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Buscar doenças, sintomas, genes...
Trombocitopenia sindrômica MYH9-relacionada
ORPHA:182050CID-10 · D69.4CID-11 · 3B64.01OMIM 153640DOENÇA RARA

É uma doença hereditária que afeta as plaquetas, tornando-as maiores que o normal. Ela apresenta um conjunto de características variadas, incluindo trombocitopenia congênita (contagem baixa de plaquetas desde o nascimento), e pode, mais tarde, desenvolver perda de audição, catarata (que surge antes da velhice), aumento das enzimas do fígado e/ou uma doença nos rins que piora com o tempo (nefropatia progressiva), muitas vezes levando à falência renal terminal (DRCT), quando os rins param de funcionar. A síndrome de Epstein, a síndrome de Fechtner, a anomalia de May-Hegglin e a síndrome de Sebastian, que antes eram consideradas doenças separadas, são, na verdade, algumas das diferentes formas como a doença MYH9 (MYH9-RD) pode se manifestar.

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Introdução

O que você precisa saber de cara

📋

É uma doença hereditária que afeta as plaquetas, tornando-as maiores que o normal. Ela apresenta um conjunto de características variadas, incluindo trombocitopenia congênita (contagem baixa de plaquetas desde o nascimento), e pode, mais tarde, desenvolver perda de audição, catarata (que surge antes da velhice), aumento das enzimas do fígado e/ou uma doença nos rins que piora com o tempo (nefropatia progressiva), muitas vezes levando à falência renal terminal (DRCT), quando os rins param de funcionar. A síndrome de Epstein, a síndrome de Fechtner, a anomalia de May-Hegglin e a síndrome de Sebastian, que antes eram consideradas doenças separadas, são, na verdade, algumas das diferentes formas como a doença MYH9 (MYH9-RD) pode se manifestar.

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.29
Italy
Início
Adolescent
+ adult, childhood, infancy, neonatal
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: D69.4
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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

🩸
Sangue
8 sintomas
🫘
Rins
4 sintomas
🫃
Digestivo
1 sintomas
👂
Ouvidos
1 sintomas
❤️
Coração
1 sintomas
👁️
Olhos
1 sintomas

+ 9 sintomas em outras categorias

Características mais comuns

90%prev.
Trombocitopenia congênita
Muito frequente (99-80%)
55%prev.
Aumento do volume plaquetário médio
Frequente (79-30%)
55%prev.
Epistaxe espontânea e recorrente
Frequente (79-30%)
55%prev.
Concentração elevada de transaminase hepática circulante
Frequente (79-30%)
55%prev.
Nefrite
Frequente (79-30%)
55%prev.
Suscetibilidade a hematomas
Frequente (79-30%)
26sintomas
Muito frequente (1)
Frequente (14)
Muito raro (1)
Sem dados (10)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 26 características clínicas mais associadas, ordenadas por frequência.

Trombocitopenia congênitaCongenital thrombocytopenia
Muito frequente (99-80%)90%
Aumento do volume plaquetário médioIncreased mean platelet volume
Frequente (79-30%)55%
Epistaxe espontânea e recorrenteSpontaneous, recurrent epistaxis
Frequente (79-30%)55%
Concentração elevada de transaminase hepática circulanteElevated circulating hepatic transaminase concentration
Frequente (79-30%)55%
NefriteNephritis
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Últimos 10 anos124publicações
Pico202016 papers
Linha do tempo
2026Hoje · 2026🧪 2004Primeiro ensaio clínico📈 2020Ano de pico
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 dominant.

