Raras
Buscar doenças, sintomas, genes...
Malformação capilar
ORPHA:211247DOENÇA RARA

Uma malformação capilar (MC), ou mancha em vinho do Porto ou, ainda nevus flammeus (termos históricos) é, quase sempre, uma mancha, sinal ou marca de nascença. É causada por uma anomalia vascular de capilares.

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Introdução

O que você precisa saber de cara

📋

Malformação capilar é uma condição rara caracterizada por vasos sanguíneos anormais, podendo causar hemangiomas, problemas cardiovasculares, polipose intestinal e déficits neurológicos. Afeta múltiplos órgãos e está associada a mutações em genes como GDF2 e GNA11.

Medicamentos
10 registrados
PROPRANOLOL, TRANEXAMIC ACID, BEVACIZUMAB

Tem tratamento?

10 medicamentos registrados
Ver detalhes, fases e interações →
PROPRANOLOLTRANEXAMIC ACIDBEVACIZUMABIMIQUIMODDOXYCYCLINE ANHYDROUSOCTREOTIDEPOMALIDOMIDEPAZOPANIBTIMOLOLNINTEDANIB
🏥
SUS: Cobertura mínimaScore: 20%
Centros em: PA, PR, SC, RS, ES +10
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

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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

🦴
Ossos e articulações
18 sintomas
🧬
Pele e cabelo
15 sintomas
🧠
Neurológico
12 sintomas
❤️
Coração
10 sintomas
🫃
Digestivo
8 sintomas
🩸
Sangue
8 sintomas

+ 74 sintomas em outras categorias

Características mais comuns

Hemangioma capilar
Anormalidade da fisiologia do sistema cardiovascular
Polipose intestinal
Anormalidade da vasculatura cerebral
Telangiectasia retiniana
Variz esofágica
173sintomas
Sem dados (173)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 173 características clínicas mais associadas, ordenadas por frequência.

Hemangioma capilarCapillary hemangioma
Anormalidade da fisiologia do sistema cardiovascularAbnormality of cardiovascular system physiology
Polipose intestinalIntestinal polyposis
Anormalidade da vasculatura cerebralAbnormality of the cerebral vasculature
Telangiectasia retinianaRetinal telangiectasia

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa12
Últimos 10 anos156publicações
Pico202226 papers
Linha do tempo
20202014Hoje · 2026🧪 2009Primeiro ensaio clínico📈 2022Ano 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

9 genes identificados com associação a esta condição.

GDF2Growth/differentiation factor 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Potent circulating inhibitor of angiogenesis. Signals through the type I activin receptor ACVRL1 but not other Alks. Signaling through SMAD1 in endothelial cells requires TGF-beta coreceptor endoglin/ENG

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Signaling by BMP
MECANISMO DE DOENÇA

Telangiectasia, hereditary hemorrhagic, 5

A multisystemic vascular dysplasia leading to dilation of permanent blood vessels and arteriovenous malformations of skin, mucosa, and viscera. The disease is characterized by recurrent epistaxis and gastro-intestinal hemorrhage. Visceral involvement includes arteriovenous malformations of the lung, liver, and brain.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
8.3 TPM
Testículo
0.1 TPM
Coração - Átrio
0.1 TPM
Brain Nucleus accumbens basal ganglia
0.0 TPM
Hipotálamo
0.0 TPM
OUTRAS DOENÇAS (3)
telangiectasia, hereditary hemorrhagic, type 5hereditary hemorrhagic telangiectasiaheritable pulmonary arterial hypertension
HGNC:4217UniProt:Q9UK05
GNA11Guanine nucleotide-binding protein subunit alpha-11Candidate gene tested inAltamente restrito
FUNÇÃO

Guanine nucleotide-binding proteins (G proteins) function as transducers downstream of G protein-coupled receptors (GPCRs) in numerous signaling cascades (PubMed:31073061). The alpha chain contains the guanine nucleotide binding site and alternates between an active, GTP-bound state and an inactive, GDP-bound state (PubMed:31073061). Signaling by an activated GPCR promotes GDP release and GTP binding (PubMed:31073061). The alpha subunit has a low GTPase activity that converts bound GTP to GDP, t

LOCALIZAÇÃO

Cell membraneCytoplasm

VIAS BIOLÓGICAS (10)
PLC beta mediated eventsG-protein activationADP signalling through P2Y purinoceptor 1G alpha (q) signalling eventsThrombin signalling through proteinase activated receptors (PARs)
MECANISMO DE DOENÇA

Hypocalciuric hypercalcemia, familial 2

A form of hypocalciuric hypercalcemia, a disorder of mineral homeostasis that is transmitted as an autosomal dominant trait with a high degree of penetrance. It is characterized biochemically by lifelong elevation of serum calcium concentrations and is associated with inappropriately low urinary calcium excretion and a normal or mildly elevated circulating parathyroid hormone level. Hypermagnesemia is typically present. Affected individuals are usually asymptomatic and the disorder is considered benign. However, chondrocalcinosis and pancreatitis occur in some adults.

