PK‐guided personalized prophylaxis with Nuwiq® (human‐cl rhFVIII) in adults with severe haemophilia A

Nuwiq® (human‐cl rhFVIII) is a 4th generation recombinant human FVIII, without chemical modification or protein fusion, produced in a human cell‐line.

[1]  D. Andersson,et al.  Efficacy and safety , 2018 .

[2]  E. Santagostino,et al.  Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A. , 2016, Blood.

[3]  J. Oldenburg,et al.  Prophylaxis vs. on‐demand treatment with Nuwiq® (Human‐cl rhFVIII) in adults with severe haemophilia A , 2016, Haemophilia : the official journal of the World Federation of Hemophilia.

[4]  Y. T. van der Schouw,et al.  Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates , 2016, Thrombosis and Haemostasis.

[5]  R. Ljung,et al.  Practical considerations in choosing a factor VIII prophylaxis regimen: Role of clinical phenotype and trough levels , 2016, Thrombosis and Haemostasis.

[6]  J. Oldenburg,et al.  Novel, human cell line‐derived recombinant factor VIII (human‐cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety , 2016, Haemophilia : the official journal of the World Federation of Hemophilia.

[7]  R. Liesner,et al.  Novel, human cell line‐derived recombinant factor VIII (Human‐cl rhFVIII, Nuwiq®) in children with severe haemophilia A: efficacy, safety and pharmacokinetics , 2016, Haemophilia : the official journal of the World Federation of Hemophilia.

[8]  S. Garger,et al.  Pharmacokinetic properties of BAY 81‐8973, a full‐length recombinant factor VIII , 2015, Haemophilia : the official journal of the World Federation of Hemophilia.

[9]  B. Konkle,et al.  Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. , 2015, Blood.

[10]  L. Valentino Considerations in individualizing prophylaxis in patients with haemophilia A , 2014, Haemophilia : the official journal of the World Federation of Hemophilia.

[11]  E. Berntorp,et al.  Methods for individualising factor VIII dosing in prophylaxis , 2014, European journal of haematology. Supplementum.

[12]  J. Dumont,et al.  Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. , 2014, Blood.

[13]  S. Pérez-Alenda,et al.  Secondary prophylaxis vs. on‐demand treatment to improve quality of life in severe adult haemophilia A patients: a prospective study in a single centre , 2014, Vox sanguinis.

[14]  L. Valentino,et al.  The first recombinant FVIII produced in human cells – an update on its clinical development programme , 2014, Haemophilia : the official journal of the World Federation of Hemophilia.

[15]  A. Klukowska,et al.  Efficacy, Immunogenicity, Pharmacokinetics, and Safety Of Human-cl rhFVIII – a GCP Study In Children With Severe Haemophilia A , 2013 .

[16]  C. Kempton,et al.  Randomized, controlled, parallel‐group trial of routine prophylaxis vs. on‐demand treatment with sucrose‐formulated recombinant factor VIII in adults with severe hemophilia A (SPINART) , 2013, Journal of thrombosis and haemostasis : JTH.

[17]  M. Franchini The modern treatment of haemophilia: a narrative review. , 2013, Blood transfusion = Trasfusione del sangue.

[18]  J. Mahlangu,et al.  Guidelines for the management of hemophilia , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[19]  M. Ramström,et al.  Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII. , 2013, Thrombosis Research.

[20]  O. Walter,et al.  Functional characteristics of the novel, human-derived recombinant FVIII protein product, human-cl rhFVIII. , 2012, Thrombosis research.

[21]  K. Martinelle,et al.  The first recombinant human coagulation factor VIII of human origin: human cell line and manufacturing characteristics , 2012, European journal of haematology.

[22]  J. Dumont,et al.  Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. , 2012, Blood.

[23]  L. Valentino,et al.  A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management , 2012, Journal of thrombosis and haemostasis : JTH.

[24]  M. Makris Prophylaxis in haemophilia should be life-long. , 2012, Blood transfusion = Trasfusione del sangue.

[25]  T. Humphries,et al.  Consequences of Switching From Prophylactic Treatment to On-Demand Treatment in Late Teens and Early Adults with Severe Hemophilia A. the TEEN/TWEN Study , 2011 .

[26]  M. Morfini,et al.  Bioequivalence between two serum‐free recombinant factor VIII preparations (N8 and ADVATE®) – an open‐label, sequential dosing pharmacokinetic study in patients with severe haemophilia A , 2011, Haemophilia : the official journal of the World Federation of Hemophilia.

[27]  L. Mantovani,et al.  A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study) , 2011, Journal of thrombosis and haemostasis : JTH.

[28]  M. Morfini,et al.  Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia , 2011, Haemophilia : the official journal of the World Federation of Hemophilia.

[29]  A. Coppola,et al.  Treatment of hemophilia: a review of current advances and ongoing issues , 2010, Journal of blood medicine.

[30]  M. Morfini,et al.  Efficacy and safety of secondary prophylactic vs. on‐demand sucrose‐formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13‐month crossover study , 2010, Journal of thrombosis and haemostasis : JTH.

[31]  J. Stockman Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia , 2009 .

[32]  M. van den Berg,et al.  The Nijmegen Modification of the Bethesda Assay for Factor VIII:C Inhibitors: Improved Specificity and Reliability , 1995, Thrombosis and Haemostasis.

[33]  P. Mannucci,et al.  Modern treatment of haemophilia. , 1995, Bulletin of the World Health Organization.

[34]  C H Chen,et al.  A New Look at the Statistical Pattern Recognition. , 1977 .

[35]  H. Akaike A new look at the statistical model identification , 1974 .