Raras
Buscar doenças, sintomas, genes...
Hemofilia A
ORPHA:98878CID-10 · D66CID-11 · 3B10.0OMIM 134500PCDT · SUSDOENÇA RARA

É o tipo mais comum de hemofilia, que causa sangramentos que surgem sem motivo aparente ou que demoram muito para parar, por causa da falta do Fator VIII.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

É o tipo mais comum de hemofilia, que causa sangramentos que surgem sem motivo aparente ou que demoram muito para parar, por causa da falta do Fator VIII.

Pesquisas ativas
35 ensaios
839 total registrados no ClinicalTrials.gov
Publicações científicas
7.221 artigos
Último publicado: 2026 Apr 16
Medicamentos
10 registrados
SIMOCTOCOG ALFA, EMICIZUMAB, EFMOROCTOCOG ALFA

Tem tratamento?

10 medicamentos registrados
Ver detalhes, fases e interações →
SIMOCTOCOG ALFAEMICIZUMABEFMOROCTOCOG ALFAANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANTEPTACOG BETA (ACTIVATED)TRANEXAMIC ACIDTUROCTOCOG ALFAEPTACOG ALFA (ACTIVATED)MOROCTOCOG ALFAOCTOCOG ALFA

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 100 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
4.85
Worldwide
Início
Childhood
+ infancy, neonatal
🏥
SUS: Cobertura completaScore: 70%
PCDT disponível7 medicamentos CEAFCID-10: D66
🇧🇷Dados SUS / DATASUS2024
6.780
internações/ano
R$ 12.450
custo médio/internação
ESTADOS COM MAIS INTERNAÇÕES
SPRJMGBARS
PROCEDIMENTOS SIGTAP (2)
0303020032
Infusão de fator VIII (Hemofilia A)blood_product
0303020075
Emicizumabe (Hemofilia A — Hemlibra)biologic
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

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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

🩸
Sangue
14 sintomas
🦴
Ossos e articulações
5 sintomas
💪
Músculos
4 sintomas
🫘
Rins
2 sintomas
❤️
Coração
1 sintomas
🫃
Digestivo
1 sintomas

+ 22 sintomas em outras categorias

Características mais comuns

90%prev.
Artralgia
Muito frequente (99-80%)
90%prev.
Inchaço articular
Muito frequente (99-80%)
90%prev.
Atividade reduzida do fator VIII
Muito frequente (99-80%)
90%prev.
Sangramento com trauma leve ou sem trauma
Muito frequente (99-80%)
55%prev.
Hematomas espontâneos
Frequente (79-30%)
55%prev.
Tromboembolismo
Frequente (79-30%)
51sintomas
Muito frequente (4)
Frequente (3)
Ocasional (5)
Muito raro (2)
Sem dados (37)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 51 características clínicas mais associadas, ordenadas por frequência.

ArtralgiaArthralgia
Muito frequente (99-80%)90%
Inchaço articularJoint swelling
Muito frequente (99-80%)90%
Atividade reduzida do fator VIIIReduced factor VIII activity
Muito frequente (99-80%)90%
Sangramento com trauma leve ou sem traumaBleeding with minor or no trauma
Muito frequente (99-80%)90%
Hematomas espontâneosSpontaneous hematomas
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico7.221PubMed
Últimos 10 anos200publicações
Pico2026152 papers
Linha do tempo
2026Hoje · 2026🧪 1978Primeiro 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: X-linked recessive.

F8Coagulation factor VIIIDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Factor VIII, along with calcium and phospholipid, acts as a cofactor for F9/factor IXa when it converts F10/factor X to the activated form, factor Xa

LOCALIZAÇÃO

Secreted, extracellular space

VIAS BIOLÓGICAS (1)
COPII-mediated vesicle transport
MECANISMO DE DOENÇA

Hemophilia A

A disorder of blood coagulation characterized by a permanent tendency to hemorrhage. About 50% of patients have severe hemophilia resulting in frequent spontaneous bleeding into joints, muscles and internal organs. Less severe forms are characterized by bleeding after trauma or surgery.

EXPRESSÃO TECIDUAL(Ubíquo)
Adipose Visceral Omentum
19.6 TPM
Tecido adiposo
15.7 TPM
Coração - Átrio
13.5 TPM
Mama
12.6 TPM
Ovário
12.6 TPM
OUTRAS DOENÇAS (6)
hemophilia Athrombophilia, X-linked, due to factor 8 defectmoderately severe hemophilia Asevere hemophilia A
HGNC:3546UniProt:P00451

Medicamentos e terapias

SIMOCTOCOG ALFAPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

EMICIZUMABPhase 4

Mecanismo: Coagulation factor IX and X other

EFMOROCTOCOG ALFAPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANTPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

EPTACOG BETA (ACTIVATED)Phase 4

Mecanismo: Coagulation factor VII exogenous protein

TRANEXAMIC ACIDPhase 4

Mecanismo: Plasminogen inhibitor

TUROCTOCOG ALFAPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

EPTACOG ALFA (ACTIVATED)Phase 4

Mecanismo: Coagulation factor VII exogenous protein

MOROCTOCOG ALFAPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

OCTOCOG ALFAPhase 4

Mecanismo: Coagulation factor VIII exogenous protein

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

1,066 variantes patogênicas registradas no ClinVar.

🧬 F8: NM_000132.4(F8):c.7016G>T (p.Arg2339Met) ()
🧬 F8: NM_000132.4(F8):c.310G>A (p.Val104Ile) ()
🧬 F8: GRCh38/hg38 Xq28(chrX:154881139-155396181)x2 ()
🧬 F8: GRCh38/hg38 Xq26.3-28(chrX:137491159-155700385)x2 ()
🧬 F8: NM_000132.4(F8):c.908C>A (p.Ala303Glu) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 5 variantes classificadas pelo ClinVar.