EXPRESSÃO TECIDUAL(Ubíquo)
Cerebelo
153.0 TPM
Cérebro - Hemisfério cerebelar
140.5 TPM
Testículo
104.7 TPM
Esôfago - Muscular
97.3 TPM
Fallopian Tube
94.3 TPM
OUTRAS DOENÇAS (8)
autosomal dominant hypocalcemia 2familial hypocalciuric hypercalcemia 2anastomosing haemangiomauveal melanoma
HGNC:4379UniProt:P29992
ARL6IP6ADP-ribosylation factor-like protein 6-interacting protein 6Candidate gene tested inTolerante
LOCALIZAÇÃO

Nucleus inner membrane

OUTRAS DOENÇAS (1)
cutis marmorata telangiectatica congenita
HGNC:24048UniProt:Q8N6S5
SMAD4SMAD family member 4Candidate gene tested inAltamente restrito
FUNÇÃO

In muscle physiology, plays a central role in the balance between atrophy and hypertrophy. When recruited by MSTN, promotes atrophy response via phosphorylated SMAD2/4. MSTN decrease causes SMAD4 release and subsequent recruitment by the BMP pathway to promote hypertrophy via phosphorylated SMAD1/5/8. Acts synergistically with SMAD1 and YY1 in bone morphogenetic protein (BMP)-mediated cardiac-specific gene expression. Binds to SMAD binding elements (SBEs) (5'-GTCT/AGAC-3') within BMP response el

LOCALIZAÇÃO

CytoplasmNucleus

VIAS BIOLÓGICAS (10)
Transcriptional regulation of pluripotent stem cellsSMAD2/SMAD3:SMAD4 heterotrimer regulates transcriptionDownregulation of SMAD2/3:SMAD4 transcriptional activitySignaling by ActivinSignaling by NODAL
MECANISMO DE DOENÇA

Pancreatic cancer

A malignant neoplasm of the pancreas. Tumors can arise from both the exocrine and endocrine portions of the pancreas, but 95% of them develop from the exocrine portion, including the ductal epithelium, acinar cells, connective tissue, and lymphatic tissue.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
37.2 TPM
Ovário
34.9 TPM
Cervix Endocervix
34.5 TPM
Cérebro - Hemisfério cerebelar
32.5 TPM
Nervo tibial
32.3 TPM
OUTRAS DOENÇAS (7)
Myhre syndromejuvenile polyposis syndromejuvenile polyposis/hereditary hemorrhagic telangiectasia syndromefamilial pancreatic carcinoma
HGNC:6770UniProt:Q13485
GNAQGuanine nucleotide-binding protein G(q) subunit alphaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Guanine nucleotide-binding proteins (G proteins) function as transducers downstream of G protein-coupled receptors (GPCRs) in numerous signaling cascades (PubMed:34556863, PubMed:35672283, PubMed:37991948). The alpha chain contains the guanine nucleotide binding site and alternates between an active, GTP-bound state and an inactive, GDP-bound state (PubMed:37991948). Signaling by an activated GPCR promotes GDP release and GTP binding (PubMed:37991948). The alpha subunit has a low GTPase activity

LOCALIZAÇÃO

Cell membraneGolgi apparatusNucleusNucleus membrane

VIAS BIOLÓGICAS (10)
PLC beta mediated eventsG-protein activationADP signalling through P2Y purinoceptor 1G alpha (q) signalling eventsThrombin signalling through proteinase activated receptors (PARs)
MECANISMO DE DOENÇA

Capillary malformations, congenital

A form of vascular malformations that are present from birth, tend to grow with the individual, do not regress spontaneously, and show normal rates of endothelial cell turnover. Capillary malformations are distinct from capillary hemangiomas, which are highly proliferative lesions that appear shortly after birth and show rapid growth, slow involution, and endothelial hypercellularity.

EXPRESSÃO TECIDUAL(Ubíquo)
Artéria tibial
54.7 TPM
Brain Frontal Cortex BA9
54.1 TPM
Brain Spinal cord cervical c-1
53.3 TPM
Pulmão
51.6 TPM
Aorta
48.3 TPM
OUTRAS DOENÇAS (5)
Sturge-Weber syndromefamilial multiple nevi flammeianastomosing haemangiomauveal melanoma
HGNC:4390UniProt:P50148
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
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
ENGEndoglinDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Vascular endothelium glycoprotein that plays an important role in the regulation of angiogenesis (PubMed:21737454, PubMed:23300529). Required for normal structure and integrity of adult vasculature (PubMed:7894484). Regulates the migration of vascular endothelial cells (PubMed:17540773). Required for normal extraembryonic angiogenesis and for embryonic heart development (By similarity). May regulate endothelial cell shape changes in response to blood flow, which drive vascular remodeling and est

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (1)
N-glycan trimming in the ER and Calnexin/Calreticulin cycle
MECANISMO DE DOENÇA

Telangiectasia, hereditary hemorrhagic, 1

A multisystemic vascular dysplasia leading to dilation of permanent blood vessels and arteriovenous malformations of skin, mucosa, and viscera. The disease is characterized by recurrent epistaxis and gastro-intestinal hemorrhage. Visceral involvement includes arteriovenous malformations of the lung, liver, and brain.

