Enhanced Half-Life Recombinant Factor VIII Concentrates for Hemophilia A: Final Results from Extension Studies

Minno, Matteo Nicola Dario Di; Minno, Alessandro Di; Calcaterra, Ilenia; Cimino, Ernesto; Dell'Aquila, Francesco; Franchini, Massimo

doi:10.1055/s-0041-1740148

Cited by 4 publications

(1 citation statement)

References 7 publications

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“…6 7 Most of the currently available VWF(/FVIII) concentrates are plasma derived, but recombinant VWF and recombinant FVIII, and indeed extended life FVIII products, are separately available in some geographic locations. 40 41 42 The PFA is not very sensitive to plasma-derived VWF concentrates, since most of these concentrates are deficient in HMW VWF, and so the PFA does not have a role in monitoring plasma VWF concentrate therapy. The main assays used in monitoring plasma VWF concentrate therapy are VWF:RCo and FVIII:C, since these assays are often the assays use to define “specific activity” in the commercial concentrates.…”

Section: The Heterogeneity In Tests Used To Diagnose/exclude Vwd and ...mentioning

confidence: 99%

Laboratory Diagnosis of von Willebrand Disease (VWD): Geographical Perspectives

Favaloro

Pasalic

2022

Semin Thromb Hemost

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Abstractvon Willebrand disease (VWD) is reportedly the most common inherited bleeding disorder, and can also arise as an acquired (von Willebrand) syndrome (AVWS). The hemostasis laboratory plays a key role in the diagnosis or exclusion of VWD/AVWS, which may otherwise be suspected due to the patient's clinical (bleeding) history. VWD/AVWS arise from deficiency and/or defects in the adhesive plasma protein, von Willebrand factor (VWF). VWF undertakes various roles within hemostasis, but principally acts within primary hemostasis to anchor platelets to sites of vascular damage, thereby facilitating thrombus formation to arrest bleeding. The diagnosis or exclusion of VWD/AVWS requires laboratory testing for both VWF level and activity, with the latter potentially comprising several of a potential plethora of different assays. Complete diagnosis of VWD also requires the differentiation of VWD type, with six types comprising the current classification (i.e., qualitative [types 2A, 2B, 2M, 2N VWD] vs. quantitative [types 1 and 3 VWD] deficiency/defects). Although appropriate diagnosis and type classification hold important therapeutic consequences, these remain problematic and sometimes elusive for some laboratories to achieve. This report reviews the laboratory aided diagnosis or exclusion of VWD from a geographic perspective, and focuses on the disparities of approaches and methods in different regions of the world. This is primarily done from the perspective of data available from published reports related to external quality assessment (or proficiency testing) from different geographic localities. Moreover, differences in approaches between laboratories may arise due to differential adherence of particular guidelines, as well as regulatory aspects and predominance of local manufacturers and suppliers.

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Section: The Heterogeneity In Tests Used To Diagnose/exclude Vwd and ...mentioning

confidence: 99%