A hipoplasia cartilagem-cabelo é uma doença que afeta as extremidades dos ossos onde eles crescem, e causa baixa estatura desde o nascimento.
Introdução
O que você precisa saber de cara
A hipoplasia cartilagem-cabelo é uma doença que afeta as extremidades dos ossos onde eles crescem, e causa baixa estatura desde o nascimento.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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
Partes do corpo afetadas
+ 44 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 123 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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.
Variantes genéticas (ClinVar)
418 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 3 variantes classificadas pelo ClinVar.
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 — Hipoplasia cartilagem-cabelo
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
Ensaios em destaque
Pesquisa e ensaios clínicos
2 ensaios clínicos encontrados.
Publicações mais relevantes
Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.
Cartilage-hair hypoplasia (CHH) is a rare autosomal recessive skeletal dysplasia. It is often associated with short stature, metaphyseal abnormalities, hair hypoplasia and immune dysfunction. This case describes a pregnant woman in her mid-30s with two previous children diagnosed with CHH with molecular confirmation of two pathogenic variants of the ribonuclease mitochondrial RNA processing gene. The couple is healthy and non-consanguineous. Routine ultrasound examination of the current pregnancy suggested this fetus was also likely to be affected. The couple opted not to perform invasive prenatal diagnosis. Umbilical cord blood was collected at birth for genetic testing, confirming the diagnosis. CHH has a significant impact on the quality of life of those affected. Genetic counselling may help parents understand the disease and its prognosis. Obstetric ultrasound plays an important role in the diagnosis by allowing early detection of suspected cases as well as assessing fetal growth.
Neonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.
Cartilage hair hypoplasia (CHH) syndrome (OMIM #250250) is a rare autosomal recessive metaphyseal dysplasia, characterized by disproportionate short stature, hypotrichosis and variable extra-skeletal manifestations, including immunodeficiency, anemia, intestinal diseases, and predisposition to malignancies. CHH results from homozygous or compound heterozygous mutations in the RMRP gene on chromosome 9p13, which encodes an untranslated RNA component of mitochondrial RNA-processing endoribonuclease. RMRP pathogenic variants can also lead to Omenn Syndrome (OS) (OMIM #603554), a systemic inflammatory condition displaying neonatal erythroderma and immunodeficiency. This report highlights the genotypic and phenotypic overlap between CHH and OS, by presenting a newborn with skeletal dysplasia, immunodeficiency and neonatal onset erythroderma, carrying the homozygous NR_003051:n.35C > A variant in the RMRP gene.
Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.
Cartilage-hair hypoplasia is a rare autosomal recessive skeletal dysplasia. It is particularly prevalent in the Finnish and Amish populations but increasing reports have been documented worldwide. It is caused by pathogenic variants in the RMRP gene. The clinical presentation is highly variable and may include short-limbed short stature, metaphyseal abnormalities, hypotrichosis, and immune deficiency, among other features. Some of the manifestations may present early in the prenatal period and ultrasound assessment is often the tool that raises suspicion for this condition. This review aims to summarize the current knowledge regarding the prenatal diagnosis of cartilage-hair hypoplasia, focusing on its molecular basis and the role of imaging and genetic testing. A comprehensive literature search was conducted in the PubMed/MEDLINE database using the terms 'Prenatal diagnosis', 'Cartilage-hair hypoplasia', 'Skeletal dysplasias', 'Osteochondrodysplasias' and 'RMRP mutation'. Prenatal diagnosis of this condition remains challenging, as ultrasound findings may overlap with other skeletal dysplasias, including lethal forms. Lethality predictors and the potential of molecular testing are also explored. A structured prenatal approach, combined with timely genetic counselling, may allow for an earlier diagnosis and support informed reproductive decisions. Given the recent advances in reproductive technologies and the potential impact of cartilage-hair hypoplasia on affected individuals, this condition should be actively considered in future studies addressing the prenatal diagnosis of skeletal dysplasias.
Cartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.