EXPRESSÃO TECIDUAL(Ubíquo)
Aorta
476.7 TPM
Pulmão
392.6 TPM
Ovário
380.3 TPM
Artéria coronária
376.9 TPM
Coração - Átrio
355.2 TPM
OUTRAS DOENÇAS (5)
telangiectasia, hereditary hemorrhagic, type 1generalized juvenile polyposis/juvenile polyposis colihereditary hemorrhagic telangiectasiaintracranial berry aneurysm
HGNC:3349UniProt:P17813
ACVRL1Activin receptor type-1-likeDisease-causing germline mutation(s) inRestrito
FUNÇÃO

Type I receptor for TGF-beta family ligands BMP9/GDF2 and BMP10 and important regulator of normal blood vessel development. On ligand binding, forms a receptor complex consisting of two type II and two type I transmembrane serine/threonine kinases. Type II receptors phosphorylate and activate type I receptors which autophosphorylate, then bind and activate SMAD transcriptional regulators. May bind activin as well

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (1)
Signaling by BMP
MECANISMO DE DOENÇA

Telangiectasia, hereditary hemorrhagic, 2

A multisystemic vascular dysplasia leading to dilation of permanent blood vessels and arteriovenous malformations of skin, mucosa, and viscera. The disease is characterized by recurrent epistaxis and gastro-intestinal hemorrhage. Visceral involvement includes arteriovenous malformations of the lung, liver, and brain.

VIAS REACTOME (1)
OUTRAS DOENÇAS (3)
telangiectasia, hereditary hemorrhagic, type 2hereditary hemorrhagic telangiectasiaheritable pulmonary arterial hypertension
HGNC:175UniProt:P37023

Medicamentos e terapias

PROPRANOLOLPhase 3

Mecanismo: Beta-1 adrenergic receptor antagonist

TRANEXAMIC ACIDPhase 3

Mecanismo: Plasminogen inhibitor

BEVACIZUMABPhase 3

Mecanismo: Vascular endothelial growth factor A inhibitor

IMIQUIMODPhase 2

Mecanismo: Toll-like receptor 7 agonist

DOXYCYCLINE ANHYDROUSPhase 2

Mecanismo: Matrix metalloproteinase 8 inhibitor

OCTREOTIDEPhase 2

Mecanismo: Somatostatin receptor agonist

POMALIDOMIDEPhase 2

Mecanismo: CRL4(CRBN) E3 ubiquitin ligase inhibitor

PAZOPANIBPhase 2

Mecanismo: Vascular endothelial growth factor receptor inhibitor

TIMOLOLPhase 2

Mecanismo: Beta-1 adrenergic receptor antagonist

NINTEDANIBPhase 2

Mecanismo: Fibroblast growth factor receptor inhibitor

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

158 variantes patogênicas registradas no ClinVar.

🧬 GDF2: NM_016204.4(GDF2):c.1262T>C (p.Met421Thr) ()
🧬 GDF2: NM_016204.4(GDF2):c.217_220del (p.Ser73fs) ()
🧬 GDF2: NM_016204.4(GDF2):c.647G>A (p.Arg216Gln) ()
🧬 GDF2: NM_016204.4(GDF2):c.265A>C (p.Met89Leu) ()
🧬 GDF2: NM_016204.4(GDF2):c.178G>T (p.Glu60Ter) ()
Ver todas no ClinVar

Vias biológicas (Reactome)

47 vias biológicas associadas aos genes desta condição.

Signaling by BMP PLC beta mediated events G-protein activation Acetylcholine regulates insulin secretion G alpha (q) signalling events ADP signalling through P2Y purinoceptor 1 Thromboxane signalling through TP receptor Fatty Acids bound to GPR40 (FFAR1) regulate insulin secretion Thrombin signalling through proteinase activated receptors (PARs) Cooperation of PDCL (PhLP1) and TRiC/CCT in G-protein beta folding High laminar flow shear stress activates signaling by PIEZO1 and PECAM1:CDH5:KDR in endothelial cells Turbulent (oscillatory, disturbed) flow shear stress activates signaling by PIEZO1 and integrins in endothelial cells Signaling by NODAL Signaling by Activin TGF-beta receptor signaling activates SMADs Downregulation of SMAD2/3:SMAD4 transcriptional activity SMAD2/SMAD3:SMAD4 heterotrimer regulates transcription SMAD4 MH2 Domain Mutants in Cancer SMAD2/3 MH2 Domain Mutants in Cancer Transcriptional regulation of pluripotent stem cells Ub-specific processing proteases RUNX2 regulates bone development RUNX3 regulates CDKN1A transcription RUNX3 regulates BCL2L11 (BIM) transcription FOXO-mediated transcription of oxidative stress, metabolic and neuronal genes FOXO-mediated transcription of cell cycle genes Cardiogenesis SARS-CoV-1 targets host intracellular signalling and regulatory pathways Germ layer formation at gastrulation Formation of definitive endoderm TGFBR3 expression Transcriptional regulation of brown and beige adipocyte differentiation by EBF2 EPH-Ephrin signaling EPHB-mediated forward signaling Ephrin signaling EPH-ephrin mediated repulsion of cells Downstream signal transduction VEGFR2 mediated cell proliferation Regulation of RAS by GAPs PTK6 Regulates RHO GTPases, RAS GTPase and MAP kinases Toll Like Receptor 5 (TLR5) Cascade Assembly of the SARS-CoV-2 Replication-Transcription Complex (RTC) SARS-CoV-2 Genome Replication and Transcription Transcription of SARS-CoV-2 sgRNAs Replication of the SARS-CoV-2 genome PI3K/AKT Signaling in Cancer PTEN Loss of Function in Cancer

