Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
Condition: Hemophilia A Intervention: Biological: valoctocogene roxaparvovec Sponsor: BioMarin Pharmaceutical Not yet recruiting
An experimental gene therapy for hemophilia A from BioMarin Pharmaceutical Inc. — promising a one-shot fix for the genetic cause of the bleeding disorder — continues to hold patients' bleeds to less than one a year after four years. The results of the ongoing study are important as San Rafael-based BioMarin's (NASDAQ: BMRN) gene therapy, called Roctavian and known scienti fically as valoctogene roxaparvovec, nears an Aug. 21 decision date from the Food and Drug Administration. If approved, it…
roup Abstract BACKGROUND: von Willebrand factor (VWF) is crucial for optimal dosing of factor VIII (FVIII) concentrate in hemophilia A patients as it protects FVIII from premature clearance. To date, it is unknown how VWF behaves and what its impact is on FVIII clearance in the perioperative setting. AIM: To investigate VWF kinetics (VWF antigen [VWF:Ag]), VWF glycoprotein Ib binding (VWF:GPIbM), and VWF propeptide (VWFpp) in severe and moderate perioperative hemophilia A patients included in the randomized controlled perioperative OPTI-CLOT trial. METHODS: Linear mixed effects modeling...
Conditions: Hemophilia; Arthropathy of Knee; Arthropathy of Ankle or Foot Intervention: Sponsor: Necmettin Erbakan University Completed
This article describes a patient successfully treated with recombinant factor VIIa, porcine factor VIII, plasmapheresis, rituximab, and high-dose corticosteroids.
Patients with hemophilia are at a high risk of developing arthropathy and so are more likely to require surgery during their lifetime; in particular, major orthopedic surgery . The management of patients with hemophilia undergoing surgery has additional considerations, as they are more likely to experience excessive or prolonged bleeding and associated complications, compared with patients without hemophilia .
Classification of the X-linked recessive bleeding disorder Hemophilia A (HA) is based on residual Factor VIII activity level (FVIII:C) that largely correlates with bleeding severity. Understanding the underlying basis for bleeding phenotypes differing from those predicted by FVIII:C should refine classification of patients, clarify genotype-phenotype correlations, and improve clinical management of persons with HA.
Hemophilia A is a bleeding disorder due to the deficiency of coagulation factor VIII (FVIII). According to resident FVIII activity, the disease was divided into mild type (FVIII:c > 5 IU/dL), moderate type (FVIII:c 1–5 IU/dL) and severe type (FVIII:c
Female hemophilia is an intriguing rare disorder and few larger reports on its genetic etiology are available. While historically the diagnosis was satisfactorily reached by factor VIII activity assays, the clinical and potentially therapeutic heterogeneity of female hemophilia calls for comprehensive molecular diagnosis in each case. Currently, the genetic investigations are not a part of routine, state-funded, diagnostics in Poland, and thus molecular epidemiological data are missing.