ObjectivesThe objective of this study was to compare total thromboembolic complications between 4-factor prothrombin complex concentrate (4F-PCC) with factor VIII inhibitor bypassing activity (FEIBA) when utilized during cardiac surgery.DesignA quasi-experimental analysis of retrospective data from consecutive patients.SettingA tertiary care university hospital.ParticipantsPatients undergoing cardiac surgery with cardiopulmonary bypass.InterventionsPatients received either 4F-PCC or FEIBA after discontinuation of cardiopulmonary bypass and reversal of heparin with protamine.Measurements and main resultsMedical records were reviewed for thromboembolic events (stroke, arterial or venous thrombosis, pulmonary embolism, myocardial infarction), acute kidney injury, ischemic bowel, death, duration of intensive care unit and hospital stay, clinical and surgical characteristics and blood product utilization. A comparison of the clinical and surgical variables demonstrated a mean effect size of 0.33 imbalance between groups that was reduced to 0.18 after propensity score weighting. The propensity scores weighted analysis found an incidence of composite thromboembolic events of 39% in the 4F-PCC (n = 90) and 47% in the FEIBA (n = 50) group, difference −8 (−24% to 12%), P = 0.13. Individual thromboembolic events, acute kidney injury, ischemic bowel, mortality, and length of intensive care unit or hospital stay was not different among groups. Patients who received FEIBA had greater chest tube drainage and received more cryoprecipitate intraoperatively. Patients who received 4F-PCC received more fresh frozen plasma transfusions postoperatively.ConclusionsAmong cardiac surgery patients, there was no difference in thromboembolic events between patients who received 4F-PCC or FEIBA when used as an adjunct to blood product administration.

31 Dec 2024·Hematology

Real-world long-term safety and effectiveness of turoctocog alfa in the treatment of haemophilia A in Japan: results from a multicentre, non-interventional, post-marketing study

Article

Author: Deguchi, Ayumi ; Nagao, Azusa ; Nogami, Keiji

OBJECTIVESTo assess the safety and effectiveness of turoctocog alfa in previously treated patients (PTPs) and previously untreated patients (PUPs) with haemophilia A in a real-world setting in Japan.METHODSThis multicentre, non-interventional, post-marketing study recruited patients with haemophilia A who initiated treatment with turoctocog alfa from 18 sites (08/2014-12/2018). The primary endpoint was adverse events (AEs) during the 2-year study period.RESULTSThe safety and effectiveness analysis set included 39 patients. In total, 13 (33.3%) patients reported ≥1 AE; incidence rate was 60.4 events/100 patient-years of exposure (PYE). Treatment was withdrawn in two cases: pruritus in a PTP and factor VIII inhibitor development in a PUP. Inhibitor development occurred in 2.6% of all patients, with an incidence rate of 3.8 events/100 PYE. The rate of inhibitor development was 0%, 25% and 20% in PTPs, PUPs and PUPs with severe type, respectively. The haemostatic success rate was 91.4% for 383 bleeding episodes and 85.7% for 14 surgeries. The negative binomial annualised bleeding rate for the prophylaxis regimen was 6.19 episodes/year (95% CI, 3.69-10.38). The mean (SD) total consumption of turoctocog alfa (n = 34; excluding FVIII inhibitors) was 5,382.6 (7,180.1) IU/kg/year/patient; consumption was 4,133.1 (1,452.4) IU/kg/year/patient for prophylaxis.DISCUSSIONThe effectiveness and safety profiles were comparable to those observed in other turoctocog alfa trials; effectiveness analysis and consumption were not affected by treatment regimens.CONCLUSIONLong-term use of turoctocog alfa therapy in clinical practice posed no newly identified safety issues and was effective for prophylaxis and treatment of bleeds in patients with haemophilia A in Japan.

01 Oct 2024·Journal of Thrombosis and Haemostasis

Development of a rapid and fully automated factor VIII inhibitor assay, insensitive to emicizumab, and a lowest level of quantification of 0.2 BU/mL

Article

Author: Verbruggen, Bert ; Sobas, Frédéric ; Binder, Nikolaus B ; Moore, Gary W ; Polenewen, Robert ; van Velp, Petra J C ; Knöbl, Paul

BACKGROUNDFactor (F)VIII inhibitors are measured using labor- and resource-expensive Nijmegen or Bethesda assays, which lack sensitivity for low-titer inhibitors and show high variations in quality surveys, mainly because of manual assay procedures.OBJECTIVESThe goal of this study was the development of a fast and fully automated FVIII inhibitor assay by using recombinant (r)FVIII as substrate and dedicated equipment for execution of the test.METHODSA new rapid, fully automated, FVIII inhibitor assay is presented, the core of which is use of full-length recombinant FVIII (rFVIII; Kovaltry, Bayer) as inhibitor substrate instead of plasma FVIII, resulting in rapid binding of inhibitors to rFVIII due to absence of von Willebrand factor. Dramatic shortening of incubation time facilitated full automation on an analyzer capable of 3 subsequent sample dilution steps and 3 reagent additions. Equal volume mixtures of sample and rFVIII (1.0 U/mL) were incubated for 10 minutes at 37 °C, whereafter remaining FVIII activity was analyzed with a kinetic chromogenic assay, allowing inhibitor activity calculation without preceding FVIII activity calibration, using a Ceveron s100 analyzer (Technoclone).RESULTSMean titer in 60 nonhemophiliacs was 0.0 BU/mL (SD, 0.1), yielding a limit of blank of 0.1 BU/mL and lower limit of quantification of 0.2 BU/mL. Analyses were performed with the new method and a Nijmegen assay in 28 inhibitor-positive clinical samples, 14 containing emicizumab and 14 without. Correlation coefficient in emicizumab-free type I inhibitor samples was 1.0. Emicizumab dependency of the method was excluded in spiking experiments with inhibitor-positive samples. Reproducibility was tested by analyzing 7 samples in 3 laboratories for 5 days, twice daily; coefficients of variation of all samples were <15%.CONCLUSIONWe present development data of a sensitive and specific rapid, automated FVIII inhibitor assay generating results within 20 minutes that is less resource-intensive than standard assays with potential to improve assay variability.

100 Deals associated with Factor VIII(Tonrol Bio-Pharmaceutical Co Ltd)

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KEGG	Wiki	ATC	Drug Bank
-	-	B02BD02	DB14473

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Indication	Country/Location	Organization	Date
Hemophilia A	China	Tonrol Biological Pharmaceutical Co. Ltd.	23 Aug 2013

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASGCT2022	Not Applicable	Hemophilia A	-	Factor VIII Transgene (Wild-type FVIII-BDD)	ysaenbggim(bzqmozmukw) = qwyrmlrwes mflnqcuqyu (wdkowwxbes ) View more	Positive	02 May 2022
				Factor VIII Transgene (Engineered FVIII-BDD variants)	ysaenbggim(bzqmozmukw) = iupibuujel mflnqcuqyu (wdkowwxbes ) View more
ASGCT2017	Not Applicable	FVIII-specific	-	Factor VIII-CAR Retroviruses (F8CAR-mFoxp3-RV transduced cells)	rlbtyhonol(ypgbxlhscf) = F8CAR-mFoxp3 Tregs significantly suppressed the proliferation of F8CAR Teffs in an antigen-specific fashion xxrqbztcng (oplgbbsofu )	Positive	01 May 2017
				Factor VIII-CAR Retroviruses (Untransduced cells)

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