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 27
·Pré-clínico1
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 — Malformação capilar

Centros de Referência SUS

24 centros habilitados pelo SUS para Malformação capilar

Centros para Malformação capilar

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.

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Publicações mais relevantes

Timeline de publicações
0 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

Cerebrovascular Malformations Associated With Hereditary Hemorrhagic Telangiectasia and HHT-Like Syndromes: A Comparative Overview.

European journal of neurology2026 Feb

Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant vascular disorder marked by mucocutaneous telangiectasias, recurrent epistaxis, and visceral arteriovenous malformations (AVMs). Neurologic risks include brain AVMs and hemorrhagic stroke. Several rare genetic and sporadic syndromes ("HHT-like" syndromes) share overlapping vascular features, complicating diagnosis. Differentiating these conditions is essential for accurate neurovascular risk assessment. A comprehensive literature review (PubMed, Scopus, Embase, Google Scholar; 1990-2025) targeted cerebrovascular manifestations of HHT and related syndromes. Key entities included Wyburn-Mason syndrome, Cobb syndrome, Klippel-Trénaunay syndrome (KTS), neurofibromatosis type 1 (NF1), PHACE(S) syndrome, capillary malformation-AVM (CM-AVM), Parkes Weber syndrome (PWS), juvenile polyposis/HHT overlap (JP-HHT), HHT type 5 (BMP9/GDF2), PTEN hamartoma tumor syndrome (PHTS), and blue rubber bleb nevus syndrome (BRBNS). Data on gene variants, lesion types, neuroimaging, stroke risk, and neurologic outcomes were synthesized. High-flow cerebrovascular malformations similar to HHT are prominent in Wyburn-Mason syndrome, CM-AVM, and PWS, conferring a substantial hemorrhagic stroke risk. NF1 and PHACE(S) primarily feature occlusive arteriopathies linked to ischemic events. KTS, BRBNS, and PHTS predominantly show low- or mixed-flow anomalies with lower CNS hemorrhagic risk but increased thrombotic complications. JP-HHT carries added gastrointestinal cancer risk via SMAD4 variants, while HHT type 5 often presents incompletely. Genetic testing and tailored neuroimaging are critical for differentiation. Although many syndromes mimic HHT, few combine mucosal telangiectasias, high-flow AVMs, and recurrent hemorrhage. Integrating clinical, imaging, and genetic data enables precise diagnosis, risk stratification, and personalized management.

#3

Clinical, Psychosocial, and Care Coordination Concerns in Macrocephaly Capillary Malformation Syndrome: Insights From a Large International Survey.

Pediatric blood & cancer2026 Apr

Macrocephaly capillary malformation syndrome (M-CM or MCAP) is a rare overgrowth disorder characterized by primary megalencephaly, overgrowth, and a range of additional anomalies. This report presents findings from a survey of 101 caregivers or individuals with M-CM, collected by the M-CM Network. Respondents shared medical and psychosocial concerns across the age spectrum, with common issues including motor delays, speech deficits, hydrocephalus, and vascular abnormalities. The survey highlighted significant developmental and behavioral challenges for individuals. The report advocates for continued research focused on the comprehensive needs of patients with M-CM.

#4

Total hip replacement in Klippel-Trenaunay syndrome with massive AV Malformations. A Case report.

La Clinica terapeutica2026

KTS, known as Klippel-Trenaunay syndrome, is a rare condition present at birth that includes a combination of capillary malformation, varicose veins, or venous malformation in the lower limbs, and excessive growth of bone and soft tissue. This report describes a female patient has 53-year-old suffering from Klippel-Trenaunay syndrome with arteriovenous malformations who underwent total hip arthroplasty surgery. The treatment included preoperative embolization, intraoperative angiography to place an iliac artery occlusive balloon, and the use of modular hip arthroplasty components to address femoral and acetabular dysplasia. In the case of patients with the syndrome (KT), all problems that the patient suffers from must be discussed with a team of specialists and experienced people, and they also, require a radiologic evaluation for malformations to determine the most suitable surgical approach and make the right decision about whether to operate or not, avoid failure, and obtain the best results.

#5

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.

Publicações recentes

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📚 EuropePMCmostrando 156

2026

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

BMJ case reports
2026

Cerebrovascular Malformations Associated With Hereditary Hemorrhagic Telangiectasia and HHT-Like Syndromes: A Comparative Overview.

