Long-term efficacy and safety of a pasteurized, plasma-derived factor VIII concentrate (Beriate® P) in patients with haemophilia A

Abstract

Beriate(®) P has an excellent efficacy and safety profile. Many patients who were initiated on Beriate(®) P at our centre remain on the treatment today.

Quantitative HLA‐class‐II/factor VIII (FVIII) peptidomic variation in dendritic cells correlates with the immunogenic potential of therapeutic FVIII proteins in hemophilia A

“…In summary, our findings from the MAPPs-analyses performed herein demonstrate its utility for identifying and characterizing the molecular-determinants of FVIII-immunogenicity. Consistent-with our Gate-Keeper-hypothesis, we have shown that significant HLAcII/FVIII-peptidome-level differences likely mediate the distinct risks of inhibitor-development observed clinically for various FVIIIs including: 1) pdFVIIIs vs.-rFVIIIs; 2) non-engineered-vs.-engineered-rFVIIIs; and-3) next-generation-vs.-original-FDA-approved-rFVIIIs [34][35][36][37][38][39]75]. We showed that pre-incubation of FVIIIs with pdVWF significantly-decreases their HLAcIIbound/FVIII-derived-peptide counts, and that this protective-effect is differentially-manifested across rFVIIIs.…”

“…The pdFVIII studied originated as Beriate ® (CSL Behring GmbH), which contains pdVWF (Figure S1) at concentrations varying lot to lot . As described previously, pdVWF in the combined reconstituted Beriate was first removed and the VWF‐free pdFVIII (Figure ) was DC‐PPPA‐tested as pdFVIII + pdVWF after add‐back of pdVWF at 13.4‐molar‐excess using a FVIII‐free pdVWF concentrate (see Formulations of FVIIIs) .…”

“…The pdFVIII-studied originated as Beriate ® (CSL-Behring-GmbH) which contains-pdVWF-(Figure -S1) at concentrations varying lot-to-lot [34].…”

“…The pdFVIII studied originated as Beriate ® (CSL Behring GmbH), which contains pdVWF (Figure S1) at concentrations varying lot to lot. 34 As described previously, 24,32 pdVWF in the combined reconstituted Beriate was first removed and the VWF-free pdFVIII (Figure 1) was DC-PPPAtested as pdFVIII + pdVWF after add-back of pdVWF at 13.4-molar-excess using a FVIII-free pdVWF concentrate (see Formulations of FVIIIs). [22][23][24]32,33 The five rFVIIIs studied (Figure 1) originated as Advate ® (Takeda), a full-length (FL) product termed FL-rFVIII, 35 and Xyntha ® (Pfizer), NovoEight ® (Novo-Nordisk), Nuwiq ® (Octapharma), and Afstyla ® (CSL Behring GmbH), engineered B-domain (BD)-deleted (BDD) products termed BDD-rFVIII 1 , BDD-rFVIII 2 , BDD-rFVIII 3 , and BDD-rFVIII 4 .…”

Platelet concentrates: reducing the risk of transfusion-transmitted bacterial infections

“…Well-known examples of the latter are chikungunya 70 and dengue virus, 71 but many other viruses, often without a disease caused by the virus, have been repeatedly mentioned in this respect. [72][73][74] Due to the nature of the methods in use for plasma products, like pasteurization, 75 solvent-detergent treatment, 70 freeze-drying 76 and nanofiltration, 77,78 these methods cannot be applied to cellular products.…”