Investigational drugs for coagulation disorders

Bibliography

• Darby SC, Kan SW, Spooner RJ, et al. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV. Blood 2007;110:815-25
   PubMed Web of Science ®Google Scholar
• Soucie JM, Nuss R, Evatt B, et al. Mortality among males with hemophilia: relations with source of medical care. The Hemophilia Surveillance System Project Investigators. Blood 2000;96:437-42
   PubMed Web of Science ®Google Scholar
• Plug I, Van Der Bom JG, Peters M, et al. Mortality and causes of death in patients with hemophilia, 1992-2001: a prospective cohort study. J Thromb Haemost 2006;4:510-16
   PubMed Web of Science ®Google Scholar
• Tagliaferri A, Rivolta GF, Iorio A, et al. Mortality and causes of death in Italian persons with haemophilia, 1990-2007. Haemophilia 2010;16:437-46
   PubMed Web of Science ®Google Scholar
• Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357:535-44
   PubMed Web of Science ®Google Scholar
• Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011;9:700-10
   PubMed Web of Science ®Google Scholar
• Available from: www.clinicaltrials.gov [Last access on 9 April 2013]
   Google Scholar
• Negrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011;118:2695-701
   PubMed Web of Science ®Google Scholar
• Collins PW, Møss J, Knobe K, et al. Population pharmacokinetic modelling for dose setting of nonacog beta pegil (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost 2012;10:2305-12
   PubMed Web of Science ®Google Scholar
• Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant fusion IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012;119:666-72
   PubMed Web of Science ®Google Scholar
• Metzner HJ, Weimer T, Kronthaler U, et al. Genetic fusion to albumin improves the pharmacokinetic properties of factor IX. Thromb Haemost 2009;102:634-44
   PubMed Web of Science ®Google Scholar
• Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood 2012;120:2405-11
   PubMed Web of Science ®Google Scholar
• Thim L, Vandhal B, Karlsson J, et al. Purification and characterization of a new recombinant factor VIII (N8). Haemophilia 2010;16:349-59
   PubMed Web of Science ®Google Scholar
• Agersø H, Stennicke HR, Pelzer H, et al. Pharmacokinetics and pharmacodynamics of turoctocog alfa and N8-GP in haemophilia dogs. Haemophilia 2012;18:941-7
   PubMed Web of Science ®Google Scholar
• Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood 2012;119:3031-7
   PubMed Web of Science ®Google Scholar
• Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-man trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost 2013; Epub ahead of print
   PubMed Web of Science ®Google Scholar
• Baru M, Carmal-Goren L, Barenholz Y, et al. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost 2005;93:1061-8
   PubMed Web of Science ®Google Scholar
• Spira J, Plyushch OP, Andreeva TA, et al. Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes. Blood 2006;108:3668-73
   PubMed Web of Science ®Google Scholar
• Powell JS, Nugent DJ, Harrison JA, et al. Safety and pharmacokinetics of a recombinant factor VIII with pegylated liposomes in severe hemophilia A. J Thromb Haemost 2008;6:277-83
   PubMed Web of Science ®Google Scholar
• Powell J, Martinowitz U, Windyga J, et al. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients. Thromb Haemost 2012;108:913-22
   PubMed Web of Science ®Google Scholar
• Mei B, Pan C, Jiang H, et al. Rational design of a fully active, long-active PEGylated factor VIII for hemophilia A treatment. Blood 2010;116:270-9
   PubMed Web of Science ®Google Scholar
• Kaliwoda M, Graninger M, Matthiessen P, et al. Structural characterization of BAX 855, a PEGylated recombinant FVIII. Blood 2011;118:abstract 4356
   Google Scholar
• Schrenk G, Varadi K, Gritsch H, et al. Functional characterization of BAX 855, a PEGylated recombinant FVIII. Blood 2011;118:abstract 4359
   Google Scholar
• Dietrich B, Spatzenegger M, Stidl R, et al. Safety of Baxter's longer acting rFVIII (BAX 855) after repeated application in rats and cynomolgus monkeys. Blood 2011;118:abstract 4345
   Google Scholar
• Hoebarth G, Kubik S, Wolfsegger M, et al. Pharmacokinetics of Baxter's longer acting rFVIII (BAX 855) in factor VIII KO mice, rats and cynomolgus monkeys. Blood 2011;118:abstract 4346
   Google Scholar
• Dietrich B, Kubik S, Ehrlich HJ, et al. Preclinical safety pharmacology of Baxter's longer acting rFVIII (BAX 855). Blood 2011;118:abstract 4357