5
Patogênica (100.0%)
VARIANTES MAIS SIGNIFICATIVAS
F8: NM_000132.4(F8):c.7016G>T (p.Arg2339Met) [Likely pathogenic]
F8: NM_000132.4(F8):c.954_955del (p.Leu319fs) [Pathogenic]
F8: NM_000132.4(F8):c.2113+461_2113+473del [Pathogenic]
F8: NM_000132.4(F8):c.1648C>T (p.Arg550Cys) [Pathogenic/Likely pathogenic]
F8: NM_000132.4(F8):c.902G>A (p.Arg301His) [Pathogenic]

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.
Aprovado11
3Fase 38
2Fase 23
1Fase 13
·Pré-clínico5
Medicamentos catalogadosEnsaios clínicos· 10 medicamentos · 20 ensaios
✓ Aprovados — podem ser usados hoje
SIMOCTOCOG ALFAEMICIZUMABEFMOROCTOCOG ALFAANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANTEPTACOG BETA (ACTIVATED)TRANEXAMIC ACIDTUROCTOCOG ALFAEPTACOG ALFA (ACTIVATED)MOROCTOCOG ALFAOCTOCOG ALFA
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Hemofilia A

🗺️

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

🟢 Recrutando agora

17 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

NCT06820515 · ATHNdataset RegistryRecrutando
NCT07285460 · A Study to Investigate the Efficacy and Safety of Fitusiran …Recrutando
PHASE3
NCT06222697 · A Study to Learn More About the Safety of Damoctocog-alfa-pe…Recrutando
NCT05145127 · Open-Label Extension Study of Marstacimab in Hemophilia Part…Recrutando
PHASE3
NCT07416526 · A Clinical Study to Evaluate the Effects of NXT007 Compared …Recrutando
PHASE3
NCT07523399 · Joint Health, Balance and Quality of Life in Adults With Hem…Recrutando
NCT05568719 · Safety and Effectiveness of Giroctocogene Fitelparvovec or F…Recrutando
PHASE3
NCT07416604 · A Clinical Study to Evaluate the Effects of NXT007 Compared …Recrutando
PHASE3
NCT04278404 · Pharmacokinetics, Pharmacodynamics, and Safety Profile of Un…Recrutando
NCT05987449 · A Study to Evaluate the Safety, Tolerability, Pharmacokineti…Recrutando
PHASE1, PHASE2
NCT05500807 · Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/H…Recrutando
PHASE1
NCT05611801 · A Clinical Trial of Study Medicine (Marstacimab) in Pediatri…Recrutando
PHASE3
NCT06703606 · A Study to Learn About How Changing Therapy From Emicizumab …Recrutando
PHASE1
NCT06940830 · Long-term Study Evaluating Joint Health in People With Haemo…Recrutando
PHASE4
NCT07101926 · Monitoring of Anti-TFPI in HemophiliaRecrutando
NCT06111638 · Evaluation of the Safety and Efficacy of Hemophilia A Gene T…Recrutando
PHASE2, PHASE3
NCT06349473 · A Study of Safety, Tolerability, Pharmacokinetics, and Pharm…Recrutando
PHASE1

Outros ensaios clínicos

839 ensaios clínicos encontrados, 35 ativos.

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

🥈Melhor nível de evidência: Observacional
Timeline de publicações
3.730 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 3.730

#1

FIXa-triggered thrombin generation correlates with FVIII levels less than 1% in reconstituted plasma mimicking samples obtained from patients with severe hemophilia A.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis2026 Mar 17

More than 10% of severe hemophilia A patients are paradoxically mild bleeders despite factor VIII (FVIII) levels being <1 IU/dl. Quantitation of FVIII levels <1 IU/dl is a challenge due to the detection limits of traditional one-stage activated partial thromboplastin time, two-stage chromogenic assay, and tissue factor-initiated thrombin generation assay (TF-TGA), impeding a precise characterization of the bleeding phenotype in severe hemophilia patients. The study aimed to enhance the sensitivity of TGA by modifying the trigger reagent for FVIII measurement. We optimized the fluorometric quantitation of thrombin generation triggered by factor IXa (FIXa) in artificial reconstituted plasma by blending varying proportions of normal pooled plasma (NPP) and FVIII-immunodepleted plasma containing a normal level of von Willebrand factor. FIXa-initiated thrombin generation depended on FVIII level, while TF could bypass FVIII deficiency to activate thrombin, making TF-TGA incapable of quantifying low levels of FVIII. When triggering with 0.6 nM FIXa, in the presence of 4 μM phospholipids and 40 μg/ml corn trypsin inhibitor, thrombin generation was highly dependent on FVIII levels even below 1 IU/dl. Among the five routinely used TGA parameters, peak thrombin, endogenous thrombin potential, and velocity index demonstrated the strongest linear correlation with FVIII levels down to 0.1 IU/dl. The study evaluated the performance of a modified TGA activated with FIXa. In addition to traditional TF-TGA, FIXa-TGA may provide additional information when assessing the bleeding tendency of patients with severe hemophilia A, facilitating personalized factor replacement therapy.

#2

Deleterious NKAP Mutations Are Associated with Musculoskeletal Abnormalities in Hemizygous Males and Skewed X Chromosome Inactivation in Heterozygous Females.

International journal of molecular sciences2026 Mar 02

NKAP (NF-kappa-B-activating protein) is a ubiquitously expressed nuclear protein involved in multiple biological processes. Males with missense NKAP mutations have been reported to present with marfanoid features and behavioral and musculoskeletal abnormalities. We have previously reported that a disruptive NKAP mutation resulted in extremely skewed X chromosome inactivation (XCI), leading to phenotypic manifestation of hemophilia A (HA) in a HA carrier. In this study, with the aim of exploring the phenotypic manifestations of deleterious NKAP mutations in males, as well as their involvement in the mechanism of XCI regulation in females, we generated NKAP mutant mice using CRISPR/Cas9 technology. Gait analysis studies conducted in male mice hemizygous for mutant NKAP by the CatWalk XT system revealed significant alterations in gait parameters, consistent with hypotonia reported in human mutant NKAP patients. By breeding mutant NKAP mice with HA mice, we generated a double heterozygous mutant NKAP/HA mouse model, i.e., female mice carrying mutant NKAP with a WT F8 copy on one X chromosome, and WT NKAP with a mutant F8 copy on the other X chromosome. XCI pattern analysis using methylation-sensitive restriction enzymes demonstrated that mutant NKAP/HA females exhibited significant XCI skewing of the X chromosome bearing the mutant NKAP copy. Furthermore, these females exhibited significantly reduced F8 mRNA levels and FVIII (factor VIII) antigen levels, as demonstrated by quantitative RT-PCR and ELISA, respectively. Murine embryonic fibroblasts (MEFs) derived from a hemizygous mutant NKAP embryo exhibited markedly reduced proliferation rate and increased senescence compared to WT NKAP MEFs, suggesting that XCI skewing induced by mutant NKAP results from secondary selection against cells with an active X chromosome bearing the mutant NKAP copy.

