A Epidermólise Bolhosa Distrófica Pruriginosa é um subtipo raro de Epidermólise Bolhosa Distrófica (EBD). Essa condição se caracteriza por lesões na pele que podem ser generalizadas (espalhadas pelo corpo) ou localizadas (em pontos específicos), associadas a uma coceira intensa e, muitas vezes, muito difícil de aliviar ou controlar.
Introdução
O que você precisa saber de cara
A Epidermólise Bolhosa Distrófica Pruriginosa é um subtipo raro de Epidermólise Bolhosa Distrófica (EBD). Essa condição se caracteriza por lesões na pele que podem ser generalizadas (espalhadas pelo corpo) ou localizadas (em pontos específicos), associadas a uma coceira intensa e, muitas vezes, muito difícil de aliviar ou controlar.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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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
+ 15 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 24 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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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 dominant, Autosomal recessive.
Stratified squamous epithelial basement membrane protein that forms anchoring fibrils which may contribute to epithelial basement membrane organization and adherence by interacting with extracellular matrix (ECM) proteins such as type IV collagen
Secreted, extracellular space, extracellular matrix, basement membrane
Medicamentos aprovados (FDA)
2 medicamentos encontrados nos registros da FDA americana.
Variantes genéticas (ClinVar)
1,204 variantes patogênicas registradas no ClinVar.
Vias biológicas (Reactome)
10 vias biológicas associadas aos genes desta condição.
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 — Epidermólise bolhosa pruriginosa distrófica
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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.
Publicações mais relevantes
Case Report: Effective treatment of dystrophic epidermolysis bullosa pruriginosa with tofacitinib.
Epidermolysis bullosa (EB) is a heterogeneous group of hereditary skin diseases caused by mutations in structural proteins at the dermal-epidermal junction. Dystrophic epidermolysis bullosa (DEB), one of its main types, is characterized by recurrent pruritic blisters, bullae, atrophy, and scarring, often accompanied by nail dystrophy. Dystrophic epidermolysis bullosa pruriginosa, also known as epidermolysis bullosa pruriginosa (EBP), is a rare clinical subtype of DEB. In addition to the common manifestations of skin blisters and ulcers, patients with EBP also present severe pruritus. Traditional treatments for EBP have limited efficacy. In this study, we report the case of a 59-year-old male patient with EBP who showed significant improvement in skin lesions and pruritus after 10 months of treatment with tofacitinib, a pan-JAK inhibitor. This case highlights the potential of JAK inhibitors in treating EBP, although long-term safety requires further investigation.
Pruritus Anesis in Dystrophic Epidermolysis Bullosa Pruriginosa with Dupilumab.
Dystrophic epidermolysis bullosa pruriginosa (DEB-Pr) is a rare subtype of dystrophic epidermolysis bullosa, and traditional treatments have limited efficacy. Dupilumab has demonstrated remarkable efficacy in relieving pruritus. In this case study, after traditional treatment failed, providers recommended the patient begin dupilumab to treat his pruritus. The patient was administrated a loading dose of 600 mg of dupilumab and a dose of 300 mg every 2 weeks. The Dermatology Life Quality Index and Pruritic Numeric Rating Scale were used to assess the patient's situation. After several months, the patient's DEB-Pr was considered in remission. Dupilumab may be a better choice than immunosuppressants for the treatment of pruritus in patients with DEB-Pr.
Th2 response drives itch in dystrophic epidermolysis bullosa pruriginosa: A case-control study.
Novel compound heterozygous mutations of the COL7A1 gene in a Chinese patient with recessive dystrophic epidermolysis bullosa pruriginosa and digestive symptoms successfully treated with tofacitinib.
Missense Variant c.3301C>T (p.R1101W) in von Willebrand Factor A Sequence in a Patient with Recessive Dystrophic Epidermolysis Bullosa Pruriginosa with Compound Heterozygous COL7A1 Variants.
Dystrophic epidermolysis bullosa (DEB) pruriginosa is a rare subtype of DEB characterized by multiple, violaceous, and severe pruritic lichenified nodules along with blisters. Here, we report the case of a Korean male who, since the age of 3 years, had multiple pruritic nodules with blisters on both lower extremities. Genetic testing is required to diagnose DEB pruriginosa because its clinical and histologic features are inconclusive. We identified compound heterozygous COL7A1 variants of c.5797C>T (p.R1933*) and c.3301C>T (p.R1101W) in the patient, leading to a diagnosis of recessive DEB pruriginosa. Among the variants identified, c.3301C>T is a novel missense variant that has not been reported previously. This variant is in exon 26, which encodes von Willebrand factor A (vWFA) in collagen type VII. vWFA is known to preserve normal dermal structures by interacting with dermal collagens and basement membranes. Considering that this variant contradicts the general concept that autosomal dominant inheritance is more common and that variants typically occur in the triple helical collagenous domain of COL7A1 in DEB pruriginosa, we focus on the rarity of this case and the possible pathogenic role of the c.3301C>T (p.R1101W) variant.
Publicações recentes
Case Report: Effective treatment of dystrophic epidermolysis bullosa pruriginosa with tofacitinib.
