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Juvenile hyaline fibromatosis
ORPHA:2028CID-10 · M72.8CID-11 · EE6YDOENÇA RARA

A fibromatose hialina juvenil (FJ) é um tumor raro de tecidos moles, caracterizado por lesões cutâneas papulonodulares (especialmente ao redor da cabeça e pescoço), massas de tecidos moles, hipertrofia gengival, contraturas articulares e lesões ósseas osteolíticas em graus variáveis. As contraturas articulares podem incapacitar os pacientes e atrasar o desenvolvimento motor normal se ocorrerem na infância. A hiperplasia gengival grave pode interferir na alimentação e atrasar a dentição. A análise histopatológica dos tecidos envolvidos revela cordões de células fusiformes incorporadas em um material amorfo e hialino. A ICJ é uma forma leve de hialinose sistêmica infantil.

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Introdução

O que você precisa saber de cara

📋

A fibromatose hialina juvenil (FJ) é um tumor raro de tecidos moles, caracterizado por lesões cutâneas papulonodulares (especialmente ao redor da cabeça e pescoço), massas de tecidos moles, hipertrofia gengival, contraturas articulares e lesões ósseas osteolíticas em graus variáveis. As contraturas articulares podem incapacitar os pacientes e atrasar o desenvolvimento motor normal se ocorrerem na infância. A hiperplasia gengival grave pode interferir na alimentação e atrasar a dentição. A análise histopatológica dos tecidos envolvidos revela cordões de células fusiformes incorporadas em um material amorfo e hialino. A ICJ é uma forma leve de hialinose sistêmica infantil.

Publicações científicas
178 artigos
Último publicado: 1993

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
Worldwide
Casos conhecidos
70
pacientes catalogados
Início
Childhood
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: M72.8
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Entender a doença

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Sinais e sintomas

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

Partes do corpo afetadas

🧬
Pele e cabelo
4 sintomas
💪
Músculos
2 sintomas
🦴
Ossos e articulações
2 sintomas
🫃
Digestivo
1 sintomas
😀
Face
1 sintomas
📏
Crescimento
1 sintomas

+ 5 sintomas em outras categorias

Características mais comuns

90%prev.
Morfologia anormal da diáfise
Muito frequente (99-80%)
90%prev.
Morfologia anormal do crânio
Muito frequente (99-80%)
90%prev.
Anormalidade da face
Muito frequente (99-80%)
90%prev.
Pápula
Muito frequente (99-80%)
90%prev.
Nódulo subcutâneo
Muito frequente (99-80%)
90%prev.
Anormalidade do cabelo
Muito frequente (99-80%)
16sintomas
Muito frequente (6)
Frequente (3)
Ocasional (7)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 16 características clínicas mais associadas, ordenadas por frequência.

Morfologia anormal da diáfiseAbnormal diaphysis morphology
Muito frequente (99-80%)90%
Morfologia anormal do crânioAbnormal skull morphology
Muito frequente (99-80%)90%
Anormalidade da faceAbnormality of the face
Muito frequente (99-80%)90%
PápulaPapule
Muito frequente (99-80%)90%
Nódulo subcutâneoSubcutaneous nodule
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico178PubMed
Últimos 10 anos46publicações
Pico20226 papers
Linha do tempo
2026Hoje · 2026🧪 2010Primeiro ensaio clínico📈 2022Ano de pico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

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

ANTXR2Anthrax toxin receptor 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Necessary for cellular interactions with laminin and the extracellular matrix (Microbial infection) Receptor for the protective antigen (PA) of B.anthracis (PubMed:12700348, PubMed:15243628, PubMed:15326297). Binding of PA leads to heptamerization of the receptor-PA complex (PubMed:12700348, PubMed:15243628, PubMed:15326297). Upon binding of the PA of B.anthracis, the complex moves to glycosphingolipid-rich lipid rafts, where it is internalized via a clathrin-dependent pathway (PubMed:12551953,