#3

Genotype-Specific Postural Control Deficits in Hemophilia A: Insights from Center of Pressure Analysis Beyond Radiographic Arthropathy.

International journal of molecular sciences2026 Mar 01

Hemophilia is an X-linked inherited bleeding disorder characterized by joint hemorrhages and progressive arthropathy. While mutation type is known to influence disease severity, its impact on postural balance strategies has remained unclear. This cross-sectional study investigated the relationship between gene mutation type and postural control in hemophilia A patient using center of pressure (CoP) analysis and radiographic joint assessment with the Pettersson score. Thirty-five participants were divided into an INV group (intron 22 or intron 1 inversion of the F8 gene) and a NonINV group (other mutations). While the Pettersson scores and traditional time-domain CoP parameters (sway area, velocity) were comparable between groups, frequency domain analysis revealed a significant difference. INV group exhibited significantly higher energy content above 2 Hz in the anteroposterior direction compared to NonINV group. This genotype-specific spectral signature emerged despite comparable radiographic arthropathy and conventional CoP metrics, suggesting that frequency-domain CoP analysis can uncover subclinical postural adaptations in hemophilia A. These findings highlight the need for targeted proprioceptive training in this specific subpopulation to prevent subclinical instability and potential falls.

#4

Monochorionic diamniotic twin brothers with severe hemophilia A: a case report.

Frontiers in pediatrics2026

Congenital hemophilia A is a recessive inherited hemorrhagic disorder caused by factor VIII (FVIII) deficiency. According to the activity of functional coagulation FVIII, the severity of hemophilia A is divided into three levels: mild, moderate and severe. The characteristic phenotype in hemophilia is the bleeding tendency. Clinical severity depends on the extent of the FVIII deficiency and the first bleeding episode in severe and moderate congenital hemophilia A usually occurs in early childhood. At present, there are few reports on symptomatic severe congenital hemophilia A in the neonatal period. We describe a pair of monozygotic twin brothers with severe hemophilia A. Patient-related factors, including a birth weight discrepancy of 10%, the need for non-invasive respiratory support due to mild respiratory distress, duration of breastfeeding, and vaccinations, were similar in both twins. Anti-hemorrhagic prophylaxis was carried out after birth with IM vitamin K. Due to the presence of prolonged bleeding at the sampling site after performing EGA (Blood Gas Analysis) and neonatal screening, a coagulation test was carried out and a coagulation factor assay (dosage of activity of factors VII, IX, XI, XII) was performed accordingly: activated partial thromboplastin time (APTT) was prolonged without extended prothrombin time (PT). Factor VIII activity was completely absent (0.7%) in both twins. Hematological consultancy was requested and a diagnosis of severe congenital hemophilia A was established. Emicizumab was started as the primary anti-hemorrhagic prophylaxis, with good response and no major bleeding events in the first year of therapy. The coagulation system is not fully developed at birth, complicating clinical decision-making and the correct interpretation of coagulation testing. Targeted coagulation profiling and factor assays are mission-critical for newborns from twin pregnancies when a hematological disorder is suspected. Coagulation factor assays are essential to confirm the diagnosis of hemophilia A. An early diagnosis of hemophilia is crucial for the timely initiation of an appropriate management plan.

#5

A combinatorial CAAR-T cell strategy to eliminate factor VIII inhibitors in preclinical models of hemophilia A.

iScience2026 Mar 20

Chimeric autoantibody receptor T (CAAR-T) cell therapy extends CAR-T technology to target autoreactive immune cells, offering a novel approach for treating autoimmune disorders. In hemophilia A, the development of anti-factor VIII (FVIII) inhibitors poses a major clinical challenge. This study investigates the therapeutic potential of CAAR-T cells engineered with FVIII C1 or A2 domains to eliminate inhibitor-producing B cells. In vitro cytotoxicity assays confirmed that C1 and A2 CAAR-T cells exhibited comparable killing efficiency against their respective targets. In an F8KO hemophilia A mouse model, both CAAR-T cells significantly reduced circulating FVIII inhibitors, while their combination further suppressed inhibitor re-emergence after FVIII rechallenge. In a humanized NCG mouse model, combined CAAR-T therapy effectively inhibited in vivo target cell expansion. These findings demonstrate that CAAR-T therapy, particularly the combinatorial approach, holds promise for addressing FVIII inhibitor formation in hemophilia A, offering a targeted and effective treatment strategy.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC10.797 artigos no totalmostrando 198

2026

Hemorrhagic Shock and Massive Abdominal Wall Hematoma After Cesarean Section in a Patient with Peripartum Acquired Hemophilia A:A Case Report.

International journal of women's health
2026

Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method.

Advances in therapy
2026

Clinical Experience with Emicizumab and Rituximab as First-Line Treatment in a Case Series of Acquired Hemophilia A.

Hematology reports
2026

Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.

The Cochrane database of systematic reviews
2026

Postpartum Acquired Hemophilia A in Two Women With Previously Undiagnosed Carrier Status for von Willebrand Disease Type 2N and Heterozygous p.R854Q Mutation.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Case Report: Differential diagnosis and clinical management of isolated prolonged activated partial thromboplastin time.

Frontiers in immunology
2026

ABO blood group-mediated VWF kinetics associated with early phenotypic divergence in dizygotic twins with severe hemophilia A.