MYH9Myosin-9Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Cellular myosin that appears to play a role in cytokinesis, cell shape, and specialized functions such as secretion and capping. Required for cortical actin clearance prior to oocyte exocytosis (By similarity). Promotes cell motility in conjunction with S100A4 (PubMed:16707441). During cell spreading, plays an important role in cytoskeleton reorganization, focal contact formation (in the margins but not the central part of spreading cells), and lamellipodial retraction; this function is mechanic

LOCALIZAÇÃO

Cytoplasm, cytoskeletonCytoplasm, cell cortexCytoplasmic vesicle, secretory vesicle, Cortical granuleCell membrane

VIAS BIOLÓGICAS (10)
RHO GTPases activate PAKsRHO GTPases Activate ROCKsRHO GTPases activate PKNsRHO GTPases activate CITSema4D induced cell migration and growth-cone collapse
MECANISMO DE DOENÇA

Macrothrombocytopenia and granulocyte inclusions with or without nephritis or sensorineural hearing loss

An autosomal dominant disorder characterized by thrombocytopenia, giant platelets and Dohle body-like inclusions in peripheral blood leukocytes with variable ultrastructural appearance. Some affected individuals lack leukocyte inclusion bodies on classic staining of peripheral blood smears. Alport syndrome-like features of nephritis, hearing loss, and eye abnormalities are present in some patients.

EXPRESSÃO TECIDUAL(Ubíquo)
Aorta
1592.3 TPM
Artéria tibial
1091.7 TPM
Útero
820.6 TPM
Artéria coronária
818.3 TPM
Pulmão
601.2 TPM
OUTRAS DOENÇAS (4)
autosomal dominant nonsyndromic hearing loss 17macrothrombocytopenia and granulocyte inclusions with or without nephritis or sensorineural hearing lossnodular fasciitisautosomal dominant nonsyndromic hearing loss
HGNC:7579UniProt:P35579

Variantes genéticas (ClinVar)

298 variantes patogênicas registradas no ClinVar.

🧬 MYH9: NM_002473.6(MYH9):c.3493C>A (p.Arg1165Ser) ()
🧬 MYH9: NM_002473.6(MYH9):c.3463A>G (p.Thr1155Ala) ()
🧬 MYH9: NM_002473.6(MYH9):c.563T>G (p.Ile188Ser) ()
🧬 MYH9: NM_002473.6(MYH9):c.5769del (p.Asp1925fs) ()
🧬 MYH9: NM_002473.6(MYH9):c.5833G>T (p.Glu1945Ter) ()
Ver todas no ClinVar

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

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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 — Trombocitopenia sindrômica MYH9-relacionada

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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.

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Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

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Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

Inherited Platelet Disorders During Pregnancy and Delivery: Overview of Management Strategies and Emerging Therapeutic Considerations.

Hematology reports2026 Feb 26

Inherited platelet disorders (IPDs) comprise a heterogeneous group of rare conditions that present particular challenges during pregnancy, with bleeding risk increasing during labor and the immediate postpartum period. These disorders require coordinated, multidisciplinary management to mitigate maternal and neonatal bleeding risk. Although data remains limited, individuals with IPD, including Bernard-Soulier syndrome, Glanzmann thrombasthenia, MYH9-related disorders, Hermansky-Pudlak syndrome, and platelet storage pool disorders, are at an increased risk for obstetrical bleeding, with the degree of risk varying by underlying diagnosis. In severe inherited platelet disorders such as Glanzmann thrombasthenia, peripartum hemorrhage is common, with up to half of the deliveries in some series requiring red cell or platelet transfusion. Because these conditions are congenital, the fetus may also be affected, placing neonates at risk for serious bleeding complications, including intracranial hemorrhage, although available data is limited. Despite the considerable morbidity and mortality risk associated with inherited platelet disorders, management strategies during pregnancy and delivery remain poorly defined. This stands in contrast to other bleeding disorders, such as factor deficiencies, for which multiple therapeutic approaches have been evaluated in the peripartum setting. In this review, we summarize the available evidence and current management strategies for individuals with inherited platelet disorders during pregnancy and delivery.

#2

The Clinical Details of MYH9-Related Disease and DFNA17 in a Large Japanese Hearing Loss Cohort.