Cartilage-hair hypoplasia (CHH) is an autosomal recessive disorder caused by homozygous or compound heterozygous mutations in the RMRP gene, which is extremely rare in the population, and its most common feature is disproportionately short limb shortness with short and thickened long bones, usually found in newborns, occasionally found in the prenatal stage, and other clinical features include a series of extracutaneous manifestations, such as hypoglycemia, gastrointestinal dysfunction, immunodeficiency, anemia and increased risk of malignant tumors, etc, the specific pathogenesis is unknown. The case of a 1.5-year-old male patient with severe short stature (height 70 cm, <-3 SD), sparse scalp hair, and short limbs, without ligamentous laxity or anemia. Family-based whole-exome sequencing revealed compound heterozygous variants in RMRP: NR_003051.3: n.-21_-9dup and n.5C > T. Classified as pathogenic or likely pathogenic according to ACMG guidelines, these variants confirm the diagnosis of CHH. Due to the elevated tumor predisposition associated with CHH, and because growth hormone therapy is currently contraindicated, no disease-specific interventions have been initiated. We have confirmed that the child has been diagnosed with CHH, but no intervention has been given, and we will do further follow-up and look forward to finding a treatment for the disease. This report describes a rare case of RMRP-associated CHH. This case broadens the clinical understanding of the RMRP mutational spectrum and phenotypic variability, providing new insights into genotype-phenotype correlations in RMRP-associated disorders. We were currently unable to take effective treatment measures for this child, and we hope that in the future, there will be treatments such as gene therapy to bring hope to children and their families.
Refractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.
Cartilage hair hypoplasia-anauxetic dysplasia (CHH-AD) spectrum disorders are rare skeletal dysplasias caused by pathogenic variants in RMRP, associated with immune dysfunction and cancer predisposition. While non-Hodgkin lymphoma is more commonly seen, Hodgkin lymphoma (HL) is rarely reported, and its management in this setting remains unclear. We describe 2 siblings with genetically confirmed CHH-AD who developed relapsed/refractory EBV-positive classic HL. Both presented with short stature, atopy, recurrent infections, and elevated IgE. The brother was diagnosed with stage IV disease, and the sister with stage IIB. Despite receiving frontline chemotherapy, both relapsed within a year. Salvage therapy with gemcitabine/vinorelbine induced metabolic responses, followed by radiotherapy and consolidation with brentuximab vedotin. Autologous transplant was considered but declined by the family due to perceived risks. At 30 months follow-up, both remain in complete remission. Genetic testing confirmed a shared homozygous pathogenic RMRP variant. These cases expand the oncologic spectrum of CHH-AD to include HL, highlight the risk of aggressive disease and early relapse, and demonstrate that durable remission may be achieved without transplantation when consolidation with targeted therapy is feasible. Early recognition of CHH-AD features in HL patients may allow risk-adapted therapy and informed genetic counseling.
Publicações recentes
Expanding the Phenotypic Spectrum of Anauxetic Dysplasia Type 3: Reporting an Iranian Family With Unique Systemic Features and NEPRO Gene Variant.
Successful Treatment of Isolated Bone Marrow Classic Hodgkin Lymphoma in a Patient With Cartilage Hair Hypoplasia.
Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.
📖 RevisãoNeonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.
Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.
🥉 Relato de caso📚 EuropePMC194 artigos no totalmostrando 89
Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.
BMJ case reportsNeonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.
European journal of medical geneticsPrenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.
Acta medica portuguesaCartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.
MedicineRefractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.
Journal of pediatric hematology/oncologyPrecision T cell correction platform for inborn errors of immunity.
Molecular therapy : the journal of the American Society of Gene TherapyNeonatal erythroderma in a patient with cartilage-hair hypoplasia: Identification of a novel RMRP mutation.
Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDGRMRP variants inhibit the cell cycle checkpoints pathway in cartilage‑hair hypoplasia.
Molecular medicine reportsAnemia in patients with cartilage hair hypoplasia: a narrative review and recommendations.
Laboratory medicineIdentification of a founder effect involving n.197C>T variant in RMRP gene associated to cartilage-hair hypoplasia syndrome in Brazilian patients.