Thrombosis research
2026

Investigation of kinesiophobia, physical activity, quality of life, depression and anxiety level in adult people with hemophilia: A frequency-matched case-control study.

Journal of back and musculoskeletal rehabilitation
2026

In Vitro Stability Study Supports the Use of Efmoroctocog Alfa and Rurioctocog Alfa Pegol for Continuous Infusion in Hemophilia A.

Seminars in thrombosis and hemostasis
2026

FIXa-triggered thrombin generation correlates with FVIII levels less than 1% in reconstituted plasma mimicking samples obtained from patients with severe hemophilia A.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2026

Delayed diagnosis of acquired hemophilia a complicating hepatocellular carcinoma: a rare case of hepatectomy following atezolizumab and bevacizumab therapy.

Clinical journal of gastroenterology
2026

Acute recurrent appendicitis in a hemophiliac Nepalese girl: beyond just… avoidance of formidable cuts! - A case report.

International journal of surgery case reports
2026

New Methods for Activated Partial Thromboplastin Time -Based Clot Waveform Analysis: Normalization and Multi-Parameter Combination.

International journal of general medicine
2026

An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study.

Therapeutic advances in hematology
2026

Deleterious NKAP Mutations Are Associated with Musculoskeletal Abnormalities in Hemizygous Males and Skewed X Chromosome Inactivation in Heterozygous Females.

International journal of molecular sciences
2026

Genotype-Specific Postural Control Deficits in Hemophilia A: Insights from Center of Pressure Analysis Beyond Radiographic Arthropathy.

International journal of molecular sciences
2026

Psychosocial Outcomes and Quality of Life in Patients with Hemophilia a Without Inhibitors: The HemoLIFE Study.

Journal of clinical medicine
2026

Bleeding rates, healthcare utilization, and costs among patients with hemophilia a without inhibitors treated with concomitant octocog alfa or extended half-life factor VIII while on emicizumab prophylaxis.

Journal of medical economics
2026

Mechanisms of thrombin inhibition by protein S and the TFPIα-fVshort-protein S complex.

Biophysical journal
2026

In Vitro Spiking Comparison of Coagulation Potential Between Emicizumab and Mim8 in Whole Blood and Plasma From a Single Patient With Severe Hemophilia A Receiving FVIII Prophylaxis and Warfarin.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Persons with high Hemophilia Joint Health Scores have reduced functional range of motion during simulated sports activities.

Research and practice in thrombosis and haemostasis
2026

[Acquired hemophilia A secondary to marginal zone lymphoma].

Revue medicale de Liege
2026

Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity.

Signal transduction and targeted therapy
2026

Monochorionic diamniotic twin brothers with severe hemophilia A: a case report.

Frontiers in pediatrics
2026

Comprehensive Care for People With Hemophilia: A Dental Perspective From an Indian Scenario.

Special care in dentistry : official publication of the American Association of Hospital Dentists, the Academy of Dentistry for the Handicapped, and the American Society for Geriatric Dentistry
2026

Subclinical Cardiac Diseases and the Role of Extracellular Vesicles in Patients with Hemophilia A Treated on-Demand.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2026

A Case of Acquired Hemophilia A Following Robot-Assisted Minimally Invasive Esophagectomy.

Surgical case reports
2026

A combinatorial CAAR-T cell strategy to eliminate factor VIII inhibitors in preclinical models of hemophilia A.

iScience
2026

Enhanced Peripartum Hemostatic Management Does Not Decrease Postpartum Hemorrhage Incidence in Hemophilia Carriers: the Pregnancy and Inherited Bleeding Disorders study (PRIDES).

Journal of thrombosis and haemostasis : JTH
2026

Development of a Novel Hemophilia A Mouse Model to Study Emicizumab and Factor VIII Inhibitor Risk.

Blood advances
2026

Kinesiophobia in Parents of Children with Haemophilia.

Journal of the College of Physicians and Surgeons--Pakistan : JCPSP
2026

Discrepancies of results for post infusion levels of extended half-life factor VIII/IX concentrates - a cause of concern, awaiting for solution.

Clinical chemistry and laboratory medicine
2026

Comparative Efficacy and Safety of Non-Clotting Factor Prophylaxis Versus. on-Demand Therapy in Hemophilia: A Meta-Analysis of Randomized Controlled Trials.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2026

Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.

Journal of blood medicine
2026

COVID-19-Associated Acquired Hemophilia A With an Exceptionally High Inhibitor Titer: A Case of Remission and Overwhelming Sepsis.

Cureus
2026

Immune Response Associated Hepatotoxicity in Hemophilia Gene Therapy: Mechanisms, Management, and Challenges.

Journal of hematology
2026

Emicizumab in the treatment of acquired hemophilia A: A Two-Center experience.

Annals of hematology
2026

Machine learning estimation of FVIII pharmacokinetic parameters in Chinese children with severe Hemophilia A.

NPJ systems biology and applications
2026

Is marstacimab the next step in personalized and effective haemophilia management?

Expert review of clinical pharmacology
2026

Association of Patient-Reported Outcomes with Hemophilia A Inhibitor Status and Treatment Product Type.

Journal of clinical medicine
2026

Managing massive gastrointestinal and abdominal haemorrhage in inherited bleeding disorders: experience from a pediatric cohort.

International journal of hematology
2026

Two decades of prenatal diagnosis in hemophilia A and B: a systematic review of global trends and current practices.

Thrombosis journal
2026

Pathogen-Reduce Cryoprecipitate: An Overview of Method(s) in Pathogen Reduction, Transfusion-Transmitted Infection Risk, and Inventory Management Considerations.

Clinics in laboratory medicine
2026

Understanding Treatment Care and Delays in Acquired Hemophilia A: A Population-Based Study From Nova Scotia, Canada.

EJHaem
2026

Factor-VIII mimetic bispecific antibodies for the treatment of hemophilia A: an update.

Expert review of clinical pharmacology
2026

Health Related Quality of Life (HRQoL) and the Factors Affecting it in Children with Hemophilia.