Genes2026 Jan 29

Background/Objectives: MYH9 gene variants cause MYH9-related disease (MYH9-RD), which is also known as Epstein syndrome, Fechtner syndrome, May-Hegglin anomaly, and Sebastian syndrome. MYH9-RD is characterized by sensorineural hearing loss, macrothrombocytopenia, thrombocytopenia, hematuria/proteinuria, glomerulonephritis, cataracts purpura, and mucosal bleeding. In addition, the MYH9 gene is also known to be causative of autosomal dominant non-syndromic hearing loss (DFNA17). MYH9-RD is a relatively rare disorder, and the detailed clinical features and mutational spectra remain unclear. Methods: In this study, we performed next-generation sequencing analysis for 15,684 hearing loss patients and identified MYH9-associated hearing loss patients. Detailed clinical information was collected for these patients and summarized. Results: In this study, we identified 24 patients from 18 families with MYH9-associated hearing loss. We clarified the details of hearing deterioration observed in patients based on collected serial audiogram data. Some cases showed rapid hearing deterioration that worsened by about 50 dB within 5 years. Hearing loss is more likely to progress in patients with myosin head domain variants than in patients with myosin tail domain variants, but hearing loss in each set of patients finally deteriorates to bilateral profound hearing loss. Conclusions: In this study, we were able to clarify the detailed characteristics of MYH9-RD- and DFNA17-related hearing loss in a relatively large number of patients, particularly in some cases that showed rapid and asymmetrical hearing deterioration progressing to bilateral profound hearing loss. Our data will be useful for providing more appropriate treatment and follow-up for MYH9-associated hearing loss.

#3

A Case Report of Thrombocytopenia Caused by a May-Hegglin Anomaly in a Young Saudi Female.

Journal of investigative medicine high impact case reports2026

Low platelet count is rarely caused by inherited thrombocytopenia. May-Hegglin anomaly is an uncommon condition that falls under the umbrella of familial thrombocytopenia. The condition is under-reported in Saudi Arabia; therefore, we report the current case. This is a 24-year-old Saudi lady, presented to the emergency room with vaginal bleeding. No bleeding occurred at any other sites. She has a positive family history of thrombocytopenia among her father and 2 of her siblings. Her platelet count was 16 × 103/µL with normal other blood count as well as renal and liver panels. She was admitted to the regular bed for investigation as sever thrombocytopenia with suspicion of either familial or immune thrombocytopenia. Further studies showed normal hemostatic, virology, and connective tissue disease markers. Peripheral blood film showed low platelet distribution with occasional large/giant platelets and basophilic inclusion bodies in some neutrophils (Dohle body-like). A picture suggestive of May-Hegglin related thrombocytopenia that was confirmed by the presence of a positive myosin heavy chain 9 (MYH9) gene mutation. In conclusion, there are many difficulties in diagnosing and treating May-Hegglin disorders in females of reproductive age. More research and guidelines are needed to manage inherited thrombocytopenia before and throughout pregnancy.

#4

De Novo MYH9-Related Macrothrombocytopenia in a Toddler: Insights From Platelet Mass Index.

British journal of hospital medicine (London, England : 2005)2026 Jan 26

Myosin heavy chain 9-related disease (MYH9-RD) is a rare inherited disorder characterised by macrothrombocytopenia, often misdiagnosed as immune thrombocytopenia (ITP). Early identification is crucial to prevent unnecessary treatments and to ensure appropriate monitoring. The present case aims to highlight the diagnostic challenges and clinical management of MYH9-RD in a toddler, emphasising the importance of early genetic testing. We discuss a 13.5-month-old girl with macrothrombocytopenia lacking Döhle bodies, who initially received intravenous immunoglobulin (IVIg) and corticosteroids without any response. Within two months, whole-exome sequencing identified a pathogenic MYH9 mutation (c.287C>T; p.Ser96Leu). One year later, the patient remains clinically stable without significant bleeding. The occurrence of petechial rash exhibited a more pronounced correlation with platelet mass index (PMI) values compared to platelet count (PLT), underscoring its significance in clinical evaluation. MYH9-RD should be considered in cases of IVIg-resistant thrombocytopenia accompanied by macrothrombocytes. Timely genetic testing can facilitate accurate diagnosis and may help avoid unnecessary procedures, while routine renal and auditory monitoring is important for managing the S96L variant.

