Raras
Buscar doenças, sintomas, genes...
Macroaneurisma arterial retiniano familiar
ORPHA:284247CID-10 · H35.0CID-11 · 9B7YOMIM 614224DOENÇA RARA
Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Hemorragia intraocular é o sangramento dentro do olho. O sangramento pode ocorrer a partir de qualquer estrutura do olho onde haja vasculatura ou fluxo sanguíneo, incluindo a câmara anterior, a cavidade vítrea, a retina, a coroide, o espaço supracoroidiano ou o disco óptico.

Publicações científicas
14 artigos
Último publicado: 2023 Sep

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
<1 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Europe
Início
Childhood
🏥
SUS: Cobertura mínimaScore: 5%
Triagem neonatal (Fase 5)CID-10: H35.0
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

Encontrou um erro ou informação desatualizada? Sugira uma correção →

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

Características mais comuns

100%prev.
Estenose pulmonar
Frequência: 13/13
100%prev.
Macroaneurismas arteriais retinianos
Frequência: 22/22
Herança autossômica recessiva
Descolamento exsudativo da retina
4sintomas
Muito frequente (2)
Sem dados (2)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 4 características clínicas mais associadas, ordenadas por frequência.

Estenose pulmonarPulmonic stenosis
Frequência: 13/13100%
Macroaneurismas arteriais retinianosRetinal arterial macroaneurysms
Frequência: 22/22100%
Herança autossômica recessivaAutosomal recessive inheritance
Descolamento exsudativo da retinaExudative retinal detachment

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa4desde 2022
Total histórico14PubMed
Últimos 10 anos6publicações
Pico20203 papers
Linha do tempo
2022Hoje · 2026📈 2020Ano de pico
Publicações por ano (últimos 10 anos)

Triagem neonatal (Teste do Pezinho)

👶
Teste: qPCR para deleção de SMN1 em sangue seco
Fase 5 do PNTNpending
Incidência no Brasil: 1:10.000

A triagem neonatal permite diagnóstico precoce e início imediato do tratamento.

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

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.

IGFBP7Insulin-like growth factor-binding protein 7Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Binds IGF1 and IGF2 with a relatively low affinity. Stimulates prostacyclin (PGI2) production. Stimulates cell adhesion. Acts as a ligand for CD93 to play a role in angiogenesis (PubMed:38218180)

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
Post-translational protein phosphorylationRegulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)
MECANISMO DE DOENÇA

Retinal arterial macroaneurysm with supravalvular pulmonic stenosis

An autosomal recessive condition characterized by the bilateral appearance of 'beading' along the major retinal arterial trunks, with the subsequent formation of macroaneurysms. Affected individuals also have supravalvular pulmonic stenosis, often requiring surgical correction.

EXPRESSÃO TECIDUAL(Ubíquo)
Aorta
5638.9 TPM
Artéria tibial
4250.8 TPM
Artéria coronária
4211.3 TPM
Tireoide
1485.1 TPM
Cervix Endocervix
1146.8 TPM
OUTRAS DOENÇAS (1)
familial retinal arterial macroaneurysm
HGNC:5476UniProt:Q16270

Variantes genéticas (ClinVar)

20 variantes patogênicas registradas no ClinVar.

🧬 IGFBP7: NM_001553.3(IGFBP7):c.830-24T>G ()
🧬 IGFBP7: GRCh37/hg19 4p12-q35.2(chr4:45455621-191003541)x3 ()
🧬 IGFBP7: GRCh37/hg19 4q12-13.3(chr4:57584845-72430996)x1 ()
🧬 IGFBP7: GRCh37/hg19 4q11-13.1(chr4:52685685-61903883)x1 ()
🧬 IGFBP7: GRCh38/hg38 4p16.3-q12(chr4:85624-57073230)x3 ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 3 variantes classificadas pelo ClinVar.

2
1
Patogênica (66.7%)
Benigna (33.3%)
VARIANTES MAIS SIGNIFICATIVAS
IGFBP7: NM_001553.3(IGFBP7):c.829G>A (p.Gly277Ser) [Pathogenic]
IGFBP7: NM_001553.3(IGFBP7):c.830-1G>A [Pathogenic/Likely pathogenic]
IGFBP7: NM_001553.3(IGFBP7):c.585+4T>A [Benign]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Macroaneurisma arterial retiniano familiar

🗺️

Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

Nenhum ensaio clínico registrado para esta condição.

🧪 Está conduzindo uma pesquisa?
Divulgue para pacientes e familiares que acompanham esta doença.
Divulgar pesquisa →

Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

Giant coronary artery aneurysm associated with familial retinal artery macroaneurysm: a case report.

