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Insônia fatal esporádica
ORPHA:586130CID-10 · A81.9CID-11 · 6D85.5DOENÇA RARA

Insônia fatal é uma doença neurodegenerativa hereditária ou esporádica causada pela alteração dos PrPC.

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Introdução

O que você precisa saber de cara

📋

Doença priônica neurodegenerativa rara e fatal, caracterizada por insônia progressiva e intratável, disautonomia e declínio neurológico. Ocorre esporadicamente, sem histórico familiar conhecido.

Publicações científicas
46 artigos
Último publicado: 2024 Dec

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
<1 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Casos conhecidos
27
pacientes catalogados
Início
Adult
+ elderly
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: A81.9
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Entender a doença

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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 pesquisa1desde 2025
Total histórico46PubMed
Últimos 10 anos20publicações
Pico20194 papers
Linha do tempo
2025Hoje · 2026🧪 2005Primeiro ensaio clínico📈 2019Ano 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

🧬

Nenhum gene associado encontrado

Os dados genéticos desta condição ainda estão sendo catalogados.

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

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.
2Fase 21
Medicamentos catalogadosEnsaios clínicos· 0 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 — Insônia fatal esporádica

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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.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

0 ensaios clínicos encontrados.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

Timeline de publicações
20 papers (10 anos)
#1

Sporadic fatal insomnia.

Practical neurology2025 Mar 14

We describe a 63-year-old man diagnosed with sporadic Creutzfeldt-Jakob disease (sCJD), specifically sporadic fatal insomnia, confirmed through real-time quaking-induced conversion (RT-QuIC) analysis of cerebrospinal fluid and polysomnography. He presented with rapid cognitive decline, behavioural changes, sleep disturbances and dysautonomic symptoms. Initial MR imaging, electroencephalogram and cerebrospinal fluid analyses were inconclusive, highlighting the difficulty in diagnosing this rare subtype of CJD. Clinical evaluation is fundamental in defining the diagnosis of sCJD. When clinical suspicion is strong, the diagnostic work-up should be continued. In this case, the combination of comprehensive clinical evaluations and advanced diagnostic tools, including RT-QuIC and polysomnography, proved essential in making a definitive diagnosis.

#2

Efficient transmission of human prion diseases to a glycan-free prion protein-expressing host.

Brain : a journal of neurology2024 Apr 04

It is increasingly evident that the association of glycans with the prion protein (PrP), a major post-translational modification, significantly impacts the pathogenesis of prion diseases. A recent bioassay study has provided evidence that the presence of PrP glycans decreases spongiform degeneration and disease-related PrP (PrPD) deposition in a murine model. We challenged (PRNPN181Q/197Q) transgenic (Tg) mice expressing glycan-free human PrP (TgGlyc-), with isolates from sporadic Creutzfeldt-Jakob disease subtype MM2 (sCJDMM2), sporadic fatal insomnia and familial fatal insomnia, three human prion diseases that are distinct but share histotypic and PrPD features. TgGlyc- mice accurately replicated the basic histotypic features associated with the three diseases but the transmission was characterized by high attack rates, shortened incubation periods and a greatly increased severity of the histopathology, including the presence of up to 40 times higher quantities of PrPD that formed prominent deposits. Although the engineered protease-resistant PrPD shared at least some features of the secondary structure and the presence of the anchorless PrPD variant with the wild-type PrPD, it exhibited different density gradient profiles of the PrPD aggregates and a higher stability index. The severity of the histopathological features including PrP deposition appeared to be related to the incubation period duration. These findings are clearly consistent with the protective role of the PrP glycans but also emphasize the complexity of the conformational changes that impact PrPD following glycan knockout. Future studies will determine whether these features apply broadly to other human prion diseases or are PrPD-type dependent.

#3

Sporadic Creutzfeldt-Jakob Disease: A Case Report and Literature Review.

Cureus2024 Dec

Prion disease is an uncommon entity characterized by exceptionally rapid neurodegenerative deterioration. There are three categories of prion disease: (1) sporadic: sporadic Creutzfeldt-Jakob disease (sCJD), sporadic fatal insomnia, and protease-sensitive prionopathy; (2) genetic: genetic Creutzfeldt-Jakob disease, familial fatal insomnia, and Gerstmann-Sträussler-Scheinker syndrome; and (3) acquired: Kuru, iatrogenic Creutzfeldt-Jakob disease, and variant Creutzfeldt-Jakob disease. Although it is an incurable disease, a specific pathophysiological mechanism exists involving neuronal loss, glial cell proliferation, absence of inflammatory response, development of vacuoles leading to a spongiform appearance, and the presence of prions. This case report describes the approach to a patient with progressive cognitive deterioration, later developing motor ataxia and difficulty in language expression. The patient was hospitalized for the diagnostic approach of autoimmune or paraneoplastic encephalitis at Hospital Ángeles del Pedregal, Mexico City, Mexico, with poor response to medical treatment and clinical worsening. Finally, a diagnosis of Creutzfeldt-Jakob disease was concluded through cerebrospinal fluid analysis. This demonstrates the diagnostic challenge this entity presents.

#4

Sporadic fatal insomnia: a rapidly progressive phenotype resembling progressive supranuclear palsy.

Neurologia2024
#5

Clinical profiles and ethnic heterogeneity of sporadic fatal insomnia.

