Eptacog alfa activated: a recombinant product to treat rare congenital bleeding disorders
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Di Minno, 2009, Glanzmann's thrombasthenia (defective platelet integrin alphaIIb-beta3): proposals for management between evidence and open issues, Thromb Haemost, 102, 1157, 10.1160/TH09-04-0225
Lapecorella, 2008, Factor VII deficiency: defining the clinical picture and optimizing therapeutic options, Haemophilia, 14, 1170, 10.1111/j.1365-2516.2008.01844.x
Di Minno
Poon, 2013, Treatment modalities and outcomes in 204 surgical procedures in 96 Glanzmann's thrombasthenia (GT) patients: the International Prospective Glanzmann's Thrombasthenia Registry (GTR), J Thromb Haemost, 11, 161
Poon, 2006, Glanzmann's thrombasthenia treatment: a prospective observational registry on the use of recombinant human activated factor VII and other hemostatic agents, Semin Hematol, 43, S33, 10.1053/j.seminhematol.2005.11.009
Mariani, 2013, Replacement therapy for bleeding episodes in factor VII deficiency. A prospective evaluation, Thromb Haemost, 109, 238, 10.1160/TH12-07-0476
Napolitano, 2013, Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER), Haematologica, 98, 538, 10.3324/haematol.2012.074039
Bellucci, 2002, Molecular basis of Glanzmann's Thrombasthenia and current strategies in treatment, Blood Rev, 16, 193, 10.1016/S0268-960X(02)00030-9
Nurden, 2013, Glanzmann thrombasthenia: state of the art and future directions, Semin Thromb Hemost, 39, 642, 10.1055/s-0033-1353393
Toogeh, 2004, Presentation and pattern of symptoms in 382 patients with Glanzmann thrombasthenia in Iran, Am J Hematol, 77, 198, 10.1002/ajh.20159
George, 1990, Glanzmann's thrombasthenia: the spectrum of clinical disease, Blood, 75, 1383, 10.1182/blood.V75.7.1383.1383
Bolton-Maggs, 2006, A review of inherited platelet disorders with guidelines for their management on behalf of the UKHCDO, Br J Haematol, 135, 603, 10.1111/j.1365-2141.2006.06343.x
Rajpurkar, 2014, Use of recombinant activated factor VII in patients with Glanzmann's thrombasthenia: a review of the literature, Haemophilia, 20, 464, 10.1111/hae.12473
Spiess, 2010, Platelet transfusions: the science behind safety, risks and appropriate applications, Best Pract Res Clin Anaesthesiol, 24, 65, 10.1016/j.bpa.2009.11.001
MacLennan, 2006, Risks of fresh frozen plasma and platelets, J Trauma, 60, S46
Hedner, 2008, Factor VIIa and its potential therapeutic use in bleeding-associated pathologies, Thromb Haemost, 100, 557, 10.1160/TH08-07-0434
Lisman, 2004, Recombinant factor VIIa restores aggregation of alphaIIbbeta3-deficient platelets via tissue factor-independent fibrin generation, Blood, 103, 1720, 10.1182/blood-2003-07-2287
Poon, 1999, Recombinant factor VIIa is effective for bleeding and surgery in patients with Glanzmann thrombasthenia, Blood, 94, 3951, 10.1182/blood.V94.11.3951
Poon, 2004, Prophylactic and therapeutic recombinant factor VIIa administration to patients with Glanzmann's thrombasthenia: results of an international survey, J Thromb Haemost, 2, 1096, 10.1111/j.1538-7836.2004.00767.x
Di Minno, 2015, The international prospective Glanzmann Thrombasthenia Registy: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients, Haematologica, 10.3324/haematol.2014.121475
Poon, 2015, The international prospective Glanzmann Throbasthenia Registry: treatment and outcomes in surgical intervention, Haematologica, 10.3324/haematol.2014.121384
Borhany, 2010, Bleeding disorders in the tribe: result of consanguineous in breeding, Orphanet J Rare Dis, 5, 23, 10.1186/1750-1172-5-23
Mariani, 2005, Clinical phenotypes and factor VII genotype in congenital factor VII deficiency, Thromb Haemost, 93, 481, 10.1160/TH04-10-0650
Di Minno, 2011, At disease presentation, severity of the bleeding symptoms predicts the following bleeds in patients with FVII deficiency, Blood, 118, 543, 10.1182/blood.V118.21.30.30
Giansily-Blaizot, 2002, Inherited factor VII deficiency and surgery: clinical data are the best criteria to predict the risk of bleeding, Br J Haematol, 117, 172, 10.1046/j.1365-2141.2002.03408.x
Mariani, 2012, Invasive procedures and minor surgery in factor VII deficiency, Haemophilia, 18, e63, 10.1111/j.1365-2516.2012.02751.x
Mariani, 2006, Congenital factor VII deficiency: therapy with recombinant activated factor VII – a critical appraisal, Haemophilia, 12, 19, 10.1111/j.1365-2516.2006.01180.x
Berrettini, 2001, Pharmacokinetic evaluation of recombinant, activated factor VII in patients with inherited factor VII deficiency, Haematologica, 86, 640
Greenberg, 2001, Blood coagulation factors: Their complementary DNAs, genes and expression, 135
Gopalakrishnan, 2010, Bio-distribution of pharmacologically administered recombinant factor VIIa (rFVIIa), J Thromb Haemost, 8, 301, 10.1111/j.1538-7836.2009.03696.x
Lopez-Vilchez, 2011, Redistribution and hemostatic action of recombinant activated factor VII associated with platelets, Am J Pathol, 178, 2938, 10.1016/j.ajpath.2011.02.026
Mathijssen, 2013, Increased volume of distribution for recombinant activated factor VII and longer plasma-derived factor VII half-life may explain their long lasting prophylactic effect, Thromb Res, 132, 256, 10.1016/j.thromres.2013.05.027
Morfini, 2014, Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect, Thromb Haemost, 112, 424, 10.1160/TH13-12-1045
Di Minno, 2013, Bleeding symptoms at disease presentation and prediction of ensuing bleeding in inherited FVII deficiency, Thromb Haemost, 109, 1051, 10.1160/TH12-10-0740
Nicolaisen, 1998, Antigenicity of activated recombinant factor VII followed through nine years of clinical experience, Blood Coagul Fibrinolysis, 9, S119
Rodeghiero, 2005, The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study, J Thromb Haemost, 3, 2619, 10.1111/j.1538-7836.2005.01663.x
Tosetto, 2006, A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD), J Thromb Haemost, 4, 766, 10.1111/j.1538-7836.2006.01847.x
Batorova, 2014, Inhibitors to factor VII in congenital factor VII deficiency, Haemophilia, 20, e188, 10.1111/hae.12376
Neufeld, 2015, Safety update on the use of recombinant activated factor VII in approved indications, Blood Reviews, 29, S34, 10.1016/S0268-960X(15)30006-0
Mariani, 2011, Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation – the surgical STER, Br J Haematol, 152, 340, 10.1111/j.1365-2141.2010.08287.x
Monroe, 1997, Platelet activity of high-dose factor VIIa is independent of tissue factor, Br J Haematol, 99, 542, 10.1046/j.1365-2141.1997.4463256.x
Weeterings, 2008, The glycoprotein Ib-IX-V complex contributes to tissue factor-independent thrombin generation by recombinant factor VIIa on the activated platelet surface, Blood, 112, 3227, 10.1182/blood-2008-02-139113
Poon, 2013, Factor VIIa, 1257