Indian journal of hematology &amp; blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion
2026

Pharmacokinetics of Factor VIII in Adults with Hemophilia: A 24-Hour Single-Sample Study Focused on Trough Levels.

Indian journal of hematology &amp; blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion
2026

Exercise Programmes for People With Haemophilia: A Scoping Review.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

GTH 2026: Connected Science - Advanced Solutions.

Hamostaseologie
2026

A Multifactorial Case of Acquired Hemophilia A.

Cureus
2026

Gene therapy in the treatment of hemophilia A: a systematic review and meta-analysis.

Expert review of hematology
2026

Corrigendum to 'Deleterious variants cluster in the A3 domain of factor VIII in people with severe hemophilia A and inhibitors': [Research and Practice in Thrombosis and Haemostasis Volume 9, Issue 6, August 2025, 103006].

Research and practice in thrombosis and haemostasis
2026

Factor VIII in vitro bioequivalence of denecimig (Mim8) hemostatic effect by thrombin generation assays.

Research and practice in thrombosis and haemostasis
2026

Thrombin Generation Assays: Possibilities and Limitations.

Hamostaseologie
2026

Immune Response Mechanisms in Haemophilia A.

Hamostaseologie
2026

Gene Therapy of Haemophilia: Current Status and Future Directions.

Hamostaseologie
2026

Severe hemophilia A with inhibitors and pancreatic cancer - when emicizumab is not enough.

Blood transfusion = Trasfusione del sangue
2026

Twelve-month follow-up of a real-world, noninterventional study evaluating the impact of emicizumab on bleeding episodes, joint health, and quality of life in people with hemophilia A.

Therapeutic advances in hematology
2026

A chemiluminescent microfluidic thrombin generation assay for real-time monitoring in patient plasma.

Journal of thrombosis and haemostasis : JTH
2026

MLDP-AS: an optimized next-generation sequencing assay for enhanced detection of technically challenging variants in expanded carrier screening.

Journal of translational medicine
2026

Country-level prediction of blood type distribution in hemophilia A in support of factor VIII consumption.

Research and practice in thrombosis and haemostasis
2026

Challenges in Balancing Hemostasis and Thrombosis in Therapy Tailoring for Hemophilia: A Narrative Review.

International journal of molecular sciences
2026

Enhancing Hemophilia A Care Through Home-Based Prophylaxis: Real-World Outcomes of a National Patient Support Program in Mexico.

Journal of clinical medicine
2026

Postpartum acquired hemophilia presenting as compartment syndrome: a diagnostic challenge in the emergency department.

International journal of emergency medicine
2026

Integrative modeling to improve bleeding risk prediction in adult female hemophilia A carriers.

Journal of thrombosis and haemostasis : JTH
2026

A Revised Classification of FVIII Concentrates: Rationale and Novel Metrics.

Blood advances
2026

FVIII-containing platelets modulate immune responses and attenuate inhibitor development in hemophilia A mice.

Haematologica
2025

Impact of prophylaxis, inhibitors, and genetics on joint outcomes according to the IPSG-MRI score in hemophilia A, B and vWD type 3.

Frontiers in medicine
2026

Clinical Spectrum, Subtype Distribution, and Treatment Outcomes in von Willebrand Disease: A Prospective Study from a Hemophilia Treatment Center in Pakistan.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2026

Mutations of six amino acid residues in a B domain-deleted blood coagulation factor VIII have a cumulative effect on increasing its secretion.

Research and practice in thrombosis and haemostasis
2026

TNF‑α Gene Polymorphisms as Determinants of Alloantibody Emergence in Hemophilia: A Systematic Review and Meta-Analysis.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Factors contributing to the development of hemophilic arthropathy: A real-world study.

Annals of hematology
2026

A first-in-human study assessing the safety, pharmacokinetics, and pharmacodynamics of TU7710, a recombinant factor VIIa-transferrin fusion protein, in warfarin-pretreated healthy male participants.

Journal of thrombosis and haemostasis : JTH
2026

Monitoring the Hemostatic Balance: measuring thrombin generation in a patient with acquired hemophilia A on combined pro- and anticoagulant therapy.

Thrombosis research
2026

rFVIIIa-platelet binding enhances platelet procoagulant activity independently of thrombin generation.

Blood vessels, thrombosis &amp; hemostasis
2026

Human leukocyte antigen alleles associated with inhibitor development in severe hemophilia A: analysis of the "My Life, Our Future" hemophilia A cohort.

Journal of thrombosis and haemostasis : JTH
2026

The MAPTO survey: worldwide approaches on unmasking factor VIII inhibitors in children with emicizumab treatment: communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders.

Journal of thrombosis and haemostasis : JTH
2026

NXTAGE: a phase 1/2 study of NXT007 to assess safety, pharmacokinetics, and efficacy in hemophilia A without inhibitors.

Blood
2026

A specific FX activator for bleeding treatment in hemophilia with inhibitors: multicenter, open-label, phase I/II trials.

Blood advances
2026

Real-world adherence to and persistence with emicizumab in people with hemophilia A using a national claims database.

Journal of managed care &amp; specialty pharmacy
2026

Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.

Blood advances
2026

Frequency of T Regulatory Cells Subpopulations in Hemophilia A Patients with Inhibitors.

Hamostaseologie
2026

A Case Report on Emergency Medicine: Suspected Acquired Hemophilia A in an Older Adult Male.

Cureus
2026

Augmented emicizumab-driven coagulation potential in hemophilia A state by in vitro and in vivo supplementation of combined factors IX and X.

Research and practice in thrombosis and haemostasis
2026

Direct oral anticoagulant interference and removal in the factor VIII inhibitor assay.

Research and practice in thrombosis and haemostasis
2026

Corrigendum to "Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study" [Thromb. Res. 253 (2025) 109402].

Thrombosis research
2026

Evidence-based dental management strategies for individuals with congenital hemophilia: a systematic review.

