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Miocardiopatia histiocitoide
ORPHA:137675CID-10 · I42.0CID-11 · BC43.00OMIM 500000DOENÇA RARA

A cardiomiopatia histiocitóide é um distúrbio arritmogênico caracterizado por cardiomegalia, arritmias cardíacas graves ou morte súbita e presença de células semelhantes a histiócitos no miocárdio.

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Introdução

O que você precisa saber de cara

📋

A cardiomiopatia histiocitóide é um distúrbio arritmogênico caracterizado por cardiomegalia, arritmias cardíacas graves ou morte súbita e presença de células semelhantes a histiócitos no miocárdio.

Publicações científicas
55 artigos
Último publicado: 2025 Mar 1

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
100
pacientes catalogados
Início
Infancy
+ neonatal
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: I42.0
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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

❤️
Coração
11 sintomas
🧠
Neurológico
5 sintomas
🫁
Pulmão
2 sintomas
🫃
Digestivo
2 sintomas
👁️
Olhos
2 sintomas
📏
Crescimento
2 sintomas

+ 20 sintomas em outras categorias

Características mais comuns

90%prev.
Taquicardia
Muito frequente (99-80%)
55%prev.
Taquicardia supraventricular
Frequente (79-30%)
55%prev.
Taquicardia ventricular
Frequente (79-30%)
17%prev.
Bloqueio do ramo direito
Ocasional (29-5%)
17%prev.
Tosse
Ocasional (29-5%)
17%prev.
Síndrome de Wolff-Parkinson-White
Ocasional (29-5%)
48sintomas
Muito frequente (1)
Frequente (2)
Ocasional (17)
Muito raro (25)
Sem dados (3)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 48 características clínicas mais associadas, ordenadas por frequência.

TaquicardiaTachycardia
Muito frequente (99-80%)90%
Taquicardia supraventricularSupraventricular tachycardia
Frequente (79-30%)55%
Taquicardia ventricularVentricular tachycardia
Frequente (79-30%)55%
Bloqueio do ramo direitoRight bundle branch block
Ocasional (29-5%)17%
TosseCough
Ocasional (29-5%)17%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2025
Total histórico55PubMed
Últimos 10 anos14publicações
Pico20174 papers
Linha do tempo
2025Hoje · 2026📈 2017Ano de pico🧪 2022Primeiro ensaio clínico
Publicações por ano (últimos 10 anos)

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, Unknown, X-linked dominant.

MT-CYBCytochrome bCandidate gene tested inDesconhecido
FUNÇÃO

Component of the ubiquinol-cytochrome c reductase complex (complex III or cytochrome b-c1 complex) that is part of the mitochondrial respiratory chain. The b-c1 complex mediates electron transfer from ubiquinol to cytochrome c. Contributes to the generation of a proton gradient across the mitochondrial membrane that is then used for ATP synthesis

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (3)
Respiratory electron transportComplex III assemblyMitochondrial translation termination
OUTRAS DOENÇAS (4)
mitochondrial diseaseLeber hereditary optic neuropathyhistiocytoid cardiomyopathymitochondrial complex III deficiency
HGNC:7427UniProt:P00156

Variantes genéticas (ClinVar)

87 variantes patogênicas registradas no ClinVar.

🧬 MT-CYB: NC_012920.1(MT-CYB):m.15041G>A ()
🧬 MT-CYB: NC_012920.1:m.8944_15057del ()
🧬 MT-CYB: Single allele ()
🧬 MT-CYB: NC_012920.1(MT-CYB):m.15215G>A ()
🧬 MT-CYB: NC_012920.1(MT-CYB):m.10950_15540del ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 5 variantes classificadas pelo ClinVar.

3
1
1
Patogênica (60.0%)
VUS (20.0%)
Benigna (20.0%)
VARIANTES MAIS SIGNIFICATIVAS
NDUFB11: NM_001135998.3(NDUFB11):c.262C>T (p.Arg88Ter) [Pathogenic]
MT-ATP6: NC_012920.1(MT-ATP8):m.8528T>C [Likely pathogenic]
MT-TL1: NC_012920.1(MT-TL1):m.3243A>G [Pathogenic]
MT-CYB: NC_012920.1(MT-CYB):m.15498G>A [Uncertain significance]
FAM135A: NM_001162529.3(FAM135A):c.474C>G (p.Tyr158Ter) [Likely benign]

Vias biológicas (Reactome)

3 vias biológicas associadas 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

Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Miocardiopatia histiocitoide

🗺️

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.

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Publicações mais relevantes

Timeline de publicações
14 papers (10 anos)
#1

Conduction System Hamartoma: Autopsy Case Series.

