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[PMID]:29257899
[Au] Autor:Castaman G; Linari S
[Ad] Endereço:a Center for Bleeding Disorders, Department of Oncology , Careggi University Hospital , Florence , Italy.
[Ti] Título:Pharmacokinetic drug evaluation of recombinant factor VIII for the treatment of hemophilia A.
[So] Source:Expert Opin Drug Metab Toxicol;14(2):143-151, 2018 Feb.
[Is] ISSN:1744-7607
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:INTRODUCTION: The prevention of bleeding by prophylactic factor replacement is the recommended approach for the treatment of severe hemophilia. Prophylaxis should be individualized to provide the best clinical benefit to each patient. Therefore, a pharmacokinetic approach is crucial. Areas covered: This review aims to concisely describe the basic principles of pharmacokinetics of FVIII, the role of population pharmacokinetic, the available different recombinant FVIII concentrates and the new extended half-life FVIII molecules with possible improvement in hemophilia A treatment. Expert opinion: Pharmacokinetic is a useful tool to predict the outcome of replacement therapy, even though a large inter-individual variability exists, becauseof several factors: age, weight, von Willebrand factor level, blood group, active bleed, presence of inhibitors to FVIII, FVIII concentrate. Among the different recombinant FVIII concentrates pharmacokinetic differences are minor and clinically not significant. The extended half-life FVIII products brings only moderate advances, as half life extension is limited to 1.5-1.8-fold in comparison to that of native FVIII. Thus, infusions could be done every fourth, rarely fifth day to ensure a safe through level and a significant benefit can be offered only to patients treated every other day or three times weekly.
[Mh] Termos MeSH primário: Coagulantes/administração & dosagem
Fator VIII/administração & dosagem
Hemofilia A/tratamento farmacológico
[Mh] Termos MeSH secundário: Animais
Coagulantes/farmacocinética
Esquema de Medicação
Fator VIII/farmacocinética
Meia-Vida
Seres Humanos
Medicina de Precisão/métodos
Proteínas Recombinantes
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
0 (Coagulants); 0 (Recombinant Proteins); 9001-27-8 (Factor VIII)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180222
[Lr] Data última revisão:
180222
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:171220
[St] Status:MEDLINE
[do] DOI:10.1080/17425255.2018.1420161


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[PMID]:29270992
[Au] Autor:Ljung RCR
[Ad] Endereço:Department of Clinical Sciences - Paediatrics, Lund University, Lund, Sweden.
[Ti] Título:How I manage patients with inherited haemophilia A and B and factor inhibitors.
[So] Source:Br J Haematol;180(4):501-510, 2018 02.
[Is] ISSN:1365-2141
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:Development of inhibitors to coagulation factor VIII or IX is still the most challenging complication in haemophilia care. 'Bypassing agents' may be used to treat a bleed but the eradication of the inhibitor by immune tolerance induction (ITI) is the main objective in the treatment of a patient with haemophilia who has developed neutralizing antibodies. Several options exist for ITI and the patient may be at 'good' or 'bad risk' for successful outcome with different regimens. This paper offers a review of current regimens to be considered in the treatment of a bleed in a patient with an inhibitor but the main focus is the aspects of different choices in the management of the child or the adult with severe or mild forms of haemophilia A or B, who has developed an inhibitor. There are also some final outlooks on new and emerging treatment possibilities.