Scientific reportsCartilage-hair hypoplasia-anauxetic dysplasia spectrum disorders harboring RMRP mutations in two Korean children: A case report.
MedicineValidation of Risk Factors for Early Mortality in Cartilage-Hair Hypoplasia.
Journal of clinical immunologyAltered oral microbiome, but normal human papilloma virus prevalence in cartilage-hair hypoplasia patients.
Orphanet journal of rare diseasesPrediction of LncRNA-protein Interactions Using Auto-Encoder, SE-ResNet Models and Transfer Learning.
MicroRNA (Shariqah, United Arab Emirates)Extensive and deep granulomatous ulcers as an atypical manifestation of cartilage-hair hypoplasia syndrome: A diagnostic and therapeutic challenge.
The Australasian journal of dermatologyRMRP-related short stature: A report of six additional Japanese individuals with cartilage hair hypoplasia and literature review.
American journal of medical genetics. Part AShorter birth length and decreased T-cell production and function predict severe infections in children with non-severe combined immunodeficiency cartilage-hair hypoplasia.
The journal of allergy and clinical immunology. GlobalLong noncoding RNA RMRP ameliorates doxorubicin-induced apoptosis by interacting with PFN1 in a P53-Dependent manner.
Molecular and cellular probesJapanese siblings with cartilage-hair hypoplasia exhibiting different severity.
Pediatrics international : official journal of the Japan Pediatric SocietyOral findings in patients with cartilage-hair hypoplasia - cross-sectional observational study.
Orphanet journal of rare diseasesExpanding the phenotype of anauxetic dysplasia caused by biallelic NEPRO mutations: A case report.
American journal of medical genetics. Part AHomozygous RMRP Promoter Duplications Cause Severely Reduced Transcript Abundance and SCID Associated with Cartilage Hair Hypoplasia.
Journal of clinical immunologyNatural history and genetic spectrum of the Turkish metaphyseal dysplasia cohort, including rare types caused by biallelic COL10A1, COL2A1, and LBR variants.
BoneLymphomas in cartilage-hair hypoplasia - A case series of 16 patients reveals advanced stage DLBCL as the most common form.
Frontiers in immunologyReduced Intensity Conditioning Allogeneic Transplant for SCID Associated with Cartilage Hair Hypoplasia.
Journal of clinical immunologySirolimus Restores Erythropoiesis and Controls Immune Dysregulation in a Child With Cartilage-Hair Hypoplasia: A Case Report.
Frontiers in immunologyCartilage-hair hypoplasia with T-cell dysfunction.
Pediatrics international : official journal of the Japan Pediatric SocietyImmunologic Heterogeneity in 2 Cartilage-Hair Hypoplasia Patients With a Distinct Clinical Course.
Journal of investigational allergology & clinical immunologyA novel experimental approach for the selective isolation and characterization of human RNase MRP.
RNA biologyA disease-linked lncRNA mutation in RNase MRP inhibits ribosome synthesis.
Nature communicationsUncovering pathways regulating chondrogenic differentiation of CHH fibroblasts.
Non-coding RNA researchCraniofacial and Craniocervical Features in Cartilage-Hair Hypoplasia: A Radiological Study of 17 Patients and 34 Controls.
Frontiers in endocrinology[Cartilage hair hypoplasia with severe combined immunodeficiency caused by a novel RMRP gene variant].
Zhonghua er ke za zhi = Chinese journal of pediatricsLong Non-coding RNA RMRP in the Pathogenesis of Human Disorders.
Frontiers in cell and developmental biologyCartilage-hair Hypoplasia Complicated with Liver Cirrhosis Due to Chronic Intrahepatic Cholestasis.
Internal medicine (Tokyo, Japan)Partial recapitulation of fetal thymic T-cell constitution postnatally in a patient with cartilage hair hypoplasia-anauxetic dysplasia spectrum disorder: A case report.
