low-Dose Adrenocorticotrophic Hormone Therapy for Infantile Epileptic Spasms Syndrome With Down Syndrome.

Pediatric neurology
2024

Population-based study of rare epilepsy incidence in a US urban population.

Epilepsia
2024

Epilepsies with onset during the first year of life: A prospective study on syndromes, etiologies, and outcomes.

Epilepsia open
2024

Total Corpus Callosotomy via Posterior Approach with Endoscopic-Alone Technique.

World neurosurgery
2024

Infantile epileptic spasms syndrome in a child with lissencephaly associated with de novo PAFAH1B1 variant and coincidental CMV infection.

Epilepsy &amp; behavior reports
2024

Epileptic spasms relapse is associated with response latency but not conventional attributes of post-treatment EEG.

Epilepsia open
2024

Electrographic screening for infantile epileptic spasms syndrome in a single sleep-wake cycle.

Epilepsia
2024

Novel copy number variations and phenotypes of infantile epileptic spasms syndrome.

Clinical genetics
2024

Genetic Advancements in Infantile Epileptic Spasms Syndrome and Opportunities for Precision Medicine.

Genes
2024

Diagnosis and management of infantile epileptic spasms syndrome (IESS) in Gulf Cooperation Council (GCC) countries: Expert consensus statement.

Seizure
2024

Real-life data comparing the efficacy of vigabatrin and oral steroids given sequentially or combined for infantile epileptic spasms syndrome.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2024

Efficacy and safety of corticosteroids and ACTH in epileptic syndromes beyond Infantile Epileptic Spasms Syndrome (IESS): A systematic review and meta-analysis.

Epilepsia
2024

Severe Hemolytic Anemia: Atypical Presentation of Cobalamin Deficiency.

Journal of pediatric hematology/oncology
2024

A Potential Mechanism of Neurological Impairment in Children With Infantile Spasm: Based on Microanatomic Structure Analysis Employing Voxel-Based Morphometry and Surface-Based Morphometry.

Pediatric neurology
2024

Timing the clinical onset of epileptic spasms in infantile epileptic spasms syndrome: A tertiary health center's experience.

Epilepsia
2025

Electroclinical Landscape of Infantile Epileptic Spasms Syndrome.

Indian journal of pediatrics
2024

Genotype and phenotype correlation of PHACTR1-related neurological disorders.

Journal of medical genetics
2024

Purified cannabidiol as add-on therapy in children with treatment-resistant infantile epileptic spasms syndrome.

Seizure
2024

Initial combination versus early sequential standard therapies for Infantile Epileptic Spasms Syndrome-Feedback from stakeholders.

Epilepsia open
2024

Societal costs of illness for infantile epileptic spasms syndrome and evolutionary cost prediction in the era of WHO's IGAP.

Epilepsy research
2024

Super-Refractory Status Epilepticus Progressing to Infantile Epileptic Spasms Syndrome Secondary to Very Long Chain Acyl-CoA Dehydrogenase Deficiency.

Indian journal of pediatrics
2024

Does the absence or presence of sleep spindles on EEG have prognostic value for cognitive outcome in children with infantile epileptic spasms syndrome? A systematic literature review.

Epileptic disorders : international epilepsy journal with videotape
2024

Adrenocorticotropic hormone combined with magnesium sulfate therapy for infantile epileptic spasms syndrome: a real-world study.

World journal of pediatrics : WJP
2023

Infantile epileptic spasms syndrome: a cohort study of 88 children.

Italian journal of pediatrics
2024

Mechanisms of infantile epileptic spasms syndrome: What have we learned from animal models?

Epilepsia
2024

Delays to care in infantile epileptic spasms syndrome: Racial and ethnic inequities.

Epilepsia
2023

Clinical and electroencephalographic characteristics of 34 infant with onset of epileptic spasms before three months of age.

Epilepsy &amp; behavior : E&amp;B
2023

Crucial involvement of fast waves and Delta band in the brain network attributes of infantile epileptic spasms syndrome.

Frontiers in pediatrics
2023

Case report: De novo variant of SETD1A causes infantile epileptic spasms syndrome.

Frontiers in neurology
2023

The Response of Synthetic Adrenocorticotropic Hormone (ACTH) Treatment in Pediatric Drug-Resistant Epilepsy Other Than Infantile Epileptic Spasms Syndrome: A Retrospective Observational Study.

Cureus
2024

Safety and efficacy of melatonin supplementation as an add-on treatment for infantile epileptic spasms syndrome: A randomized, placebo-controlled, double-blind trial.

Journal of pineal research
2024

Fifteen years of real-world data on the use of vigabatrin in individuals with infantile epileptic spasms syndrome.

Epilepsia
2023

Brazilian experts' consensus on the treatment of infantile epileptic spasm syndrome in infants.

Arquivos de neuro-psiquiatria
2024

Clinical phenotype and genotype of NPRL2-related epilepsy: Four cases reports and literature review.