[Mh] Termos MeSH primário: Inibidores dos Fatores de Coagulação Sanguínea
Hemofilia A/diagnóstico
Hemofilia A/terapia
Hemofilia B/diagnóstico
Hemofilia B/terapia
Isoanticorpos
[Mh] Termos MeSH secundário: Adulto
Fatores Etários
Inibidores dos Fatores de Coagulação Sanguínea/sangue
Inibidores dos Fatores de Coagulação Sanguínea/imunologia
Criança
Gerenciamento Clínico
Fator IX/genética
Fator IX/imunologia
Fator IX/uso terapêutico
Fator VIII/genética
Fator VIII/imunologia
Fator VIII/uso terapêutico
Hemofilia A/complicações
Hemofilia A/genética
Hemofilia B/complicações
Hemofilia B/genética
Hemorragia/etiologia
Hemorragia/prevenção & controle
Hemorragia/terapia
Seres Humanos
Isoanticorpos/sangue
Isoanticorpos/imunologia
Medição de Risco
Índice de Gravidade de Doença
[Pt] Tipo de publicação:JOURNAL ARTICLE; RESEARCH SUPPORT, NON-U.S. GOV'T; REVIEW
[Nm] Nome de substância:
0 (Blood Coagulation Factor Inhibitors); 0 (Isoantibodies); 9001-27-8 (Factor VIII); 9001-28-9 (Factor IX)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180221
[Lr] Data última revisão:
180221
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:171223
[St] Status:MEDLINE
[do] DOI:10.1111/bjh.15053


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[PMID]:29320623
[Au] Autor:Kulic A; Cvetkovic Z; Libek V
[Ti] Título:Primary hyperfibrinolysis as the presenting sign of prostate cancer: A case report.
[So] Source:Vojnosanit Pregl;73(9):877-80, 2016 Sep.
[Is] ISSN:0042-8450
[Cp] País de publicação:Serbia
[La] Idioma:eng
[Ab] Resumo:Introduction: A bleeding syndrome in the setting of primary hyperfibrinolysis in a prostate cancer patient is only 0.40­ 1.65% of cases. The laboratory diagnosis of primary hyperfibrinolysis is based on the increase of biomarkers like D-dimer, fibrinogen split products, plasminogen, and euglobulin lysis test. These tests are not specific for primary hyperfibrinolysis. We reported a rare case of hemorrhagic syndrome caused by primary hyperfibrinolysis as the first clinical symptom of metastatic prostate cancer. Case report: A 64-year-old male was admitted to our hospital with large hematomas in the right pectoral and axillary areas (20 x 7 cm), right hemiabdomen (30 x 30 cm) and the left lumbal area, (25 x 5 cm). The patient had no subjective symptoms nor used any medication. Initial coagulation testing, prothrombin time (PT), and activated partial thromboplastin time (APTT) were within the normal range, while fibrinogen level was extremely low (1.068 g/L) (normal range 2.0­5.0) and the D-dimer assay result was high 1.122 mg/L (normal range < 0.23). The results obtained by rotation thrombelastometry pointed to primary fibrinolysis. Further clinical and laboratory examination indicated progressive malignant prostate disease. First line treatment for the patient was a combined administration of tranexamic acid (3 x 500 mg iv) and transfusion of ten units of cryoprecipitate (400 mL). Next day, fibrinolytic function measurements by rotation thrombelastometry were within the normal ranges. Fibrinogen level was normalized within two days (2.4 g/L). There were no newly developed hematomas. Conclusion: This case report shows primary hyperfibrinolysis with bleeding symptoms, which is an uncommon paraneoplastic phenomenon within expanded prostate malignancy. Rotation thrombelastometry in this severe complication helped to achieve the prompt and proper diagnosis and treatment.
