scholarly journals Clinical evaluation of safety and efficacy of a plasma-derived factor VIII (CROSS EIGHT M) in previously untreated patients with hemophilia A

2006 ◽  
Vol 17 (6) ◽  
pp. 682-694
Author(s):  
Akira YOSHIOKA ◽  
Akira SHIRAHATA ◽  
Katsuyuki FUKUTAKE ◽  
Tadashi KAMIYA ◽  
Michio FUJIMAKI
Keyword(s):  
Factor Viii ◽  
Clinical Evaluation ◽  
Hemophilia A ◽  
Safety And Efficacy

Sucrose Formulated Recombinant Human Antihemophilic Factor VIII Is Safe and Efficacious for Treatment of Hemophilia A in Home Therapy

2000 ◽  
Vol 83 (06) ◽  
pp. 811-816 ◽  
Author(s):  
E. Gorina ◽  
E. Kellermann ◽  
E. Vosburgh ◽  
T. C. Abshire ◽  
H.-H. Brackmann ◽  
...  
Keyword(s):  
Factor Viii ◽  
Hemophilia A ◽  
De Novo ◽  
Pharmacokinetic Profile ◽  
Full Length ◽  
Home Therapy ◽  
Safety And Efficacy ◽  
Previously Treated Patients ◽  
New Formulation ◽  
Antihemophilic Factor

SummaryTo add an increased level of safety to antihemophilic factor replacement therapy, a full-length, recombinant Factor VIII (rFVIII) product has been developed without human-derived plasma proteins during purification and formulation and using an additional solvent/detergent viral inactivation step. This first clinical trial of a sucrose-formulated full-length rFVIII (rFVIII-FS) was conducted in previously treated patients (≥100 prior exposure days) with severe (<2% FVIII) hemophilia A in North America (NA) and Europe (EU). Pharmacokinetic profiles for rFVIII-FS were compared with those of currently licensed rFVIII product (Kogenate®) in 35 patients. Safety and efficacy during home therapy were evaluated in 71 patients. The new formulation displayed a pharmacokinetic profile similar to that of rFVIII. Patients on home therapy received a cumulative total of 11,867 exposure days, 12,546 infusions, and 22,443,694 IU of rFVIII-FS. Of 2585 bleeds, 93.5% were treated with 1-2 infusions and 80.5% of responses were rated as excellent or good. No evidence of de novo inhibitor formation was observed. Only 0.27% of infusions were associated with any drugrelated adverse event. Except for an episode of intermittent chest pain with palpitations which ceased after treatment with analgesics, associated adverse events were mild or moderate. Overall, rFVIII-FS provided excellent hemostatic control, was well-tolerated, and caused no significant adverse effects, thus demonstrating safety and efficacy for treatment of bleeds in patients with hemophilia A.

scholarly journals Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A: Results of the PROTECT VIII clinical trial

2019 ◽  
Vol 183 ◽  
pp. 13-19 ◽  
Author(s):  
Elena Santagostino ◽  
Shadan Lalezari ◽  
Mark T. Reding ◽  
Jonathan Ducore ◽  
Heng Joo Ng ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Factor Viii ◽  
Half Life ◽  
Hemophilia A ◽  
Severe Hemophilia ◽  
Safety And Efficacy ◽  
Life Factor

Recombinant B-Domain Deleted Porcine Factor VIII (OBI-1) Safety and Efficacy in the Treatment of Acquired Hemophilia A: Interim Results.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2224-2224
Author(s):  
Jean St. Louis ◽  
Rebecca Kruse-Jarres ◽  
Anne Greist ◽  
Amy D. Shapiro ◽  
Hedy Smith ◽  
...  
Keyword(s):  
Factor Viii ◽  
Clinical Assessment ◽  
Research Funding ◽  
Hemophilia A ◽  
Cross Reactivity ◽  
Acquired Hemophilia A ◽  
Acquired Hemophilia ◽  
Open Label ◽  
Serious Adverse Events ◽  
Safety And Efficacy

Abstract Abstract 2224 Introduction OBI-1 is an investigational B-domain deleted recombinant porcine factor VIII (FVIII) with low cross-reactivity to anti-human FVIII antibodies. Acquired hemophilia A (AHA) is caused by autoantibodies (inhibitors) against human FVIII. Patients are predominantly elderly and have co-morbidities. Current pharmacologic treatment of bleeds is guided by clinical assessment alone as there is no laboratory surrogate for efficacy. Importantly, OBI-1 efficacy can be monitored by FVIII levels in addition to clinical assessment. Methods Accur8 Auto-antibody trial (NCT01178294) is a prospective, open label, Phase 2/3 study. The primary objective is to evaluate efficacy of OBI-1 treatment for serious (life- or limb-threatening) bleeds in patients ≥18 years with AHA. FVIII levels are obtained before and within 10–20 min following initial OBI-1 dose (200U/kg) and at 2–3 h. Additional OBI-1 doses (≤400U/kg every 2–3 h) are administered to achieve target FVIII levels. The primary efficacy outcome is the control of bleeding 24 h after starting OBI-1. Results As of July, 2012, fifteen patients with severe bleeds were entered into the trial along with one individual treated under compassionate use and all had successful control of hemorrhage at 24 h and subsequent resolution of the bleed. Therapeutic FVIII activity levels were achieved and maintained with intermittent OBI-1 administration based on FVIII levels. Six serious adverse events were reported including four deaths after treatment was discontinued, all being unrelated to OBI-1 as determined by the investigators. Antibodies to OBI-1 developed in two subjects indicated with an * in the table below. However, both responded toOBI-1. Conclusions These interim results provide support for the safety and efficacy of OBI-1 in the treatment of serious bleeding episodes in AHA. Additional confirming data could establish OBI-1 as a useful treatment option for AHA. Disclosures: St. Louis: Inspiration Biopharmaceuticals Inc: Research Funding. Kruse-Jarres:Inspiration Biopharmaceiticals Inc: Research Funding. Greist:Inspiration Biopharmaceuticals Inc: Research Funding. Shapiro:Inspiration Biopharmaceuticals Inc: Research Funding. Smith:Inspiration Biopharmaceuticals Inc: Research Funding. Drebes:Inspiration Biopharmaceuticals Inc: Research Funding. Gomperts:Inspiration Biopharmaceuticals Inc: Consultancy.

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