#5

Pulled-in-two syndrome in a young woman with restrictive strabismus and rare genetic mutation.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus2026 Feb

Pulled-in-two syndrome (PITS) is a rare but serious complication of strabismus surgery, with a reported incidence of once every decade among strabismus surgeons. It occurs more commonly in patients of advanced age and those with cranial nerve palsies or who have undergone prior surgeries. We present a case of PITS in a young female, diagnosed following surgery with a hereditary myosin myopathy.

Publicações recentes

Ver todas no PubMed

📚 EuropePMCmostrando 122

2026

Inherited Platelet Disorders During Pregnancy and Delivery: Overview of Management Strategies and Emerging Therapeutic Considerations.

Hematology reports
2026

The Clinical Details of MYH9-Related Disease and DFNA17 in a Large Japanese Hearing Loss Cohort.

Genes
2026

A Case Report of Thrombocytopenia Caused by a May-Hegglin Anomaly in a Young Saudi Female.

Journal of investigative medicine high impact case reports
2026

De Novo MYH9-Related Macrothrombocytopenia in a Toddler: Insights From Platelet Mass Index.

British journal of hospital medicine (London, England : 2005)
2026

Pulled-in-two syndrome in a young woman with restrictive strabismus and rare genetic mutation.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2026

Research Advances in the Physiological and Pathological Function of MYH9 and NMIIA.

International journal of immunogenetics
2026

Re-evaluating the MYH9 p.I1816V variant in a patient with atypical clinical presentation.

Pediatric nephrology (Berlin, Germany)
2025

A novel variant of MYH9 mutation associated macro-thrombocytopenia: A case series.

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis
2025

Successful kidney transplantation using eltrombopag in a patient with MYH9-related disease.

CEN case reports
2025

Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease.

Hematology (Amsterdam, Netherlands)
2025

Thrombocytopenia, renal failure and hearing loss in a young patient: MYH9-related disorder.

BMJ case reports
2025

Natural history of the severe subtype of MYH9-related disease (Epstein syndrome).

European journal of internal medicine
2025

Kidney pathological findings of MYH9-related disease: a cross-sectional nationwide survey in Japan.

Pediatric nephrology (Berlin, Germany)
2025

Bariatric Surgery for a Patient With Myosin Heavy Chain 9-Related Disorders (MYH9RD): A Case Report.

Journal of metabolic and bariatric surgery
2025

[Refractory immune thrombocytopenia revealing MYH9 related disease in a 64-year-old man].

La Revue de medecine interne
2025

Clinical and genetic characteristics of 40 patients with nonmuscle myosin heavy chain 9-related disease (MYH9-RD) misdiagnosed as immune thrombocytopenia: a retrospective analysis in China.

Journal of thrombosis and haemostasis : JTH
2025

MYH9-related disease with a normal platelet count.

CEN case reports
2024

Identification of a novel MYH9-related disease-associated mutation with multiple faintly staining Döhle-like bodies.

Pediatric blood &amp; cancer
2024

Successful hematopoietic stem cell transplantation in MYH9-related congenital thrombocytopenia.

Pediatric blood &amp; cancer
2025

MYH9-related disorder with sole presentation of end-stage kidney disease and long-term, recurrence-free living after living donor renal transplantation: a case report.

CEN case reports
2024

MYH9-related disease misdiagnosed for a decade: lessons from diagnostic pitfalls.

QJM : monthly journal of the Association of Physicians
2024

Diagnostic delay of MYH9-related disorder in Japan.

British journal of haematology
2024

Marked Underestimation of Platelet Count and a Characteristic Platelet Histogram as Clues to MYH9-Related Disorders.

