Prophylaxis vs. on‐demand treatment with Nuwiq® (Human‐cl rhFVIII) in adults with severe haemophilia A

Haemophilia A is treated with FVIII, either prophylactically or on demand. Prophylaxis is the gold standard in children and evidence is accumulating in adults.

[1]  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.

[2]  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.

[3]  C. Peterfy,et al.  Effect on Joint Health of Routine Prophylaxis with Bayer’s Sucrose-Formulated Recombinant Factor VIII (rFVIII-FS) in Adolescents and Adults Previously Treated on Demand: MRI Analyses from the 3-Year Spinart Study , 2014 .

[4]  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.

[5]  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.

[6]  K. Fischer,et al.  Treatment for life for severe haemophilia A– A cost‐utility model for prophylaxis vs. on‐demand treatment , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[7]  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.

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

[9]  J. P. Dijk,et al.  A survey of the outcome of prophylaxis, on‐demand treatment or combined treatment in 18–35‐year old men with severe haemophilia in six countries , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

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

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

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

[13]  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.

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

[15]  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.

[16]  R. Engelbert,et al.  Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: Validity of the hemophilia joint health score , 2011, Arthritis care & research.

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

[18]  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.

[19]  A. Joshi,et al.  Health‐related quality of life and productivity impact in haemophilia patients with inhibitors , 2009, Haemophilia : the official journal of the World Federation of Hemophilia.

[20]  P. Mannucci,et al.  Effects of secondary prophylaxis started in adolescent and adult haemophiliacs , 2008, Haemophilia : the official journal of the World Federation of Hemophilia.

[21]  G. Dolan,et al.  European Study on Orthopaedic Status of haemophilia patients with inhibitors , 2007, Haemophilia : the official journal of the World Federation of Hemophilia.

[22]  Alan R. Cohen,et al.  Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. , 2007, The New England journal of medicine.

[23]  B. Feldman,et al.  Hemophilia joint health score reliability study , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[24]  S. Darby,et al.  The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977–99 , 2004, Journal of thrombosis and haemostasis : JTH.

[25]  L. Mantovani,et al.  Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. , 2003, Blood.

[26]  F. Rosendaal,et al.  Definitions in Hemophilia , 2001, Thrombosis and Haemostasis.

[27]  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.

[28]  A. Kobata,et al.  Comparative study of the sugar chains of factor VIII purified from human plasma and from the culture media of recombinant baby hamster kidney cells. , 1992, The Journal of biological chemistry.

[29]  J. Kamerling,et al.  Sialylated carbohydrate chains of recombinant human glycoproteins expressed in Chinese hamster ovary cells contain traces of N‐glycolylneuraminic acid , 1990, FEBS letters.

[30]  Prophylaxis in haemophilia. , 1971, Lancet.