Raras
Buscar doenças, sintomas, genes...
Malformação capilar-malformação arteriovenosa
ORPHA:137667CID-10 · Q27.3CID-11 · LA90.3YDOENÇA RARA

Esta síndrome é caracterizada pela associação de múltiplas malformações capilares (MC) com malformação arteriovenosa (MAV) e fístulas arteriovenosas.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Esta síndrome é caracterizada pela associação de múltiplas malformações capilares (MC) com malformação arteriovenosa (MAV) e fístulas arteriovenosas.

Pesquisas ativas
1 ensaio
4 total registrados no ClinicalTrials.gov
Publicações científicas
161 artigos
Último publicado: 2026

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Casos conhecidos
261
pacientes catalogados
Início
Infancy
+ neonatal
🏥
SUS: Cobertura mínimaScore: 35%
Centros em: PA, PR, SC, RS, ES +10CID-10: Q27.3
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (5)
0202010503
Cariótipo — bandas G, Q ou Rgenetic_test
0202010600
Pesquisa de microdeleções/microduplicações por FISHlab_test
0202010694
Sequenciamento completo do exoma (WES)rehabilitation
0202010260
Dosagem de alfa-fetoproteína
0301070040
Atendimento em reabilitação — doenças raras
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

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Sinais e sintomas

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

Partes do corpo afetadas

🦴
Ossos e articulações
7 sintomas
❤️
Coração
7 sintomas
🧠
Neurológico
6 sintomas
🩸
Sangue
5 sintomas
💪
Músculos
3 sintomas
🧬
Pele e cabelo
3 sintomas

+ 30 sintomas em outras categorias

Características mais comuns

90%prev.
Malformação capilar
Muito frequente (99-80%)
55%prev.
Anormalidade vascular da pele
Frequente (79-30%)
17%prev.
Sangramento anormal
Ocasional (29-5%)
17%prev.
Fístula arteriovenosa periférica
Ocasional (29-5%)
17%prev.
Malformação arteriovenosa
Ocasional (29-5%)
17%prev.
Fístula arteriovenosa
Ocasional (29-5%)
66sintomas
Muito frequente (1)
Frequente (1)
Ocasional (14)
Muito raro (13)
Sem dados (37)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 66 características clínicas mais associadas, ordenadas por frequência.

Malformação capilarCapillary malformation
Muito frequente (99-80%)90%
Anormalidade vascular da peleVascular skin abnormality
Frequente (79-30%)55%
Sangramento anormalAbnormal bleeding
Ocasional (29-5%)17%
Fístula arteriovenosa periféricaPeripheral arteriovenous fistula
Ocasional (29-5%)17%
Malformação arteriovenosaArteriovenous malformation
Ocasional (29-5%)17%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico161PubMed
Últimos 10 anos135publicações
Pico202119 papers
Linha do tempo
2026Hoje · 2026🧪 2006Primeiro ensaio clínico📈 2021Ano 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

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

RASA1Ras GTPase-activating protein 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

GTPase-activating protein (GAP) that stimulates the intrinsic GTPase activity of Ras proteins, such as NRAS, facilitating their transition from the active GTP-bound state to the inactive GDP-bound state, thereby terminating Ras signaling

LOCALIZAÇÃO

Cytoplasm

VIAS BIOLÓGICAS (5)
VEGFR2 mediated cell proliferationRegulation of RAS by GAPsPTK6 Regulates RHO GTPases, RAS GTPase and MAP kinasesDownstream signal transductionEPHB-mediated forward signaling
EXPRESSÃO TECIDUAL(Ubíquo)
Ovário
39.3 TPM
Fibroblastos
38.2 TPM
Nervo tibial
37.2 TPM
Linfócitos
34.6 TPM
Testículo
33.9 TPM
OUTRAS DOENÇAS (2)
capillary malformation-arteriovenous malformation 1basal cell carcinoma, susceptibility to, 1
HGNC:9871UniProt:P20936
EPHB4Ephrin type-B receptor 4Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Receptor tyrosine kinase which binds promiscuously transmembrane ephrin-B family ligands residing on adjacent cells, leading to contact-dependent bidirectional signaling into neighboring cells. The signaling pathway downstream of the receptor is referred to as forward signaling while the signaling pathway downstream of the ephrin ligand is referred to as reverse signaling. Together with its cognate ligand/functional ligand EFNB2 it is involved in the regulation of cell adhesion and migration, an

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (3)
EPH-ephrin mediated repulsion of cellsEPH-Ephrin signalingEphrin signaling
MECANISMO DE DOENÇA

Lymphatic malformation 7

A form of primary lymphedema, a disease characterized by swelling of body parts due to developmental anomalies and functional defects of the lymphatic system. Patients with lymphedema may suffer from recurrent local infections. LMPHM7 is an autosomal dominant form with variable expressivity. Some individuals present with severe non-immune hydrops fetalis, which may cause perinatal demise or fully resolve after the neonatal period. Others present with no edema and have milder clinical features, such as atrial septal defect or varicose veins as adults.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
133.8 TPM
Cervix Endocervix
110.2 TPM
Baço
84.6 TPM
Ovário
83.5 TPM
Fallopian Tube
73.2 TPM
OUTRAS DOENÇAS (5)
lymphatic malformation 7capillary malformation-arteriovenous malformation 2EPHB4-related lymphatic-related hydrops fetalisvein of Galen aneurysm
HGNC:3395UniProt:P54760

Variantes genéticas (ClinVar)

601 variantes patogênicas registradas no ClinVar.