[Mh] Termos MeSH primário: Adenocarcinoma/complicações
Transtornos da Coagulação Sanguínea/etiologia
Fibrinólise
Síndromes Paraneoplásicas/etiologia
Neoplasias da Próstata/complicações
[Mh] Termos MeSH secundário: Adenocarcinoma/sangue
Adenocarcinoma/secundário
Adenocarcinoma/terapia
Antifibrinolíticos/uso terapêutico
Biomarcadores/sangue
Transtornos da Coagulação Sanguínea/sangue
Transtornos da Coagulação Sanguínea/diagnóstico
Transtornos da Coagulação Sanguínea/tratamento farmacológico
Testes de Coagulação Sanguínea
Neoplasias Ósseas/secundário
Fator VIII/uso terapêutico
Fibrinogênio/uso terapêutico
Fibrinólise/efeitos dos fármacos
Hematoma/etiologia
Hemorragia/etiologia
Seres Humanos
Masculino
Meia-Idade
Síndromes Paraneoplásicas/sangue
Síndromes Paraneoplásicas/diagnóstico
Síndromes Paraneoplásicas/tratamento farmacológico
Neoplasias da Próstata/sangue
Neoplasias da Próstata/patologia
Neoplasias da Próstata/terapia
Ácido Tranexâmico/uso terapêutico
Resultado do Tratamento
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Antifibrinolytic Agents); 0 (Biomarkers); 0 (cryoprecipitate coagulum); 6T84R30KC1 (Tranexamic Acid); 9001-27-8 (Factor VIII); 9001-32-5 (Fibrinogen)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180213
[Lr] Data última revisão:
180213
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:180111
[St] Status:MEDLINE
[do] DOI:10.2298/VSP150525076K


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[PMID]:29384900
[Au] Autor:Zupancic-Salek S; Vodanovic M; Pulanic D; Skoric B; Matytsina I; Klovaite J
[Ad] Endereço:Unit for Haemostasis, Thrombosis and Benign Diseases of Haematopoietic System, Division of Hematology, Department of Internal Medicine, University Hospital Centre Zagreb.
[Ti] Título:A case report of acute inferior myocardial infarction in a patient with severe hemophilia A after recombinant factor VIII infusion.
[So] Source:Medicine (Baltimore);96(52):e9075, 2017 Dec.
[Is] ISSN:1536-5964
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:RATIONALE: The extent of protective effects of hemophilia against thrombotic events such as myocardial infarction (MI) and other acute coronary syndromes remains to be determined, as major risk factors for cardiovascular disease exist despite factor VIII (FVIII) deficiency. We present a case report of a 41-year-old male with severe hemophilia A and several cardiovascular risk factors. PATIENT CONCERNS: This morbidly obese patient developed chest pressure, followed by chest pain and difficulty in breathing shortly after receiving on-demand treatment with intravenous recombinant FVIII (rFVIII) (turoctocog alfa) dosed per body weight. DIAGNOSES: An electrocardiogram revealed a diagnosis of inferior ST-segment elevation MI. INTERVENTIONS: The patient underwent an urgent coronary angiography using a radial artery approach. During the next 12 months, he received dual antiplatelet treatment, acetylsalicylic acid 100 mg, and clopidogrel 75 mg daily. His treatment for severe hemophilia A was changed to plasma-derived FVIII replacement therapy. OUTCOMES: During this 12-month period, he experienced several small bleeds in his elbows. CONCLUSIONS: The temporal relationship between rFVIII infusion and onset of the MI suggests a possible association; however, apart from obesity, the patient also had other major risk factors for arterial thrombosis, such as hypertension and smoking. Furthermore, atherosclerotic disease and underlying atherosclerotic changes could not be excluded with certainty. This case highlights the importance of studies assessing the impact of excess body weight on rFVIII dosing.
[Mh] Termos MeSH primário: Fator VIII/uso terapêutico
Hemofilia A/complicações
Hemofilia A/tratamento farmacológico
Infarto Miocárdico de Parede Inferior/diagnóstico
Infarto Miocárdico de Parede Inferior/etiologia
[Mh] Termos MeSH secundário: Adulto
Seres Humanos
Masculino
Obesidade Mórbida/complicações
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (recombinant factor VIII N8); 9001-27-8 (Factor VIII)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180209
[Lr] Data última revisão:
180209
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:180201
[St] Status:MEDLINE
[do] DOI:10.1097/MD.0000000000009075


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[PMID]:29381944
[Au] Autor:Bastida JM; Cano-Mozo MT; Lopez-Cadenas F; Vallejo VE; Merchán S; Santos-Montón C; González-Calle D; Carrillo J; Martín AA; Torres-Hernández JA; González M; Martín-Herrero F; Pabón P; González-Porras JR
[Ad] Endereço:Department of Hematology, Hospital Universitario de Salamanca-IBSAL, Salamanca.