LOCALIZAÇÃO

Cell membraneEndoplasmic reticulum membraneSecreted

VIAS BIOLÓGICAS (1)
Uptake and function of anthrax toxins
MECANISMO DE DOENÇA

Hyaline fibromatosis syndrome

An autosomal recessive syndrome characterized by abnormal growth of hyalinized fibrous tissue usually affecting subcutaneous regions on the scalp, ears, neck, face, hands, and feet. The lesions appear as pearly papules or fleshy nodules. Additional features include gingival hypertrophy, progressive joint contractures resulting in severe limitation of mobility, osteopenia, and osteoporosis. Disease severity is variable. Some individuals manifest symptoms in infancy and have additional visceral or systemic involvement. Hyaline deposits in multiple organs, recurrent infections and intractable diarrhea often lead to early death. Surviving children may suffer from severely reduced mobility due to joint contractures. Other patients have later onset of a milder disorder affecting only the face and digits.

INTERAÇÕES PROTEICAS (4)
OUTRAS DOENÇAS (3)
hyaline fibromatosis syndromeinfantile systemic hyalinosisjuvenile hyaline fibromatosis
HGNC:21732UniProt:P58335

Variantes genéticas (ClinVar)

67 variantes patogênicas registradas no ClinVar.

🧬 ANTXR2: NM_058172.6(ANTXR2):c.1347+1G>T ()
🧬 ANTXR2: NM_058172.6(ANTXR2):c.54_60dup (p.Leu21fs) ()
🧬 ANTXR2: NC_000004.11:g.(?_80822299)_(80906018_80929674)del ()
🧬 ANTXR2: NM_058172.6(ANTXR2):c.1114_1115dup (p.Trp373fs) ()
🧬 ANTXR2: NM_058172.6(ANTXR2):c.1179+1G>C ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 2 variantes classificadas pelo ClinVar.

2
Patogênica (100.0%)
VARIANTES MAIS SIGNIFICATIVAS
ANTXR2: NC_000004.11:g.(?_80822299)_(80906018_80929674)del [Likely pathogenic]
ANTXR2: NM_058172.6(ANTXR2):c.652T>C (p.Cys218Arg) [Pathogenic/Likely pathogenic]

Vias biológicas (Reactome)

1 via biológica associada aos genes desta condição.

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.
·Pré-clínico1
Medicamentos catalogadosEnsaios clínicos· 0 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 — Juvenile hyaline fibromatosis

🗺️

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

3 ensaios clínicos encontrados.

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

Timeline de publicações
47 papers (10 anos)
#1

Juvenile Hyaline Fibromatosis Syndrome: A Novel Variant in the ANTXR2 Gene Causing Severe Phenotype.

Pediatric dermatology2026

Hyaline fibromatosis syndrome (HFS) is an autosomal recessive disorder caused by variants in the ANTXR2 gene. Clinically, HFS is characterized by papular and nodular skin lesions, gingival hyperplasia, joint contractures, and bone involvement in variable degrees. In this report, we present a 3-year-old Syrian boy with HFS, detailing his clinical and genetic profile, furthering the understanding of genotype-phenotype correlation in the ANTXR2 gene and HFS.

#2

Unraveling Juvenile Hyaline Fibromatosis: A Case of Novel ANTXR2 Mutations Associated with Subcutaneous Masses and Hydronephrosis.

Pediatric blood &amp; cancer2025 Mar
#3

Genetic Insights Into Hyaline Fibromatosis Syndrome: A Case Report of an ANTXR2 Mutation Featuring a Rare Variant c.697+1G>A.