European journal of neurology
2026

Clinical, Psychosocial, and Care Coordination Concerns in Macrocephaly Capillary Malformation Syndrome: Insights From a Large International Survey.

Pediatric blood & cancer
2026

Total hip replacement in Klippel-Trenaunay syndrome with massive AV Malformations. A Case report.

La Clinica terapeutica
2025

An unusual case of Kippel-Trenaunay syndrome complicated with intrahepatic cholestasis of pregnancy and disseminated intravascular coagulation.

BMC pregnancy and childbirth
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

Not Just a Birthmark: A Case Report of a Subtle Port-Wine Stain Heralding Sturge-Weber Syndrome.

Cureus
2025

An Extremely Preterm Infant With PIK3CA-Related Overgrowth Spectrum (PROS): Alpelisib Treatment and Outcome.

Case reports in genetics
2026

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

Journal of pain and symptom management
2025

Necrobiosis lipoidica arising in a port wine stain treated with topical ruxolitinib.

Journal of osteopathic medicine
2025

Parkes Weber syndrome, a rare case of pulmonary hypertension: a case report.

European heart journal. Case reports
2025

Management of Chronic Non-Healing Ulcer in Child With Capillary Malformation.

Clinical pediatrics
2025

Autism Spectrum Disorder in a Child with Megalencephaly-capillary Malformation-polymicrogyria Syndrome: A Case Report.

Clinical psychopharmacology and neuroscience : the official scientific journal of the Korean College of Neuropsychopharmacology
2025

Sturge Weber syndrome in a multinational pediatric cohort: a systematic analysis of different types.

Orphanet journal of rare diseases
2025

Refractory Diarrhea Related to EPHB4 Mutation in a Patient With Capillary Malformation-Arteriovenous Type 2 Syndrome.

ACG case reports journal
2025

Bilateral Sturge-Weber syndrome with soft tissue hypertrophy and trichomegaly.

BMJ case reports
2025

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

Cureus
2025

A Case of Normal-pressure Hydrocephalus Caused by Cauda Equina Capillary Malformation.

NMC case report journal
2025

Evaluating the therapeutic efficacy of the 595 nm pulsed dye laser system for treating capillary malformations.

Dermatology reports
2025

Phakomatosis pigmentovascularis type 2a: a rare case report.

AME case reports
2025

Multidisciplinary, multicenter consensus for the care of patients affected with Sturge-Weber syndrome.

Orphanet journal of rare diseases
2025

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

International journal of dermatology
2025

PIK3CA-Related Overgrowth Spectrum: Exploring brain growth from fetus to infant.

Pediatric neurology
2024

A Review of Sturge-Weber Syndrome Brain Involvement, Cannabidiol Treatment and Molecular Pathways.

Molecules (Basel, Switzerland)
2024

Fegeler Syndrome Mimicking Kaposi Sarcoma: A Case Report.

Cureus
2024

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

Clinical case reports
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

Lobular Capillary Hemangioma of the Buccal Mucosa: A Rare Presentation.

Cureus
2024

Infantile Hemangioma with Minimal or Arrested Growth (IHMAG): A Retrospective Analysis of Clinical and Dermoscopic Diagnostic Clues.

Dermatology practical & conceptual
2025

Capillary malformation types are a marker of venous malformation severity.

Journal of the European Academy of Dermatology and Venereology : JEADV
2024

Delineation of the phenotypes and genotypes of PIK3CA-related overgrowth spectrum in East asians.

Molecular genetics and genomics : MGG
2024

STAMBP is Required for Long-Term Maintenance of Neural Progenitor Cells Derived from hESCs.

Stem cell reviews and reports
2024

Rare Presentation of the Vein of Servelle in a Case of Klippel-Trenaunay Syndrome.

Cureus
2023

Subepidermal Basal Cell carcinoma Following Laser Treatment of Congenital Capillary Malformation: A Case Report.

Acta dermatovenerologica Croatica : ADC
2024

De Novo Brain Vascular Malformations in Hereditary Hemorrhagic Telangiectasia.

Pediatric neurology
2024

RASopathies for Radiologists.

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

Coexistence of kaposiform hemangioendothelioma and capillary malformation: More than a coincidence? Two case reports.

Heliyon
2024

A Rare Case of Klippel Trenaunay Syndrome with Von Willebrand Factor Deficiency and Multiple Accessory Spleens: A Case Report and Brief Literature Review.

Advanced biomedical research
2024

A Case of Klippel-Trenaunay Syndrome Complicated by Group A Streptococcemia and Multiple Organ Failure.

Cureus
2024

Rare scrotal chylous effusion: A case report.

SAGE open medical case reports
2024

Epilepsy with faint capillary malformation or reticulated telangiectasia associated with mosaic AKT3 pathogenic variants.

American journal of medical genetics. Part A
2023

Sturge-Weber Syndrome: A Case Report.

JNMA; journal of the Nepal Medical Association
2024

Infrequent associations of cutis marmorata telangiectatica congenita: a two-case report.

