Vladimir Vdovin

Novel, human cell line‐derived recombinant factor VIII (Human‐cl rhFVIII, Nuwiq®) in children with severe haemophilia A: efficacy, safety and pharmacokinetics

Nuwiq® (Human‐cl rhFVIII) is a new‐generation recombinant factor VIII (rFVIII) protein, without chemical modification or fusion to any other protein, produced in a human cell line.

First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study

Development of neutralizing inhibitors against factor VIII (FVIII) is a major complication of haemophilia A treatment.

Efficacy and safety of OCTANINE F in children with haemophilia B

Summary.  OCTANINE F is a high‐purity blood clotting factor IX concentrate that has been shown to be effective and safe in adults with haemophilia B. At present, there are no prospective clinical study data on FIX replacement therapy in young children. The primary objective of this trial was to investigate the immunogenicity of OCTANINE F in children aged <6 years with haemophilia B. Secondary objectives were to assess the efficacy, viral safety and tolerability of OCTANINE F in this patient population. Twenty‐five children aged <6 years with moderate or severe haemophilia B, including six who were previously untreated and 13 who had less than previous 50 exposure days were assigned to prophylactic or on‐demand treatment with OCTANINE F over a 12‐ to 24‐month period. Immunogenicity was assessed at baseline, during the treatment period and at the end of treatment by monitoring the levels of inhibitor. OCTANINE F was not associated with the development of an inhibitor in any patient during the study; all patients had a FIX inhibitor level of <0.4 Bethesda units (BU) for all samples taken throughout the study. The efficacy of OCTANINE F was rated as excellent in 96.4% of 499 bleeding episodes and tolerability was rated as very good in 97% of 1684 injections. OCTANINE F was shown to be effective and well tolerated in children aged <6 years with moderate or severe haemophilia B, including previously untreated patients, with no reported cases of FIX inhibitors or thrombotic events.

Immunogenicity, efficacy and safety of Nuwiq® (human-cl rhFVIII) in previously untreated patients with severe haemophilia A—Interim results from the NuProtect Study

Nuwiq® (Human‐cl rhFVIII) is a fourth generation recombinant FVIII, produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq® in 201 previously treated patients with haemophilia A (HA). The immunogenicity, efficacy and safety of Nuwiq® in previously untreated patients (PUPs) with severe HA are being assessed in the ongoing NuProtect study.

Low incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A treated with octanate®: Final report from a prospective study

Octanate® is a human, plasma‐derived, von Willebrand factor‐stabilized coagulation factor VIII (FVIII) concentrate with demonstrated haemostatic efficacy in previously treated patients with haemophilia A.

Long-term tolerability, immunogenicity and efficacy of Nuwiq® (human-cl rhFVIII) in children with severe haemophilia A

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