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Miastenia gravis com início no adulto
ORPHA:391490CID-10 · G70.0CID-11 · 8C60PCDT · SUSDOENÇA RARA

A miastenia gravis adquirida (MG) é uma doença autoimune da junção neuromuscular caracterizada por fraqueza muscular fatigável com sinais oculares frequentes e/ou fraqueza muscular generalizada, e ocasionalmente associada a timoma.

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Introdução

O que você precisa saber de cara

📋

A miastenia gravis adquirida (MG) é uma doença autoimune da junção neuromuscular caracterizada por fraqueza muscular fatigável com sinais oculares frequentes e/ou fraqueza muscular generalizada, e ocasionalmente associada a timoma.

Publicações científicas
8 artigos
Último publicado: 2020 Dec 15

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-5 / 10 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
31.8
Serbia
Início
Adult
🏥
SUS: Cobertura parcialScore: 65%
PCDT disponível4 medicamentos CEAFCID-10: G70.0
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (2)
0202010694
Sequenciamento completo do exoma (WES)genetic_test
0301070040
Atendimento em reabilitação — doenças rarasrehabilitation
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Sinais e sintomas

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

Características mais comuns

Início na idade adulta
1sintomas
Sem dados (1)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 1 características clínicas mais associadas, ordenadas por frequência.

Início na idade adultaAdult onset

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa6desde 2020
Total histórico8PubMed
Últimos 10 anos4publicações
Pico20192 papers
Linha do tempo
20202020Hoje · 2026🧪 2010Primeiro ensaio clínico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

3 genes identificados com associação a esta condição. Padrão de herança: Multigenic/multifactorial, Not applicable.

CTLA4Cytotoxic T-lymphocyte protein 4Major susceptibility factor inAltamente restrito
FUNÇÃO

Inhibitory receptor acting as a major negative regulator of T-cell responses (PubMed:11279501, PubMed:11279502, PubMed:16551244, PubMed:1714933, PubMed:18641304, PubMed:28484017). Acts as a decoy receptor: the affinity of CTLA4 for its natural B7 family ligands, CD80 and CD86, is considerably stronger than the affinity of their cognate stimulatory coreceptor CD28 (PubMed:11279501, PubMed:11279502, PubMed:16551244, PubMed:1714933, PubMed:28484017)

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (3)
Co-stimulation by CD28Co-inhibition by CTLA4RUNX1 and FOXP3 control the development of regulatory T lymphocytes (Tregs)
MECANISMO DE DOENÇA

Systemic lupus erythematosus

A chronic, relapsing, inflammatory, and often febrile multisystemic disorder of connective tissue, characterized principally by involvement of the skin, joints, kidneys and serosal membranes. It is of unknown etiology, but is thought to represent a failure of the regulatory mechanisms of the autoimmune system. The disease is marked by a wide range of system dysfunctions, an elevated erythrocyte sedimentation rate, and the formation of LE cells in the blood or bone marrow.

EXPRESSÃO TECIDUAL(Tecido-específico)
Baço
5.8 TPM
Intestino delgado
4.9 TPM
Pulmão
4.3 TPM
Testículo
2.5 TPM
Sangue
1.8 TPM
OUTRAS DOENÇAS (6)
autoimmune lymphoproliferative syndrome due to CTLA4 haploinsufficiencysystemic lupus erythematosusgranulomatosis with polyangiitismycosis fungoides
HGNC:2505UniProt:P16410
HLA-DQA1HLA class II histocompatibility antigen, DQ alpha 1 chainMajor susceptibility factor inTolerante
FUNÇÃO

Binds peptides derived from antigens that access the endocytic route of antigen presenting cells (APC) and presents them on the cell surface for recognition by the CD4 T-cells. The peptide binding cleft accommodates peptides of 10-30 residues. The peptides presented by MHC class II molecules are generated mostly by degradation of proteins that access the endocytic route, where they are processed by lysosomal proteases and other hydrolases. Exogenous antigens that have been endocytosed by the APC

LOCALIZAÇÃO

Cell membraneEndoplasmic reticulum membraneGolgi apparatus, trans-Golgi network membraneEndosome membraneLysosome membrane

