A síndrome da fenda palatina-orelhas grandes-cabeça pequena é uma síndrome genética rara caracterizada por fenda palatina, orelhas grandes e salientes, microcefalia e baixa estatura (início pré-natal). Outras anormalidades esqueléticas (idade óssea tardia, dedos afilados distalmente, falanges distais hipoplásicas, polegares posicionados proximalmente, clinodactilia do quinto dedo), sequência de Pierre Robin, displasia renal cística, acidose tubular renal proximal, hipospádia, anomalias cerebrais em exames de imagem (aumento dos ventrículos laterais, atrofia cortical leve), convulsões, hipotonia e atraso no desenvolvimento também são observados.
Introdução
O que você precisa saber de cara
A síndrome da fenda palatina-orelhas grandes-cabeça pequena é uma síndrome genética rara caracterizada por fenda palatina, orelhas grandes e salientes, microcefalia e baixa estatura (início pré-natal). Outras anormalidades esqueléticas (idade óssea tardia, dedos afilados distalmente, falanges distais hipoplásicas, polegares posicionados proximalmente, clinodactilia do quinto dedo), sequência de Pierre Robin, displasia renal cística, acidose tubular renal proximal, hipospádia, anomalias cerebrais em exames de imagem (aumento dos ventrículos laterais, atrofia cortical leve), convulsões, hipotonia e atraso no desenvolvimento também são observados.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 5 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 25 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
Triagem neonatal (Teste do Pezinho)
A triagem neonatal permite diagnóstico precoce e início imediato do tratamento.
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Nenhum gene associado encontrado
Os dados genéticos desta condição ainda estão sendo catalogados.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Síndrome de fenda palatina-pavilhões auriculares grandes-microcefalia
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Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Pesquisa e ensaios clínicos
Nenhum ensaio clínico registrado para esta condição.
Publicações mais relevantes
Multiple rhabdomyomatous mesenchymal hamartomas in a patient with mosaic Barber-Say syndrome.
Barber-Say syndrome (BSS) is a rare congenital ectodermal dysplasia with few cases reported in the literature. We describe a 9-year-old boy with congenital generalized hypertrichosis and multiple rhabdomyomatous mesenchymal hamartomas (RMHs) on his nose and periocular region. Next-generation sequencing, performed in DNA from a blood sample, and RMH tissue, revealed a pathogenic variant in the TWIST2 gene, which was not detected in a salivary sample of the patient, nor in his parents. Therefore, we consider this variant as de novo mosaicism. To our knowledge, this is the first case of multiple RMHs associated with BSS.
High-power laser as a treatment of recurrent gingival fibromatosis in a patient with a rare syndrome: A case report.
Ocular adnexal phenotype and management of a patient with mosaic expression of a mutation in TWIST2.
Ablepharon-macrostomia syndrome (AMS) and Barber-Say syndrome (BSS) are congenital ectodermal dysplasias associated with mutations in the TWIST2 gene. Among the ophthalmic anomalies that occur in these syndromes, underdevelopment of the anterior lamella of the eyelid is a defining feature. Reports of mosaic expression of TWIST2 mutations are extremely rare, with only five confirmed or suspected cases described to date. Mosaic expression of TWIST2 variants is correlated with a less severe phenotype than that reported for the typical expression of TWIST2 variants associated with BSS or AMS. Abnormal development of the anterior lamella appears to be a common feature in all cases of AMS with mosaic expression. Here, we describe the phenotype of a patient with mosaic expression of a TWIST2 mutation that is typically associated with AMS. We additionally describe the surgical approach employed in the treatment of this patient.
Barber Say Syndrome (A New Case Report).
Barber Say syndrome (BSS) is a rare ectodermal dysplasia with neonatal onset characterized by congenital generalized hypertrichosis, atrophic skin, ectropion and macrostomia. A literature review showed less than 20 previously reported cases of Barber Say syndrome. This presentation reports a one day old female with syndrome face, low hairline, coarse face, macrostomia, thin upper lip, bilateral ectropion and hypertelorism, hypertrichosis, senile skin appearance, hypoplastic nipples and one area of mild skin atrophy. These findings are consistent with BSS.
Multidisciplinary eyelid reconstruction in Barber-Say syndrome: A case report.