European heart journal. Case reports2022 Feb

The term 'Giant' coronary aneurysm is usually defined as any coronary aneurysm more than 8 mm in maximum diameter. The form of familial retinal arterial macroaneurysms (FRAMs) is a rare autosomal recessive disease that is described by arterial aneurysm formation in the retina. Here, we report an association of coronary artery aneurysms with FRAM in a young male who presented with the acute coronary syndrome. A 31-year-old male smoker presented to the emergency department with atypical chest pain for 5 days. Blood investigations showed raised troponin enzymes. Review of his past medical history revealed decreased vision in the left eye, starting at the age of 10 years which progressed to blindness. He was diagnosed to have IGFBP7 mutation which causes eye manifestations in the form of FRAM. Fundoscopy showed bleeding retinal artery macroaneurysms in the right retina and sub-retinal gliosis suggesting laser treatment for the prior retinal arterial aneurysm. Coronary angiogram revealed a large aneurysm in the proximal segment of the left anterior descending (LAD) artery. Cardiac computed tomography scan with contrast was done which showed a 2.28 × 1.64 cm coronary aneurysm at the proximal segment of the LAD artery with peripheral calcification with a narrow neck of about 0.6 cm. After the heart team discussion, he underwent surgical ligation of the LAD and coronary aneurysm with implantation of the saphenous venous graft to distal LAD. Coronary artery aneurysms can be a part of multisystem diseases like FRAM. The management should be individualized based on symptoms at presentation, size of coronary aneurysms, and local expertise.

#2

Left anterior descending artery aneurysm in a young patient with familial retinal arterial macroaneurysm: A case report.

American journal of ophthalmology case reports2022 Jun

Familial retinal arterial macroaneurysm (FRAM) is a rare genetic disorder caused by a gene mutation in the insulin-like growth factor binding protein 7 (IGFBP7). We report a 30-year-old male with FRAM and IGFBP7 gene mutation who presented with an acute coronary syndrome (ACS). Invasive coronary angiography revealed a large aneurysm at the proximal part of the left anterior descending (LAD) artery. Few cases with systemic vascular involvement in patients with FRAM have been described before; however, our case represents the first documentation of a LAD artery aneurysm in a patient with FRAM and IGFBP7 gene mutation.

#3

Retinal arteriolar macroaneurysms with supravalvular pulmonic stenosis in the United Arab Emirates.

Ophthalmic genetics2022 Feb

Retinal arteriolar macroaneurysms with supravalvular pulmonic stenosis (RAMSVPS) is a rare syndrome that to date has only been reported in Saudi Arabian families. All tested patients have been homozygous for a single IGFBP7 splice variant (NM_001553.2:c.830-1G>A). We report our experience with RAMSVPS in the United Arab Emirates. Retrospective case series. Five affected individuals (two males and three females) from two unrelated Emirati families were known to our institution (age of first signs 6 months to 10 years of age, with one asymptomatic 6-year-old boy identified by sibling screening examination). Initial ophthalmic diagnoses had been Coats disease or traumatic retinal bleeding. Characteristic retinal arteriolar trunk beading and macroaneurysms led to the actual diagnosis of RAMSVPS. One child with esotropia at 6 months of age seemed to have unilateral Coats disease until retinal signs became apparent in the contralateral eye at 4 years old. One family consented to genetic testing, and both affected siblings were homozygous for the Saudi IGFBP7 splice variant (c.830-1G>A). The three children who underwent echocardiography were all confirmed to have cardiac valvular abnormalities (two supravalvular pulmonic stenosis and one tricuspid stenosis). The distinct ophthalmic phenotype of RAMSVPS is important to recognize because of systemic implications. Retinal findings can be misinterpreted as sequelae of trauma or Coats disease and can seem unilateral in very young children until changes in the contralateral eye become apparent years later. The homozygous IGFBP7 splice variant associated with the disease likely represents an ancestral founder effect for the Arabian Peninsula.

#4

Phenotypic delineation of the retinal arterial macroaneurysms with supravalvular pulmonic stenosis syndrome.

Clinical genetics2020 Mar

Retinal arterial macroaneurysms with supravalvular pulmonic stenosis (RAMSVPS), also known as Familial Retinal Arterial Macroaneurysms (FRAM) syndrome, is a very rare multisystem disorder. Here, we present a case series comprising ophthalmologic and systemic evaluation of patients homozygous for RAMSVPS syndrome causative IGFBP7 variant. New clinical details on 22 previously published and 8 previously unpublished patients are described. Age at first presentation ranged from 1 to 34 years. The classical feature of macroaneurysms and vascular beading involving the retinal arteries was universal. Follow up extending up to 14 years after initial diagnosis revealed recurrent episodes of bleeding and leakage from macroaneurysms in 55% and 59% of patients, respectively. The majority of patients who underwent echocardiography (18/23) showed evidence of heart involvement, most characteristically pulmonary (valvular or supravalvular) stenosis, often requiring surgical correction (12/18). Four patients died in the course of the study from complications of pulmonary stenosis, cerebral hemorrhage, and cardiac complications. Liver involvement (usually cirrhosis) was observed in eight patients. Cerebral vascular involvement was observed in one patient, and stroke was observed in two. We conclude that RAMSVPS is a recognizable syndrome characterized by a high burden of ocular and systemic morbidity, and risk of premature death. Recommendations are proposed for early detection and management of these complications.