🧬 RASA1: NM_002890.3(RASA1):c.1528G>T (p.Glu510Ter) ()
🧬 RASA1: NM_002890.3(RASA1):c.2555T>C (p.Leu852Pro) ()
🧬 RASA1: NM_002890.3(RASA1):c.1049+1G>A ()
🧬 RASA1: NM_002890.3(RASA1):c.2285_2291del (p.His762fs) ()
🧬 RASA1: NM_002890.3(RASA1):c.2185-1G>C ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 1,541 variantes classificadas pelo ClinVar.

154
308
1079
Patogênica (10.0%)
VUS (20.0%)
Benigna (70.0%)
VARIANTES MAIS SIGNIFICATIVAS
CCNH: NM_002890.3(RASA1):c.1777-1G>C [Likely pathogenic]
CCNH: NM_002890.3(RASA1):c.1528G>T (p.Glu510Ter) [Pathogenic]
CCNH: NM_002890.3(RASA1):c.1527C>A (p.Ser509Arg) [Uncertain significance]
CCNH: NM_002890.3(RASA1):c.2315C>T (p.Thr772Ile) [Uncertain significance]
CCNH: NM_002890.3(RASA1):c.3023A>T (p.Glu1008Val) [Uncertain significance]

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.
Aprovado1
·Pré-clínico2
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 3 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Malformação capilar-malformação arteriovenosa

Centros de Referência SUS

24 centros habilitados pelo SUS para Malformação capilar-malformação arteriovenosa

Centros para Malformação capilar-malformação arteriovenosa

Detalhes dos centros

Hospital Universitário Prof. Edgard Santos (HUPES)

R. Dr. Augusto Viana, s/n - Canela, Salvador - BA, 40110-060 · CNES 0003808

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Infantil Albert Sabin

R. Tertuliano Sales, 544 - Vila União, Fortaleza - CE, 60410-794 · CNES 2407876

Serviço de Referência

Rota
Anomalias CongênitasDeficiência Intelectual

Hospital de Apoio de Brasília (HAB)

AENW 3 Lote A Setor Noroeste - Plano Piloto, Brasília - DF, 70684-831 · CNES 0010456

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Estadual Infantil e Maternidade Alzir Bernardino Alves (HIABA)

Av. Min. Salgado Filho, 918 - Soteco, Vila Velha - ES, 29106-010 · CNES 6631207

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital das Clínicas da UFG

Rua 235 QD. 68 Lote Área, Nº 285, s/nº - Setor Leste Universitário, Goiânia - GO, 74605-050 · CNES 2338424

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Universitário da UFJF

R. Catulo Breviglieri, Bairro - s/n - Santa Catarina, Juiz de Fora - MG, 36036-110 · CNES 2297442

Atenção Especializada

Rota
Anomalias Congênitas

Hospital das Clínicas da UFMG

Av. Prof. Alfredo Balena, 110 - Santa Efigênia, Belo Horizonte - MG, 30130-100 · CNES 2280167

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Julio Müller (HUJM)

R. Luis Philippe Pereira Leite, s/n - Alvorada, Cuiabá - MT, 78048-902 · CNES 2726092

Atenção Especializada

Rota
Anomalias Congênitas

Hospital Universitário João de Barros Barreto

R. dos Mundurucus, 4487 - Guamá, Belém - PA, 66073-000 · CNES 2337878

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Lauro Wanderley (HULW)

R. Tabeliao Estanislau Eloy, 585 - Castelo Branco, João Pessoa - PB, 58050-585 · CNES 0002470

Atenção Especializada

Rota
Anomalias Congênitas

Instituto de Medicina Integral Prof. Fernando Figueira (IMIP)

R. dos Coelhos, 300 - Boa Vista, Recife - PE, 50070-902 · CNES 0000647

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Pequeno Príncipe

R. Des. Motta, 1070 - Água Verde, Curitiba - PR, 80250-060 · CNES 3143805

Serviço de Referência

Rota
Anomalias CongênitasDeficiência Intelectual

Hospital Universitário Regional de Maringá (HUM)

Av. Mandacaru, 1590 - Parque das Laranjeiras, Maringá - PR, 87083-240 · CNES 2216108

Atenção Especializada

Rota
Anomalias Congênitas

Hospital de Clínicas da UFPR

R. Gen. Carneiro, 181 - Alto da Glória, Curitiba - PR, 80060-900 · CNES 2364980

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Pedro Ernesto (HUPE-UERJ)

Blvd. 28 de Setembro, 77 - Vila Isabel, Rio de Janeiro - RJ, 20551-030 · CNES 2280221

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira (IFF/Fiocruz)