Clinical case reports2025 Apr

Hyaline fibromatosis syndrome (HFS) is a rare genetic disorder encompassing juvenile hyaline fibromatosis (JHF) and infantile systemic hyalinosis (ISH), caused by mutations in the anthrax toxin receptor 2 gene (ANTXR2). This condition leads to the accumulation of hyaline plaques in the skin and organs, resulting in symptoms such as skin lesions, joint contractures, and digestive issues, often culminating in early mortality due to infections or diarrhea. By 2005, 20 mutations in ANTXR2 linked to ISH and JHF had been documented, impairing cellular adhesion to the laminin matrix. In this study, we present a case of a 6-month-old Iranian female of western Asian ethnicity, born to consanguineous parents. She exhibited hyperpigmentation of the proximal interphalangeal (PIP) joints, knee flexion contractures, persistent diarrhea, and failure to thrive. Initial assessments suggested arthrogryposis; however, the presence of hyperpigmented nodules and perianal plaques prompted further investigation. Genetic analysis confirmed a homozygous mutation in ANTXR2 and incidental heterozygous mutations in the HEXA and PAH genes. The patient will undergo regular monitoring and may require immunosuppressive therapy and orthopedic interventions. Hyaline fibromatosis syndrome presents unique diagnostic challenges due to its overlap with other conditions like arthrogryposis. While arthrogryposis typically lacks systemic symptoms, HFS is marked by significant pain and systemic manifestations due to hyaline deposits in tissues. The case presented aligns with existing literature regarding HFS characteristics, including joint contractures and skin lesions. The identification of the c.697+1G>A mutation at a splice site within the ANTXR2 gene highlights potential mechanisms contributing to HFS pathology. This finding emphasizes the necessity for comprehensive genetic profiling when diagnosing rare syndromes. Furthermore, low allele frequencies of this variant across population databases underscore its rarity and potential significance in disease manifestation. Hyaline fibromatosis syndrome (HFS) is an uncommon autosomal recessive disorder that is marked by notable clinical features, such as the deposition of hyaline material in various tissues, joint contractures, and systemic complications. The case discussed illustrates the diagnostic challenges associated with HFS, particularly in a young patient who presented with atypical symptoms that initially indicated arthrogryposis. Genetic analysis revealed a homozygous mutation in the ANTXR2 gene, specifically the c.697+1G>A variant, which interferes with normal splicing and results in the absence of functional protein. This observation emphasizes the critical role of genetic testing in the precise diagnosis of rare syndromes and in differentiating them from other hereditary disorders.

#4

Recurrent Giant Subcutaneous Tumor in Juvenile Hyaline Fibromatosis.

The Journal of craniofacial surgery2025 Jun 01

This case report presents a 5-year-old boy diagnosed with juvenile hyaline fibromatosis (JHF), an extremely rare autosomal recessive disorder characterized by the abnormal accumulation of collagen. The patient exhibited a recurrent giant subcutaneous tumor measuring 20 cm in diameter, along with multiple tumors in the oral cavity, gingiva, and joints, leading to significant facial deformity and functional impairments. Previous surgeries at ages 1 and 2 for tumor removal resulted in recurrence. Surgical intervention was performed to excise the large tumor and alleviate symptoms. Pathologic analysis confirmed the diagnosis of JHF. This case highlights the challenges in managing JHF and the need for multidisciplinary approaches in treatment.

#5

Hyaline Fibromatosis Syndrome Presenting with Nasal Mass: A Case Report.

Ear, nose, &amp; throat journal2025 Mar

Hyaline fibromatosis syndrome (HFS) is a rare, autosomally-recesfvsive disease characterized by papulonodular skin lesions, soft tissue masses, joint contractures, gingival overgrowth, and osteolytic bone lesions. Mutations in capillary morphogenesis gene 2 are responsible for both these conditions. Generally, an autosomal recessive pattern is assumed to be the most common mode of inheritance. Here, we report an unusual case of a twenty-three-year-old female patient with HFS who reported with a chief complaint of growing nasal mass for three months. There was no history of pain or bleeding associated with the nasal mass. Due to the growing mass, she experienced right nasal obstruction, which compromised her quality of life. There was an unremarkable family history. Her physical examination revealed multiple asymptomatic pinkish-white papulonodular lesions located at multiple sites. Intra orally, she had generalized gingival enlargement. Her nasal examination revealed a right sided nasal mass, bright red in color. The lesion was soft on palpation. All the results of hematological and biochemical tests were normal. However, skeletal radiographic examination showed the joint contractures on her knees and elbows without the presence of osteolytic bone lesions. The nasal lesion was surgically excised and histopathological examination revealed features suggestive of HFS.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC138 artigos no totalmostrando 45

2026

Juvenile Hyaline Fibromatosis Syndrome: A Novel Variant in the ANTXR2 Gene Causing Severe Phenotype.