#5

Familial Retinal Arterial Macroaneurysms with Peripheral Retinal Ischemia: A Characterization on Ultra-Widefield Fluorescein Angiography.

Middle East African journal of ophthalmology2020

Retinal arterial macroaneurysm with supravalvular pulmonic stenosis (RAMSVPS), also known as familial retinal arterial macroaneurysm syndrome, is an autosomal recessive disorder associated with many life-threatening complications. The disease is characterized by progressive multiple retinal arterial macroaneurysms, retinal arterial beading, and recurrent vitreous hemorrhage. Here, we describe the first case of RAMSVPS associated with peripheral ischemia evident on ultra-widefield fluorescein angiography. A 23-year-old otherwise healthy woman presented to our ophthalmology emergency room with a 1-week history of right eye floaters and decreased vision. Dilated fundoscopic examination showed right eye vitreous and preretinal hemorrhage with bilateral active and involuted retinal arterial macroaneurysms, arterial beading, and sclerosed vessels. Ultra-widefield fluorescein angiography demonstrated multiple macroaneurysms on the optic discs and along the major retinal arteries in addition to significant peripheral retinal ischemia in both eyes. Echocardiography showed severe supravalvular pulmonic stenosis that required urgent pulmonary valvuloplasty. Indeed, peripheral retinal ischemia is an additional finding in RAMSVPS.

Publicações recentes

Ver todas no PubMed

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

Ainda não temos associações cadastradas para Macroaneurisma arterial retiniano familiar.

É de uma associação que acompanha esta doença? Fale com a gente →

Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

Ainda não existe comunidade no Raras para Macroaneurisma arterial retiniano familiar

Pacientes, familiares e cuidadores se organizam em comunidades pra compartilhar experiências, fazer perguntas e se apoiar. Você pode ser o primeiro.

Tire suas dúvidas

Perguntas, dicas e experiências compartilhadas aqui na página

Participe da discussão

Faça login para postar dúvidas, compartilhar experiências e interagir com especialistas.

Fazer login

Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

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

  1. Giant coronary artery aneurysm associated with familial retinal artery macroaneurysm: a case report.
    European heart journal. Case reports· 2022· PMID 35299703mais citado
  2. Left anterior descending artery aneurysm in a young patient with familial retinal arterial macroaneurysm: A case report.
    American journal of ophthalmology case reports· 2022· PMID 35514799mais citado
  3. Retinal arteriolar macroaneurysms with supravalvular pulmonic stenosis in the United Arab Emirates.
    Ophthalmic genetics· 2022· PMID 34519236mais citado
  4. Phenotypic delineation of the retinal arterial macroaneurysms with supravalvular pulmonic stenosis syndrome.
    Clinical genetics· 2020· PMID 31730227mais citado
  5. Familial Retinal Arterial Macroaneurysms with Peripheral Retinal Ischemia: A Characterization on Ultra-Widefield Fluorescein Angiography.
    Middle East African journal of ophthalmology· 2020· PMID 33814820mais citado
  6. Venous Tortuosity in COL4A2-Associated Gould Syndrome.
    Ophthalmic Surg Lasers Imaging Retina· 2023· PMID 37642429recente
  7. Retinal arterial macroaneurysms with supravalvular pulmonic stenosis syndrome can be associated with coronary and major systemic arterial disease.
    Am J Ophthalmol Case Rep· 2022· PMID 35464689recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:284247(Orphanet)
  2. OMIM OMIM:614224(OMIM)
  3. MONDO:0013640(MONDO)
  4. GARD:12779(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q55784267(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

Compêndio · Raras BR

Macroaneurisma arterial retiniano familiar

ORPHA:284247 · MONDO:0013640
🇧🇷 Brasil SUS
Triagem
qPCR para deleção de SMN1 em sangue seco
PNTN
Fase 5
Incidência BR
1:10.000
Geral
Prevalência
<1 / 1 000 000
Herança
Autosomal recessive
CID-10
H35.0 · Retinopatias de fundo e alterações vasculares da retina
CID-11
Início
Childhood
Prevalência
0.0 (Europe)
MedGen
UMLS
C0423402
EuropePMC
Wikidata
DiscussaoAtiva

Nenhuma novidade ainda. O agente esta monitorando.

0membros
0novidades