Cirugia pediatrica : organo oficial de la Sociedad Espanola de Cirugia Pediatrica
2024

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

Pediatric dermatology
2023

Novel STAMBP mutations in a Chinese girl with rare symptoms of microcephaly-capillary malformation syndrome and Mowat-Wilson syndrome.

Heliyon
2023

An expanded clinical spectrum of hypoinsulinaemic hypoketotic hypoglycaemia.

Orphanet journal of rare diseases
2024

Molecular characterization of 13 patients with PIK3CA-related overgrowth spectrum using a targeted deep sequencing approach.

American journal of medical genetics. Part A
2023

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

Journal of neurosurgery. Case lessons
2023

Gastrointestinal involvement in Klippel-Trénaunay syndrome: pathophysiology, evaluation, and management.

Orphanet journal of rare diseases
2023

Targeted next-generation sequencing for detection of PIK3CA mutations in archival tissues from patients with Klippel-Trenaunay syndrome in an Asian population : List the full names and institutional addresses for all authors.

Orphanet journal of rare diseases
2023

Verification of the efficacy of topical sirolimus gel for systemic rare vascular malformations: a pilot study.

The Journal of dermatology
2023

Isolated diffuse choroidal hemangioma without systemic symptoms: a case report.

BMC ophthalmology
2023

High-Output Heart Failure in a Patient With Klippel-Trénaunay Syndrome: A Case Report.

Cureus
2023

Sturge-Weber Syndrome: A Review of Pathophysiology, Genetics, Clinical Features, and Current Management Approache.

The application of clinical genetics
2023

Developmental expression of the Sturge-Weber syndrome-associated genetic mutation in Gnaq: a formal test of Happle's paradominant inheritance hypothesis.

Genetics
2023

Diffuse Capillary Malformation With Overgrowth (DCMO): A Case Report and Literature Review.

Cureus
2023

Sociodemographic characteristics of pediatric patients with vascular malformations: Results of a single site study.

Frontiers in pediatrics
2023

A rare case of Klippel-Trenaunay syndrome presenting with chronic myeloid leukemia.

The Turkish journal of pediatrics
2023

Easily misdiagnosed complex Klippel-Trenaunay syndrome: A case report.

World journal of clinical cases
2023

Genetic testing in the evaluation of individuals with clinical diagnosis of atypical Sturge-Weber syndrome.

American journal of medical genetics. Part A
2022

Prenatal diagnosis and delivery of megalencephaly-capillary malformation syndrome.

BMJ case reports
2022

A Rare Case of Klippel-Trénaunay Syndrome.

Cureus
2022

Updates on Sturge-Weber Syndrome.

Stroke
2022

Further clinical delineation of microcephaly-capillary malformation syndrome.

American journal of medical genetics. Part A
2022

Pregnancy management for a woman with extensive vulvar and pelvic malformations caused by Klippel-Trénaunay syndrome.

Clinical case reports
2022

Qualitative research with patients and caregivers of patients with PIK3CA related overgrowth spectrum: content validity of clinical outcome assessments.

Journal of patient-reported outcomes
2022

Quality of life of children with capillary malformations of the lower limbs: Evolution and associated factors. Data from the French national paediatric cohort, CONAPE.

Annales de dermatologie et de venereologie
2022

An interesting case of nevus flammeus with loss of vision and hemiparesis.

Journal of family medicine and primary care
2022

Clinical and Molecular Spectrum of Sporadic Vascular Malformations: A Single-Center Study.

Biomedicines
2022

Megalencephaly-Capillary Malformation-Polymicrogyria Syndrome (MCAP): A Rare Dynamic Genetic Disorder.

Cureus
2022

Dermatologic manifestations in paediatric neurofibromatosis type 2: a cross sectional descriptive multicentric study.

Orphanet journal of rare diseases
2022

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

International journal of dermatology
2022

Brain overgrowth associated with megalencephaly-capillary malformation syndrome causing progressive Chiari and syringomyelia.

Surgical neurology international
2022

A novel somatic mutation in GNAQ in a capillary malformation provides insight into molecular pathogenesis.

Angiogenesis
2022

[Cutaneous capillary malformations with cerebral implementation].

Ugeskrift for laeger
2022

PIK3CA-related overgrowth with an uncommon phenotype: case report.

Italian journal of pediatrics
2022

Mapping the PIK3CA-related overgrowth spectrum (PROS) patient and caregiver journey using a patient-centered approach.

Orphanet journal of rare diseases
2022

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

Pediatric dermatology
2022

A rare case of persistent lateral marginal vein of Servelle in Klippel Trenaunay Syndrome: A successful surgical management.

International journal of surgery case reports
2022

Angioma serpiginosum: Two cases in children and review of literature.

Dermatology reports
2022

Phenotypic and molecular characterization of five patients with PIK3CA-related overgrowth spectrum (PROS).

American journal of medical genetics. Part A
2021

Undiagnosed Case of Klippel-Trenaunnay Syndrome Presenting as Extensive Heterotrophic Ossification and Flexion Deformity of Right Lower Limb Requiring Amputation : A Case Report.