Av. Rui Barbosa, 716 - Flamengo, Rio de Janeiro - RJ, 22250-020 · CNES 2269988

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital São Lucas da PUCRS

Av. Ipiranga, 6690 - Jardim Botânico, Porto Alegre - RS, 90610-000 · CNES 2232928

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital de Clínicas de Porto Alegre (HCPA)

Rua Ramiro Barcelos, 2350 Bloco A - Av. Protásio Alves, 211 - Bloco B e C - Santa Cecília, Porto Alegre - RS, 90035-903 · CNES 2237601

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário da UFSC (HU-UFSC)

R. Profa. Maria Flora Pausewang - Trindade, Florianópolis - SC, 88036-800 · CNES 2560356

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital das Clínicas da FMUSP

R. Dr. Ovídio Pires de Campos, 225 - Cerqueira César, São Paulo - SP, 05403-010 · CNES 2077485

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Base de São José do Rio Preto

Av. Brg. Faria Lima, 5544 - Vila Sao Jose, São José do Rio Preto - SP, 15090-000 · CNES 2079798

Atenção Especializada

Rota
Anomalias Congênitas

Hospital de Clínicas da UNICAMP

R. Vital Brasil, 251 - Cidade Universitária, Campinas - SP, 13083-888 · CNES 2748223

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas de Ribeirão Preto (HCRP-USP)

R. Ten. Catão Roxo, 3900 - Vila Monte Alegre, Ribeirão Preto - SP, 14015-010 · CNES 2082187

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

UNIFESP / Hospital São Paulo

R. Napoleão de Barros, 715 - Vila Clementino, São Paulo - SP, 04024-002 · CNES 2688689

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo
Sobre os centros SUS: Estes centros são habilitados pelo Ministério da Saúde como Serviços de Referência em Doenças Raras ou Serviços de Atenção Especializada. O atendimento é pelo SUS, com encaminhamento da rede de atenção básica.

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

🟢 Recrutando agora

1 pesquisa recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

4 ensaios clínicos encontrados, 1 ativos.

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

Timeline de publicações
127 papers (10 anos)
#1

Progressive non-immune hydrops fetalis associated with RASA1 mutation: prenatal imaging and genomic insights.

BMJ case reports2026 Feb 27

Hydrops fetalis (HF) is the pathological accumulation of fluid in two or more fetal compartments. While immune-mediated HF was historically predominant, non-immune hydrops fetalis (NIHF) is now increasingly common. Advances in genetic testing have revealed monogenic causes, including RASopathies (a group of genetic syndromes caused by dysregulation of the RAS-mitogen-activated protein kinase signalling pathway). We report a young primigravida, referred at 20+3 weeks for unilateral fetal pleural effusion. Serial ultrasound scans showed rapid progression to bilateral effusion, hepatomegaly, polyhydramnios and NIHF. First-trimester screening and non-invasive prenatal testing were low-risk, Toxoplasmosis, Other agents, Rubella, Cytomegalovirus, Herpes simplex serologies and PCR ruled out infection. Whole exome sequencing identified a likely pathogenic heterozygous frameshift mutation in the RASA1 gene (c.723dupT; p.Gly242TrpfsTer23), associated with capillary malformation-arteriovenous malformation type 1 (CM-AVM1). The mother had a subtle congenital capillary haemangioma on her left hand and revealed the same heterozygous variant of RASA1 gene, indicating variable expressibility. This case highlights the importance of considering rare monogenic causes like RASA1-related CM-AVM1 in NIHF, especially when early findings precede florid hydrops and classical RASopathy features are absent.

#2

COVID-19 Outcomes and Risk Factors for Hospitalization in Adult Patients With Primary Immunodeficiency.

Allergy, asthma &amp; immunology research2026 Jan

The impact of coronavirus disease 2019 (COVID-19) on patients with primary immunodeficiency (PID) remains insufficiently characterized. This study aimed to describe the clinical manifestations, disease course, and outcomes of COVID-19 in patients with PID. Adult patients with PID who had COVID-19 infection between March 2020 and August 2022 were screened. Demographic and clinical data were retrospectively collected from institutional databases, and additional information was obtained through a patient questionnaire. A total of 36 patients (19 males, 17 females; median age: 36.5 years) with various PID subtypes were included: 24 with common variable immunodeficiency (CVID), 3 with cytotoxic T-lymphocyte-associated protein-4 haploinsufficiency, 3 with X-linked agammaglobulinemia (XLA), 2 with hypogammaglobulinemia, 1 with lipopolysaccharide-responsive and beige-like anchor protein deficiency, 1 with DiGeorge syndrome, 1 with mitochondrial neurogastrointestinal encephalomyopathy syndrome, and 1 with CVID-like capillary malformation-arteriovenous malformation syndrome 2. Overall, 63.9% (n = 23) were managed as outpatients, while 36.1% (n = 13) required hospitalization. Admission to the intensive care unit was required in 19.4% (n = 7) of the cases. The overall case fatality rate was 8.3% (n = 3), which is higher than the rate observed in the general population. Although the majority experienced a mild clinical course, patients with XLA exhibited prolonged symptoms and persistent seropositivity. Risk factors associated with hospitalization included lymphopenia, elevated C-reactive protein and ferritin levels, dyspnea, COVID-19 Reporting and Data System score ≥ 4 on imaging, need for supplemental oxygen, prolonged symptoms, and extended polymerase chain reaction positivity. A subset of adult patients with PID may be at increased risk for severe COVID-19.