Cytometry. Part B, Clinical cytometryPulmonary Follow-Up Imaging in Cartilage-Hair Hypoplasia: a Prospective Cohort Study.
Journal of clinical immunologyCrystal structure of human RPP20-RPP25 proteins in complex with the P3 domain of lncRNA RMRP.
Journal of structural biologyEarly prenatal presentation of the cartilage-hair hypoplasia / anauxetic dysplasia spectrum of disorders mimicking recurrent thanatophoric dysplasia.
European journal of medical geneticsHomozygous n.64C>T mutation in mitochondrial RNA-processing endoribonuclease gene causes cartilage hair hypoplasia syndrome in two siblings.
European journal of medical geneticsOutcomes of 42 pregnancies in 14 women with cartilage-hair hypoplasia: a retrospective cohort study.
Orphanet journal of rare diseasesHow Altered Ribosome Production Can Cause or Contribute to Human Disease: The Spectrum of Ribosomopathies.
CellsThe Safety and Efficacy of Live Viral Vaccines in Patients With Cartilage-Hair Hypoplasia.
Frontiers in immunologyImmunodeficiency in cartilage-hair hypoplasia: Pathogenesis, clinical course and management.
Scandinavian journal of immunologyRituximab and intense chemotherapy in a patient with defective cell mediated immunity due to cartilage-hair hypoplasia and Burkitt lymphoma.
Pediatric blood & cancerIdentification of Novel and Recurrent RMRP Variants in a Series of Brazilian Patients with Cartilage-Hair Hypoplasia: McKusick Syndrome.
Molecular syndromologyAbnormal Newborn Screening Follow-up for Severe Combined Immunodeficiency in an Amish Cohort with Cartilage-Hair Hypoplasia.
Journal of clinical immunology[Cartilage-hair hypoplasia. A case report].
Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)The human long non-coding RNA gene RMRP has pleiotropic effects and regulates cell-cycle progression at G2.
Scientific reports'Metaphyseal dysplasia without hypotrichosis' can present with late-onset extraskeletal manifestations.
Journal of medical geneticsA 30-Year Prospective Follow-Up Study Reveals Risk Factors for Early Death in Cartilage-Hair Hypoplasia.
Frontiers in immunologyAn emerging ribosomopathy affecting the skeleton due to biallelic variations in NEPRO.
American journal of medical genetics. Part ARmrp Mutation Disrupts Chondrogenesis and Bone Ossification in Zebrafish Model of Cartilage-Hair Hypoplasia via Enhanced Wnt/β-Catenin Signaling.
Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral ResearchA Systematic Review on Predisposition to Lymphoid (B and T cell) Neoplasias in Patients With Primary Immunodeficiencies and Immune Dysregulatory Disorders (Inborn Errors of Immunity).
Frontiers in immunologyCutaneous granulomas with primary immunodeficiency in children: a report of 17 new patients and a review of the literature.
Journal of the European Academy of Dermatology and Venereology : JEADVThe antiviral protein viperin regulates chondrogenic differentiation via CXCL10 protein secretion.
The Journal of biological chemistryRubella Virus-Associated Cutaneous Granulomatous Disease: a Unique Complication in Immune-Deficient Patients, Not Limited to DNA Repair Disorders.
Journal of clinical immunologyGynecologic health in cartilage-hair hypoplasia: A survey of 26 adult females.
American journal of medical genetics. Part AGynecologic assessment of 19 adult females with cartilage-hair hypoplasia - high rate of HPV positivity.
Orphanet journal of rare diseasesA Wide Spectrum of Autoimmune Manifestations and Other Symptoms Suggesting Immune Dysregulation in Patients With Cartilage-Hair Hypoplasia.
Frontiers in immunologyAnauxetic dysplasia: A rare clinical entity.
The Turkish journal of pediatrics[Syndromic Hirschsprung′s disease and its mode of inheritance].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatricsCartilage hair hypoplasia with cutaneous lymphomatoid granulomatosis.
Clinical and experimental dermatologyDiffuse large B-cell lymphoma chemotherapy reveals a combined immunodeficiency syndrome in cartilage hair hypoplasia.
Swiss medical weeklyDiversity of Pubertal Development in Cartilage-Hair Hypoplasia; Two Illustrative Cases.
Journal of pediatric and adolescent gynecologyQuantum Language of MicroRNA: Application for New Cancer Therapeutic Targets.
Methods in molecular biology (Clifton, N.J.)Expression of RMRP RNA is regulated in chondrocyte hypertrophy and determines chondrogenic differentiation.
Scientific reportsDiverse Autoantibody Reactivity in Cartilage-Hair Hypoplasia.
Journal of clinical immunologyAnalysis of clinical and immunologic phenotype in a large cohort of children and adults with cartilage-hair hypoplasia.
The Journal of allergy and clinical immunologyPregnancy Outcome in Cartilage-Hair Hypoplasia, a Rare Form of Dwarfism.
Case reports in obstetrics and gynecologyDefects in lymphocyte telomere homeostasis contribute to cellular immune phenotype in patients with cartilage-hair hypoplasia.
The Journal of allergy and clinical immunologyTargeted CRISPR disruption reveals a role for RNase MRP RNA in human preribosomal RNA processing.
Genes & developmentCartilage-hair hypoplasia with normal height in childhood-4 patients with a unique genotype.
Clinical geneticsBroadening the phenotypic spectrum of POP1-skeletal dysplasias: identification of POP1 mutations in a mild and severe skeletal dysplasia.
Clinical geneticsDecreased telomere length in children with cartilage-hair hypoplasia.
Journal of medical geneticsClinical and molecular diagnosis of a cartilage-hair hypoplasia with IGF-1 deficiency.
American journal of medical genetics. Part AThe Finnish founder mutation c.70 A>G in RMRP causes cartilage-hair hypoplasia in a Pakistani family.
Clinical dysmorphologyHirschsprung's disease associated with alopecia universalis congenita: a case report.
Journal of medical case reportsHigh prevalence of bronchiectasis in patients with cartilage-hair hypoplasia.
The Journal of allergy and clinical immunologyFurther evidence of POP1 mutations as the cause of anauxetic dysplasia.
American journal of medical genetics. Part ACartilage-hair hypoplasia associated with isolated hypoganglionosis: A case report.
Congenital anomaliesHypo- and Hyper-Assembly Diseases of RNA-Protein Complexes.
Trends in molecular medicineCartilage Hair Hypoplasia: Two Unrelated Cases with g.70 A > G Mutation in RMRP Gene.
Indian journal of pediatricsDDX5 and its associated lncRNA Rmrp modulate TH17 cell effector functions.
NatureNovel Mutation and Structural RNA Analysis of the Noncoding RNase MRP Gene in Cartilage-Hair Hypoplasia.
Molecular syndromologyCartilage hair hypoplasia: characteristics and orthopaedic manifestations.
Journal of children's orthopaedicsVariable phenotype of severe immunodeficiencies associated with RMRP gene mutations.
Journal of clinical immunology[Clinical and molecular diagnostics of a cartilage-hair hypoplasia: Two new cases].
Anales de pediatria (Barcelona, Spain : 2003)Widespread intracranial calcifications in the follow-up of a patient with cartilage-hair hypoplasia--anauxetic dysplasia spectrum disorder: a coincidental finding?
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology SocietyAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Hipoplasia cartilagem-cabelo.
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 Hipoplasia cartilagem-cabelo
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 loginDoenças 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.
- Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.
- Neonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.
- Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.
- Cartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.
- Refractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.
- Expanding the Phenotypic Spectrum of Anauxetic Dysplasia Type 3: Reporting an Iranian Family With Unique Systemic Features and NEPRO Gene Variant.
- Successful Treatment of Isolated Bone Marrow Classic Hodgkin Lymphoma in a Patient With Cartilage Hair Hypoplasia.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:175(Orphanet)
- OMIM OMIM:250250(OMIM)
- MONDO:0009595(MONDO)
- GARD:6996(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q2964438(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