Pediatric dermatology
2025

Genetic Insights Into Hyaline Fibromatosis Syndrome: A Case Report of an ANTXR2 Mutation Featuring a Rare Variant c.697+1G>A.

Clinical case reports
2025

Recurrent Giant Subcutaneous Tumor in Juvenile Hyaline Fibromatosis.

The Journal of craniofacial surgery
2025

Unraveling Juvenile Hyaline Fibromatosis: A Case of Novel ANTXR2 Mutations Associated with Subcutaneous Masses and Hydronephrosis.

Pediatric blood &amp; cancer
2024

Anti-proliferative impact of resveratrol on gingival fibroblasts from juvenile hyaline fibromatosis.

Clinical oral investigations
2024

Hyaline Fibromatosis Syndrome: Early Outcomes Following Major Craniofacial Mass Excision.

The Journal of craniofacial surgery
2024

Beyond Skin Deep: A Case Report of Infantile Systemic Hyalinosis in a Six-Month-Old Infant.

Cureus
2024

Hyaline fibromatosis syndrome: a rare, yet recognizable syndrome.

The Turkish journal of pediatrics
2024

Investigating the Influence of ANTXR2 Gene Mutations on Protective Antigen Binding for Heightened Anthrax Resistance.

Genes
2024

Hyaline Fibromatosis Syndrome Diagnosed by Whole Genome Sequencing.

Journal of pediatric health care : official publication of National Association of Pediatric Nurse Associates &amp; Practitioners
2023

Atypical Presentation of Lip Nodules in Clinically Diagnosed Juvenile Hyaline Fibromatosis.

Cureus
2023

Hypercalcemia as a rare presentation of hyaline fibromatosis syndrome from different Sudanese families: two case reports.

Journal of medical case reports
2023

Anesthetic Management of a Patient With Juvenile Hyaline Fibromatosis: A Case Report Written With the Assistance of the Large Language Model ChatGPT.

Cureus
2025

Hyaline Fibromatosis Syndrome Presenting with Nasal Mass: A Case Report.

Ear, nose, &amp; throat journal
2022

Overlapping Hyaline Fibromatosis Syndrome: A Rare Case of Juvenile Hyaline Fibromatosis and Infantile Systemic Hyalinosis.

Cureus
2022

Imaging manifestations of juvenile hyaline fibromatosis: a case report and literature review.

BJR case reports
2022

Juvenile Hyaline Fibromatosis: Report of a Case with a Novel ANTXR2 Gene Mutation.

The American journal of case reports
2022

Hyaline fibromatosis syndrome with a novel 4.41-kb deletion in ANTXR2 gene: A case report and literature review.

Molecular genetics &amp; genomic medicine
2022

The diagnostic challenge of juvenile hyaline fibromatosis, a case report with literature reviews.

International journal of surgery case reports
2021

Juvenile hyaline fibromatosis: a rare oral disease case report and literature review.

Translational pediatrics
2021

Juvenile hyaline fibromatosis: A clinicopathological study of five cases.

Annals of diagnostic pathology
2021

Multiple Scalp Tumors in Juvenile Hyaline Fibromatosis with Antxr-2 Mutation in a Family.

Indian journal of dermatology
2021

Anesthetic Management of a Juvenile Hyaline Fibromatosis Patient With Trismus and Cervical Movement Limitation.

Anesthesia progress
2022

Atypical Presentation of Juvenile Hyaline Fibromatosis of Hands.

The Journal of hand surgery
2020

Juvenile Hyaline Fibromatosis: Literature Review and a Case Treated With Surgical Excision and Corticosteroid.

Cureus
2021

Hyaline fibromatosis syndrome: A rare case of multifocal intra-articular involvement.

Clinical imaging
2020

Clinical aspects of Hyaline Fibromatosis Syndrome and identification of a novel mutation.