#3

Addressing Palliative Care Gaps for Rare Congenital Disease in Adults: CM-AVM2 as an Example.

Journal of pain and symptom management2026 Jan

Capillary malformation-arteriovenous malformation type 2 (CM-AVM2) is a rare vascular disorder marked by complex, progressive symptoms and limited treatment options. As more individuals with rare diseases reach adulthood, palliative care plays an essential role in supporting quality of life when disease-directed therapies begin to fail. We present a young adult male with EPHB4-positive CM-AVM2, transferred to our tertiary care center for progressive malnutrition, refractory diarrhea, and worsening abdominal pain despite extensive subspecialty care. His hospitalization was marked by escalating symptom burden, limited response to medical therapies, and increasing emotional distress for both patient and family. Palliative care was consulted to assist with pain control and symptom management, with initial reluctance for discussing goals of care (GOC). Through medication optimization, dynamic communication, and consistent interdisciplinary collaboration, the team helped stabilize aspects of his comfort and gradually facilitated GOC discussions. Our case emphasizes the value of integrating palliative care in rare disease management, particularly when longstanding treatments are refractory. We further discuss how palliative care in this setting differs from standard frameworks, requiring proactive use of disease-specific resources and protracted, anticipatory care planning to optimize quality of life.

#4

Double Genetic Diagnosis Involving MECP2 and EPHB4 in a Child with Neurodevelopmental Delay and Vascular Anomalies: A Case Report.

Molecular syndromology2025 Oct 23

Double genetic diagnoses are increasingly identified with the advent of genome-wide sequencing techniques. While MECP2 mutations are associated with Rett syndrome and EPHB4 mutations with vascular malformation syndromes, their co-occurrence has not been previously described. We describe an 8-year-and-2-month-old girl presenting with global developmental delay, autism spectrum disorder, and stereotypic behaviors, along with multiple well-demarcated cutaneous vascular lesions. Although she had no clinical seizures, electroencephalogram revealed epileptiform discharges. Physical examination showed dysmorphic features and vascular anomalies, including telangiectatic pink-to-red macular vascular lesions. Whole exome sequencing (WES) identified two de novo heterozygous pathogenic variants: a missense mutation in MECP2 (c.433C>T; p.Arg145Cys), a gene classically implicated in Rett syndrome, and a nonsense mutation in EPHB4 (c.1093C>T; p.Arg365Ter), which has been previously associated with capillary malformation-arteriovenous malformation syndrome type 2. The neurodevelopmental findings, while consistent with the broader spectrum of MECP2-related disorders, along with coexisting vascular anomalies, were best accounted for by a dual genetic diagnosis involving both MECP2 and EPHB4. This case underscores the diagnostic value of considering dual genetic diagnoses in patients with complex phenotypes and highlights the role of WES in uncovering multilocus variation, thereby expanding the known phenotypic spectrum associated with MECP2 and EPHB4 mutations.

#5

Capillary malformation-arteriovenous malformation syndrome (CM-AVM): a systematic review of cerebrovascular manifestations.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery2025 Dec 16

Capillary malformation-arteriovenous malformation (CM-AVM) syndrome is a rare genetic disorder characterized by cutaneous capillary malformations and fast-flow vascular lesions, including arteriovenous malformations (AVMs) and arteriovenous fistulas (AVFs). CM-AVM is caused by mutations in RASA1 and EPHB4, leading to aberrant Ras-MAPK signaling. A systematic search of PubMed and Scopus was conducted for studies published until June 2025. The inclusion criteria were studies reporting cerebrovascular malformations in genetically confirmed CM-AVM cases. A total of 37 studies were included in the final analysis. The review included 148 patients diagnosed with CM-AVM, with 86% carrying RASA1 mutations and 14% carrying EPHB4 mutations. The most common cerebrovascular lesions were pial AVFs (43.3%) and AVMs (36.0%), with a notable distinction between the two genetic subtypes. RASA1 mutations were associated with a broader range of lesions, including AVMs, pAVFs, and vGaMs, whereas EPHB4 mutations were predominantly linked to vGaMs. Nearly 25% of patients required endovascular embolization, and 5.3% underwent surgery. A significant difference in the cerebrovascular phenotype was observed between RASA1 and EPHB4 mutations, with the latter group presenting a narrower vascular phenotype. This review highlights the crucial need for screening cerebrovascular anomalies in CM-AVM patients due to potential misdiagnosis with HHT. Genetic testing is essential for confirmation, but regular imaging and clinical evaluation are key to detecting vascular lesions early, preventing severe neurological complications. Further research into additional genetic mutations may improve diagnostic accuracy and management strategies.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC76 artigos no totalmostrando 134

2026

Progressive non-immune hydrops fetalis associated with RASA1 mutation: prenatal imaging and genomic insights.

BMJ case reports
2026

COVID-19 Outcomes and Risk Factors for Hospitalization in Adult Patients With Primary Immunodeficiency.