[Ti] Título:Hemorrhagic pericardial effusion as the debut of acquired hemophilia in a chronic lymphocytic leukemia patient: A case report, and a review of acquired hemophilia A-related hematological malignancies.
[So] Source:Medicine (Baltimore);96(47):e8669, 2017 Nov.
[Is] ISSN:1536-5964
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:BACKGROUND: Acquired hemophilia A (AHA) is a rare bleeding disease caused by autoantibodies against factor VIII. Spontaneous bleeding symptoms usually affect the skin and muscle, while pericardial effusion is an extremely rare manifestation. In the elderly, anticoagulant treatment is frequent and bleeding symptoms are usually associated with this. CLINICAL FINDINGS: We report a hemorrhagic pericardial effusion as the AHA debut in a patient with untreated chronic lymphocytic leukemia and anticoagulated with apixaban for atrial fibrillation and chronic arterial ischemia. The patient was treated with recombinant activated factor VII to control the active bleeding and corticosteroids and cyclophosphamide to eradicate the inhibitor. In addition, a briefly review of hematological malignancies associated to acquired hemophilia was performed. PARTICULARITIES:: a) anticoagulant treatment may confuse the suspicion of AHA and its diagnosis; b) hemorrhagic pericardial effusion is an extremely rare presentation; c) bypassing agents raise the risk of thromboembolism; d) hematological malignancies rarely cause AHA (<20% of cases). CONCLUSION: A multidisciplinary team is needed to diagnose and manage AHA effectively. The use of anticoagulants may lead to the misdiagnosis of clinical symptoms. Chronic lymphocytic leukemia is one of the main causes of hematological malignancies associated. The specific treatment of CLL is still recommended in the event of active disease.
[Mh] Termos MeSH primário: Fator VIII
Fator VIIa/administração & dosagem
Hemofilia A
Leucemia Linfocítica Crônica de Células B
Derrame Pericárdico
Pericardiectomia/métodos
[Mh] Termos MeSH secundário: Idoso
Anticorpos/sangue
Testes de Coagulação Sanguínea/métodos
Coagulantes/administração & dosagem
Ciclofosfamida/administração & dosagem
Ecocardiografia/métodos
Fator VIII/análise
Fator VIII/imunologia
Hemofilia A/sangue
Hemofilia A/complicações
Hemofilia A/etiologia
Seres Humanos
Imunossupressores/administração & dosagem
Leucemia Linfocítica Crônica de Células B/complicações
Leucemia Linfocítica Crônica de Células B/diagnóstico
Masculino
Derrame Pericárdico/diagnóstico
Derrame Pericárdico/etiologia
Derrame Pericárdico/fisiopatologia
Prednisona/administração & dosagem
Radiografia Torácica/métodos
Proteínas Recombinantes/administração & dosagem
Resultado do Tratamento
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Antibodies); 0 (Coagulants); 0 (Immunosuppressive Agents); 0 (Recombinant Proteins); 8N3DW7272P (Cyclophosphamide); 9001-27-8 (Factor VIII); AC71R787OV (recombinant FVIIa); EC 3.4.21.21 (Factor VIIa); VB0R961HZT (Prednisone)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180208
[Lr] Data última revisão:
180208
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:180201
[St] Status:MEDLINE
[do] DOI:10.1097/MD.0000000000008669


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[PMID]:29224506
[Au] Autor:Rangarajan S; Walsh L; Lester W; Perry D; Madan B; Laffan M; Yu H; Vettermann C; Pierce GF; Wong WY; Pasi KJ
[Ad] Endereço:From Hampshire Hospitals NHS Foundation Trust, Basingstoke (S.R.), University Hospitals Birmingham NHS Foundation Trust, Edgbaston (W.L.), Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Hospital, Cambridge (D.P.), and the Centre for Haemostasis and Thrombosis, St. Thomas' Hospital
[Ti] Título:AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.
[So] Source:N Engl J Med;377(26):2519-2530, 2017 12 28.
[Is] ISSN:1533-4406
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia B, the large size of the factor VIII coding region has precluded improved outcomes with gene therapy in patients with hemophilia A. METHODS: We infused a single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding a B-domain-deleted human factor VIII (AAV5-hFVIII-SQ) in nine men with severe hemophilia A. Participants were enrolled sequentially into one of three dose cohorts (low dose [one participant], intermediate dose [one participant], and high dose [seven participants]) and were followed through 52 weeks. RESULTS: Factor VIII activity levels remained at 3 IU or less per deciliter in the recipients of the low or intermediate dose. In the high-dose cohort, the factor VIII activity level was more than 5 IU per deciliter between weeks 2 and 9 after gene transfer in all seven participants, and the level in six participants increased to a normal value (>50 IU per deciliter) that was maintained at 1 year after receipt of the dose. In the high-dose cohort, the median annualized bleeding rate among participants who had previously received prophylactic therapy decreased from 16 events before the study to 1 event after gene transfer, and factor VIII use for participant-reported bleeding ceased in all the participants in this cohort by week 22. The primary adverse event was an elevation in the serum alanine aminotransferase level to 1.5 times the upper limit of the normal range or less. Progression of preexisting chronic arthropathy in one participant was the only serious adverse event. No neutralizing antibodies to factor VIII were detected. CONCLUSIONS: The infusion of AAV5-hFVIII-SQ was associated with the sustained normalization of factor VIII activity level over a period of 1 year in six of seven participants who received a high dose, with stabilization of hemostasis and a profound reduction in factor VIII use in all seven participants. In this small study, no safety events were noted, but no safety conclusions can be drawn. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov number, NCT02576795 ; EudraCT number, 2014-003880-38 .).
[Mh] Termos MeSH primário: Dependovirus
Fator VIII/genética
Terapia Genética
Vetores Genéticos
Hemofilia A/terapia
[Mh] Termos MeSH secundário: Adulto
Anticorpos Antivirais/sangue
DNA Viral
Dependovirus/imunologia
Fator VIII/administração & dosagem
Fator VIII/metabolismo
Terapia Genética/efeitos adversos
Terapia Genética/métodos
Hemofilia A/genética
Hemofilia A/imunologia
Hemofilia A/metabolismo
Hemorragia/prevenção & controle
Seres Humanos
Infusões Intravenosas
Masculino
Eliminação de Partículas Virais
Adulto Jovem
[Pt] Tipo de publicação:CLINICAL TRIAL, PHASE I; CLINICAL TRIAL, PHASE II; JOURNAL ARTICLE; MULTICENTER STUDY; RESEARCH SUPPORT, NON-U.S. GOV'T
[Nm] Nome de substância:
0 (Antibodies, Viral); 0 (DNA, Viral); 9001-27-8 (Factor VIII)
[Em] Mês de entrada:1801
[Cu] Atualização por classe:180207
[Lr] Data última revisão:
180207
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:171212
[St] Status:MEDLINE
[do] DOI:10.1056/NEJMoa1708483


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[PMID]:29217699
[Au] Autor:Pratt KP
[Ad] Endereço:UNIFORMED SERVICES UNIVERSITY OF THE HEALTH SCIENCES.
[Ti] Título:Marginal immunogenicity of factor VIII.
[So] Source:Blood;130(23):2450-2451, 2017 12 07.