Molecular genetics &amp; genomic medicine
2020

Juvenile Hyaline Fibromatosis Management With a Diode Laser: A Rare Case Report.

Journal of lasers in medical sciences
2020

Juvenile Hyaline Fibromatosis.

Mayo Clinic proceedings
2019

Juvenile hyaline fibromatosis: an unusual clinical presentation.

Dermatology online journal
2019

Juvenile hyaline fibromatosis in siblings.

Indian journal of pathology &amp; microbiology
2018

Hyaline fibromatosis syndrome: Clinical update and phenotype-genotype correlations.

Human mutation
2018

Protein-losing enteropathy and joint contractures caused by a novel homozygous ANTXR2 mutation.

Advances in genomics and genetics
2017

[Identification of novel compound heterozygous mutations in the ANTXR2 gene in a Chinese patient with juvenile hyaline fibromatosis].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2018

Hyaline fibromatosis syndrome (juvenile hyaline fibromatosis): whole-body MR findings in two siblings with different subcutaneous nodules distribution.

Skeletal radiology
2017

Juvenile Hyaline Fibromatosis- A Rare Autosomal Recessive Disease.

Journal of clinical and diagnostic research : JCDR
2017

Juvenile Hyaline Fibromatosis: A 10-year Follow-up.

Indian journal of dermatology
2017

Diagnosis implications of the whole genome sequencing in a large Lebanese family with hyaline fibromatosis syndrome.

BMC genetics
2016

Hyaline Fibromatosis Syndrome: A Rare Inherited Disorder.

Indian journal of dermatology
2016

Hyaline fibromatosis syndrome: cutaneous manifestations.

Anais brasileiros de dermatologia
2016

Juvenile hyaline fibromatosis: report of a rare case at an advanced stage with osteosclerosis and scoliosis.

International journal of dermatology
2016

Systemic Hyalinosis With Heterozygous CMG2 Mutations: A Case Report and Review of Literature.

The American Journal of dermatopathology
2015

Unusual cause for gum hypertrophy and skin nodules in a child.

BMJ case reports
2015

Juvenile Hyaline Fibromatosis: Impact of Periodontal Care on Quality of Life and a Patient Perspective.

Dental update
2015

[Hyaline fibromatosis syndrome: case report of two siblings].

Archivos argentinos de pediatria
Ver todos os 138 no EuropePMC

Associações

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

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

  1. Juvenile Hyaline Fibromatosis Syndrome: A Novel Variant in the ANTXR2 Gene Causing Severe Phenotype.
    Pediatric dermatology· 2026· PMID 40907988mais citado
  2. Unraveling Juvenile Hyaline Fibromatosis: A Case of Novel ANTXR2 Mutations Associated with Subcutaneous Masses and Hydronephrosis.
    Pediatric blood &amp; cancer· 2025· PMID 39704527mais citado
  3. Genetic Insights Into Hyaline Fibromatosis Syndrome: A Case Report of an ANTXR2 Mutation Featuring a Rare Variant c.697+1G&gt;A.
    Clinical case reports· 2025· PMID 40177156mais citado
  4. Recurrent Giant Subcutaneous Tumor in Juvenile Hyaline Fibromatosis.
    The Journal of craniofacial surgery· 2025· PMID 39960441mais citado
  5. Hyaline Fibromatosis Syndrome Presenting with Nasal Mass: A Case Report.
    Ear, nose, &amp; throat journal· 2025· PMID 36219393mais citado
  6. Hyaline Fibromatosis Syndrome.
    · 1993· PMID 20301698recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:2028(Orphanet)
  2. MONDO:0016071(MONDO)
  3. GARD:16583(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q6318955(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

Juvenile hyaline fibromatosis
Compêndio · Raras BR

Juvenile hyaline fibromatosis

ORPHA:2028 · MONDO:0016071
Prevalência
<1 / 1 000 000
Casos
70 casos conhecidos
Herança
Autosomal recessive
CID-10
M72.8 · Outros transtornos fibroblásticos
CID-11
Início
Childhood
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0406578
EuropePMC
Wikidata
Papers 10a
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