Allergy, asthma &amp; immunology research
2025

Double Genetic Diagnosis Involving MECP2 and EPHB4 in a Child with Neurodevelopmental Delay and Vascular Anomalies: A Case Report.

Molecular syndromology
2025

Capillary malformation-arteriovenous malformation syndrome (CM-AVM): a systematic review of cerebrovascular manifestations.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2025

Efficacy and safety of endovascular embolization for capillary malformation-arteriovenous malformation of the auricular and periauricular region in pediatric patients.

Quantitative imaging in medicine and surgery
2025

Capillary malformation-arteriovenous malformation: atypical neonatal presentation.

Anales de pediatria
2026

Addressing Palliative Care Gaps for Rare Congenital Disease in Adults: CM-AVM2 as an Example.

Journal of pain and symptom management
2025

Early arteriovenous malformation mimicking pediatric capillary malformation: diagnostic value of infrared thermography.

Hereditas
2025

RASopathies. Part II: Cutaneous and extracutaneous manifestations.

Journal of the American Academy of Dermatology
2025

Erythematous patches in a female teenager: a novel mutation of RASA1 in capillary malformation-arteriovenous malformation syndrome type 1.

Dermatology online journal
2025

RASopathies. Part I: Genetics and therapeutic considerations.

Journal of the American Academy of Dermatology
2025

Isolated Cutis Marmorata Telangiectatica Congenita in a Full-Term Neonate: A Case Report.

Cureus
2025

Arteriovenous cerebral high-flow shunts: genetic analysis of patients from a pediatric tertiary care center.

Frontiers in genetics
2025

Unusual Presentation of Coronary Artery Fistula in Capillary Malformation Arteriovenous Malformation 2 Syndrome: A Case Report.

American journal of medical genetics. Part A
2025

Basic Knowledge and Overview of Brain AVMs.

Journal of neuroendovascular therapy
2025

Biallelic postzygotic RASA1 variants in a germline-negative patient with capillary malformation-arteriovenous malformation.

International journal of dermatology
2025

Variations in RASA1 and EPHB4 in Chinese patients with capillary malformation-arteriovenous malformation.

The Journal of dermatology
2024

Novel postzygotic RASA1 mutation in a patient with Parkes Weber syndrome: A case report and literature review.

Clinical case reports
2025

Second Report of the p.Leu874Pro Missense Variant in  EPHB4 in a Family With Capillary Malformation-Arteriovenous Malformation Syndrome (CM-AVM) Syndrome.

American journal of medical genetics. Part A
2024

Case report: MEK inhibitor as treatment for multi-lineage mosaic KRAS G12D-associated epidermal nevus syndrome in a pediatric patient.

Frontiers in neurology
2025

Genotypes and phenotypes of capillary malformation-arteriovenous malformation: characterization and correlation analysis.

International journal of dermatology
2025

5q14.3 Microdeletion Syndrome With Simultaneous Involvement of MEF2C and RASA1. Clinical Case and Review of the Literature.

Pediatric dermatology
2024

Another face of RASA1: Report of familial germline variant in RASA1 with dysmorphic features.

American journal of medical genetics. Part A
2024

RASopathies for Radiologists.

Radiographics : a review publication of the Radiological Society of North America, Inc
2024

Single dominant lesion in capillary malformation-arteriovenous malformation (CM-AVM) RASA1 syndrome.

Pediatric dermatology
2024

Macrocheilia as an Atypical Clinical Presentation of Capillary Malformation-Arteriovenous Malformation Type 2.

JAMA dermatology
2024

SAM domain variants of EPHB4 associated with aberrant signaling are linked to lymphatic-related fetal hydrops and facial dysmorphology.

Clinical genetics
2024

Unilateral segmental presentation and a novel EPHB4 gene variant in capillary malformation-arteriovenous malformation type 2.

Pediatric dermatology
2024

Arteriovenous malformations as a presenting sign of PTEN hamartoma tumor syndrome: A case series.

Pediatric dermatology
2024

Prevalence and Predictors of Hereditary Hemorrhagic Telangiectasia and Capillary-Malformation Arteriovenous Malformation Syndrome Among Children with Neurovascular Malformations.

The Journal of pediatrics
2023

Spectrum of lymphatic anomalies in patients with RASA1-related CM-AVM.

Pediatric dermatology
2023

Large saccular intracranial aneurysm in a child with RASA1-associated capillary malformation-arteriovenous malformation syndrome: illustrative case.

Journal of neurosurgery. Case lessons
2023

Halo Phenomenon in Lobular Capillary Hemangioma: A Case Report of a Pyogenic Granuloma With Surrounding Cutaneous Hypopigmentation and Review of Tumors With Halo Phenomenon.

Cureus
2024

A temporally-restricted pattern of endothelial cell collagen 4 alpha 1 expression during embryonic development determined with a novel knockin Col4a1-P2A-eGFP mouse line.

Genesis (New York, N.Y. : 2000)
2023

EPHB4-RASA1-Mediated Negative Regulation of Ras-MAPK Signaling in the Vasculature: Implications for the Treatment of EPHB4- and RASA1-Related Vascular Anomalies in Humans.