Clinical laboratory
2023

Unveiling the hidden clues: Döhle body-like inclusions as morphological markers for MYH9-related disorders: A case report.

Medicine
2023

[Analysis of clinical phenotype and gene mutation characteristics of MYH9-related disorder].

Zhonghua yi xue za zhi
2024

A nationwide survey of MYH9-related disease in Japan.

Clinical and experimental nephrology
2023

Anesthetic considerations for May-Hegglin anomaly.

Minerva anestesiologica
2023

Successful living-related kidney transplantation in MYH9-related disorder with macrothrombocytopenia: lessons for the clinical nephrologist.

Journal of nephrology
2023

Hereditary thrombocytopenia with familial novel mutation in MYH9 gene: A familial case report.

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis
2023

Successful administration of eltrombopag in preparation for peritoneal dialysis catheter placement in a girl with MYH9-related disease.

CEN case reports
2022

Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia.

Children (Basel, Switzerland)
2022

A familial case of MYH9 gene mutation associated with multiple functional and structural platelet abnormalities.

Scientific reports
2022

Post-Kidney Transplant Brief Psychosis in a Patient With MYH9-Related Disease: A Case Report.

Transplantation proceedings
2022

Avatrombopag improves thrombocytopenia in MYH9-related disorder following eltrombopag treatment failure.

Platelets
2022

Defective VWF secretion due to expression of MYH9-RD E1841K mutant in endothelial cells disrupts hemostasis.

Blood advances
2022

Umbilical cord blood transplantation for MYH9-related disorders.

Pediatric blood &amp; cancer
2022

A gain-of-function variant in the Wiskott-Aldrich syndrome gene is associated with a MYH9-related disease-like syndrome.

Blood advances
2022

Lower Leg Hyperpigmentation in MYH9-Related Disorder.

Cutis
2022

Characterization of Sensorineural Hearing Loss in Patients With MYH9-Related Disease: A Systematic Review.

Otology &amp; neurotology : official publication of the American Otological Society, American Neurotology Society [and] European Academy of Otology and Neurotology
2021

[Clinical and genetic features of seven children with MYH9-related disease].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2021

[Gene analysis and clinical features of MYH9-related disease].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2021

Individualized Bleeding Risk Assessment through Thromboelastography: A Case Report of May-Hegglin Anomaly in Preterm Twin Neonates.

Children (Basel, Switzerland)
2021

Myosin Heavy Chain 9 (MYH9)-Related Congenital Macrothrombocytopenia.

Cureus
2021

MYH9-related Disease Caused by an R1165C Mutation in a Child With Previous Diagnosis of Immune Thrombocytopenic Purpura.

Journal of pediatric hematology/oncology
2021

Spontaneous and recurrent subdural haematoma in a patient with May-Hegglin anomaly.

BMJ case reports
2021

Role of Thrombopoietin Receptor Agonists in Inherited Thrombocytopenia.

International journal of molecular sciences
2021

Renal Biopsy-induced Hematoma and Infection in a Patient with Asymptomatic May-Hegglin Anomaly.

Journal of Nippon Medical School = Nippon Ika Daigaku zasshi
2021

The May-Hegglin anomaly: a rare cause of a common complaint.

BMJ case reports
2020

The curious incident of a cavum velum interpositum cyst in twins of a mother carrying May-Hegglin anomaly: a case report and short literature review.

BMC pregnancy and childbirth
2020

Anesthetic Management of Living-Donor Renal Transplantation in a Patient With Epstein Syndrome Using Rotational Thromboelastometry: A Case Report.

A&amp;A practice
2021

Obstetrical bleeding in women with MYH9-related disease-A systematic review.

Haemophilia : the official journal of the World Federation of Hemophilia
2022

Successful cochlear implantation in a patient with Epstein syndrome during long-term follow-up.

Auris, nasus, larynx
2020

A family with an MYH9-related disorder with different phenotypes masquerading as immune thrombocytopaenia: an underreported disorder in Taiwan.

