Nonacog Beta Pegol: A Review in Haemophilia B

Syed, Yahiya Y.

doi:10.1007/s40265-017-0836-8

Cited by 9 publications

(7 citation statements)

References 28 publications

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“…More studies are required to confirm whether this also applies to human tissues. However, this difference in distribution may explain the higher FIX:C levels but comparable efficacy observed with N9-GP; both N9-GP and rIX-FP have AsBRs of 0 in adult and paediatric patients 3,4,10. Of patients treated with rIX-FP, >95% maintained a mild haemophilia phenotype (FIX:C > 5%) at all time points and on all regimens.Patients may find that an extended dosing regimen would still provide protection from bleeds but be easier to maintain.…”

mentioning

confidence: 95%

Sustained high trough factor IX activity levels with continued use of rIX‐FP in adult and paediatric patients with haemophilia B

Gill

Roberts

et al. 2019

Haemophilia

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mentioning

confidence: 95%

Sustained high trough factor IX activity levels with continued use of rIX‐FP in adult and paediatric patients with haemophilia B

Gill

Roberts

et al. 2019

Haemophilia

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“…Nonacog beta pegol (Refixia) is a recombinant, site-specific (glyco)PEGylated FIX molecule. 21 Regarding the OSC assays, the experiments revealed (concentration-dependent) underestimation of the molecule by AFS as well as by AFSL, while the application of PSL demonstrated substantial overestimation of FIX activities. Indeed, only the FIX CS assay yielded acceptable results (►Fig.…”

Section: Nonacog Beta Pegol (Refixia Extended Half-life Factor Ix)mentioning

confidence: 88%

Extended Half-Life Factor VIII/Factor IX Products: Assay Discrepancies and Implications for Hemophilia Management

et al. 2020

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Due to structural differences between extended half-life (EHL) factor VIII (FVIII) or FIX products and equivalent plasma wild-type molecules used for assay calibration, reagent-dependent discrepancies during monitoring of FVIII- and FIX-replacement therapies with EHL products have been described. To assess the performance of available one-stage clotting and chromogenic substrate assays on the Siemens Atellica COAG 360 analyzer, an in vitro study using spiked plasma samples was performed. The described results confirm previously described findings and allowed allocation of each EHL product to an appropriate assay. In addition, corresponding EHL product–specific analytes were defined within the order entry system of the University Hospital Bonn. The requirement of product-specific FVIII and FIX assays complicates patient monitoring and demonstrates the need for both continuous education and communication between treating physicians and the coagulation laboratory.

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“…rFIXFc was well tolerated in all studies [13,52,60,69,74,75]. N9-GP has been assessed in phase III trials and an extension study [71,76,77]. The most recent data available from the extension study shows that adult (n = 52) and pediatric patients (n = 25) on N9-GP (40 IU/kg weekly) had a median ABR of 1 and an AsBR of 0 [71,76,77].…”

Section: Other Long-acting Rfix Productsmentioning

confidence: 99%

Switching patients in the age of long-acting recombinant products?

Escobar¹,

Santagostino

Mancuso

et al. 2019

Expert Review of Hematology

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Introduction: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. Areas covered: This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis-Scientific and Standardization Committee annual meetings. The pharmacokinetic, safety and efficacy data for longacting recombinant products are reviewed, with a focus on recombinant factor IX albumin fusion protein (rIX-FP) and rVIII-SingleChain. This overview also provides a guide for managing a patient's switch to long-acting products. Expert opinion: Long-acting products may allow patients to maintain or decrease bleeding rates whilst increasing their dosing interval, which may in turn reduce the burden on patients and caregivers. When switching patients to long-acting products health-care professionals should provide balanced and thorough education to the patient, whilst supporting their emotional well-being. Regimens should address patients' needs and goals but should also be guided by clinical phenotype and pharmacokinetic assessment. Follow-up should assess safety concerns, bleeding rates, joint health and the impact of the regimen on patients' lifestyle.

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Nonacog Beta Pegol: A Review in Haemophilia B

Cited by 9 publications

References 28 publications

Sustained high trough factor IX activity levels with continued use of rIX‐FP in adult and paediatric patients with haemophilia B

Sustained high trough factor IX activity levels with continued use of rIX‐FP in adult and paediatric patients with haemophilia B

Extended Half-Life Factor VIII/Factor IX Products: Assay Discrepancies and Implications for Hemophilia Management

Switching patients in the age of long-acting recombinant products?

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