Pharmaceuticals (Basel, Switzerland)
2023

Prenatal Clinical Findings in RASA1-Related Capillary Malformation-Arteriovenous Malformation Syndrome.

Genes
2023

Seven cases of hereditary haemorrhagic telangiectasia-like hepatic vascular abnormalities associated with EPHB4 pathogenic variants.

Journal of medical genetics
2023

Importance of Arachnoid Dissection in Arteriovenous Malformation Microsurgery: A Technical Note.

World neurosurgery
2022

Recurrent Vein of Galen Aneurysmal Malformation as a Presentation of Hereditary Hemorrhagic Telangiectasia.

Molecular syndromology
2022

Capillary Malformation-Arteriovenous Malformation Syndrome Due to EPHB4 Gene Mutation: Clinical, Dermoscopic, Ecographic and Histopathological Features.

Indian journal of dermatology
2022

Dermatoscopic features differentiating among port wine stain, arteriovenous malformation, and capillary malformation-arteriovenous malformation syndrome: to detect potential fast-flow vascular malformations at an early stage.

Journal of the American Academy of Dermatology
2022

Disseminated bluish nodules with a protruded right eye: untypical presentation of capillary malformation-arteriovenous malformation (CM-AVM).

International journal of dermatology
2022

Cellular and molecular mechanisms of EPH/EPHRIN signaling in evolution and development.

Current topics in developmental biology
2022

[Cutaneous capillary malformations with cerebral implementation].

Ugeskrift for laeger
2022

Capillary malformation-arteriovenous malformation syndrome associated with basilar artery aneurysm.

Pediatric dermatology
2022

The genetics of vascular birthmarks.

Clinics in dermatology
2022

Inherited Pial Arteriovenous Fistula in Capillary Malformation-Arteriovenous Malformation Family.

Annals of neurology
2022

RASopathies: Dermatologists' viewpoints.

Indian journal of dermatology, venereology and leprology
2022

The RASopathies: Biology, genetics and therapeutic options.

Advances in cancer research
2022

Capillary Malformation-arteriovenous Malformation Type 2: A Case Report and Review.

Acta dermato-venereologica
2022

Angiogenesis depends upon EPHB4-mediated export of collagen IV from vascular endothelial cells.

JCI insight
2022

Successful management of an arteriovenous malformation with trametinib in a patient with capillary-malformation arteriovenous malformation syndrome and cardiac compromise.

Pediatric dermatology
2022

Central nervous system screening in capillary malformation-arteriovenous malformation syndrome: An observational study.

Journal of the American Academy of Dermatology
2021

PELVIS and CM-AVM syndromes: Vascular anomalies that require imaging studies.

Pediatrics international : official journal of the Japan Pediatric Society
2021

Telangiectasias on the lips and hands of a teenage girl.

Pediatric dermatology
2021

Vascular Birthmarks as a Clue for Complex and Syndromic Vascular Anomalies.

Frontiers in pediatrics
2021

Misdiagnosis of capillary malformations in darker skin phototypes.

Pediatric dermatology
2021

A case of capillary malformation-arteriovenous malformation and Ebstein's anomaly in a child with EphB4 mutation.

Pediatric dermatology
2022

Leg ulcers in childhood: A multicenter study in France.

Annales de dermatologie et de venereologie
2021

CM-AVM syndrome - A prospective observational study of unrelated paediatric cases.

The Australasian journal of dermatology
2021

RASopathies: The musculoskeletal consequences and their etiology and pathogenesis.

Bone
2021

Coexistence of RASA1 and COL4A2 variants caused pial arteriovenous fistula (AVF) in a patient with capillary malformation-arteriovenous malformation.

Clinical neurology and neurosurgery
2021

Treatment of capillary malformations in capillary malformation-arteriovenous malformation syndrome with pulsed dye laser.

Journal of cosmetic dermatology
2021

Systemic and CNS manifestations of inherited cerebrovascular malformations.

Clinical imaging
2021

Capillary Malformation-Arteriovenous Malformation Syndrome.

Cureus
2021

RhoA activation-mediated vascular permeability in capillary malformation-arteriovenous malformation syndrome: a hypothesis.

Drug discovery today
2021

Multiple lentigines in RASA1-associated capillary malformation-arteriovenous malformation syndrome.

JAAD case reports
2021

RASopathies: A significant cause of polyhydramnios?

Prenatal diagnosis
2021

RASA1 phenotype overlaps with hereditary haemorrhagic telangiectasia: two case reports.

Journal of medical genetics
2021

Capillary malformation-arteriovenous malformation syndrome: a multicentre study.

Clinical and experimental dermatology
2020

Prenatal pleural effusions and chylothorax: An unusual presentation for CM-AVM syndrome due to RASA1.

American journal of medical genetics. Part A
2021

Management challenges of a large upper extremity vascular malformation in a patient with capillary malformation-arteriovenous malformation syndrome.

Journal of vascular surgery. Venous and lymphatic disorders
2020

Unilateral and segmental distribution of facial erythema: is it a real port-wine stain?