International journal of hematology
2020

An interesting case of thrombocytopenia in pregnancy.

International journal of hematology
2020

Linking the Landscape of MYH9-Related Diseases to the Molecular Mechanisms that Control Non-Muscle Myosin II-A Function in Cells.

Cells
2021

A De Novo Mutation in MYH9 in a Child With Severe and Prolonged Macrothrombocytopenia.

Journal of pediatric hematology/oncology
2020

Familial macro thrombocytopenia: role of genetics where morphology fails.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2020

Megakaryocyte migration defects due to nonmuscle myosin IIA mutations underlie thrombocytopenia in MYH9-related disease.

Blood
2020

A case report of optic neuropathy following dacryocystorhinostomy in a 57-year-old female patient with May-Hegglin anomaly.

BMC ophthalmology
2020

Rare inherited kidney diseases: an evolving field in Nephrology.

Jornal brasileiro de nefrologia
2020

A novel de novo MYH9 mutation in MYH9-related disease: A case report and review of literature.

Medicine
2020

[Molecular diagnosis of a family with May-Hegglin anomaly].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2019

The use of pan-retinal photocoagulation to treat recurrent vitreous haemorrhage with neovascularisation in the context of Epstein syndrome: an MYH9-related disorder.

BMJ case reports
2020

A sporadic MYH9-related disease in a Chinese boy with p.A95T mutation.

Hematology (Amsterdam, Netherlands)
2019

Cellular defects resulting from disease-related myosin II mutations in Drosophila.

Proceedings of the National Academy of Sciences of the United States of America
2019

Safety of clozapine in patient with treatment resistant schizophrenia & asymptomatic constitutional macrothrombocytopenia (Harris syndrome).

Asian journal of psychiatry
2020

Next-generation sequencing for the diagnosis of MYH9-RD: Predicting pathogenic variants.

Human mutation
2019

Pay attention to neutrophil inclusions in pediatric patients with thrombocytopenia.

Blood
2020

Genetic classification and confirmation of inherited platelet disorders: current status in Korea.

Clinical and experimental pediatrics
2019

MYH9-related disorders display heterogeneous kidney involvement and outcome.

Clinical kidney journal
2019

Renin-angiotensin System Blockade Therapy for Early Renal Involvement in MYH9-related Disease with an E1841K Mutation.

Internal medicine (Tokyo, Japan)
2019

Cell surface expression of HLA I molecules as a marker of young platelets.

Journal of thrombosis and haemostasis : JTH
2019

Malta (MYH9 Associated Elastin Aggregation) Syndrome: Germline Variants in MYH9 Cause Rare Sweat Duct Proliferations and Irregular Elastin Aggregations.

The Journal of investigative dermatology
2019

[Clinical features and MYH9 gene variant in two Chinese siblings with Fechtner syndrome].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2019

MYH9-related disease mutations cause abnormal red blood cell morphology through increased myosin-actin binding at the membrane.

American journal of hematology
2019

MYH9 Disorders (May-Hegglin Anomaly) the Role of the Blood Smear.

Journal of pediatric hematology/oncology
2018

[Hereditary thrombocytopenia associated with a mutation in the MYH-9 gene. Report of one case].

Revista medica de Chile
2020

Identification of a Novel MYH9 Mutation in a Young Adult With Inherited Thrombocytopenia and Recurrent Seizures by Targeted Exome Sequencing.

Journal of pediatric hematology/oncology
2019

Genotype-phenotype correlation of a novel MYH9 mutation (p.G736L) in a patient with macrothrombocytopenia and end-stage renal disease.

Annals of hematology
2019

MYH9 Associated nephropathy.

Nefrologia
2018

Indication of total parathyroidectomy for an Epstein syndrome patient with end-stage renal disease.

Romanian journal of morphology and embryology = Revue roumaine de morphologie et embryologie
2018

Isolated thrombocytopenia in childhood: what if it is not immune thrombocytopenia?