JNMA; journal of the Nepal Medical Association
2022

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

Acta dermato-venereologica
2021

Coexistence of Neonatal Lupus Erythematous and Sturge-Weber Syndrome.

Case reports in dermatological medicine
2021

Autism spectrum disorder in a child with megalencephaly-capillary malformation-polymicrogyria syndrome (MCAP).

BMJ case reports
2022

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

Journal of the American Academy of Dermatology
2022

Hematoporphyrin monomethyl ether-mediated photodynamic therapy for phakomatosis pigmentovascularis type Ⅱ: A case report.

Photodiagnosis and photodynamic therapy
2021

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

Frontiers in pediatrics
2022

A rare case of cerebral proliferative angiopathy with acute cerebral infarction and facial capillary malformation.

The neuroradiology journal
2021

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

Pediatric dermatology
2022

A standard of care for individuals with PIK3CA-related disorders: An international expert consensus statement.

Clinical genetics
2020

Conservative management of knee arthropathy in a patient with Klippel Trenaunay syndrome.

Jornal vascular brasileiro
2021

Megalencephaly-capillary malformation syndrome and associated hydrocephalus: treatment options and revision of the literature.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2021

Proteus Syndrome: A Rare Case in An Adult Ward.

European journal of case reports in internal medicine
2021

Itchy Capillary Malformations: Unusual Appearance of Meyerson Phenomenon, a Case Series.

Pediatric reports
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

Progressive retinal vessel malformation in a premature infant with Sturge-Weber syndrome: a case report and a literature review of ocular manifestations in Sturge-Weber syndrome.

BMC ophthalmology
2021

Capillary Malformation-Arteriovenous Malformation Syndrome.

Cureus
2021

Sirolimus Treatment in Sturge-Weber Syndrome.

Pediatric neurology
2020

Gingival Biopsy to Detect Mosaicism in Overgrowth Syndromes: Report of Two Cases of Megalencephaly-Capillary Malformation Syndrome with Periodontal Anomalies.

Case reports in dentistry
2020

Introduction to phacomatoses (neurocutaneous disorders) in childhood.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2020

[Resistant epileptic encephalopathy in a child with microcephalic capillary malformation syndrome].

Zhurnal nevrologii i psikhiatrii imeni S.S. Korsakova
2021

Capillary malformation-arteriovenous malformation syndrome: a multicentre study.

Clinical and experimental dermatology
2020

Detailed analysis of phenotypes and genotypes in megalencephaly-capillary malformation-polymicrogyria syndrome caused by somatic mosaicism of PIK3CA mutations.

Orphanet journal of rare diseases
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
2020

Long-term follow-up of intracranial arteriovenous malformations with frontal capillary malformation (Wyburn-Mason syndrome or Bonnet-Dechaume-Blanc syndrome): three case reports.

International journal of dermatology
2020

Recurrent Venous Thromboembolism in a Patient with Klippel-Trenaunay Syndrome Despite Adequate Anticoagulation with Warfarin.

Cureus
2020

Intraneural hemangioma in Klippel-Trenaunay syndrome: role of musculo-skeletal ultrasound in diagnosis-case report and review of the literature.

Journal of ultrasound
2020

One of the First Cases with PIK3CA-related Overgrowth Spectrum (PROS) in Saudi Arabia: A Case Report and Literature Review.

Cureus
2020

A PIK3CA mutation in an acquired capillary malformation.

Pediatric dermatology
2019

Cutis marmorata telangiectatica congenita: a literature review.

Orphanet journal of rare diseases
2020

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

Pediatric dermatology
2019

Lower Extremity Skin Ulcer Associated With Neurofibromatosis Type 1: A Case Report.

Wounds : a compendium of clinical research and practice
2019

Klippel-Trénaunay syndrome: a case report of a rare vascular disorder identified in a rural Canadian hospital.

Rural and remote health
2019

Orthopaedic diagnoses in patients with Klippel-Trenaunay syndrome.

Journal of children's orthopaedics
2019

Clinical characteristics and treatment of 52 cases of phakomatosis pigmentovascularis.

The Journal of dermatology
2019

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

Brain & development
2019

Phakomatosis Pigmentovascularis: A Clinical Profile of 11 Indian Patients.

Indian journal of dermatology
2019

Idiopathic inflammatory myopathy comorbid with Sturge-Weber syndrome: a case report.

BMC neurology
2019

An Engorged Vein over the Lower Abdominal Wall.

The Journal of pediatrics
2019

Extracutaneous manifestations in phacomatosis cesioflammea and cesiomarmorata: Case series and literature review.

American journal of medical genetics. Part A
2019

[Arterial sinus pericranii. First case report].

Cirugia pediatrica : organo oficial de la Sociedad Espanola de Cirugia Pediatrica
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
2019

Association of Somatic GNAQ Mutation With Capillary Malformations in a Case of Choroidal Hemangioma.

JAMA ophthalmology
2018

Clinical pitfalls in the diagnosis of segmental overgrowth syndromes: a child with the c.2740G > A mutation in PIK3CA gene.