   Google Scholar
• Holmberg H, Elm T, Karpf D, et al. GlycoPEGylated rFVIIa (N7-GP) has a prolonged hemostatic effect in hemophilic mice compared with rFVIIa. J Thromb Haemost 2011;9:1070-2
   PubMed Web of Science ®Google Scholar
• Møss J, Rosholm A, Laurén A. Safety and pharmacokinetics of a glycoPEGylated recombinant activated factor VII derivative: a randomized first human dose trila in healthy subjects. J Thromb Haemost 2011;9:1368-74
   PubMed Web of Science ®Google Scholar
• Ljung R, Karim FA, Saxena K, et al. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics, and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost 2013; doi: 10.1111/jth.12237
   Web of Science ®Google Scholar
• Allen GA, Persson E, Campbell RA, et al. A variant of recombinant FVIIa with enhanced procoagulant and antifibrinolytic activities in an in vitro model of hemophilia. Arterioscler Thromb Vasc Biol 2007;27:683-9
   PubMed Web of Science ®Google Scholar
• Sørensen B, Persson E, Ingerslev J. Factor VIIa analogue (V158D/E296V/M298Q-FVIIa) normalizes clot formation in whole blood from patients with severe haemophilia A. Br J Haematol 2007;137:158-65
   PubMed Web of Science ®Google Scholar
• Møss J, Scharling B, Ezban M, et al. Evaluation of the safety and pharmacokinetics of a fast-acting recombinant FVIIa analogue, NN1731, in healthy male subjects. J Thromb Haemost 2009;7:299-305
   PubMed Web of Science ®Google Scholar
• Liu T, Zhang X, Pan J, et al. Enhanced and prolonged efficacy of a novel recombinant FVIIa variant (BAY86-6150) for acute and prophylactic treatments in hemophilia A (HemA) mice. J Thromb Haemost 2009;7(Suppl 2):abstract OC-WE-057
   Google Scholar
• Sinclair AM, Elliott S. Glycoengineering: the effect of glycosylation on the properties of therapeutic proteins. J Pharm Sci 2005;94:1626-35
   PubMed Web of Science ®Google Scholar
• Mahlangu JN, Coetzee MJ, Laffan M, et al. Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. J Thromb Haemost 2012;10:773-80
   PubMed Web of Science ®Google Scholar
• Yatuv R, Dayan I, Carmel-Goren L, et al. Enhancement of factor VIIa haemostatic efficacy by formulation with PEGylated liposomes. Haemophilia 2008;14:476-83
   PubMed Web of Science ®Google Scholar
• Yatuv R, Robinson M, Dayan I, et al. Enhancement of the efficacy of therapeutic proteins by formulation with PEGylated liposomes; a case of FVIII, FVIIa and G-CSF. Expert Opin Drug Deliv 2010;7:187-201
   PubMed Web of Science ®Google Scholar
• Spira J, Plyushch O, Zozulya N, et al. Safety, pharmacokinetics and efficacy of factor VIIa formulated with PEGylated liposomes in haemophilia A patients with inhibitors to factor VIII – an open label, exploratory, cross-over, phase I/II study. Haemophilia 2010;16:910-18
   PubMed Web of Science ®Google Scholar
• Doering CB, Healey JF, Parker ET, et al. High level expression of recombinant porcine coagulation Factor VIII. J Biol Chem 2002;277:38345-9
   PubMed Web of Science ®Google Scholar
• Kernoff PBA, Thomas ND, Lilley PA, et al. Clinical experience with polyelectrolyte-fractionated porcine factor VIII concentrate in the treatment of hemophiliacs with antibodies to factor VIII. Blood 1984;63:31-41
   PubMed Web of Science ®Google Scholar
• Kempton CL, Abshire TC, Deveras RA, et al. Pharmacokinetics and safety of OBI-1, a recombinant B domain-deleted porcine factor VIII, in subjects with haemophilia A. Haemophilia 2012;12:798-804
   Google Scholar
• Guidelines on the clinical investigation of recombinant and human plasma-derived factor VIII products; 21 July 2011 EMA/CHMP/BPWP/144533/2009
   Google Scholar
• Guidelines on the clinical investigation of recombinant and human plasma-derived factor IX products; 21 July 2011 EMA/CHMP/BPWP/144552/2009
   Google Scholar
• Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor X concentrates. Blood 2001;98:3600-6
   PubMed Web of Science ®Google Scholar
• Szebeni J. Complement activation-related pseudoallergy: a new class of drug-induced acute immune toxicity. Toxicology 2005;216:106-21
   PubMed Web of Science ®Google Scholar
• Szebeni J, Muggia F, Gabizon A, et al. Activation of complement by therapeutic liposomes and other lipid excipient-based therapeutic products: prediction and prevention. Adv Drug Deliv Rev 2011;63:1020-30
   PubMed Web of Science ®Google Scholar