VIAS BIOLÓGICAS (7)
Generation of second messenger moleculesTranslocation of ZAP-70 to Immunological synapsePhosphorylation of CD3 and TCR zeta chainsCo-inhibition by PD-1Downstream TCR signaling
EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
456.0 TPM
Pulmão
91.4 TPM
Baço
90.3 TPM
Tecido adiposo
34.6 TPM
Nervo tibial
30.4 TPM
OUTRAS DOENÇAS (3)
adult-onset myasthenia gravisidiopathic achalasiaceliac disease, susceptibility to, 1
HGNC:4942UniProt:P01909
TNFRSF11ATumor necrosis factor receptor superfamily member 11AMajor susceptibility factor inTolerante
FUNÇÃO

Receptor for TNFSF11/RANKL/TRANCE/OPGL; essential for RANKL-mediated osteoclastogenesis (PubMed:9878548). Its interaction with EEIG1 promotes osteoclastogenesis via facilitating the transcription of NFATC1 and activation of PLCG2 (By similarity). Involved in the regulation of interactions between T-cells and dendritic cells (By similarity)

LOCALIZAÇÃO

Cell membraneMembrane raft

VIAS BIOLÓGICAS (2)
TNFR2 non-canonical NF-kB pathwayTNF receptor superfamily (TNFSF) members mediating non-canonical NF-kB pathway
MECANISMO DE DOENÇA

Familial expansile osteolysis

Rare autosomal dominant bone disorder characterized by focal areas of increased bone remodeling. The osteolytic lesions develop usually in the long bones during early adulthood. FEO is often associated with early-onset deafness and loss of dentition.

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula salivar
10.6 TPM
Pituitária
9.9 TPM
Cólon transverso
8.4 TPM
Intestino delgado
7.9 TPM
Skin Sun Exposed Lower leg
2.8 TPM
OUTRAS DOENÇAS (6)
familial expansile osteolysisautosomal recessive osteopetrosis 7adult-onset myasthenia gravisdysosteosclerosis
HGNC:11908UniProt:Q9Y6Q6

Variantes genéticas (ClinVar)

263 variantes patogênicas registradas no ClinVar.

🧬 TNFRSF11A: NM_003839.4(TNFRSF11A):c.1075C>T (p.Gln359Ter) ()
🧬 TNFRSF11A: GRCh38/hg38 18q11.1-23(chr18:20966775-80255845)x3 ()
🧬 TNFRSF11A: GRCh37/hg19 18q21.2-22.2(chr18:52640210-68070259)x4 ()
🧬 TNFRSF11A: GRCh37/hg19 18q21.2-23(chr18:53564430-74587425)x1 ()
🧬 TNFRSF11A: NM_003839.4(TNFRSF11A):c.428-8del ()
Ver todas no ClinVar

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.
Aprovado1
2Fase 21
·Pré-clínico2
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 4 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Miastenia gravis com início no adulto

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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

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4 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

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Publicações mais relevantes

Timeline de publicações
4 papers (10 anos)
#1

Comparison of anti-acetylcholine receptor profiles between Chinese cases of adult- and juvenile-onset myasthenia gravis using cell-based assays.