BMC oral health
2026

Investigation of the Suitability of the ROTEM Assay to Measure Coagulation Potential in Blood From Patients on Concizumab Prophylaxis.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Sleep and Haemophilia-A Case-Control Analysis of Associated Factors.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Lifetime Cost-Utility Analysis of Emicizumab Prophylaxis in Severe Haemophilia A Without Inhibitors in China: A Markov Model Analysis.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Nine areas with outstanding challenges for hemophilia B research.

Therapeutic advances in hematology
2026

Coexistence of two X-linked disorders: hemophilia A and G6PD deficiency.

Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis
2026

Complex Triple Deformity of the Knee Managed with Knee Arthrodesis in a Severe Hemophilia A Patient: A Case Report.

JBJS case connector
2026

Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII-lentiviral vector.

Blood vessels, thrombosis &amp; hemostasis
2025

Case Report: Intracranial hemophilic pseudotumor mimicking an aggressive neoplasm: a rare skull-invasive presentation.

Frontiers in surgery
2026

Extraarticular and Intraarticular Key Lesions in Knee Joints of Adult Persons with Hemophilia-A Case-Control Ultrasound Study.

Hamostaseologie
2026

Acquired Hemophilia A After Neoadjuvant Immunotherapy for Renal Cell Carcinoma.

Rhode Island medical journal (2013)
2026

Key Findings From the World Federation of Haemophilia National Member Organization Survey on Women and Girls With Bleeding Disorders.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

Transplanted gene-modified placental cells boost FVIII activity in pediatric sheep without eliciting immunity, toxicity, or adverse events.

Frontiers in immunology
2026

Redefining hemophilia management: treatment goals, nonfactor replacement therapies, and the role of fitusiran.

The American journal of managed care
2026

Feasibility and outcomes of switching factor VIII therapies: post-hoc analysis of patients transitioning from older plasma-derived and first-generation recombinant factor VIII to newer third-generation recombinant factor VIII replacement therapies within the Takeda factor VIII portfolio.

Expert review of hematology
2026

Platelet function abnormalities in acquired hemophilia A.

Research and practice in thrombosis and haemostasis
2026

The IL-33 receptor ST2 as a therapeutic target for hemophilic arthropathy.

Expert opinion on therapeutic targets
2026

On Factor VIII Assay Discrepancies in Post-infusion Samples Obtained from Patients Treated with Efanesoctocog Alfa.

Hamostaseologie
2026

Anti-emicizumab antibodies and their relevance in clinical practice.

Research and practice in thrombosis and haemostasis
2026

Joint tissue plasticity in hemophilia: insights from the Joint Activity and Damage Exam ultrasound protocol.

Research and practice in thrombosis and haemostasis
2026

Interfacility Collaboration for Hemophilia Care in Japan: A Retrospective Database Study Using a Japanese Healthcare Claims Database.

Health science reports
2026

Balancing Promise and Peril: Hemophilia Gene Therapy Insights.

IUBMB life
2026

Bone Health in Adults With Severe Haemophilia Receiving Different Prophylactic Treatments.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Evaluation of the Burden of Bone Fractures in People Living With Haemophilia: A Registry-Based Matched Cohort Study.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Bridging the Gap: Efficacy of Low-Dose Emicizumab in Haemophilia A With or Without Inhibitors.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

An Initial Diagnosis of Acquired Hemophilia A in an Elderly Male With a Neck Hematoma.

Cureus
2026

Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.

Research and practice in thrombosis and haemostasis
2026

Comparative safety of drug therapies used in hemophilia A and B in Canada: a multi-center, retrospective study.

Research and practice in thrombosis and haemostasis
2026

Emicizumab in Acquired Hemophilia A: A Real-World Case Series with Patient-Level Outcome Analysis.

Seminars in thrombosis and hemostasis
2026

Performance of Factor VIII Extended Half-Life Product Measurements in External Quality Assessment Programmes.

International journal of laboratory hematology
2026

Plain Language Summary on Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study.

European journal of haematology
2026

Reference Values for the Haemophilia Joint Health Score in Patients With Severe Haemophilia Derived From the Canadian Bleeding Disorder Registry.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

florio HAEMO-A Digital Medical Device for Monitoring of Treatment, Symptoms and Physical Activities for People Living With Haemophilia.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Impact of antiplatelet therapy on the hemostatic efficacy of platelet-targeted FVIII gene therapy in hemophilia A mice.

Blood advances
2026

Immunogenicity of Gene and Cell Therapies.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
2025

Therapeutic Antibodies in Hematology: Advances in Malignant and Non-Malignant Disorders.

Cells
2025

Engineering Liver-Specific Promoters: A Comprehensive Review of Design, Mechanisms, and Clinical Applications in Gene Therapy.

Cells
2026

Characteristics and posttransplant outcomes of patients with congenital and acquired von Willebrand disease and hemophilia A and with renal transplants.

Research and practice in thrombosis and haemostasis
2026

Characterization of a factor VIII/immunoglobulin heavy chain μ double-knockout mouse model of hemophilia A for long-term exposure to factor VIII proteins.

Research and practice in thrombosis and haemostasis
2026

Efficacy of radiosynoviorthesis on clinical outcomes in persons with haemophilia. A systematic review and meta-analysis.

Thrombosis research
2026

Noncarrier mothers of hemophilia A patients with Intron 22 inversions often have other rearrangements.

Journal of thrombosis and haemostasis : JTH
2026

Concizumab in hemophilia with inhibitors: longer-term efficacy and safety results from the phase 3 explorer7 study.

Blood advances
2026

Massive Intra-Abdominal Pseudotumor in a Patient With Hemophilia A Treated With Emicizumab: A Case Report.

Clinical case reports
2026

Remarkable Prolongation of Irreversible APTT and Non-Significant Hemorrhage: A Rare High Titer of FVIII Inhibitor Case Report and Literature Review.

Journal of clinical laboratory analysis
2026

Race, Ethnicity, and Hemophilia: A Scoping Review.

Journal of racial and ethnic health disparities
2026

Barriers to dental care and parental attitudes toward oral health in children with haemophilia: a cross-sectional study in China.

European archives of paediatric dentistry : official journal of the European Academy of Paediatric Dentistry
2026

mGem: AAV, from almost a virus to an awesome vector-or is it?

mBio
2025

Post-Infectious Acquired Hemophilia A: A Case Report and Review of the Literature.