European journal of neurology2023 Apr

This study was undertaken to elucidate the clinical profile of sporadic fatal insomnia (sFI), assess the similarities and differences between sFI and fatal familial insomnia (FFI), and evaluate the influence of ethnicity on the phenotype of sFI patients. The data of sFI and FFI patients were retrieved from our case series and through literature review. The clinical and diagnostic features of sFI and FFI were compared, as were the phenotypes of Asian and Caucasian sFI patients. We identified 44 sFI and 157 FFI cases. The prevalence of sleep-related, neuropsychiatric, and autonomic symptoms among the sFI patients were 65.9%, 100.0%, and 43.2%, respectively. Compared to FFI, sFI exhibited longer disease duration and a higher proportion of neuropsychiatric symptoms, whereas FFI was characterized by a higher incidence of sleep-related and autonomic symptoms in the early stages of the disease or throughout its course. In addition, a higher proportion of the sFI patients showed hyperintensity on magnetic resonance imaging (MRI) and periodic sharp wave complexes on electroencephalography compared to the FFI patients, especially those presenting with pathological changes associated with MM2-cortical type sporadic Creutzfeldt-Jakob disease. The Asian sFI patients had a higher proportion of males and positivity for cerebrospinal fluid 14-3-3 protein, and fewer sleep-related symptoms compared to Caucasian sFI patients. The age at onset and duration of sFI differed between ethnic groups, but the difference failed to reach statistical significance. Despite its similarities to FFI, sFI is characterized by longer disease duration, higher proportion of neuropsychiatric symptoms, and hyperintensity on MRI, along with differences in the clinical characteristics based on ethnicity.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC25 artigos no totalmostrando 17

2024

Sporadic Creutzfeldt-Jakob Disease: A Case Report and Literature Review.

Cureus
2024

Sporadic fatal insomnia: a rapidly progressive phenotype resembling progressive supranuclear palsy.

Neurologia
2024

Efficient transmission of human prion diseases to a glycan-free prion protein-expressing host.

Brain : a journal of neurology
2023

Sporadic Fatal Insomnia presenting with agrypnia excitata and rapidly progressive dementia: a case report.

Journal of neurology
2023

Clinical profiles and ethnic heterogeneity of sporadic fatal insomnia.

European journal of neurology
2022

Sporadic Fatal Insomnia Presenting with Initial Symptoms of Parkinsonism and Abnormal Dopamine Transporter Imaging.

Movement disorders clinical practice
2021

Sporadic Creutzfeldt-Jakob Disease Appears to Be Sporadic Fatal Insomnia: A Case Report and Review of the Literature.

Nature and science of sleep
2019

Two distinct prions in fatal familial insomnia and its sporadic form.

Brain communications
2020

Diagnosis of prion diseases by RT-QuIC results in improved surveillance.

Neurology
2019

Molecular Characterization of the Danish Prion Diseases Cohort With Special Emphasis on Rare and Unique Cases.

Journal of neuropathology and experimental neurology
2019

Cortical and bithalamic hypometabolism by FDG-PET/CT in a patient with sporadic fatal insomnia.

Neurology
2018

Sporadic Fatal Insomnia in Europe: Phenotypic Features and Diagnostic Challenges.

Annals of neurology
2018

Fatal familial insomnia and sporadic fatal insomnia.

Handbook of clinical neurology
2018

Variably protease-sensitive prionopathy.

Handbook of clinical neurology
2017

A review of drug therapy for sporadic fatal insomnia.

Prion
2017

Novel strain properties distinguishing sporadic prion diseases sharing prion protein genotype and prion type.

Scientific reports
2016

Human prion diseases: surgical lessons learned from iatrogenic prion transmission.

Neurosurgical focus
Ver todos os 25 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

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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. Sporadic fatal insomnia.
    Practical neurology· 2025· PMID 39322409mais citado
  2. Efficient transmission of human prion diseases to a glycan-free prion protein-expressing host.
    Brain : a journal of neurology· 2024· PMID 38000783mais citado
  3. Sporadic Creutzfeldt-Jakob Disease: A Case Report and Literature Review.
    Cureus· 2024· PMID 39881893mais citado
  4. Sporadic fatal insomnia: a rapidly progressive phenotype resembling progressive supranuclear palsy.
    Neurologia· 2024· PMID 38387778mais citado
  5. Clinical profiles and ethnic heterogeneity of sporadic fatal insomnia.
    European journal of neurology· 2023· PMID 36617541mais citado
  6. Correction to: Sporadic Fatal Insomnia presenting with agrypnia excitata and rapidly progressive dementia: a case report.
    J Neurol· 2023· PMID 36976330recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:586130(Orphanet)
  2. MONDO:0035614(MONDO)
  3. GARD:22349(GARD (NIH))
  4. Busca completa no PubMed(PubMed)
  5. 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

Insônia fatal esporádica
Compêndio · Raras BR

Insônia fatal esporádica

ORPHA:586130 · MONDO:0035614
Prevalência
<1 / 1 000 000
Casos
27 casos conhecidos
CID-10
A81.9 · Infecção não especificada do sistema nervosos central por vírus atípicos
CID-11
Início
Adult, Elderly
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C5567889
Repurposing
6 candidatos
etizolambenzodiazepine receptor agonist
inositol-hexanicotinatedopamine receptor antagonist
L-glutaminemelatonin receptor agonist
+3 outros
EuropePMC
Wikipedia
Papers 10a
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