HEMA Biologics(TM) Announces FDA Approval of SEVENFACT(R) coagulation factor VIIa (recombinant)-jncw for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors
First new bypassing agent approved for the treatment and control of bleeding episodes in hemophilia A and B patients with inhibitors in over 2 decades Median number of infusions required to achieve bleeding control in the first 12 hours was 1 (225 mcg/k... Biopharmaceuticals, FDA HEMA Biologics, SEVENFACT, coagulation factor VIIa, hemophilia
ConclusionsEmicizumab pharmacokinetics in PwHA was described with dose-independent parameters. Body weight was an important predictor of emicizumab pharmacokinetics. All three dosing regimens are predicted to achieve similar exposure associated with clinically meaningful prevention of bleeding.
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...
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
Iliacus hematoma syndrome (IHS) is characterized as a retroperitoneal compartment neuropathy caused by bleeding within the iliacus muscle leading to hematoma formation and compression upon the femoral nerve . Subsequently, it shows symptoms such as weakness of the iliopsoas muscle, numbness, loss of knee-jerk, and sensory loss of anteromedial thigh . IHS may be precipitated by trauma alone but have characteristically been described in patients with hemophilia and those on anticoagulation therapy [3, 4].
Condition: Hemophilia A Intervention: Biological: Moroctocog-alfa (AF-CC) Sponsor: Pfizer Recruiting