Hereditas
2021

De novo intracranial arteriovenous malformation development after endovascular treatment for a pial arteriovenous fistula in capillary malformation-arteriovenous malformation syndrome.

Interventional neuroradiology : journal of peritherapeutic neuroradiology, surgical procedures and related neurosciences
2020

[Neonatal capillary malformation-arteriovenous malformation complicated with acute heart failure: a case report and literature review].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2021

Characterization of vascular stains associated with high flow.

Journal of the American Academy of Dermatology
2020

A novel mutation of RASA1 in capillary malformation-arteriovenous malformation syndrome in a case with spinal cord involvement.

Clinical neurology and neurosurgery
2020

Role of colour-Doppler high-frequency ultrasonography in capillary malformation-arteriovenous malformation syndrome: a case series.

The Australasian journal of dermatology
2020

Broadly distributed vascular macules in a pediatric patient.

Cutis
2020

Histopathological hallmarks of cutaneous lesions of capillary malformation-arteriovenous malformation syndrome.

Journal of the European Academy of Dermatology and Venereology : JEADV
2020

Pediatric intracranial arteriovenous shunts: Advances in diagnosis and treatment.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2020

Good response to pulsed dye laser in patients with capillary malformation-arteriovenous malformation syndrome (CM-AVM).

Pediatric dermatology
2019

Capillary Malformation-Arteriovenous Malformation Combined Alagille Syndrome in a Patient With Double Gene Variations of RASA1 and NOTCH2.

Frontiers in genetics
2020

Atypical capillary malformations with subsequent diplegia: A difficult case of capillary malformation-arteriovenous malformation syndrome.

Pediatric dermatology
2019

Molecular regulation of arteriovenous endothelial cell specification.

F1000Research
2020

RASA1 mosaic mutations in patients with capillary malformation-arteriovenous malformation.

Journal of medical genetics
2019

Capillary malformations in a child with Kabuki syndrome: A case report.

JAAD case reports
2019

Novel RASA1 mutations in Japanese pedigrees with capillary malformation-arteriovenous malformation.

Brain &amp; development
2019

RASopathy in Patients With Isolated Sagittal Synostosis.

Global pediatric health
2019

RASA1-dependent cellular export of collagen IV controls blood and lymphatic vascular development.

The Journal of clinical investigation
2019

Pathogenesis of Growth Failure in Rasopathies.

Pediatric endocrinology reviews : PER
2019

Rasopathies case report: concurrence of two pathogenic variations de novo in NF1 and KRAS genes in a patient.

BMC pediatrics
2019

Phenotype of CM-AVM2 caused by variants in EPHB4: how much overlap with hereditary hemorrhagic telangiectasia (HHT)?

Genetics in medicine : official journal of the American College of Medical Genetics
2019

Constitutional mosaicism in RASA1-related capillary malformation-arteriovenous malformation.

Clinical genetics
2019

The protean manifestations of RASA1 gene mutation.

Clinical and experimental dermatology
2018

[New insight of craniofacial and oral findings of the RASopathies].

Zhonghua kou qiang yi xue za zhi = Zhonghua kouqiang yixue zazhi = Chinese journal of stomatology
2019

Multiple arteriovenous malformations caused by RASA1 gene mutation presenting during pregnancy - a case report and review of the literature.

VASA. Zeitschrift fur Gefasskrankheiten
2019

A Pathogenic Homozygous Mutation in The Pleckstrin Homology Domain of RASA1 Is Responsible for Familial Tricuspid Atresia in An Iranian Consanguineous Family.

Cell journal
2018

RASA-1 somatic "second hit" mutation in capillary malformation-arteriovenous malformation.

The Journal of dermatology
2018

Multiple capillary malformations of progressive onset: Capillary malformation-arteriovenous malformation syndrome (CM-AVM).

Annales de dermatologie et de venereologie
2018

A novel RASA1 mutation causing capillary malformation-arteriovenous malformation (CM-AVM): the first genetic clinical report in East Asia.

Hereditas
2018

Expanding the clinical and molecular findings in RASA1 capillary malformation-arteriovenous malformation.

European journal of human genetics : EJHG
2018

RAS signalling in energy metabolism and rare human diseases.

Biochimica et biophysica acta. Bioenergetics
2018

Loss of function mutations in EPHB4 are responsible for vein of Galen aneurysmal malformation.

Brain : a journal of neurology
2018

Human genetics and molecular mechanisms of vein of Galen malformation.

Journal of neurosurgery. Pediatrics
2017

Clinical and genetic findings in children with central nervous system arteriovenous fistulas.

Annals of neurology
2018

RASA1 mutation in a family with capillary malformation-arteriovenous malformation syndrome: A discussion of the differential diagnosis.

Pediatric dermatology
2018

Search for RASA1 Variants in Capillary Malformations of the Legs in 113 Children: Results from the French National Paediatric Cohort CONAPE.

Acta dermato-venereologica
2018

Somatic second hit mutation of RASA1 in vascular endothelial cells in capillary malformation-arteriovenous malformation.

European journal of medical genetics
2017

Angioarchitecture of Hereditary Arteriovenous Malformations.