Journal of neuroimmunology2020 Dec 15

Juvenile-onset myasthenia gravis (JOMG) is a unique clinical subtype in China, featured by a higher prevalence of ocular myasthenia gravis (OMG), higher seronegativity of acetylcholine receptor (AChR) antibodies, and better prognosis than that in adult-onset myasthenia gravis (AOMG). We previously identified low-affinity AChR antibodies in Chinese JOMG patients using cell-based assays (CBAs), indicating a predominantly AChR antibody-positive profile. Here, we further screened AChR antibodies in both Chinese AOMG and JOMG patients by CBAs. We recruited patients with MG who had not received prednisone or immunosuppressive therapies between June 2015 and June 2019, and divided them into AOMG and JOMG subgroups according to their ages at the time of recruitment. Clinical information and blood samples were collected. Serum AChR antibodies were detected by CBAs in HEK293T cells expressing clustered adult and fetal AChRs, as well as by enzyme-linked immunosorbent assays (ELISAs). Differences in AChR antibody profiles between AOMG and JOMG subgroups were determined. A total of 239 patients with MG were enrolled in the present study, including 121 AOMG and 118 JOMG patients. Based on ELISAs, 74.4% of AOMG (90/121) and 59.3% of JOMG (70/118) patients were anti-AChR positive (p = 0.02). However, CBAs yielded equal anti-AChR positivities (p = 0.64), as indicated by 80.2% of AOMG patients (97/121) and 77.1% of JOMG patients (91/118). Furthermore, among AOMG patients, 67.8% (82/121) were positive for both adult and fetal AChR antibodies, 5.8% (7/121) were positive for only adult AChR antibodies, and 6.6% (8/121) were positive for only fetal AChR antibodies, while these rates were 50.8% (60/118), 21.2% (25/118), and 5.1% (6/118), respectively, in JOMG cohorts (p < 0.01). Twenty-nine AOMG patients and 10 JOMG patients underwent IgG subclassification of AChR antibodies, which were all confirmed to be predominantly IgG1. The positive rates of AChR antibodies did not differ between Chinese AOMG and JOMG patients, as revealed by CBAs. Furthermore, the screened AChR antibodies were predominantly IgG1 in both AOMG and JOMG patients.

#2

Cell-Based Versus Enzyme-Linked Immunosorbent Assay for the Detection of Acetylcholine Receptor Antibodies in Chinese Juvenile Myasthenia Gravis.

Pediatric neurology2019 Sep

Patients in China with juvenile-onset myasthenia gravis present early, with a high prevalence of purely ocular symptoms, spontaneous remission rates, and low antibody seropositivity. Antibody detection using a cell-based assay has been reported to increase the diagnostic sensitivity in adult-onset myasthenia gravis. However, this method in patients with juvenile-onset myasthenia gravis has not been investigated. Patients with juvenile-onset myasthenia gravis who had not received prednisone or immunosuppressive therapy were recruited between June 2015 and April 2018 at the Huashan Hospital. Clinical information was collected. Serum anti-acetylcholine receptor antibodies were detected via cell-based assay with HEK293T cells expressing acetylcholine receptor subunits and rapsyn. Additionally, the IgG antibody subclass was identified. Eighty-two patients with juvenile-onset myasthenia gravis were enrolled in the current study. Among them, 48 patients were anti-acetylcholine receptor positive (58.5%) and 34 were seronegative (41.5%), as assessed via enzyme-linked immunosorbent assay. Cell-based assay yielded 63 positive subjects (76.8%) and 19 seronegative subjects (23.2%). All the enzyme-linked immunosorbent assay-positive samples showed robust immunofluorescence in the cell-based assay, whereas 15 of 34 enzyme-linked immunosorbent assay-negative patients (44.1%) were found to have low-affinity acetylcholine receptor antibodies. Among all the cell-based assay-positive patients, 41 were positive for both adult and fetal acetylcholine receptor antibodies (50.0%), 18 were found positive for only adult acetylcholine receptor antibodies (21.9%), and four were found to possess only fetal acetylcholine receptor antibodies (4.9%). Fifteen antibody-positive samples underwent subclassification and were confirmed to be IgG1 subclass predominant (n = 15, including eight adult and fetal acetylcholine receptor antibody positive, five only adult acetylcholine receptor antibody positive, and two only fetal acetylcholine receptor antibody positive). There were no significant differences in clinical features among patients with different antibody profiles. The cell-based assay showed increased sensitivity in acetylcholine receptor antibody detection in Chinese patients with juvenile-onset myasthenia gravis, and most cases of Chinese juvenile-onset myasthenia gravis are still acetylcholine receptor autoantibody mediated. Furthermore, the antibodies detected are predominately of the IgG1 subclass.

#3

HLA typing using next-generation sequencing for Chinese juvenile- and adult-onset myasthenia gravis patients.

Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia2019 Jan

To compare HLA typing between juvenile- and adult-onset myasthenia gravis (MG), we enrolled 101 children (age ≤12 years) and 168 adults (age ≥20 years) with MG. We excluded patients with histories of thymoma, thyroid disease, or other autoimmune disease. We selected 41 seronegative juvenile-onset patients with ocular symptoms only, and 41 seropositive adult-onset patients with generalized symptoms. We used next-generation sequencing for typing and analysis of HLA genes (Loci: A, B, C, DPA1, DPB1, DQA1, DQB1 and DRB1). Haplotypes HLA-A∗02:07:01-B∗46:01:01-C∗01:02:01-DQA1∗01:01:01-DQB1∗03:03:02-DRB1∗09:01:02, HLA-A∗11:01:01, HLA-A∗24:02:01, and HLA-DPA1∗02:02:02 were found to be related to juvenile-onset MG and HLA-A∗01:01:01, HLA-A∗02:03:01, HLA-C∗03:04:01, and HLA-DQB1∗06:02:01 to adult-onset MG. Therefore, our findings suggested that HLA typing might determine the heterogeneity between AChR-Ab negative juvenile-onset and AChR-Ab positive adult-onset Chinese MG patients.

#4

Survival and mortality of adult-onset myasthenia gravis in the population of Belgrade, Serbia.

Muscle &amp; nerve2018 Nov

The objective of this study was to estimate mortality and survival in a large cohort of myasthenia gravis (MG) patients from Belgrade, Serbia, during the period 1979-2008. Data for all patients with MG were collected from hospital records and the Belgrade MG Registry. Within the 30-year study period, death occurred in 107 (20%) of 562 patients with MG, with MG-related fatality below 2%. The average MG mortality rate was 1.76 per 1,000,000 population (1.26/1,000,000 women, 2.45/1,000,000 men). A statistically significant increase was recorded for the average standardized mortality rate for all patients (P < 0.01). The mean survival from disease onset was 34.3 ± 2.0 years. Significantly shorter survival was observed in men compared with women and in patients older than 50 years compared with younger ones (P < 0.01). We observed long survival and low frequency of MG-related fatalities but increasing average standardized mortality rate, most notably in older men with MG. Muscle Nerve 58: 708-712, 2018.

Publicações recentes

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Associações

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Comunidades

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

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. Comparison of anti-acetylcholine receptor profiles between Chinese cases of adult- and juvenile-onset myasthenia gravis using cell-based assays.
    Journal of neuroimmunology· 2020· PMID 32992216mais citado
  2. Cell-Based Versus Enzyme-Linked Immunosorbent Assay for the Detection of Acetylcholine Receptor Antibodies in Chinese Juvenile Myasthenia Gravis.
    Pediatric neurology· 2019· PMID 31307830mais citado
  3. HLA typing using next-generation sequencing for Chinese juvenile- and adult-onset myasthenia gravis patients.
    Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia· 2019· PMID 30595166mais citado
  4. Survival and mortality of adult-onset myasthenia gravis in the population of Belgrade, Serbia.
    Muscle &amp; nerve· 2018· PMID 29572981mais citado
  5. Epidemiological study of adult-onset myasthenia gravis in the area of Belgrade (Serbia) in the period 1979-2008.
    Neuroepidemiology· 2013· PMID 23363926recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:391490(Orphanet)
  2. MONDO:0018324(MONDO)
  3. Miastenia Gravis(PCDT · Ministério da Saúde)
  4. GARD:21623(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q55787955(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

Miastenia gravis com início no adulto
Compêndio · Raras BR

Miastenia gravis com início no adulto

ORPHA:391490 · MONDO:0018324
🇧🇷 Brasil SUS
CEAF
1BPiridostigmina1AAzatioprinaEculizumabeRavulizumabe
Geral
Prevalência
1-5 / 10 000
Herança
Multigenic/multifactorial, Not applicable
CID-10
G70.0 · Miastenia gravis
CID-11
Início
Adult
Prevalência
31.8 (Serbia)
MedGen
UMLS
C5680024
Repurposing
5 candidatos
ambenoniumcholinesterase inhibitor
edrophoniumacetylcholinesterase inhibitor
mestinonacetylcholine receptor antagonist
+2 outros
EuropePMC
Wikidata
Papers 10a
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