[Is] ISSN:1528-0020
[Cp] País de publicação:United States
[La] Idioma:eng
[Mh] Termos MeSH primário: Fator VIII
Hemofilia A
[Mh] Termos MeSH secundário: Seres Humanos
[Pt] Tipo de publicação:JOURNAL ARTICLE; COMMENT
[Nm] Nome de substância:
9001-27-8 (Factor VIII)
[Em] Mês de entrada:1801
[Cu] Atualização por classe:180118
[Lr] Data última revisão:
180118
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:171209
[St] Status:MEDLINE
[do] DOI:10.1182/blood-2017-10-811109


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[PMID]:28457068
[Au] Autor:Ben Haim G; Manor U; Appel S; Lalezari S; Margalit-Yehuda R; Steinlauf S
[Ad] Endereço:Department of Internal Medicine C, Sheba Medical Center, Tel Hashomer, Israel.
[Ti] Título:Acquired Hemophilia A in a Patient with Non-Small Cell Lung Carcinoma: A Rare Paraneoplastic Phenomenon.
[So] Source:Isr Med Assoc J;19(2):128-130, 2017 Feb.
[Is] ISSN:1565-1088
[Cp] País de publicação:Israel
[La] Idioma:eng
[Mh] Termos MeSH primário: Carcinoma Pulmonar de Células não Pequenas
Hemofilia A
Neoplasias Pulmonares
Metilprednisolona/administração & dosagem
Síndromes Paraneoplásicas
[Mh] Termos MeSH secundário: Idoso
Carcinoma Pulmonar de Células não Pequenas/complicações
Carcinoma Pulmonar de Células não Pequenas/patologia
Tratamento Conservador
Fator VIII/análise
Hemorragia Gastrointestinal/diagnóstico
Hemorragia Gastrointestinal/etiologia
Hematúria/diagnóstico
Hematúria/etiologia
Hemofilia A/diagnóstico
Hemofilia A/etiologia
Hemofilia A/fisiopatologia
Seres Humanos
Imunossupressores/administração & dosagem
Neoplasias Pulmonares/complicações
Neoplasias Pulmonares/patologia
Masculino
Imagem Multimodal/métodos
Estadiamento de Neoplasias
Síndromes Paraneoplásicas/diagnóstico
Síndromes Paraneoplásicas/fisiopatologia
Resultado do Tratamento
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Immunosuppressive Agents); 9001-27-8 (Factor VIII); X4W7ZR7023 (Methylprednisolone)
[Em] Mês de entrada:1711
[Cu] Atualização por classe:171130
[Lr] Data última revisão:
171130
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170501
[St] Status:MEDLINE


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[PMID]:28904675
[Au] Autor:Echahdi H; El Hasbaoui B; El Khorassani M; Agadr A; Khattab M
[Ad] Endereço:Center of Hematology and Oncology Paediatrics, Children's Hospital, Faculty of Medicine and Pharmacy, University Mohammed V Rabat, Morocco.
[Ti] Título:Von Willebrand's disease: case report and review of literature.
[So] Source:Pan Afr Med J;27:147, 2017.