Singapore medical journal
2018

MYH9-related disorders: a rare cause of neonatal thrombocytopaenia.

BMJ case reports
2019

Expert consensus guidelines for the genetic diagnosis of Alport syndrome.

Pediatric nephrology (Berlin, Germany)
2018

Anesthetic management without perioperative platelet transfusion for cervical laminectomy and laminoplasty in a case of May-Hegglin anomaly.

Journal of anesthesia
2018

Macrothrombocytopenia, renal dysfunction and nephrotic syndrome in a young male patient: a case report of MYH9-related disease.

Jornal brasileiro de nefrologia
2018

MYH9: Structure, functions and role of non-muscle myosin IIA in human disease.

Gene
2018

[Patient was wrongly diagnosed and repeatedly treated for immune thrombocytopenia for 50 years].

Lakartidningen
2019

Management of patients with severe Epstein syndrome: Review of four patients who received living-donor renal transplantation.

Nephrology (Carlton, Vic.)
2018

Macrothrombocytopenia With Congenital Bilateral Cataracts: A Phenotype of MYH9 Disorder With Exon 24 Indel Mutations.

Journal of pediatric hematology/oncology
2017

Nephroquiz 10: A 16-Year-Old Patient With Thrombocytopenia and Kidney Failure.

Iranian journal of kidney diseases
2018

Mean platelet diameter measurements to classify inherited thrombocytopenias.

International journal of laboratory hematology
2018

Diagnosis and treatment of MYH9-RD in an Australasian cohort with thrombocytopenia.

Platelets
2017

New Alert Message Settings for the XN-series Automated Hematology Analyzer Are Useful for Avoiding Falsely High WBC Counts and to Detect Specimens with Giant Platelets.

Annals of clinical and laboratory science
2017

Subarachnoid Hemorrhage Revealing Moyamoya Syndrome in a Patient With May-Hegglin Anomaly.

The neurologist
2018

Eltrombopag to Treat Thrombocytopenia During Last Month of Pregnancy in a Woman With MYH9-Related Disease: A Case Report.

A&amp;A practice
2017

MYH9-Related Thrombocytopenia.

Mayo Clinic proceedings
2017

[Renal diseases related to MYH9 disorders].

Giornale italiano di nefrologia : organo ufficiale della Societa italiana di nefrologia
2017

Case 4: Hydrocephalus, Macrothrombocytopenia, Inclusion Bodies, and Nephropathy in a 9-year-old Boy.

Pediatrics in review
2017

[Genetic analysis of a pedigree affected with inherited thrombocytopenia caused by a novel mutation of MYH9 gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2017

MYH9 gene mutations associated with bleeding.

Platelets
2017

Role for formin-like 1-dependent acto-myosin assembly in lipid droplet dynamics and lipid storage.

Nature communications
2016

Severe to profound deafness may be associated with MYH9-related disease: report of 4 patients.

Acta otorhinolaryngologica Italica : organo ufficiale della Societa italiana di otorinolaringologia e chirurgia cervico-facciale
2016

MYH9-related disorder, a probable May-Hegglin anomaly case series: A tertiary care experience.

Hematology/oncology and stem cell therapy
2016

Delayed diagnosis of MYH-9–related disorder and the role of light microscopy in congenital macrothrombocytopenias.

Blood
2016

Macrothrombocytopenia With Döhle Body-Like Granulocyte Inclusions: A Case Report of May-Hegglin Anomaly in a 33-Year-Old White Woman With an Update on the Molecular Findings of MYH9-Related Disease.

Laboratory medicine
2016

[Anesthetic Management of a Patient with May-Hegglin Anomaly].

Masui. The Japanese journal of anesthesiology
2016

Grey platelet syndrome misdiagnosed as ITP.

British journal of haematology
2016

Late onset and high-frequency dominant hearing loss in a family with MYH9 disorder.

European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery
2016

A Japanese pedigree with a p.A95V mutation in the MYH9 gene demonstrates inherited macrothrombocytopenia without Alport manifestations.