The American journal of forensic medicine and pathology2025 Mar 01

Conduction system hamartoma is a benign hamartomatous lesion arising from Purkinje and Purkinje-like cells of the heart. We aimed to investigate the cases that we detected during postmortem histopathological examination. The histopathology reports of the cases autopsied between 2012 and 2022 were reviewed retrospectively. The cases were evaluated in terms of histopathological features, demographic data, autopsy findings, causes of death, microbiological results, and accompanying cardiac anomalies. There was a total of 4 cases. The female-to-male ratio was 3/1. The mean age of the cases was 5.8 months. The heart weight was found within the normal range when evaluated according to age and gender. Microscopy revealed sharply demarcated cell groups or layers with histiocyte-like cells with foamy cytoplasm. Congenital heart disease and lung infection were given as causes of death in all cases. The lesion, which was previously called histiocytoid cardiomyopathy and many different names, is known as "conduction system hamartoma" in the latest World Health Organization classification (5th edition). Being aware of this entity that causes fatal arrhythmias and sudden cardiac deaths is important for pathologists. Autopsy findings should be evaluated by taking extra samples from the heart if necessary, especially in suspected cases below 2 years of age.

#2

Case report: severe hypertrophic cardiomyopathy in a female neonate caused by de novo variant in NDUFB11.

European heart journal. Case reports2024 Aug

Hypertrophic cardiomyopathy in the neonate has a diverse genetic background, and non-sarcomeric variants may not be identified on commercial genetic testing panels. NDUFB11 is an X-linked mitochondrial Complex I protein and is known to cause histiocytoid cardiomyopathy but has not been described in female infants with hypertrophic cardiomyopathy. We present this first reported case of obstructive hypertrophic cardiomyopathy in a female neonate secondary to a pathogenic variant in NDUFB11. A term female neonate presented following a prenatal diagnosis of biventricular hypertrophy and growth restriction. She developed lactic acidosis after birth and whole-genome sequencing identified a de novo variant in the mitochondrial Complex I gene, NDUFB11 (c.391G>A, p.Glu131Lys). There was progression of left ventricular hypertrophy and obstruction, with rapid development of heart failure symptoms. She was unresponsive to beta-blocker medical therapy and was not suitable for advanced mechanical support. There was subsequent clinical deterioration resulting in death by 3 months of age. Hemizygous variants in NDUFB11 have been associated with hypertrophic cardiomyopathy in male infants previously, and skewed X-linked inactivation likely resulted in the presentation described here in a female infant. This variant was not identifiable by commercial cardiomyopathy panels. We highlight the importance of rapid whole-genome sequencing in cases of infantile hypertrophic cardiomyopathy and the importance of genetic diagnosis in guiding prognosis and care for these individuals.

#3

Histiocytoid cardiomyopathy presenting as sudden death in an 18-month-old infant.

Forensic science, medicine, and pathology2024 Sep

Histiocytoid cardiomyopathy (HC) is an arrhythmogenic disorder, usually involving children under two years of age with a strong Caucasian and female predominance. The disease is fatal in the vast majority and diagnosis is nearly always established at autopsy, but this is only possible with adequate myocardial sampling. Meticulous gross and histological examination of the heart in collaboration with a cardiovascular-trained pathologist maximises the opportunity to make specific diagnoses (and therefore rule out the differentials of SIDS, SUDC and child abuse), guide genetic testing, and inform potentially life-saving medical interventions for blood relations. We present a typical HC case presenting as sudden death, without prodrome, in a previously healthy 18-month-old boy. The disease is characterised histologically by discrete groups of enlarged, polygonal histiocyte-like cells with distinct margins and abundant faintly eosinophilic foamy cytoplasm. Cells often contain coarse granules, microvacuoles and irregular, round nuclei. In our case, dysplastic fascicles were predominantly located immediately deep to the endocardium of the left ventricle. We report our own autopsy findings with histological images, and discuss the expected clinical, morphological and ultrastructural features of the disease.

#4

Case report: high-dose carvedilol as a potential key drug for arrhythmias in histiocytoid cardiomyopathy.

European heart journal. Case reports2023 Dec

Histiocytoid cardiomyopathy is a rare infancy cardiac disorder manifesting as severe cardiac arrhythmias or dilated cardiomyopathy. There is no specific treatment for these arrhythmias. This is the first report of infantile histiocytoid cardiomyopathy whose refractory ventricular arrhythmias were successfully controlled by high-dose carvedilol. A 4-month-old girl presented with asystole, and recurrent ventricular tachycardias. From the histological findings and clinical symptoms, she was diagnosed as histiocytoid cardiomyopathy. Sedatives were the most effective therapy for her arrhythmia, but the cardiac sympathetic denervation was not effective enough. Finally, her ventricular arrhythmias were controlled with high-dose carvedilol, and she was discharged on hospitalization Day 393. Carvedilol is the only beta blocker that directly acts on the ryanodine receptor (RyR2) and inhibits store-overload-induced Ca2+ release (SOICR) in myocardium at high dosage. The arrhythmias did not disappear with bisoprolol, landiolol, or verapamil, but high-dose carvedilol was effective. This clinical course suggested that the arrhythmias in histiocytoid cardiomyopathy might be related with SOICR. High-dose carvedilol might be a key drug for patients with histiocytoid cardiomyopathy.

#5

Biventricular Assist Device Support for Intractable Arrhythmias From Histiocytoid Cardiomyopathy.