Journal of Brown hospital medicine
2025

Season-dependent low basal CD86 expression promotes immune cell activation upon treatment with plasma-derived factor Ⅷ products.

Research and practice in thrombosis and haemostasis
2026

Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study.

European journal of haematology
2026

Real-World Diagnosis and Testing of Acquired Hemophilia A in Japan: A Web-Based Physician Survey.

Internal medicine (Tokyo, Japan)
2025

Repeated hemorrhagic ulcers of the esophagus associated with acquired hemophilia A: A case report.

World journal of gastrointestinal endoscopy
2026

Rebalancing Hemostasis: Fitusiran as a First-in-Class RNAi Therapy in Hemophilia A and B.

Health science reports
2025

A Deletion Variant of Human Factor VIII Displaying Low Immunogenicity in a Murine Model of Hemophilia A.

International journal of molecular sciences
2025

A Rare Case of Mild Hemophilia A in a Female with Mosaic Monosomy X and a De Novo F8 Variant.

International journal of molecular sciences
2026

Low-Load Quadriceps Strengthening With Blood Flow Restriction in Adults With Haemophilic Knee Arthropathy: A Randomised Controlled Trial.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Carotid Intima-Media Thickness as a Marker of Subclinical Atherosclerosis in Adolescents With Severe Haemophilia.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Five-Year Experience of Haemophilia Centre Certification Performed by the German, Austrian and Swiss Society for Thrombosis and Haemostasis Research.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

von Willebrand disease combined with other hemostasis disorders: an overlooked clinical entity.

Research and practice in thrombosis and haemostasis
2025

Fitusiran: A Novel Antithrombin-Targeting Therapy for Hemophilia A and B, With or Without Inhibitors.

The Annals of pharmacotherapy
2026

Overview of gene therapy for hemophilia: questions and answers to navigate the innovation.

Journal of thrombosis and haemostasis : JTH
2025

Recurrence of FVIII Inhibitor during Surgery in a Patient with Severe Hemophilia A Receiving Emicizumab Prophylaxis.

Acta medica Okayama
2025

Successful hemodialysis initiation in acquired hemophilia a managed with susoctocog alfa and emicizumab.

CEN case reports
2025

High Prevalence of Heterotopic Ossification in Mild Hemophilia: A Biological Inevitability or a Prophylactic Blind Spot?

Journal of ultrasound in medicine : official journal of the American Institute of Ultrasound in Medicine
2026

Inhibition of CIRBP as a Novel Strategy to Alleviate Chondrocyte Ferroptosis in Haemophilic Arthropathy.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Approaches to Identify People With Haemophilia With Medicare Claims Data.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

Safe Outpatient Dental Management of a Pediatric Patient With Mild Hemophilia A: A Case Report.

Cureus
2025

Mixing Test in a Case of Combined Acquired Hemophilia A and Coagulation Factor Deficiency: A Case Report.

Cureus
2025

Laparoscopic Cholecystectomy for Acute Cholecystitis in a Patient with Acquired Hemophilia A: A Case Report of Perioperative Hemostatic Management with Plasma Exchange.

Surgical case reports
2025

Acquired Hemophilia A in Sjögren's Syndrome: An Uncommon Bleeding Disorder Masked by Antiplatelet Use.

Annals of African medicine
2026

Current gynaecological management of women and girls with bleeding disorders in the United Kingdom: A UKHCDO haemophilia treatment centre survey and evaluation of real-world clinical practice for the British Journal of Haematology.

British journal of haematology
2025

Orthopedic manifestations of acquired hemophilia: a case report on uncontrolled bleeding and progressive swelling post-phlebotomy- should fasciotomy be considered?

BMC musculoskeletal disorders
2026

Hemophilic pseudotumor in the hand: a case report and literature review.

Skeletal radiology
2025

[How I treat acquired clotting factor deficiency].

Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi
2025

Defining the critical roles of factor VIII residues 1900 to 1908 in factor VIIIa-factor IXa complex formation and factor VIIIa activity.

Journal of thrombosis and haemostasis : JTH
2026

Acceptability and Determinants for Implementation of the EnzySystem, a Novel Near-Patient Testing Platform for Haemophilia A.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Integration of Efanesoctocog Alfa in Clinical Practice for Children, Adolescents, and Young Adults With Severe Haemophilia A.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

T cells from individuals with and without hemophilia A respond to the same epitopes in factor VIII.

Journal of thrombosis and haemostasis : JTH
2026

Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

Vildagliptin-Associated Acquired Hemophilia A.

The Annals of pharmacotherapy
2025

Real World Experience with use of Coagulation Factor VIIa at an Academic Medical Center.

Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
2025

Eight-Week Resistance Training and Manual Therapy in Young Patients with Severe Hemophilia: A Case Series Evaluating Functional, Imaging, and Immunological Outcomes.

Journal of clinical medicine
2025

Evaluation of kidney function and damage in patients with hemophilia B-insights from the B-Natural study.

Research and practice in thrombosis and haemostasis
2026

mRNA based in vivo MG1113 antibody production as a potential therapeutic approach for hemophilia.

Journal of controlled release : official journal of the Controlled Release Society
2025

Acquired Hemophilia A Following Omalizumab Treatment in a Patient With Chronic Spontaneous Urticaria.

Journal of investigational allergology &amp; clinical immunology
2025

AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis.

Research and practice in thrombosis and haemostasis
2025

Osteonecrosis of Femoral Head with Antiphospholipid Syndrome and Acquired Subclinical Hemophilia A.

Clinical laboratory
2025

Heterogeneity of active mast cells, endothelial cells, and fibroblasts in hemophilic arthritis defined by synovial single-cell sequencing.

Scientific reports
2026

Dental Neglect by Individuals With Haemophilia: Clinical Challenges and Solutions.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice Experiment.

Haemophilia : the official journal of the World Federation of Hemophilia
2026

Haemophilic Elbow Arthropathy: Mechanisms, Management and Future Perspectives.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

Recurrent hemoptysis after laryngectomy-acquired hemophilia induced by laryngeal cancer surgery and chemoradiotherapy: A case report.