Th2 response drives itch in dystrophic epidermolysis bullosa pruriginosa: A case-control study.
Pruritus Anesis in Dystrophic Epidermolysis Bullosa Pruriginosa with Dupilumab.
Missense Variant c.3301C>T (p.R1101W) in von Willebrand Factor A Sequence in a Patient with Recessive Dystrophic Epidermolysis Bullosa Pruriginosa with Compound Heterozygous COL7A1 Variants.
Genetic diagnosis of a rare COL7A1 variant causing dystrophic epidermolysis bullosa pruriginosa through whole‑exome sequencing.
📚 EuropePMC40 artigos no totalmostrando 21
Case Report: Effective treatment of dystrophic epidermolysis bullosa pruriginosa with tofacitinib.
Frontiers in medicineTh2 response drives itch in dystrophic epidermolysis bullosa pruriginosa: A case-control study.
Journal of the American Academy of DermatologyPruritus Anesis in Dystrophic Epidermolysis Bullosa Pruriginosa with Dupilumab.
Advances in skin & wound careMissense Variant c.3301C>T (p.R1101W) in von Willebrand Factor A Sequence in a Patient with Recessive Dystrophic Epidermolysis Bullosa Pruriginosa with Compound Heterozygous COL7A1 Variants.
Annals of dermatologyGenetic diagnosis of a rare COL7A1 variant causing dystrophic epidermolysis bullosa pruriginosa through whole‑exome sequencing.
Experimental and therapeutic medicineNovel compound heterozygous mutations of the COL7A1 gene in a Chinese patient with recessive dystrophic epidermolysis bullosa pruriginosa and digestive symptoms successfully treated with tofacitinib.
The Journal of dermatologyIdentification of a novel COL7A1 variant associated with dystrophic epidermolysis bullosa pruriginosa responding effectively to dupilumab.
Molecular genetics & genomic medicineDystrophic Epidermolysis Bullosa Pruriginosa: Successfully Treated With Dupilumab.
Dermatitis : contact, atopic, occupational, drug[Variation of COL7A1 gene in dystrophic epidermolysis bullosa pruriginosa].
Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical geneticsCOL7A1 G2287R mutation with two clinical phenotypes in the same family: Bullous dermolysis of the newborn and dystrophic epidermolysis bullosa pruriginosa.
The Journal of dermatologyA case of dystrophic epidermolysis bullosa pruriginosa treated with dupilumab.
Journal of the European Academy of Dermatology and Venereology : JEADVDystrophic epidermolysis bullosa pruriginosa: a new case series of a rare phenotype unveils skewed Th2 immunity.
Journal of the European Academy of Dermatology and Venereology : JEADVAmelioration of dystrophic epidermolysis bullosa pruriginosa symptoms with dupilumab: A case report.
Dermatologic therapyMisdiagnosed dystrophic epidermolysis bullosa pruriginosa: A case report.
World journal of clinical casesNovel heterozygous COL7A1 mutation in a patient with de-novo dominant dystrophic epidermolysis bullosa pruriginosa.
JAAD case reportsDominant Dystrophic Epidermolysis Bullosa Pruriginosa Responding to Naltrexone Treatment.
Acta dermato-venereologicaWhole exome sequencing identified two point mutations of COL7A1 and FLG in a Chinese family with dystrophic epidermolysis bullous pruriginosa and ichthyosis vulgaris.
The Journal of dermatologyDystrophic epidermolysis bullosa pruriginosa presenting with flagellate scarring lesions.
Clinical and experimental dermatologyDominant dystrophic epidermolysis bullosa pruriginosa with a COL7A1 exon 87 c.6898C>T mutation.
Clinical and experimental dermatologyThe clinical phenotype and a novel COL7A1 mutation in a Chinese family with dystrophic epidermolysis bullosa pruriginosa.
Journal of the European Academy of Dermatology and Venereology : JEADVDystrophic epidermolysis bullosa pruriginosa successfully treated with immunosuppressants.
The Journal of dermatologyAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
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Comunidades
Grupos ativos de quem convive com esta doença aqui no Raras
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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.
- Case Report: Effective treatment of dystrophic epidermolysis bullosa pruriginosa with tofacitinib.
- Pruritus Anesis in Dystrophic Epidermolysis Bullosa Pruriginosa with Dupilumab.
- Th2 response drives itch in dystrophic epidermolysis bullosa pruriginosa: A case-control study.
- Novel compound heterozygous mutations of the COL7A1 gene in a Chinese patient with recessive dystrophic epidermolysis bullosa pruriginosa and digestive symptoms successfully treated with tofacitinib.
- Missense Variant c.3301C>T (p.R1101W) in von Willebrand Factor A Sequence in a Patient with Recessive Dystrophic Epidermolysis Bullosa Pruriginosa with Compound Heterozygous COL7A1 Variants.
- Genetic diagnosis of a rare COL7A1 variant causing dystrophic epidermolysis bullosa pruriginosa through whole‑exome sequencing.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:89843(Orphanet)
- OMIM OMIM:604129(OMIM)
- MONDO:0011398(MONDO)
- GARD:16779(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q55783353(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