Seizure
2023

Long-term survival and factors associated with mortality among children with infantile epileptic spasms syndrome - A retrospective cohort study.

Seizure
2023

A case of infantile epileptic spasms syndrome and autism spectrum disorder with an RFX3 mutation.

Seizure
2023

Aicardi Syndrome Is a Genetically Heterogeneous Disorder.

Genes
2023

Landscape of genetic infantile epileptic spasms syndrome-A multicenter cohort of 124 children from India.

Epilepsia open
2023

Prediction model for long-term seizure and developmental outcomes among children with infantile epileptic spasms syndrome.

Frontiers in neurology
2023

Identifying risk factors for adverse events of pyridoxal phosphate in infantile epileptic spasms syndrome.

Epilepsy &amp; behavior : E&amp;B
2023

Care of Children with Infantile Epileptic Spasms Syndrome and Applicability of Telemedicine Amidst the COVID-19 Pandemic.

Indian journal of pediatrics
2023

A case-control evaluation of Spasm control and Tolerability of the Modified Atkins diet versus classic ketogenic diet in Chinese Children with infantile epileptic spasms syndrome.

Seizure
2023

The landscape of infantile epileptic spasms syndrome in South Asia: peculiarities, challenges, and way forward.

The Lancet regional health. Southeast Asia
2023

What Should be the Next Choice After Failure of Hormonal and Vigabatrin Therapy in Infantile Epileptic Spasms Syndrome?

Indian journal of pediatrics
2023

Nutritional vitamin B12 deficiency-associated Infantile epileptic spasms syndrome: Clinico-neurophysiological presentation, response to treatment, and neurodevelopmental outcome.

Seizure
2023

Long-term analysis of adrenocorticotropic hormone monotherapy for infantile epileptic spasms syndrome with periventricular leukomalacia.

Seizure
2023

The epileptology of Wiedemann-Steiner syndrome: Electroclinical findings in five patients with KMT2A pathogenic variants.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2023

The epilepsy phenotype of ST3GAL3-related developmental and epileptic encephalopathy.

Epilepsia open
2023

Mutations in plasticity-related-gene-1 (PRG-1) protein contribute to hippocampal seizure susceptibility and modify epileptic phenotype.

Cerebral cortex (New York, N.Y. : 1991)

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

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. Effectiveness, tolerability, and retention of the ketogenic diet for infantile epileptic spasms syndrome: a single-center cohort study.
    Frontiers in neurology· 2026· PMID 41821635mais citado
  2. Deep learning-based real-time seizure detection and multi-seizure classification on pediatric EEG.
    Frontiers in neurology· 2026· PMID 41809189mais citado
  3. Analysis of treatment outcome variations in infantile epileptic spasms syndrome.
    Frontiers in neurology· 2026· PMID 41783846mais citado
  4. Long-term outcome in children with infantile epileptic spasms syndrome: a multicenter retrospective study in Korea.
    Clinical and experimental pediatrics· 2026· PMID 41713210mais citado
  5. Does First-Line Treatment Impact Outcomes in Trisomy 21-Associated Infantile Epileptic Spasms Syndrome? A Multicenter North American Analysis.
    Pediatric neurology· 2026· PMID 41689988mais citado
  6. Elevated Serum Concentrations of Lacosamide and Perampanel in a Pediatric Patient Following Coadministration of Clarithromycin for Mycoplasma pneumoniae Pneumonia.
    J Pediatr Pharmacol Ther· 2026· PMID 41983010recente
  7. AFG2A-related encephalopathy, expanding the neurodevelopmental and epileptic spectrum.
    Orphanet J Rare Dis· 2026· PMID 41933351recente
  8. DTaP-induced infantile epileptic spasms syndrome high-risk window: Insights from school-age children.
    Hum Vaccin Immunother· 2026· PMID 41910094recente
  9. A randomized controlled double-blind study on the brain protection of infantile patients with epileptic spasm syndrome through atomized inhalation of hydrogen-oxygen gas.
    Front Neurol· 2026· PMID 41908285recente
  10. KCNJ11-associated infantile epileptic spasms syndrome and neonatal diabetes.
    Epileptic Disord· 2026· PMID 41874548recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:697160(Orphanet)
  2. MONDO:0018097(MONDO)
  3. GARD:7887(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Artigo Wikipedia(Wikipedia)

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

Infantile epileptic spasms syndrome
Compêndio · Raras BR

Infantile epileptic spasms syndrome

ORPHA:697160 · MONDO:0018097
CID-10
G40.4 · Outras epilepsias e síndromes epilépticas generalizadas
CID-11
Ensaios
1 ativos
Medicamentos
10 registrados
MedGen
Repurposing
20 candidatos
adiphenineacetylcholine receptor antagonist
baclofenbenzodiazepine receptor agonist
chlorphensin-carbamatemuscle relaxant
+17 outros
EuropePMC
Wikipedia
Papers 10a
Evidência
🥉 Relato de caso
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