A deficiência combinada de fator V e fator VIII é um distúrbio hemorrágico hereditário devido à redução na atividade e nos níveis de antígeno do fator V (FV) e do fator VIII (FVIII) e caracterizado por sintomas hemorrágicos leves a moderados.
Introdução
O que você precisa saber de cara
A deficiência combinada de fator V e fator VIII é uma doença genética e rara que causa tendência a sangramentos por diminuir a atividade de duas proteínas essenciais da coagulação do sangue. Quem vive com essa condição pode notar hematomas frequentes pelo corpo, sangramentos no nariz ou nas gengivas e sangramentos prolongados após cirurgias ou extrações dentárias. O diagnóstico é feito por meio de exames laboratoriais de coagulação e pode ser confirmado por testes genéticos. No Brasil, a doença ainda não conta com um protocolo clínico (PCDT) próprio publicado pelo Ministério da Saúde nem com medicamentos específicos aprovados no catálogo.
A deficiência combinada de fator V e fator VIII é um distúrbio hemorrágico hereditário devido à redução na atividade e nos níveis de antígeno do fator V (FV) e do fator VIII (FVIII) e caracterizado por sintomas hemorrágicos leves a moderados.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 6 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 20 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
2 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive.
Factor V and factor VIII combined deficiency 1
A blood coagulation disorder characterized by bleeding symptoms similar to those in hemophilia or parahemophilia, that are caused by single deficiency of FV or FVIII, respectively. The most common symptoms are epistaxis, menorrhagia, and excessive bleeding during or after trauma. Plasma levels of coagulation factors V and VIII are in the range of 5 to 30% of normal.
Factor V and factor VIII combined deficiency 2
A blood coagulation disorder characterized by bleeding symptoms similar to those in hemophilia or parahemophilia, that are caused by single deficiency of FV or FVIII, respectively. The most common symptoms are epistaxis, menorrhagia, and excessive bleeding during or after trauma. Plasma levels of coagulation factors V and VIII are in the range of 5 to 30% of normal.
Variantes genéticas (ClinVar)
119 variantes patogênicas registradas no ClinVar.
Vias biológicas (Reactome)
9 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Deficiência combinada de fator V e fator VIII
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Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.
Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Pesquisa e ensaios clínicos
Nenhum ensaio clínico registrado para esta condição.
Publicações mais relevantes
Combined deficiency of factor V and factor VIII in a pediatric patient: a case report.
Combined deficiency of factors V and VIII is a rare autosomal recessive disorder associated with an increased risk of bleeding. We present an unusual case of a 7-year-old Moroccan child with no history of consanguinity who was hospitalized owing to a hemorrhagic episode during circumcision. The 7-year-old patient, a Moroccan boy from North Africa, coming from a family of five siblings, was referred for an evaluation of prolonged activated partial thromboplastin time and prothrombin time. Coagulation factor assays revealed a combined deficiency of factors V and VIII, with normal levels of other coagulation factors. This anomaly was detected in the hematology laboratory, where hemostasis tests were performed via optical methods on the Acl Top 750 analyzer. A complete blood count was conducted on the Beckman Coulter DXH 900 analyzer. Hemostasis assessments revealed an elevated activated partial thromboplastin time at 73.2 s (normal range < 36), with a patient-to-control activated partial thromboplastin time ratio of 2.58 (normal ratio < 1.2), a low prothrombin time at 18.35 s (normal prothrombin time range: 11.4-13.5), and an international normalized ratio of 1.59 (normal range: 2-3.5). Specific coagulation factor assays demonstrated a combined deficiency of factors V and VIII at 12.4% (normal range: 55-150) and 9.1% (normal range: 50-145), respectively, whereas other coagulation factor levels remained within the normal range, including the antigenic activity of von Willebrand at 71.7% (normal range: 50-150). The complete blood count showed no abnormalities, except for a small thrombocytosis. The child was managed in the pediatric hematology department, and a family investigation among the remaining siblings was initiated to search for similar cases. Our study highlights a rare and often underdiagnosed genetic disorder that is often confused with a diagnosis of minor hemophilia A or congenital factor V deficiency. Differential diagnosis is crucial, particularly for von Willebrand disease. Combined deficiency of factors V and VIII should be suspected in patients with a suggestive clinical and laboratory profile, including prolonged prothrombin time and activated partial thromboplastin time along with a deficiency in coagulation factor V. Therefore, measuring factor VIII levels is highly recommended.
[Combined deficiency of clotting factor V and factor VIII: about three siblings].