ASAIO journal (American Society for Artificial Internal Organs : 1992)2022 Nov 01

Histiocytoid cardiomyopathy (HICMP) is a rare mitochondrial cardiomyopathy associated with recurrent life-threatening arrhythmias and variable degrees of systolic dysfunction. Successful heart transplantation for HICMP has been described, but there has been no published experience with biventricular assist device (BiVAD) support for intractable arrhythmias in HICMP. We report a 13 month old girl with left ventricular noncompaction and preserved systolic function who presented in cardiogenic shock secondary to incessant ventricular arrhythmias. After failed attempts at chemical and electrical cardioversion, she underwent BiVAD implantation as bridge to transplantation. Her BiVAD course was complicated by mechanical inflow obstruction during sinus rhythm, necessitating left-sided cannulation revision from an apical to atrial inflow cannula. This maneuver resolved the obstruction and the patient was transitioned to Berlin EXCOR (Berlin Heart Inc, The Woodlands, TX) BiVADs. On Berlin pumps, she had intermittent pauses (no fill/no eject) while in sinus rhythm, felt to be due to competition from intrinsic ejection. Despite these pauses, the patient experienced an uneventful remainder of her BiVAD course (205 days total) with minimal fibrin deposition and no device-related complications. BiVAD can support pediatric patients with hemodynamically significant arrhythmias to transplantation. Atrial cannulation strategy may be preferred in cases of preserved systolic function, ventricular noncompaction, and frequent rhythm changes.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC41 artigos no totalmostrando 14

2025

Conduction System Hamartoma: Autopsy Case Series.

The American journal of forensic medicine and pathology
2024

Case report: severe hypertrophic cardiomyopathy in a female neonate caused by de novo variant in NDUFB11.

European heart journal. Case reports
2023

Case report: high-dose carvedilol as a potential key drug for arrhythmias in histiocytoid cardiomyopathy.

European heart journal. Case reports
2024

Histiocytoid cardiomyopathy presenting as sudden death in an 18-month-old infant.

Forensic science, medicine, and pathology
2022

Biventricular Assist Device Support for Intractable Arrhythmias From Histiocytoid Cardiomyopathy.

ASAIO journal (American Society for Artificial Internal Organs : 1992)
2021

Findings suggestive of coronary microvascular dysfunction in cats with myocardial ischemia.

Open veterinary journal
2019

Successful catheter ablation of premature ventricular contractions triggering torsade de pointes in a small infant with histiocytoid cardiomyopathy: a case report.

European heart journal. Case reports
2019

Diverse phenotype in patients with complex I deficiency due to mutations in NDUFB11.

European journal of medical genetics
2017

Histiocytoid cardiomyopathy and ventricular noncompaction presenting as sudden death in an adult male.

Pathology, research and practice
2017

Histiocytoid cardiomyopathy and microphthalmia with linear skin defects syndrome: phenotypes linked by truncating variants in NDUFB11.

Cold Spring Harbor molecular case studies
2017

Sudden Death in a Male Infant Due to Histiocytoid Cardiomyopathy: An Autopsy Case and Review of the Literature.

The American journal of forensic medicine and pathology
2017

A novel mutation in NDUFB11 unveils a new clinical phenotype associated with lactic acidosis and sideroblastic anemia.

Clinical genetics
2015

[Histiocytoid cardiomyopathy concurrent with noncompact myocardium, myocarditis, and pericarditis].

Arkhiv patologii
2015

Exome sequencing of patients with histiocytoid cardiomyopathy reveals a de novo NDUFB11 mutation that plays a role in the pathogenesis of histiocytoid cardiomyopathy.

American journal of medical genetics. Part A
Ver todos os 41 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

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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. Conduction System Hamartoma: Autopsy Case Series.
    The American journal of forensic medicine and pathology· 2025· PMID 39225698mais citado
  2. Case report: severe hypertrophic cardiomyopathy in a female neonate caused by de novo variant in NDUFB11.
    European heart journal. Case reports· 2024· PMID 39132302mais citado
  3. Histiocytoid cardiomyopathy presenting as sudden death in an 18-month-old infant.
    Forensic science, medicine, and pathology· 2024· PMID 37831311mais citado
  4. Case report: high-dose carvedilol as a potential key drug for arrhythmias in histiocytoid cardiomyopathy.
    European heart journal. Case reports· 2023· PMID 38089118mais citado
  5. Biventricular Assist Device Support for Intractable Arrhythmias From Histiocytoid Cardiomyopathy.
    ASAIO journal (American Society for Artificial Internal Organs : 1992)· 2022· PMID 35439193mais citado

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:137675(Orphanet)
  2. OMIM OMIM:500000(OMIM)
  3. MONDO:0010771(MONDO)
  4. GARD:9511(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q41516644(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

Miocardiopatia histiocitoide
Compêndio · Raras BR

Miocardiopatia histiocitoide

ORPHA:137675 · MONDO:0010771
Prevalência
<1 / 1 000 000
Casos
100 casos conhecidos
Herança
Autosomal recessive, Unknown, X-linked dominant
CID-10
I42.0 · Cardiomiopatia dilatada
CID-11
Início
Infancy, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C1708371
Repurposing
1 candidato
dexrazoxanechelating agent|topoisomerase inhibitor
EuropePMC
Wikidata
Papers 10a
DiscussaoAtiva

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