Barber-Say syndrome is an unusual dysplasia caused by the mutation of the TWIST2 gene (2q37.3), which encodes a protein that acts at an epigenetic level. The case is presented of a 2-day-old male child in whom ectropion, hypertelorism, hypertrichosis and other dysmorphic features led to the clinical diagnosis of Barber-Say syndrome, which was later confirmed with genetic tests. Around 20 cases have been reported on this syndrome, of which less than half have described the surgical technique, as it represents a surgical challenge. The approach in this case included a lateral tarsorrhaphy and skin grafts taken from the volar surface of the forearm, retroauricular area and supraclavicular fossa, as well as autologous lipografts from the inner side of both thighs for palpebral reconstruction. This is the first case of Barber-Say syndrome in which the use of skin grafts are taken from supraclavicular fossa and forearms.
Publicações recentes
Prevalence estimation of a rare disease with the French National Rare Disease Registry: example of TNF receptor associated periodic syndrome (TRAPS).
Efficacy and organ protective effects of continuous renal replacement therapy in children with organic acidemia complicated by decompensated acidosis: a retrospective study in PICU.
The incidence rate and gene mutation characteristics of hyperphenylalaninemia in Yunnan Province, Southwest China.
Risk factors, stroke rates and aspirin prescribing trends in the Canadian Fabry disease initiative cohort.
Novel pathogenic variant in the deficiency in ELF4, X-linked (DEX): case report and literature review.
🥉 Relato de caso📚 EuropePMCmostrando 15
Multiple rhabdomyomatous mesenchymal hamartomas in a patient with mosaic Barber-Say syndrome.
Pediatric dermatologyHigh-power laser as a treatment of recurrent gingival fibromatosis in a patient with a rare syndrome: A case report.
Photodermatology, photoimmunology & photomedicineOcular adnexal phenotype and management of a patient with mosaic expression of a mutation in TWIST2.
Orbit (Amsterdam, Netherlands)Barber Say Syndrome (A New Case Report).
Indian dermatology online journalMultidisciplinary eyelid reconstruction in Barber-Say syndrome: A case report.
Archivos de la Sociedad Espanola de OftalmologiaAblepharon and craniosynostosis in a patient with a localized TWIST1 basic domain substitution.
American journal of medical genetics. Part AClinical Description, Molecular Analysis of TWIST2 Gene, and Surgical Treatment in a Patient With Barber-Say Syndrome.
Ophthalmic plastic and reconstructive surgeryBarber-Say Syndrome and Ablepharon-Macrostomia Syndrome: A Patient's View.
Molecular syndromologyBarber-say syndrome: a confirmed case of TWIST2 gene mutation.
Clinical case reportsThe focal facial dermal dysplasias: phenotypic spectrum and molecular genetic heterogeneity.
Journal of medical geneticsBarber-Say syndrome and Ablepharon-Macrostomia syndrome: An overview.
American journal of medical genetics. Part ATransmission of Barber-Say syndrome from a mosaic father to his child in an Indian family.
Clinical dysmorphologyGeneral anesthesia of a Japanese infant with Barber-Say syndrome: a case report.
JA clinical reportsRecurrent Mutations in the Basic Domain of TWIST2 Cause Ablepharon Macrostomia and Barber-Say Syndromes.
American journal of human geneticsA case of Barber-Say syndrome in a male Japanese newborn.
Clinical case reportsAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Síndrome de fenda palatina-pavilhões auriculares grandes-microcefalia.
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Comunidades
Grupos ativos de quem convive com esta doença aqui no Raras
Ainda não existe comunidade no Raras para Síndrome de fenda palatina-pavilhões auriculares grandes-microcefalia
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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.
- Multiple rhabdomyomatous mesenchymal hamartomas in a patient with mosaic Barber-Say syndrome.
- High-power laser as a treatment of recurrent gingival fibromatosis in a patient with a rare syndrome: A case report.
- Ocular adnexal phenotype and management of a patient with mosaic expression of a mutation in TWIST2.
- Barber Say Syndrome (A New Case Report).
- Multidisciplinary eyelid reconstruction in Barber-Say syndrome: A case report.
- Prevalence estimation of a rare disease with the French National Rare Disease Registry: example of TNF receptor associated periodic syndrome (TRAPS).
- Efficacy and organ protective effects of continuous renal replacement therapy in children with organic acidemia complicated by decompensated acidosis: a retrospective study in PICU.
- The incidence rate and gene mutation characteristics of hyperphenylalaninemia in Yunnan Province, Southwest China.
- Risk factors, stroke rates and aspirin prescribing trends in the Canadian Fabry disease initiative cohort.
- Novel pathogenic variant in the deficiency in ELF4, X-linked (DEX): case report and literature review.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:2013(Orphanet)
- OMIM OMIM:181180(OMIM)
- MONDO:0008402(MONDO)
- GARD:162(GARD (NIH))
- Busca completa no PubMed(PubMed)
- Q7429066(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