Combined deficiency of clotting factor V and factor VIII (DF5F8) is a congenital autosomal recessive disorder. This study involved a family of four children born to consanguineous parents. The eldest daughter was referred for assessment of activated partial thromboplastin time and prothrombin time associated with hemorrhagic manifestations. Coagulation factor dosing showed combined deficiency of factor V and factor VIII as well as normal levels of other coagulation factors. DF5F8 was detected in two girls and a boy. Two protein coding genes LMAN1 (lectin, mannose binding 1) and MCFD2 (multiple coagulation factor deficiency2) were involved in the intracellular passage of Factor V and Factor VIII, including some mutations which caused deficiency of Factor V and VIII. The diagnosis of DF5F8 is routinely possible, especially in patients born to consanguineous parents with a suggestive clinico-biological condition. Le déficit combiné en facteurs V et VIII de la coagulation (DF5F8) est un désordre constitutionnel de transmission autosomique récessif. C´est une famille de quatre enfants, issus de consanguinité. La fille aînée adressée pour exploration d´allongement du Temps de Céphaline avec activateur et du Temps de Quick, associé à des manifestations hémorragiques. Le dosage des facteurs de coagulation montre un déficit combiné en facteurs V et VIII, et taux normaux des autres facteurs de coagulation. On trouve un DF5F8 chez deux filles et un garçon. Deux gènes codent pour protéines LMAN1 (Lectin MANnose-Binding1) et MCFD2 (Multiple Coagulation factor deficiency2), sont impliquées dans le passage intracellulaire des FV et VIII, dont certaines mutations provoquent un déficit combiné en facteur V et VIII. Diagnostic du DF5F8 est possible en routine surtout chez des patients issus de consanguinité avec un contexte clinico-biologique évocateur.
Low factor V level ameliorates bleeding diathesis in patients with combined deficiency of factor V and factor VIII.
Combined factor V (FV) and FVIII deficiency (F5F8D) is a rare autosomal-recessive bleeding disorder caused by mutations in lectin mannose binding-1 (LMAN1) and multiple coagulation factor deficiency-2 (MCFD2). Six causative homozygous mutations (5 in LMAN1 and 1 in MCFD2) were identified in 6 patients with F5F8D. A thrombin-generation assay, triggered with tissue factor (1 pM) in F5F8D plasma, paradoxically exhibited enhanced thrombin generation compared with normal plasma. Significantly lower free tissue factor pathway inhibitor (fTFPI) was found in F5F8D patients compared with healthy controls (P < .01). Normalizing tissue factor pathway inhibitor α (TFPIα) in F5F8D plasma greatly delayed and reduced thrombin generation. Increasing FV concentrations by adding plasma FV to F5F8D plasma only caused a gradual decrease in thrombin generation, suggesting that low levels of TFPIα and FV cocontributed to the elevated thrombin generation by reducing anticoagulant effects. On the contrary, thrombin generation in F5F8D platelet-rich plasma (PRP) was significantly lower than in normal controls (P < .05); however, it was fully corrected by normalizing FVIII or after 1-deamino-8-d-arginine vasopressin (DDAVP) infusion, indicating that the hypocoagulable state of F5F8D patients is associated with low FVIII levels. In addition, plasma and platelet FV in F5F8D PRP were sufficient to support normal thrombin generation, and low TFPIα may have no effect on thrombin generation. DDAVP infusion induced a complete response in 5 F5F8D patients and a partial response in the remaining patient. Based on our findings, we suggest that DDAVP may be considered a potential substitute for FVIII concentrates, and fresh-frozen plasma (FFP) infusion may not be necessary for F5F8D patients with minor bleeding challenges.
Combined deficiency of factor V and factor VIII: management during cardiothoracic surgery.
: Combined factor V and factor VIII deficiency is an extremely rare bleeding disorder for which research is lacking. We present the case of a 33-year-old man requiring repeat mitral valve replacement. A multidisciplinary team approach was utilized to minimize his risk of bleeding which included the use of plasma exchange, intravenous factor replacement, and platelet transfusion. This approach created an operative experience that did not require blood transfusion or the use of other hemostatic medications.
Publicações recentes
Combined deficiency of factor V and factor VIII in a pediatric patient: a case report.
[Combined deficiency of clotting factor V and factor VIII: about three siblings].
Low factor V level ameliorates bleeding diathesis in patients with combined deficiency of factor V and factor VIII.
Combined deficiency of factor V and factor VIII: management during cardiothoracic surgery.
Successful Pregnancy in a Patient with Combined Deficiency of Factor V and Factor VIII.
📚 EuropePMC22 artigos no totalmostrando 4
Combined deficiency of factor V and factor VIII in a pediatric patient: a case report.
Journal of medical case reports[Combined deficiency of clotting factor V and factor VIII: about three siblings].
The Pan African medical journalLow factor V level ameliorates bleeding diathesis in patients with combined deficiency of factor V and factor VIII.
BloodCombined deficiency of factor V and factor VIII: management during cardiothoracic surgery.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosisAssociações
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Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Deficiência combinada de fator V e fator VIII e no aconselhamento genético da família.
Doenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
Perguntas frequentes
O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu
A condição é causada por alterações genéticas com padrão de herança autossômica recessiva. As mutações ocorrem nos genes LMAN1 ou MCFD2, afetando o transporte intracelular e reduzindo a atividade dos fatores V e VIII no sangue.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
5 publicações do grafo RarasNet (PubMed) · 6 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.
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.
Citar este verbete
Raras. (s.d.). Deficiência combinada de fator V e fator VIII. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/deficiencia-combinada-de-fator-v-e-fator-viii
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
Deficiência combinada de fator V e fator VIII
📋 Origem dos dados
Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.
- Doença rara (ontologia)
- fonte: Orphanet
- Identificador unificado
- fonte: MONDO
- Codificação WHO/SUS
- fonte: WHO ICD-10 / DATASUS
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata