12 research outputs found
Key Issues In Inhibitor Management In Patients With Haemophilia
[No abstract available]12SUPPL.1s319s329Bray, G.L., Gomperts, E.D., Courter, S., A multicenter study of recombinant factor VIII (recombinate): Safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The recombinate study group (1994) Blood, 83, pp. 2428-2435Lusher, J.M., Arkin, S., Abildgaard, C.F., Schwartz, R.S., Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate previously untreated patient study group (1993) N Engl J Med, 328, pp. 453-459Hay, C.R., Baglin, T.P., Collins, P.W., The diagnosis and management of factor VIII and IX inhibitors: A guideline from the UK Haemophilia Centre Doctors' Organization (UKHCDO) (2000) Br J Haematol, 111, pp. 78-90Lawrence, J.S., Johnson, J.B., The presence of a circulating anticoagulant in a male member of a hemophiliac family (1941) Trans Am Clin Climatol Assoc, 57, pp. 223-231Konkle, B.A., Ebbesen, L.S., Erhardtsen, E., Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors (2007) J Thromb Haemost, 5, pp. 1904-1913Leissinger, C., Gringeri, A., Antmen, B., Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors (2011) N Engl J Med, 365, pp. 1684-1692Gouw, S.C., Van Den Berg, H.M., Oldenburg, J., F8 gene mutation type and inhibitor development in patients with severe hemophilia A: Systematic review and meta-analysis (2012) Blood, 119, pp. 2922-2934Gouw, S.C., Van Der Bom, J.G., Auerswald, G., Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: The CANAL cohort study (2007) Blood, 109, pp. 4693-4697Iorio, A., Halimeh, S., Holzhauer, S., Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: A systematic review (2010) J Thromb Haemost, 8, pp. 1256-1265Wight, J., Paisley, S., The epidemiology of inhibitors in haemophilia A: A systematic review (2003) Haemophilia, 9, pp. 418-435Hay, C.R., The epidemiology of factor VIII inhibitors (2006) Haemophilia, 12 (SUPPL. 6), pp. 23-28. , discussion 8-9Astermark, J., Basic aspects of inhibitors to factors VIII and IX and the influence of non-genetic risk factors (2006) Haemophilia, 12 (SUPPL. 6), pp. 8-13Hay, C.R., Palmer, B., Chalmers, E., Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom (2011) Blood, 117, pp. 6367-6370Wacey, A.I., Kemball-Cook, G., Kazazian, H.H., The haemophilia A mutation search test and resource site, home page of the factor VIII mutation database: HAMSTeRS (1996) Nucleic Acids Res, 24, pp. 100-102Green, P.M., Montandon, A.J., Ljung, R., Haemophilia B mutations in a complete Swedish population sample: A test of new strategy for the genetic counselling of diseases with high mutational heterogeneity (1991) Br J Haematol, 78, pp. 390-397Viel, K.R., Ameri, A., Abshire, T.C., Inhibitors of factor VIII in black patients with hemophilia (2009) N Engl J Med, 360, pp. 1618-1627Viel, K.R., Machiah, D.K., Warren, D.M., A sequence variation scan of the coagulation factor VIII (FVIII) structural gene and associations with plasma FVIII activity levels (2007) Blood, 109, pp. 3713-3724Santos, A., Annichino-Bizzacchi, J.M., Ozelo, M.C., Inhibitors of factor VIII in hemophilia (2009) N Engl J Med, 361, pp. 309-310. , author reply 10Oldenburg, J., Pavlova, A., (2006) Genetic risk factors for inhibitors to factors VIII and IX.Haemophilia, 12 (SUPPL. 6), pp. 15-22Goodeve, A.C., Peake, I.R., The molecular basis of hemophilia A: Genotype-phenotype relationships and inhibitor development (2003) Semin Thromb Hemost, 29, pp. 23-30Jacquemin, M., Vantomme, V., Buhot, C., CD4+ T-cell clones specific for wild-type factor VIII: A molecular mechanism responsible for a higher incidence of inhibitor formation in mild/moderate hemophilia A (2003) Blood, 101, pp. 1351-1358Kane, W.H., Davie, E.W., Cloning of a cDNA coding for human factor V, a blood coagulation factor homologous to factor VIII and ceruloplasmin (1986) Proc Natl Acad Sci USA, 83, pp. 6800-6804Lacroix-Desmazes, S., Repesse, Y., Kaveri, S.V., Dasgupta, S., The role of VWF in the immunogenicity of FVIII (2008) Thromb Res, 122 (SUPPL. 2), pp. S3-6Rivard, G.E., Lillicrap, D., Poon, M.C., Can activated recombinant factor VII be used to postpone the exposure of infants to factor VIII until after 2 years of age? (2005) Haemophilia, 11, pp. 335-339Hermans, C., De Moerloose, P., Fischer, K., Management of acute haemarthrosis in haemophilia A without inhibitors: Literature review, European survey and recommendations (2011) Haemophilia, 17, pp. 383-392Kurnik, K., Bidlingmaier, C., Engl, W., New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development (2010) Haemophilia, 16, pp. 256-262Gouw, S.C., Van Den Berg, H.M., Fischer, K., Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: The RODIN study (2013) BloodKreuz, W., Ettingshausen, C.E., Zyschka, A., Inhibitor development in previously untreated patients with hemophilia A: A prospective long-term follow-up comparing plasmaderived and recombinant products (2002) Semin Thromb Hemost, 28, pp. 285-290Santagostino, E., Mannucci, P.M., Bianchi Bonomi, A., Guidelines on replacement therapy for haemophilia and inherited coagulation disorders in Italy (2000) Haemophilia, 6, pp. 1-10Rezende, S.M., Pinheiro, K., Caram, C., Registry of inherited coagulopathies in Brazil: First report (2009) Haemophilia, 15, pp. 142-149Manco-Johnson, M.J., Abshire, T.C., Shapiro, A.D., Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia (2007) N Engl J Med, 357, pp. 535-544Kruse-Jarres, R., Inhibitors: Our greatest challenge.Can we minimize the incidence? (2013) Haemophilia, 19 (SUPPL. 1), pp. 2-7Lobet, S., Detrembleur, C., Francq, B., Hermans, C., Natural progression of blood-induced joint damage in patients with haemophilia: Clinical relevance and reproducibility of threedimensional gait analysis (2010) Haemophilia, 16, pp. 813-821Leissinger, C.A., Prevention of bleeds in hemophilia patients with inhibitors: Emerging data and clinical direction (2004) Am J Hematol, 77, pp. 187-193Gringeri, A., Mantovani, L.G., Scalone, L., Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS Study Group (2003) Blood, 102, pp. 2358-2363Fischer, K., Van Der Bom, J.G., Molho, P., Prophylactic versus on-demand treatment strategies for severe haemophilia: A comparison of costs and long-term outcome (2002) Haemophilia, 8, pp. 745-752Morfini, M., Haya, S., Tagariello, G., European study on orthopaedic status of haemophilia patients with inhibitors (2007) Haemophilia, 13, pp. 606-612Soucie, J.M., Cianfrini, C., Janco, R.L., Joint range-of-motion limitations among young males with hemophilia: Prevalence and risk factors (2004) Blood, 103, pp. 2467-2473Hay, C.R., Dimichele, D.M., The principal results of the International Immune Tolerance Study: A randomized dose comparison (2012) Blood, 119, pp. 1335-1344Astermark, J., Donfield, S.M., Dimichele, D.M., A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: The FEIBA NovoSeven Comparative (FENOC) Study (2007) Blood, 109, pp. 546-551Sorensen, B., Dargaud, Y., Kenet, G., On-demand treatment of bleeds in haemophilia patients with inhibitors: Strategies for securing and maintaining predictable efficacy with recombinant activated factor VII (2012) Haemophilia, 18, pp. 255-262Kenet, G., Martinowitz, U., Single-dose recombinant activated factor VII therapy in hemophilia patients with inhibitors (2008) Semin Hematol, 45, pp. S38-41Treur, M.J., McCracken, F., Heeg, B., Efficacy of recombinant activated factor VII vs. Activated prothrombin complex concentrate for patients suffering from haemophilia complicated with inhibitors: A Bayesian meta-regression (2009) Haemophilia, 15, pp. 420-436Knight, C., Dano, A.M., Kennedy-Martin, T., Systematic review of efficacy of rFVIIa and aPCC treatment for hemophilia patients with inhibitors (2009) Adv Ther, 26, pp. 68-88Valentino, L.A., The benefits of prophylactic treatment with APCC in patients with haemophilia and high-titre inhibitors: A retrospective case series (2009) Haemophilia, 15, pp. 733-742Teitel, J., Berntorp, E., Dolan, G., A consensus statement on clinical trials of bypassing agent prophylaxis in inhibitor patients (2011) Haemophilia, 17, pp. 516-521Young, G., Auerswald, G., Jimenez-Yuste, V., When should prophylaxis therapy in inhibitor patients be considered? (2011) Haemophilia, 17, pp. e849-e857Dimichele, D., Negrier, C., A retrospective postlicensure survey of FEIBA efficacy and safety (2006) Haemophilia, 12, pp. 352-362Valentino, L.A., Assessing the benefits of FEIBA prophylaxis in haemophilia patients with inhibitors (2010) Haemophilia, 16, pp. 263-271Brackmann, H.H., Schwaab, R., Effenberger, W., Hemophilia treatment. 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Executive Committee of the German Medical Association on the Recommendation of the Scientific Advisory Board, p. 91. , German Medical AssociationCoppola, A., Margaglione, M., Santagostino, E., Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of hemophilia A patients with highresponding inhibitors (2009) J Thromb Haemost, 7, pp. 1809-1815Freiburghaus, C., Berntorp, E., Ekman, M., Immunoadsorption for removal of inhibitors: Update on treatments in Malmo-Lund between 1980 and 1995 (1998) Haemophilia, 4, pp. 16-20Auerswald, G., Spranger, T., Brackmann, H.H., The role of plasmaderived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients (2003) Haematologica, 88, pp. EREP05Dimichele, D., The North American Immune Tolerance Registry: Contributions to the thirty-year experience with immune tolerance therapy (2009) Haemophilia, 15, pp. 320-328Mariani, G., Ghirardini, A., Bellocco, R., Immune tolerance in hemophilia-principal results from the International Registry. Report of the factor VIII and IX Subcommittee (1994) Thromb Haemost, 72, pp. 155-158Mariani, G., Kroner, B., Immune tolerance in hemophilia with factor VIII inhibitors: Predictors of success (2001) Haematologica, 86, pp. 1186-1193Franchini, M., Mannucci, P.M., Inhibitors of propagation of coagulation (factors VIII, IX and XI): A review of current therapeutic practice (2011) Br J Clin Pharmacol, 72, pp. 553-562Greninger, D.A., Saint-Remy, J.M., Jacquemin, M., The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high-titre inhibitors: Association of clinical outcome with inhibitor epitope profile (2008) Haemophilia, 14, pp. 295-302Gringeri, A., Musso, R., Mazzucconi, M.G., Immune tolerance induction with a high purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response (2007) Haemophilia, 13, pp. 373-379Kurth, M.A., Dimichele, D., Sexauer, C., Immune tolerance therapy utilizing factor VIII/von Willebrand factor concentrate in haemophilia A patients with high titre factor VIII inhibitors (2008) Haemophilia, 14, pp. 50-55Astermark, J., Morado, M., Rocino, A., Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors (2006) Haemophilia, 12, pp. 363-371Garvey, B., Rituximab in the treatment of autoimmune haematological disorders (2008) Br J Haematol, 141, pp. 149-169Franchini, M., Mengoli, C., Lippi, G., Immune tolerance with rituximab in congenital haemophilia with inhibitors: A systematic literature review based on individual patients' analysis (2008) Haemophilia, 14, pp. 903-912Sorensen, B., Johansen, P., Christiansen, K., Whole blood coagulation thrombelastographic profiles employing minimal tissue factor activation (2003) J Thromb Haemost, 1, pp. 551-558De Paula, E.V., Kavakli, K., Mahlangu, J., Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: A randomized controlled trial (2012) J Thromb Haemost, 10, pp. 81-89Holmberg, H.L., Lauritzen, B., Tranholm, M., Ezban, M., Faster onset of effect and greater efficacy of NN1731 compared with rFVIIa, aPCC and FVIII in tail bleeding in hemophilic mice (2009) J Thromb Haemost, 7, pp. 1517-1522Moss, J., Scharling, B., Ezban, M., Moller Sorensen, T., Evaluation of the safety and pharmacokinetics of a fast-acting recombinant FVIIa analogue, NN1731, in healthy male subjects (2009) J Thromb Haemost, 7, pp. 299-305Sorensen, B., Persson, E., Ingerslev, J., Factor VIIa analogue (V158D/E296V/M298Q-FVIIa) normalises clot formation in whole blood from patients with severe haemophilia A (2007) Br J Haematol, 137, pp. 158-165Allen, G.A., Persson, E., Campbell, R.A., A variant of recombinant factor VIIa with enhanced procoagulant and antifibrinolytic activities in an in vitro model of hemophilia (2007) Arterioscler Thromb Vasc Biol, 27, pp. 683-689Mahlangu, J.N., Coetzee, M.J., Laffan, M., Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the rFVIIa variant BAY 86-6150 in hemophilia (2012) J Thromb Haemost, 10, pp. 773-780Karpf, D.M., Sorensen, B.B., Hermit, M.B., Prolonged halflife of glycoPEGylated rFVIIa variants compared to native rFVIIa (2011) Thromb Res, 128, pp. 191-195Sen, P., Ghosh, S., Ezban, M., Effect of glycoPEGylation on factor VIIa binding and internalization (2010) Haemophilia, 16, pp. 339-348Toschi, V., OBI-1, porcine recombinant factor VIII for the potential treatment of patients with congenital hemophilia A and alloantibodies against human Factor VIII (2010) Curr Opin Mol Ther, 12, pp. 617-625Parker, E.T., Craddock, H.N., Barrow, R.T., Lollar, P., Comparative immunogenicity of recombinant B domain-deleted porcine factor VIII and Hyate: C in hemophilia A mice presensitized to human factor VIII (2004) J Thromb Haemost, 2, pp. 605-611Kempton, C.L., Abshire, T.C., Deveras, R.A., Pharmacokinetics and safety of OBI-1, a recombinant B domain-deleted porcine factor VIII, in subjects with haemophilia A (2012) Haemophilia, 18, pp. 798-804Abshire, T.C., Brackmann, H.H., Scharrer, I., Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy-International Kogenate-FS Study Group (2000) Thromb Haemost, 83, pp. 811-816Tarantino, M.D., Collins, P.W., Hay, C.R., Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: Pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A (2004) Haemophilia, 10, pp. 428-437Young, G., Cooper, D.L., Gut, R.Z., Dosing and effectiveness of recombinant activated factor VII (rFVIIA) in congenital haemophilia with inhibitors by bleed type and location: The experience of the Haemophilia and Thrombosis Research Society (HTRS) Registry (2004-2008) (2012) Haemophilia, 18, pp. 990-99
Diagnosis And Treatment Of Congenital Hemophilia With Inhibitors. A Latin American Perspective
The Committee of Latin America on the Therapeutics of Inhibitor Groups (CLOTTING) is composed of a number of hemophilia specialists from Latin America. The group aims to encourage the adoption of a good standard of care for Latin American patients with hemophilia. The occurrence of inhibitors in patients with hemophilia poses clinical challenges, and it is estimated that between 1 000 and 3 000 patients in Latin America are affected by hemophilia with inhibitors. There is an urgent need to establish a regional consensus and clinical guidelines for the diagnosis and treatment of these patients. We present an extensive review based on best current clinical practice and published literature, as seen from a Latin American perspective, taking into account the variable nature of hemophilia care available in the various countries in this Region.683227242Ehrenforth, S., Kreuz, W., Scharrer, I., Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs (1992) Lancet, 339, pp. 594-598Katz, J., Prevalence of factor IX inhibitors among patients with haemophilia B: Results of a large-scale North American study (1996) Haemophilia, 2, pp. 28-31Goudemand, J., Pharmaco-economic aspects of inhibitor treatment (1998) Eur J Haematol, 61, pp. 24-27World Hemophilia Federation Report on Global Survey 2006. World Federation of Hemophilia, 2007. On www.wfh.orgconsulted on 23/04/2008Rieger, A., Roisenberg, I., Prevalence of factor VIII inhibitors in patients with hemophilia A in Brazil (1999) Thromb Haemost, 81, pp. 475-476Fontes, E.M., Amorim, L., Carvalho, S.M., Farah, M.B., Hemophilia care in the state of Rio de Janeiro, Brazil (2003) Rev Panam Salud Pública, 13, pp. 124-128Izquierdo-Ramírez, J., Contreras-Mulato, E.L., Sotelo-Ham, E.I., Incidence of inhibitors in children with hemophilia A (1988) Bol Med Hosp Infant Méx, 45, pp. 578-582Boadas, A., Ruiz-Sáez, A., Arguello, A., de Bosch, N., Prevalence and acute bleeding treatment of allo and auto FVIII and FIX antibodies cases in Venezuela (2004) Haemophilia, 10, p. 56Wight, J., Paisley, S., The epidemiology of inhibitors in hemophilia A: A systematic review (2003) Haemophilia, 9, pp. 418-435Delivery of treatment for hemophilia (2002) Report of a joint WHO/WFH/ISTH meeting, , World Health OrganizationHay, C.R., Brown, S., Collins, P.W., Keeling, D.M., Liesner, R., The diagnosis and management of factor VIII and IX inhibitors: A guideline from the United Kingdom Haemophilia Centre Doctors Organization (2006) Br J Haematol, 133, pp. 591-605Ewing, N.P., Kasper, C.K., In vitro detection of mild inhibitors to factor VIII in hemophilia (1982) Am J Clin Pathol, 77, pp. 749-752Verbruggen, B., Novakova, I., Wessels, H., Boezeman, J., van den Berg, M., Mauser-Bunschoten, E., The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: Improved specificity and reliability (1995) Thromb Haemost, 73, pp. 247-251Giles, A.R., Verbruggen, B., Rivard, G.E., Teitel, J., Walker, I., A detailed comparison of the performance of the standard versus the Nijmegen modification of the Bethesda assay in detecting factor VIII:C inhibitors in the hemophilia A population of Canada. Association of Hemophilia Centre Directors of Canada. Factor VIII/IX Subcommittee of Scientific and Standardization Committee of International Society on Thrombosis and Haemostasis (1998) Thromb Haemost, 79, pp. 872-875Verbruggen, B., van Heerde, W., Novakova, I., Lillicrap, D., Giles, A., A 4% solution of bovine serum albumin may be used in place of factor VIII:C deficient plasma in the control sample in the Nijmegen modification of the Bethesda factor VIII:C inhibitor assay (2002) Thromb Haemost, 88, pp. 362-364White II, G.C., Rosendaal, F., Aledort, L.M., Lusher, J.M., Rothschild, C., Ingerslev, J., Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis (2001) Thromb Haemost, 85, p. 560Kitchen, S., McCraw, A., (2000) Diagnosis of hemophilia and other bleeding disorders, , A laboratory manual. World Federation of Hemophiliahttp://www.med.unc.edu/isth, Available at:, Accessed November 2005http://www.wfh.org, Available at:, Accessed November 2005Oldenburg, J., Brackmann, H.H., Schwaab, R., Risk factors for inhibitor development in hemophilia A (2000) Haematologica, 85, pp. 7-13Rossetti, L.C., Candela, M., Pérez Bianco, R., de Tezanos Pinto, M., Western, A., Goodeve, A., Analysis of factor VIII gene intron 1 inversion in Argentinean families with severe hemophilia A and a review of the literature (2004) Blood Coagul Fibrinolysis, 15, pp. 569-572Santos, A., Montalva, O., Thomas, S., Veiga, M., De Paula, E., Ozelo, M., Genetic and ethnic aspects related to the development of inhibitors among Brazilian patients with hemophilia from five distinct geographical regions in Brazil (2006) Haemophilia, 12, pp. 1-154Mantilla-Capacho, J.M., Beltrán-Miranda, C.P., Luna-Záizar, H., Frequency of intron 1 and 22 inversions of Factor VIII gene in Mexican patients with severe Hemophilia A (2007) Am J Hematol, 82, pp. 283-287Guidelines for the management of hemophilia. World Federation of Hemophilia, 2005Girolami, A., Luzzatto, G., Varvarikis, C., Pellati, D., Sartori, R., Girolami, B., Main clinical manifestations of a bleeding diathesis: An often disregarded aspect of medical and surgical history taking (2005) Haemophilia, 11, pp. 193-202Suggestions for the management of FVIII inhibitors (2000) Treatment of Hemophilia Monograph, , Inhibitor Subcommittee of the Association of Hemophilia Clinic Directors of Canada, revised edition, World Federation of HemophiliaKasper, C., Diagnosis and management of inhibitors to factors VIII and IX (2004) Treatment of Hemophilia Monograph, , World Federation of HemophiliaGringeri, A., Mannucci, P.M., Italian Association of Haemophilia Centres. 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Factor Eight Bypassing Activity (1997) Thromb Haemost, 77, pp. 1113-1119Astermark, J., Donfield, S.M., DiMichelle, D.M., A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor - The Feiba® NovoSeven® Comparative Study (FENOC) (2007) Blood, 109, pp. 546-551Young, G., Shafer, F.E., Rojas, P., Seremetis, S., Single 270 μg kg-1 dose rFVIIa vs. standard 90 μg kg-1 dose rFVIIa and APCC for home treatment of joint bleeds in hemophilia patients with inhibitors: A randomized comparison (2008) Haemophilia, 14, pp. 287-294Ozelo, M.C., Villaça, P.R., De Almeida, J.O., A cost evaluation of treatment alternatives for mild-to-moderate bleeding episodes in patients with hemophilia and inhibitors in Brazil (2007) Haemophilia, 13, pp. 462-469Kenet, G., Lubetsky, A., Luboshitz, J., Martinowitz, U., A new approach to treatment of bleeding episodes in young hemophilia patients: A single bolus megadose of recombinant activated factor VII (NovoSeven) (2003) J Thromb Haemost, 1, pp. 450-455Parameswaran, R., Shapiro, A.D., Gill, J.C., Kessler, C.M., Dose effect and efficacy of rFVIIa in the treatment of hemophilia patients with inhibitors: Analysis from the Hemophilia and Thrombosis Research Society Registry (2005) Haemophilia, 11, pp. 100-106Kavakli, K., Makris, M., Zulfikar, B., Erhardtsen, E., Abrams, Z.S., Kenet, G., Home treatment of haemarthroses using single dose regimen of recombinant activated factor VII in patients with hemophilia and inhibitors. A multi-centre, randomized, double blind, cross-over trial (2006) Thromb Haemost, 95, pp. 600-605Santagostino, E., Mancuso, M.E., Rocino, A., Mancuso, G., Scaraggi, F., Mannucci, P.M., A prospective randomized trial of high and standard dosages of recombinant factor VIIa for treatment of haemarthroses in hemophiliacs with inhibitors (2006) J Thromb Haemost, 4, pp. 367-371Schneiderman, J., Nugent, D.J., Young, G., Sequential therapy with activated prothrombin complex concentrate and recombinant factor VIIa in patients with severe hemophilia and inhibitors (2004) Haemophilia, 10, pp. 347-351Lusher, J.M., Shapiro, S.S., Palascak, J.E., Rao, A.V., Levine, P.H., Blatt, P.M., Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: A multicenter therapeutic trial (1980) NEJM, 303, pp. 421-425Teitel, J., Berntorp, E., Collins, P., A systematic approach to controlling problem bleeds in patients with severe congenital hemophilia A and high-titre inhibitors (2007) Haemophilia, 13, pp. 256-263Lloyd Jones, M., Wight, J., Paisley, S., Knight, C., Control of bleeding in patients with hemophilia A with inhibitors: A systematic review (2003) Haemophilia, 9, pp. 464-520Ewenstein, B.M., Takemoto, C., Warrier, I., Nephrotic syndrome as a complication of immune tolerance in hemophilia B (1997) Blood, 89, pp. 1115-1116Warrier, I., Lenk, H., Saidi, P., Pollmann, H., Tengborn, L., Berntorp, E., Nephrotic syndrome in hemophilia B patients with inhibitors (1998) Haemophilia, 4, p. 248Warrier, I., Management of hemophilia B patients with inhibitors and anaphylaxis (1998) Haemophilia, 4, pp. 574-576Strawczynski, H., Stachewitsch, A., Morgenstern, G., Shaw, M.E., Delivery of care to hemophilic children: Home care versus hospitalization (1973) Pediatrics, 51, pp. 986-991Rabiner, S.F., Telfer, M.C., Fajardo, R., Home transfusions of hemophiliacs (1972) JAMA, 221, pp. 885-887Levine, P., The home therapy program at the New England area hemophilia center (1977) Scand J Haematol, 31, pp. 37-51Soucie, J.M., Symons, J.I., Evatt, B., Brettler, D., Huszti, H., Linden, J., Home-based factor infusion therapy and hospitalization for bleeding complications among males with hemophilia (2001) Haemophilia, 7, pp. 198-206Solovieva, S., Clinical severity of disease, functional disability and health-related quality of life. 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Oxford, Oxford University PressRickard, K.A., Guidelines for therapy and optimal dosages of coagulation factors for treatment of bleeding and surgery in hemophilia (1995) Haemophilia, 1, pp. 8-13Shapiro, A., Gilchrist, G.S., Hoots, W.K., Cooper, H.A., Gastineau, D.A., Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in hemophilia patients with inhibitors undergoing surgery (1998) Thromb Haemost, 80, pp. 773-778Hvid, I., Rodriguez-Merchan, E.C., Orthopaedic surgery in haemophilic patients with inhibitors: An overview (2002) Haemophilia, 8, pp. 288-291Rodriguez-Merchan, E.C., Rocino, A., Literature review of surgery management in inhibitor patients (2004) Haemophilia, 10, pp. 22-29Abshire, T., Kenet, G., Recombinant factor VIIa: Review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors (2004) J Thromb Haemost, 2, pp. 899-909Ingerslev, J., Efficacy and safety of Recombinant Factor VIIa in the prophylaxis of bleeding in various surgical procedures in hemophilic patients with factor VIII and factor IX inhibitors (2000) Semin Thromb Hemost, 26, pp. 425-432Tjønnfjord, G.E., Brinch, L., Gedde-Dahl III, T., Brosstad, F.R., Activated prothrombin complex concentrate (FEIBA) treatment during surgery in patients with inhibitors to FVIII/IX (2004) Haemophilia, 10, pp. 174-178Ingerslev, J., Sorensen, B., Role of recombinant activated factor VII as hemostatic support in orthopedic surgery (2006) TATM, 8, pp. 35-42Obergfell, A., Auvinen, M.K., Mathew, P., Recombinant activated factor VII for haemophilia patients with inhibitors undergoing orthopaedic surgery: A review of the literature (2008) Haemophilia, 14, pp. 233-241Ewenstein, B.M., Valentino, L.A., Journeycake, J.M., Consensus recommendations for use of central venous access devices in hemophilia (2004) Haemophilia, 10, pp. 629-648Morado, M., Jimenez-Yuste, V., Villar, A., Complications of central venous catheters in patients with hemophilia and inhibitors (2001) Haemophilia, 7, pp. 551-556Bollard, C.M., Teague, L.R., Berry, E.W., Ockelford, P.A., The use of central venous catheters (portacaths) in children with hemophilia (2000) Haemophilia, 6, pp. 66-70O'Connell, N., Mc Mahon, C., Smith, J., Recombinant factor VIIa in the management of surgery and acute bleeding episodes in children with hemophilia and high responding inhibitors (2002) Br J Haematol, 116, pp. 632-635Cooper, H.A., Jones, C.P., Campion, E., Roberts, H.R., Hedner, U., Rationale for the use of high dose rFVIIa in a high-titre inhibitor patient with hemophilia B during major orthopaedic procedures (2001) Haemophilia, 7, pp. 517-522Colowick, A.B., Bohn, R.L., Avorn, J., Ewenstein, B.M., Immune tolerance induction in hemophilia patients with inhibitors: Costly can be cheaper (2000) Blood, 96, pp. 1698-1702Brackmann, H.H., Gormsen, J., Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder (1977) Lancet, 2, p. 933Key, N.S., Inhibitors in congenital coagulation disorders (2004) Br J Haematol, 127, pp. 379-391Nilsson, I.M., Berntorp, E., Zettervall, O., Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII (1988) NEJM, 318, pp. 947-950Mathias, M., Khair, K., Hann, I., Liesner, R., Rituximab in the treatment of alloimmune factor VIII and IX antibodies in two children with severe hemophilia (2004) Br J Haematol, 125, pp. 366-368Stasi, R., Brunetti, M., Stipa, E., Amadori, S., Selective B-cell depletion with rituximab for the treatment of patients with acquired hemophilia (2004) Blood, 103, pp. 4424-4428Carcao, M., Ungar, W.J., Feldman, B.M., Cost-utility analysis in evaluating prophylaxis in hemophilia (2004) Haemophilia, 10, pp. 50-57Curtin, J., Misra, A., Teo, J., Webster, B., Lammi, A., Use of Rituximab as an alternative strategy for the management of difficult high titre inhibitors in children with hemophilia A (2004) Haemophilia, 10, p. 57DiMichele, D., Immune tolerance therapy dose as an outcome predictor (2003) Haemophilia, 9, pp. 382-386Mariani, G., Kroner, B., Immune tolerance in hemophilia with factor VIII inhibitors: Predictors of success (2001) Haematologica, 86, pp. 1186-1193DiMichele, D., Inhibitors: Resolving diagnostic and therapeutic dilemmas (2002) Haemophilia, 8, pp. 280-287Lenk, H., The German Registry of immune tolerance treatment in hemophilia-1999 update (2000) Haematologica, 85, pp. 45-47Haya, S., Lopez, M.F., Aznar, J.A., Batlle, J., Immune tolerance treatment in hemophilia patients with inhibitors: The Spanish Registry (2001) Haemophilia, 7, pp. 154-159DiMichele, D.M., Hoots, W.K., Pipe, S.W., Rivard, G.E., Santagostino, E., International workshop on immune tolerance induction: Consensus recomendations (2007) Haemophilia, 13, pp. 1-22Kreuz, W., Mentzer, D., Auerswald, G., Becker, S., Joseph-Steiner, J., Successful immune tolerance therapy of FVIII inhibitor in children after changing from high to intermediate purity FVIII concentrate (1996) Haemophilia, 2, p. 19Rocino, A., Papa, M.L., Salerno, E., Capasso, F., Miraglia, E., de Biasi, R., Immune tolerance induction in hemophilia A patients with high-responding inhibitors to factor VIII: Experience at a single institution (2001) Haemophilia, 7, pp. 33-38DiMichele, D., Rivard, G., Hay, C., Antunes, S., Inhibitors in hemophilia: Clinical aspects (2004) Haemophilia, 10, pp. 140-145Mauser-Bunschoten, E.P., Nieuwenhuis, H.K., Roosendaal, G., van den Berg, H.M., Low-dose immune tolerance induction in hemophilia A patients with inhibitors (1995) Blood, 86, pp. 983-988Almeida, J., Paula, J.C., Toscano, R., Immune tolerance such as salvage therapy in severe hemophilia A patient with ultra high-responders inhibitors (2002) Haemophilia, 8, p. 538Solano, M.H., Ramírez, C., Parra, L., Tratamiento de inhibidores del factor VIII en hemofilia. (1998) Acta Med Colomb, 23, p. 193Carneiro, J.D.A., Bassit, R.P., Villaça, P.R., Sandoval, E.P.N., Silva, C.S.S.S., D'amico, E.A., Low-dose immune tolerance induction in hemophilia A children with inhibitors (2002) Haemophilia, 8, pp. 538-539Wight, J., Paisley, S., Knight, C., Immune tolerance induction in patients with hemophilia A with inhibitors: A systematic review (2003) Haemophilia, 9, pp. 436-463Tengborg, L., Hansson, S., Fasth, A., Lübeck, P.O., Berg, A., Ljung, R., Anaphylactoid reactions and nephrotic syndrome - a considerable risk during factor IX treatment in patients with hemophilia B and inhibitors: A report on the outcome in two brothers (1998) Haemophilia, 4, pp. 854-859Schulman, S., Safety, efficacy and lessons from continuous infusion with rFVIIa (1998) Haemophilia, 4, pp. 564-567Schulman, S., Continuous infusion of recombinant factor VIIa in hemophilic patients with inhibitors: Safety, monitoring, and cost effectiveness (2000) Semin Thromb Hemost, 26, pp. 421-424Pruthi, R.K., Mathew, P., Valentino, L.A., Sumner, M.J., Seremetis, S., Hoots, W.K., Haemostatic efficacy and safety of bolus and continuous infusion of recombinant factor VIIa are comparable in hemophilia patients with inhibitors undergoing major surgery. Results from an open-label, randomized, multicenter trial (2007) Thromb Haemost, 98, pp. 726-732Smith, M.P., Ludlam, C.A., Collins, P.W., Elective surgery on factor VIII inhibitor patients using continuous infusion of recombinant activated factor VII: Plasma factor VII activity of 10 IU/ml is associated with an increased incidence of bleeding (2001) Thromb Haemost, 86, pp. 949-953Santagostino, E., Morfini, M., Rocino, A., Baudo, F., Scaraggi, F.A., Gringeri, A., Relationship between factor VII activity and clinical efficacy of recombinant factor VIIa given by continuous infusion to patients with factor VIII inhibitors (2001) Thromb Haemost, 86, pp. 954-958Mauser-Bunschoten, E.P., Koopman, M.M., Goede-Bolder, A.D., Efficacy of recombinant factor VIIa administered by continuous infusion to hemophilia patients with inhibitors (2002) Haemophilia, 8, pp. 649-656Ludlam, C.A., Smith, M.P., Morfini, M., Gringeri, A., Santagostino, E., Savidge, G.F., A prospective study of recombinant activated factor VII administered by continuous infusion to inhibitor patients undergoing elective major orthopaedic surgery: A pharmacokinetic and efficacy evaluation (2003) Br J Haematol, 120, pp. 808-813Escobar, M.A., Recombinant Factor VIIa: The possibilities for monitoring (2003) TATM, 5, pp. 51-54Young, G., Ebbesen, L.S., Viuff, D., Evaluation of thromboelastography for monitoring recombinant activated factor VII ex
International consensus recommendations on the management of people with haemophilia B
Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if untreated, can cause recurrent and disabling bleeding, potentially leading to severe arthropathy and/or life-threatening haemorrhage. Recent decades have brought significant improvements in haemophilia B management, including the advent of recombinant FIX and extended half-life FIX. This therapeutic landscape continues to evolve with several non-factor replacement therapies and gene therapies under investigation. Given the rarity of haemophilia B, the evidence base and clinical experience on which to establish clinical guidelines are relatively sparse and are further challenged by features that are distinct from haemophilia A, precluding extrapolation of existing haemophilia A guidelines. Due to the paucity of formal haemophilia B-specific clinical guidance, an international Author Group was convened to develop a clinical practice framework. The group comprised 15 haematology specialists from Europe, Australia, Japan, Latin America and North America, covering adult and paediatric haematology, laboratory medicine and biomedical science. A hybrid approach combining a systematic review of haemophilia B literature with discussion of clinical experience utilized a modified Delphi format to develop a comprehensive set of clinical recommendations. This approach resulted in 29 recommendations for the clinical management of haemophilia B across five topics, including product treatment choice, therapeutic agent laboratory monitoring, pharmacokinetics considerations, inhibitor management and preparing for gene therapy. It is anticipated that this clinical practice framework will complement existing guidelines in the management of people with haemophilia B in routine clinical practice and could be adapted and applied across different regions and countries
A novel use of thromboelastography in type 2B von Willebrand disease.
[No abstract available]356e11e14Hampshire, D.J., Goodeve, A.C., The international society on thrombosis and haematosis von Willebrand disease database: an update (2011) Semin Thromb Hemost, 37, pp. 470-479Von Willebrand disease mutation database, , http://www.vwf.group.shef.ac.uk/, Accessed 23 October 2011Othman, M., Favaloro, E.J., Genetics of type 2B von Willebrand disease: "true 2B", "tricky 2B", or "not 2B". What are the modifiers of the phenotype? (2008) Semin Thromb Hemost, 34, pp. 520-531Nurden, P., Debili, N., Vainchenker, W., Bobe, R., Bredoux, R., Corvazier, E., Combrie, R., Enouf, J., Impaired megakaryocytopoiesis in type 2B von Willebrand disease with severe thrombocytopenia (2006) Blood, 108, pp. 2587-2595Federici, A.B., Mannucci, P.M., Stabile, F., Canciani, M.T., Di Rocco, N., Miyata, S., Ware, J., Ruggeri, Z.M., A type 2b von Willebrand disease mutation (Ile546->Val) associated with an unusual phenotype (1997) Thromb Haemost, 78, pp. 1132-1137Takimoto, Y., Imanaka, F., Type 2B Hiroshima: a variant of von Willebrand disease characterized by chronic thrombocytopenia and the presence of all von Willebrand factor multimers in plasma (1999) Int J Hematol, 70, pp. 127-131Castaman, G., Eikenboom, J.C., ABO blood group also influences the von Willebrand factor (VWF) antigen level in heterozygous carriers of VWF null alleles, type 2N mutation Arg854GIn, and the missense mutation Cys2362Phe (2002) Blood, 100, pp. 1927-1928Yuan, Z.H., Zhao, J., Zhang, Y., Zhu, P., [Impact of vWF gene A1381T polymorphism and ABO blood group on von Willebrand factor level in plasma] (2010) Zhongguo Shi Yan Xue Ye Xue Za Zhi, 18, pp. 967-971Fleming, A.F., Ethnic variation in von Willebrand factor levels can influence the diagnosis of von Willebrand disease (2003) Clin Lab Haematol, 25, p. 413Miesbach, W., Berntorp, E., When von Willebrand disease comes into age - a matter of change? (2011) Eur J Haematol, 86, pp. 496-501Ruggeri, Z.M., Mannucci, P.M., Lombardi, R., Federici, A.B., Zimmerman, T.S., Multimeric composition of factor VIII/von Willebrand factor following administration of DDAVP: implications for pathophysiology and therapy of von Willebrand's disease subtypes (1982) Blood, 59, pp. 1272-1278Nichols, W.L., Hultin, M.B., James, A.H., Manco-Johnson, M.J., Montgomery, R.R., Ortel, T.L., Add Rick, M.E., Yawn, B.P., von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA) (2008) Haemophilia, 14, pp. 171-232Chen, A., Teruya, J., Global hemostasis testing thromboelastography: old technology, new applications (2009) Clin Lab Med, 29, pp. 391-407Stammers, A.H., Bruda, N.L., Gonano, C., Hartmann, T., Point-of-care coagulation monitoring: applications of the thromboelastography (1998) Anaesthesia, 53 (SUPPL. 2), pp. 58-59Chitlur, M., Sorensen, B., Rivard, G.E., Young, G., Ingerslev, J., Othman, M., Nugent, D., Lusher, J., Standardization of thromboelastography: a report from the TEG-ROTEM working group (2011) Haemophilia, 17, pp. 532-537Chitlur, M., Lusher, J., Standardization of thromboelastography: values and challenges (2010) Semin Thromb Hemost, 36, pp. 707-711Topf, H.G., Weiss, D., Lischetzki, G., Strasser, E., Rascher, W., Rauh, M., Evaluation of a modified thromboelastography assay for the screening of von Willebrand disease (2011) Thromb Haemost, 105, pp. 1091-109
Non-operative Management Of Blunt Major Hepatic Injury In A Young Adult With Severe Haemophilia A
[No abstract available]183e84e86Carrillo, E.H., Platz, A., Miller, F.B., Richardson, J.D., Polk Jr., H.C., Non-operative management of blunt hepatic trauma (1998) Br J Surg, 85, pp. 461-468Kozar, R.A., McNutt, M.K., Management of adult blunt hepatic trauma (2010) Curr Opin Crit Care, 16, pp. 596-601English, P.J., Sheppard, E.M., Wensley, R.T., Traumatic rupture of the liver in a haemophilic patient with factor-VIII inhibitors (1976) Lancet, 1, pp. 1299-1300Sarihan, H., Erduran, E., Abes, M., Nonsurgical therapy of hepatic injury in a hemophilic child (1994) Eur J Pediatr Surg, 4, pp. 366-367Sartorelli, K.H., Rogers, F.B., Vane, D.W., Nonoperative treatment of a major hepatic injury in a hemophiliac (1995) J Trauma, 38, pp. 246-247Tinkoff, G., Esposito, T.J., Reed, J., American Association for the Surgery of Trauma Organ Injury Scale I: spleen, liver, and kidney, validation based on the National Trauma Data Bank (2008) J Am Coll Surg, 207, pp. 646-655Jona, J.Z., Cox-Gill, J., Nonsurgical therapy of splenic rupture in a hemophiliac (1992) J Pediatr Surg, 27, pp. 523-524Fort, D.W., Bernini, J.C., Johnson, A., Cochran, C.J., Buchanan, G.R., Splenic rupture in hemophilia (1994) Am J Pediatr Hematol Oncol, 16, pp. 255-259Zieg, P.M., Cohn, S.M., Beardsley, D.S., Nonoperative management of a splenic tear in a Jehovah's Witness with hemophilia (1996) J Trauma, 40, pp. 299-301Sharma, O.P., Oswanski, M.F., Issa, N.M., Stein, D.T., Role of non-operative management of spleen injury in patients with hemophilia: report of two patients with review of literature (2011) J Emerg Med, 41, pp. e59-e6
Factor Viii Inhibitors In Patients With Congenital Severe Haemophilia A And Its Relation To Genotype
[No abstract available]186e411e414Graw, J., Brackmann, H.H., Oldenburg, J., Schneppenheim, R., Spannagl, M., Schwaab, R., Haemophilia A: from mutation analysis to new therapies (2005) Nat Rev Genet, 6, pp. 488-501Gouw, S.C., van den Berg, H.M., The multifactorial etiology of inhibitor development in haemophilia: genetics and environmental (2009) Semin Tromb Hemost, 35, pp. 723-734Oldenburg, J., Pavlova, A., Genetic risk factors for inhibitors to factors VIII and IX (2006) Haemophilia, 12 (SUPPL. 6), pp. 15-22Margaglione, M., Castaman, G., Morfini, M., The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype (2008) Haematologica, 93, pp. 722-728Antonarakis, S.E., Rossiter, J.P., Young, M., Factor VIII gene inversion in sevee hemophilia A: results of an international consortium study (1995) Blood, 86, pp. 2206-2212Leiria, L., Roisenberg, I., Salzano, F., Bandinelli, E., Intron 1 and 22 invrsions and factor VIII inhibitors patients with severe haemophilia A in southern Brazil (2009) Haemophilia, 15, pp. 309-313Gouw, S., Van Der Bom, J.G., Van Den Berg, H.M., Influence of the type F8 gene mutation on inhibitor development in a single centre cohort of severe haemopilia A patients (2011) Haemophilia, 17, pp. 275-281Viel, K.R., Ameri, A., Abshire, T.C., Inhibitors of Factor VIII in black patients with hemophilia (2009) N Engl J Med, 360, pp. 1618-1627Santos, A., Annichino-Bizzacchi, J.M., Ozelo, M.C., Inhibitors of factor VIII in haemophilia (2009) N Engl J Med, 361, pp. 309-31
Routine clinical care data for population pharmacokinetic modeling: the case for Fanhdi/Alphanate in hemophilia A patients
Fanhdi/Alphanate is a plasma derived factor VIII concentrate used for treating hemophilia A, for which there has not been any dedicated model describing its pharmacokinetics (PK). A population PK model was developed using data extracted from the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) project. WAPPS-Hemo provided individual PK profiles for hemophilia patients using sparse observations as provided in routine clinical care by hemophilia centers. Plasma factor activity measurements and covariate data from hemophilia A patients on Fanhdi/Alphanate were extracted from the WAPPS-Hemo database. A population PK model was developed using NONMEM and evaluated for suitability for Bayesian forecasting using prediction-corrected visual predictive check (pcVPC), cross validation, limited sampling analysis and external evaluation against a population PK model developed on rich sampling data. Plasma factor activity measurements from 92 patients from 12 centers were used to derive the model. The PK was best described by a 2-compartment model including between subject variability on clearance and central volume, fat free mass as a covariate on clearance, central and peripheral volumes, and age as covariate on clearance. Evaluations showed that the developed population PK model could predict the PK parameters of new individuals based on limited sampling analysis and cross and external evaluations with acceptable precision and bias. This study shows the feasibility of using real-world data for the development of a population PK model. Evaluation and comparison of the model for Bayesian forecasting resulted in similar results as a model developed using rich sampling data.4642743
Individualised Prophylaxis with Recombinant Factor IX FC Fusion Protein (RFIXFC) in Adults/Adolescents With Haemophilia B: Updated Interim Results of the B-YOND Extension Study
Poster presentatio