Seminars in interventional radiology
2017

EPHB4 Mutation Implicated in Capillary Malformation-Arteriovenous Malformation Syndrome: A Case Report.

Pediatric dermatology
2017

Germline Loss-of-Function Mutations in EPHB4 Cause a Second Form of Capillary Malformation-Arteriovenous Malformation (CM-AVM2) Deregulating RAS-MAPK Signaling.

Circulation
2017

Craniosynostosis in patients with RASopathies: Accumulating clinical evidence for expanding the phenotype.

American journal of medical genetics. Part A
2017

A review of craniofacial and dental findings of the RASopathies.

Orthodontics &amp; craniofacial research
2017

Pial Arteriovenous Fistula and Capillary Malformation-Arteriovenous Malformation Associated with RASA1 Mutation: 2 Pediatric Cases with Successful Surgical Management.

Pediatric neurosurgery
2017

RASA1 regulates the function of lymphatic vessel valves in mice.

The Journal of clinical investigation
2017

Rhodoid naevus syndrome: why is this name preferable to 'capillary malformation-arteriovenous malformation'?

Journal of the European Academy of Dermatology and Venereology : JEADV
2017

5q14.3 Microdeletions: A Contiguous Gene Syndrome with Capillary Malformation-Arteriovenous Malformation Syndrome and Neurologic Findings.

Pediatric dermatology
2018

Prenatal diagnosis of cerebral and extracerebral high-flow lesions revealing familial capillary malformation-arteriovenous malformation (CM-AVM) syndrome.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2017

Modeling RASopathies with Genetically Modified Mouse Models.

Methods in molecular biology (Clifton, N.J.)
2017

The pathobiology of vascular malformations: insights from human and model organism genetics.

The Journal of pathology
2016

Multifocal vascular lesions.

Seminars in cutaneous medicine and surgery
2016

Genetic basis for vascular anomalies.

Seminars in cutaneous medicine and surgery
2016

A Typical Vascular and Pigmentary Dermoscopic Pattern of Capillary Malformations in Capillary Malformation-Arteriovenous Malformation Syndrome: Report of Four Cases.

Pediatric dermatology
2016

Vascular Stains: Proposal for a Clinical Classification to Improve Diagnosis and Management.

Pediatric dermatology
2016

The Fourth International Symposium on Genetic Disorders of the Ras/MAPK pathway.

American journal of medical genetics. Part A
2016

RASA1 somatic mutation and variable expressivity in capillary malformation/arteriovenous malformation (CM/AVM) syndrome.

American journal of medical genetics. Part A
2016

A spectrum of intracranial vascular high-flow arteriovenous shunts in RASA1 mutations.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2015

Doctor, remember the roof. A memory connects a physician and his young patient.

Minnesota medicine
2016

Recent advances in RASopathies.

Journal of human genetics
2015

Pediatric central nervous system vascular malformations.

Pediatric radiology
2015

Maternal and fetal capillary malformation-arteriovenous malformation (CM-AVM) due to a novel RASA1 mutation presenting with prenatal non-immune hydrops fetalis.

American journal of medical genetics. Part A
2015

Capillary malformations: a classification using specific names for specific skin disorders.

Journal of the European Academy of Dermatology and Venereology : JEADV
2015

Heredity of port-wine stains: investigation of families without a RASA1 mutation.

Journal of cosmetic and laser therapy : official publication of the European Society for Laser Dermatology

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Doenças relacionadas

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Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

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

  1. Progressive non-immune hydrops fetalis associated with RASA1 mutation: prenatal imaging and genomic insights.
    BMJ case reports· 2026· PMID 41763666mais citado
  2. COVID-19 Outcomes and Risk Factors for Hospitalization in Adult Patients With Primary Immunodeficiency.
    Allergy, asthma &amp; immunology research· 2026· PMID 41592542mais citado
  3. Addressing Palliative Care Gaps for Rare Congenital Disease in Adults: CM-AVM2 as an Example.
    Journal of pain and symptom management· 2026· PMID 41052613mais citado
  4. Double Genetic Diagnosis Involving MECP2 and EPHB4 in a Child with Neurodevelopmental Delay and Vascular Anomalies: A Case Report.
    Molecular syndromology· 2025· PMID 41409310mais citado
  5. Capillary malformation-arteriovenous malformation syndrome (CM-AVM): a systematic review of cerebrovascular manifestations.
    Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery· 2025· PMID 41398316mais citado
  6. Chinese capillary malformation-arteriovenous malformation: clinical and genetic analysis of eight cases.
    Front Med (Lausanne)· 2026· PMID 41970387recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:137667(Orphanet)
  2. MONDO:0012016(MONDO)
  3. GARD:11904(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q55783570(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

Malformação capilar-malformação arteriovenosa
Compêndio · Raras BR

Malformação capilar-malformação arteriovenosa

ORPHA:137667 · MONDO:0012016
Prevalência
Unknown
Casos
261 casos conhecidos
Herança
Autosomal dominant, Not applicable
CID-10
Q27.3 · Malformação artério-venosa periférica
CID-11
Ensaios
1 ativos
Início
Infancy, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C1842180
EuropePMC
Wikidata
Papers 10a
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