World journal of clinical cases
2025

[A cohort study on cardiovascular diseases in adults with hemophilia: an interim subanalysis on comorbidities and medication from ADVANCE Japan].

[Rinsho ketsueki] The Japanese journal of clinical hematology
2026

Efficacy and safety of marstacimab prophylaxis in hemophilia A/B with inhibitors: results from the phase 3 BASIS trial.

Blood
2026

Optimized production and coagulation activity of rhFVII in HEK293 cells for bleeding disorders.

Protein expression and purification
2025

The aging hemophilia patient.

Hematology. American Society of Hematology. Education Program
2025

New therapies in hemophilia: extend the half-life, mimic, or rebalance?

Hematology. American Society of Hematology. Education Program
2025

Deconstructing gene therapy in hemophilia for the clinician.

Hematology. American Society of Hematology. Education Program
2025

Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care.

Clinical and translational science
2026

Impact of Inhibitor Development on the Cost Effectiveness of Prophylactic Treatment with Recombinant Factor VIII in Previously Untreated Patients with Severe Hemophilia A.

PharmacoEconomics - open
2025

Continuum of Care for Hemophilia: The Story of India.

Indian journal of pediatrics
2025

Case Report of acupotomy release combined with manual release under anesthesia for adhesions after unilateral total knee arthroplasty in a patient with hemophilia A.

Frontiers in surgery
2025

Critical limb ischemia leading to amputation as a presentation of acquired hemophilia A with congenital factor XII deficiency: a case report.

International journal of surgery case reports
2025

Selective embolization for recurrent gingiva bleeding associated with aneurysmal bone cyst and hemophilia: A case report.

International journal of surgery case reports
2025

Robot-assisted total hip arthroplasty following gene therapy for hemophilia a arthropathy: a case report.

Arthroplasty (London, England)
2025

Author Correction: Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs.

Experimental &amp; molecular medicine
2026

Health-Related Quality of Life in Egyptian Children With Haemophilia A and Chronic Arthropathy With the Era of Emicizumab Therapy: A Single-Centre Cohort Study.

Haemophilia : the official journal of the World Federation of Hemophilia
2025

Posterior fossa extradural haematoma with cerebral venous sinus thrombosis precipitates haemophilia a diagnosis: a paediatric case report and literature review.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2025

NXT007 enhances in vitro coagulation potential in the coexistence of emicizumab in hemophilia A through distinct complex formation.

Research and practice in thrombosis and haemostasis
2025

Emicizumab, the factor VIII mimetic bispecific monoclonal antibody: effects on thrombin generation and thromboelastometry.

Research and practice in thrombosis and haemostasis
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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. FIXa-triggered thrombin generation correlates with FVIII levels less than 1% in reconstituted plasma mimicking samples obtained from patients with severe hemophilia A.
    Blood coagulation &amp; fibrinolysis : an international journal in haemostasis and thrombosis· 2026· PMID 41846404mais citado
  2. Deleterious NKAP Mutations Are Associated with Musculoskeletal Abnormalities in Hemizygous Males and Skewed X Chromosome Inactivation in Heterozygous Females.
    International journal of molecular sciences· 2026· PMID 41828556mais citado
  3. Genotype-Specific Postural Control Deficits in Hemophilia A: Insights from Center of Pressure Analysis Beyond Radiographic Arthropathy.
    International journal of molecular sciences· 2026· PMID 41828546mais citado
  4. Monochorionic diamniotic twin brothers with severe hemophilia A: a case report.
    Frontiers in pediatrics· 2026· PMID 41809524mais citado
  5. A combinatorial CAAR-T cell strategy to eliminate factor VIII inhibitors in preclinical models of hemophilia A.
    iScience· 2026· PMID 41797925mais citado
  6. Acquired Hemophilia A During Prasugrel Therapy After Recurrent Acute Coronary Syndrome.
    JACC Case Rep· 2026· PMID 41989370recente
  7. FVIIIa Mimetics: New Approaches and Next-Generation Initiatives.
    Haemophilia· 2026· PMID 41988951recente
  8. A Jehovah's Witness with Acquired Hemophilia A Who Successfully Avoided Blood Transfusions by Receiving Emicizumab.
    Transfus Med Hemother· 2026· PMID 41982273recente
  9. Severe Hemophilia A and Congenital Antithrombin Deficiency: A Case of Natural Hemostatic Rebalancing.
    Haemophilia· 2026· PMID 41982006recente
  10. The Prevalence of Coagulation Factor Deficiency Among Pediatric Populations in Medina City, Saudi Arabia.
    Clin Lab· 2026· PMID 41979610recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:98878(Orphanet)
  2. OMIM OMIM:134500(OMIM)
  3. MONDO:0010602(MONDO)
  4. Hemofilia Hereditaria(PCDT · Ministério da Saúde)
  5. GARD:6591(GARD (NIH))
  6. Variantes catalogadas(ClinVar)
  7. Busca completa no PubMed(PubMed)
  8. Artigo Wikipedia(Wikipedia)
  9. Q2092064(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

Hemofilia A
Compêndio · Raras BR

Hemofilia A

ORPHA:98878 · MONDO:0010602
🇧🇷 Brasil SUS
CEAF
1AFator VIII recombinanteEmicizumabeEfmoroctocogue alfaEfanesoctocogue alfa+3 mais
Internações
6.780/ano
Prevalência BR
1:5000 (homens)
Custo SUS
R$ 12.450/internação
Dados
DATASUS 2024
Geral
Prevalência
1-9 / 100 000
Herança
X-linked recessive
CID-10
D66 · Deficiência hereditária do fator VIII
CID-11
Ensaios
35 ativos
Medicamentos
10 registrados
Início
Childhood, Infancy, Neonatal
Prevalência
4.85 (Worldwide)
MedGen
UMLS
C0019069
Repurposing
1 candidato
tranexamic-acidantifibrinolytic|plasminogen activator inhibitor
Wikidata
Wikipedia
Papers 10a
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