Italian journal of pediatrics
2018

Verrucous hemangioma (also known as verrucous venous malformation): A vascular anomaly frequently misdiagnosed as a lymphatic malformation.

Pediatric dermatology
2018

A female infant with phacomatosis pigmentovascularis and congenital chylous ascites: A case report.

Medicine
2018

Port-wine stain as a clue for two rare coexisting entities.

BMJ case reports
2018

RAS signalling in energy metabolism and rare human diseases.

Biochimica et biophysica acta. Bioenergetics
2018

In vitro efficacy of ARQ 092, an allosteric AKT inhibitor, on primary fibroblast cells derived from patients with PIK3CA-related overgrowth spectrum (PROS).

Neurogenetics
2018

A child with Apert syndrome and Sturge-Weber syndrome: could fibronectin or the RAS/MAPK signaling pathway be the connection?

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2018

CLAPO syndrome: identification of somatic activating PIK3CA mutations and delineation of the natural history and phenotype.

Genetics in medicine : official journal of the American College of Medical Genetics
2018

Human genetics and molecular mechanisms of vein of Galen malformation.

Journal of neurosurgery. Pediatrics
2018

A cross-sectional survey of long-term outcomes for patients with diffuse capillary malformation with overgrowth.

Journal of the American Academy of Dermatology
2018

Clinical and haemodynamic risk factors associated with discrepancies in lower limb length with capillary malformations: data from the national paediatric French cohort CONAPE.

The British journal of dermatology
2017

Wilms tumor screening in diffuse capillary malformation with overgrowth and macrocephaly-capillary malformation: A retrospective study.

Journal of the American Academy of Dermatology
2017

Successful Treatment of Unilateral Klippel-Trenaunay Syndrome With Pulsed-Dye Laser in a 2-Week Old Infant.

Journal of lasers in medical sciences
2017

Thick Corpus Callosum in Children.

Journal of clinical neurology (Seoul, Korea)
2017

Proteus Syndrome with Arteriovenous Malformation.

Advanced biomedical research
2016

Megalencephaly-capillary malformation-polymicrogyria syndrome: the first case report in Korea.

Korean journal of pediatrics
2016

Klippel Trenaunay Syndrome: A Case Report.

Mymensingh medical journal : MMJ
2017

Modeling RASopathies with Genetically Modified Mouse Models.

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

Somatic GNAQ Mutation is Enriched in Brain Endothelial Cells in Sturge-Weber Syndrome.

Pediatric neurology
2016

A rare atypical rapidly involuting congenital hemangioma combined with vascular malformation in the upper limb.

World journal of surgical oncology
2016

Combination of Sturge-Weber Syndrome and Trigonocephaly.

The Journal of craniofacial surgery
2016

Abstracts from the 3rd International Genomic Medicine Conference (3rd IGMC 2015) : Jeddah, Kingdom of Saudi Arabia. 30 November - 3 December 2015.

BMC genomics
2017

Megalencephaly-capillary malformation polymicrogyria: A review and complex pediatric case report.

Applied neuropsychology. Child
2016

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

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

Clinical Experience of the Klippel-Trenaunay Syndrome.

Archives of plastic surgery
2016

Unusual cause of lower extremity wounds: Cobb syndrome.

International wound journal
2015

PHACE syndrome misdiagnosed as a port-wine stain.

BMJ case reports
2015

Molecular and Functional Characterization of Three Different Postzygotic Mutations in PIK3CA-Related Overgrowth Spectrum (PROS) Patients: Effects on PI3K/AKT/mTOR Signaling and Sensitivity to PIK3 Inhibitors.

PloS one
2015

Phakomatosis pigmentovascularis presenting with sturge-weber syndrome and klippel-trenaunay syndrome.

Indian journal of dermatology
2014

Challenges in the management of a rare case of extensive retroperitoneal haemangioma in a pregnant woman.

Singapore medical journal

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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. Cerebrovascular Malformations Associated With Hereditary Hemorrhagic Telangiectasia and HHT-Like Syndromes: A Comparative Overview.
    European journal of neurology· 2026· PMID 41704211mais citado
  3. Clinical, Psychosocial, and Care Coordination Concerns in Macrocephaly Capillary Malformation Syndrome: Insights From a Large International Survey.
    Pediatric blood & cancer· 2026· PMID 41579065mais citado
  4. Total hip replacement in Klippel-Trenaunay syndrome with massive AV Malformations. A Case report.
    La Clinica terapeutica· 2026· PMID 41525108mais citado
  5. 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
  6. Hemodynamic and microvascular abnormalities in P450 oxidoreductase deficiency: evidence for COX-dependent dysfunction.
    Eur J Endocrinol· 2026· PMID 41693157recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:211247(Orphanet)
  2. MONDO:0016231(MONDO)
  3. Variantes catalogadas(ClinVar)
  4. Busca completa no PubMed(PubMed)
  5. Q56013802(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
Compêndio · Raras BR

Malformação capilar

ORPHA:211247 · MONDO:0016231
Medicamentos
10 registrados
MedGen
UMLS
C0340803
Wikidata
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