Annals of hematology
2015

Successful Kidney Transplantation in Epstein Syndrome With Antiplatelet Antibodies and Donor-specific Antibodies: A Case Report.

Transplantation proceedings
2015

Easy bruising due to giant platelet (possibly MYH9-related/Sebastian) syndrome.

Indian journal of dermatology, venereology and leprology
2015

[Which genetic testing in renal disease].

Giornale italiano di nefrologia : organo ufficiale della Societa italiana di nefrologia
2016

Preoperative use of platelets in a 6-year-old with acute appendicitis and a myosin heavy chain 9-related disorder: a case report and review of literature.

Transfusion
2015

Sporadic Epstein syndrome with macrothrombocytopenia, sensorineural hearing loss and renal failure.

Pediatrics international : official journal of the Japan Pediatric Society
2016

Thrombin generation in two families with MYH9-related platelet disorder.

Platelets
2015

[Hereditary macrothrombocytopenia and hearing loss].

Duodecim; laaketieteellinen aikakauskirja
2016

Nonmuscle Myosin Heavy Chain IIA Mutation Predicts Severity and Progression of Sensorineural Hearing Loss in Patients With MYH9-Related Disease.

Ear and hearing
2015

Genotype-phenotype Correlation of the p.R1165C Mutation in the MYH9 Disorder: Report of a Japanese Pedigree.

Journal of pediatric hematology/oncology
2015

Immature platelet fraction measurement is influenced by platelet size and is a useful parameter for discrimination of macrothrombocytopenia.

Hematology (Amsterdam, Netherlands)
2015

Misdiagnosed MYH9 related inherited macrothrombocytopenia with an inadvertent splenectomy.

Pathology
2015

MYH9-related thrombocytopenia and intracranial bleedings: a complex clinical/surgical management and review of the literature.

British journal of haematology

Associações

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Doenças relacionadas

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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.

  1. Inherited Platelet Disorders During Pregnancy and Delivery: Overview of Management Strategies and Emerging Therapeutic Considerations.
    Hematology reports· 2026· PMID 41874099mais citado
  2. The Clinical Details of MYH9-Related Disease and DFNA17 in a Large Japanese Hearing Loss Cohort.
    Genes· 2026· PMID 41751538mais citado
  3. A Case Report of Thrombocytopenia Caused by a May-Hegglin Anomaly in a Young Saudi Female.
    Journal of investigative medicine high impact case reports· 2026· PMID 41721658mais citado
  4. De Novo MYH9-Related Macrothrombocytopenia in a Toddler: Insights From Platelet Mass Index.
    British journal of hospital medicine (London, England : 2005)· 2026· PMID 41609159mais citado
  5. Pulled-in-two syndrome in a young woman with restrictive strabismus and rare genetic mutation.
    Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus· 2026· PMID 41587614mais citado
  6. Re-evaluating the MYH9 p.I1816V variant in a patient with atypical clinical presentation.
    Pediatr Nephrol· 2026· PMID 41243005recente
  7. A novel variant of MYH9 mutation associated macro-thrombocytopenia: A case series.
    Transfus Apher Sci· 2025· PMID 40934793recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:182050(Orphanet)
  2. OMIM OMIM:153640(OMIM)
  3. MONDO:0015912(MONDO)
  4. GARD:180(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q3843790(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

Trombocitopenia sindrômica MYH9-relacionada
Compêndio · Raras BR

Trombocitopenia sindrômica MYH9-relacionada

ORPHA:182050 · MONDO:0015912
Prevalência
1-9 / 1 000 000
Herança
Autosomal dominant
CID-10
D69.4 · Outra trombocitopenia primária
CID-11
Início
Adolescent, Adult, Childhood, Infancy, Neonatal
Prevalência
0.29 (Italy)
MedGen
UMLS
C5200934
Repurposing
2 candidatos
avatrombopagthrombopoietin receptor agonist
eltrombopag
Wikidata
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