[Is] ISSN:1937-8688
[Cp] País de publicação:Uganda
[La] Idioma:eng
[Ab] Resumo:Von Willebrand Disease (VWD) is the most common human inherited bleeding disorder due to a defect of Von Willebrand Factor (VWF), which a glycoprotein crucial for platelet adhesion to the subendothelium after vascular injury. VWD include quantitative defects of VWF, either partial (type 1 with VWF levels < 50 IU/dL) or virtually total (type 3 with undetectable VWF levels) and also qualitative defects of VWF (type 2 variants with discrepant antigenic and functional VWF levels). The most bleeding forms of VWD usually do not concern type 1 patients with the mildest VWF defects (VWF levels between 30 and 50IU/dL). Von willebrand factor is a complex multimeric protein with two functions: it forms a bridge between the platelets and areas of vascular damage and it binds to and stabilizes factor VIII, which is necessary for the clotting cascade. By taking a clinical history of bleeding (mucocutaneous bleeding symptoms suggestive of a primary haemostatic disorder, a quantitative or qualitative abnormality of VWF is possible) it is important to think about VWD and to make the appropriate diagnosis. If the VWD is suspected diagnostic tests should include an activated partial thromboplastin time, bleeding time, factor VIII: C Ristocetin cofactor and vWF antigen. Additional testing of ristocetin induced plattlet adhesion (RIPA) the multimeric structure and collagen binding test and genanalysis allow diagnosing the different types of von. Willebrand Disease. The treatment of choice in mild forms is the synthetic agent desmopressin. In patients with severe type 1, type 2B, 2N and type 3 or in people who do not response to desmopressin, the appropriate treatment is a factor VIII concentrate that is rich of VWF. We report a case of infant in 27-month-old boy who had been referred due to haemorrhagic shock. His birth histories, his familie's social history and developmental milestones were unremarkable. He was born at full term with no antenatal or perinatal complications. Prior to the symptoms, the child was on a normal diet and was thriving appropriately. The child presented one days before his admission trauma to the inner face of the lower lip that caused an external acute bleeding loss. The laboratory data showed unfortunately, the most severe form of Von Willebrand's Disease; Type 3. The management was based on erythrocyte and fresh-frozen plasma (FFP) transfusions with administration of factor VII with good evolution.
[Mh] Termos MeSH primário: Fator VIII/administração & dosagem
Hemorragia/etiologia
Doenças de von Willebrand/diagnóstico
[Mh] Termos MeSH secundário: Pré-Escolar
Desamino Arginina Vasopressina/administração & dosagem
Transfusão de Eritrócitos/métodos
Seres Humanos
Masculino
Tempo de Tromboplastina Parcial
Plasma
Índice de Gravidade de Doença
Doenças de von Willebrand/terapia
Fator de von Willebrand
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
0 (von Willebrand Factor); 9001-27-8 (Factor VIII); ENR1LLB0FP (Deamino Arginine Vasopressin)
[Em] Mês de entrada:1709
[Cu] Atualização por classe:170922
[Lr] Data última revisão:
170922
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170915
[St] Status:MEDLINE
[do] DOI:10.11604/pamj.2017.27.147.12248


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[PMID]:28895852
[Au] Autor:Nathwani AC; Davidoff AM; Tuddenham EGD
[Ad] Endereço:Department of Academic Haematology, UCL Cancer Institute, Katharine Dormandy Haemophilia and Thrombosis Centre, Rowland Hill Street, London NW3 2PF, United Kingdom; National Health Service Blood and Transplant, Oak House, Reeds Crescent, Watford, Hertfordshire, WD24 4QN, United Kingdom. Electronic address: amit.nathwani@ucl.ac.uk.
[Ti] Título:Gene Therapy for Hemophilia.
[So] Source:Hematol Oncol Clin North Am;31(5):853-868, 2017 Oct.
[Is] ISSN:1558-1977
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:The best currently available treatments for hemophilia A and B (factor VIII or factor IX deficiency, respectively) require frequent intravenous infusion of highly expensive proteins that have short half-lives. Factor levels follow a saw-tooth pattern that is seldom in the normal range and falls so low that breakthrough bleeding occurs. Most hemophiliacs worldwide do not have access to even this level of care. In stark contrast, gene therapy holds out the hope of a cure by inducing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene to replace the hemophilic patient's own defective gene.
[Mh] Termos MeSH primário: Terapia Genética
Hemofilia A/genética
Hemofilia A/terapia
Hemofilia B/genética
Hemofilia B/terapia
[Mh] Termos MeSH secundário: Animais
Ensaios Clínicos como Assunto
Dependovirus/genética
Fator IX/genética
Fator VIII/genética
Terapia Genética/efeitos adversos
Terapia Genética/economia
Terapia Genética/métodos
Vetores Genéticos/genética
Seres Humanos
Resultado do Tratamento
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
9001-27-8 (Factor VIII); 9001-28-9 (Factor IX)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171009
[Lr] Data última revisão:
171009
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170913
[St] Status:MEDLINE



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